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Novartis and Bristol Myers Squibb halt CAR-T trial for autoimmune diseases over patient deaths: a potential issue with cell-based manufacturing
Curator: Stephen J. Williams, Ph.D.
As a result of trial deaths, both Novartis and Brisol Myers have halted some key CAR-T therapy trials designed for several autoimmune diseases including their rap-cel trial for systemic lupus erythematosus/lupus nephritis and multiple sclerosis and the zola-cell trials which is a CAR-T targeting CD-19 was halted.
In previous posts we examined the toxicities and pharmacovigilence related to CAR-T therapies in various cancers and these serious adverse events, mainly the tumor lysis and cytokine release syndrome, where CAR-T efficacy results in rapid lysis of tumor cells and rapid rise of interleukins and cytokines within the blood have been documented. However in this case there may have issue related to scaling up of the manufacturing process, not merely an untoward or unknown toxicity related to the therapy itself. In fact there have been many examples, some will be highlighted here, where issues may arise during Quality Control (QC) of the manufacturing up-scaling of gene and cell-therapies. For reference the following are the two major posts on this site which discuss matters of serious adverse events and pharmacovigilence of CAR-T therapies in cancer and can be read for background information of the mechanisms of toxicity of the therapy itself:
However the issues in the following report may have arose from the ‘rapid manufacturing’ technology employed to produce both the rap-cell and zola-cell CAR-T therapies.
Three deaths have forced Novartis to pause development of its CD19 CAR-T candidate in autoimmune diseases, just as Bristol Myers Squibb has halted a similar program due to immune-related adverse events, Fierce has learned. The Swiss pharma has temporarily halted development of its autologous CD19 CAR-T therapy, rapcabtagene autoleucel (rap-cel; YTB323), for several autoimmune diseases in immunology and neuroscience, Novartis confirmed with Fierce on Monday. The decision comes after the rap-cel program recorded three deaths from complications caused by serious immune effector cell-associated hemophagocytic syndrome (IEC-HS), according to Novartis. IEC-HS is a severe systemic inflammatory side effect associated with immunotherapies like CAR-T cell therapy. The rogue immune response may be triggered when engineered T cells rapidly expand and activate inside the patient’s body. Novartis confirmed the trials included phase 2 studies of rap-cel in systemic lupus erythematosus/lupus nephritis (CYTB323J12201), systemic sclerosis (CYTB323K12201), ANCA-associated vasculitis (CYTB323I12201) and idiopathic inflammatory myopathies (CYTB323L12201), as well as phase 1/2 studies evaluating safety, cellular kinetics and efficacy in rheumatoid arthritis and Sjogren’s disease, generalized myasthenia gravis, relapsing multiple sclerosis (MS), and non-active progressive MS (CYTB323M12101B, CYTB323O12101, CYTB323N12101 and CYTB323R12101, respectively). Screening, randomization and treatment administration across the studies have been put on hold, the spokesperson said.
“The temporary halt will allow for a more comprehensive review of the evolving clinical and safety data across the program, following three serious immune effector cell-associated hemophagocytic syndrome (IEC-HS) events,” Novartis told Fierce in an email. “Patient safety remains of [the] highest priority; patients who have been treated within the trials will continue to be monitored as per protocol.” The company said the ongoing program in oncology, which is currently in phase 1/2 trial for chronic lymphocytic leukemia/small lymphocytic lymphoma, diffuse large B-cell lymphoma, adult acute lymphoblastic leukemia and high-risk large B-cell lymphoma, “is not affected by this halt.”
Rap-cel is an autologous CD19-targeted CAR-T aimed to reprogram patient’s own T cells to eliminate CD19-expressing B cells. This asset is manufactured using Novartis’ T-Charge platform, a rapid manufacturing platform that allows for fewer exhausted T cells and eliminates the need for extended culture time outside of the body, according to the website. In a Monday note to clients, William Blair analysts also flagged that BMS paused enrollment in its autoimmune trials testing an autologous CD19-targeted CAR-T, zola-cel (BMS-986353). William Blair noted that in a follow-up, the company disclosed that the enrollment was paused due to “transient and reversible inflammatory events.” “Out of an abundance of caution, we implemented a voluntary pause to review clinical data across our zola-cel program,” a BMS spokesperson told Fierce in an email. “We are focused on completing our evaluation and resuming enrollment as quickly as possible.” Also targeting CD19, zola-cel acts through the same mechanism as BMS’ Breyanzi, an FDA -approved treatment for relapsed and refractory blood cancers. Like rap-cel, zola-cel was also developed using a rapid manufacturing platform. BMS’ NEXT T platform is designed to encourage the growth of more uniform and potent T cells, which may prompt a deeper and more durable response in patients, according to its website. Since both Novartis’ and BMS’ assets were developed using rapid manufacturing platforms, analysts from William Blair suggested “that rapid manufacturing could be driving increased cell expansion and the reported toxicities.”
From the press release it appears both companies are using this rapid manufacturing process and the companies suggest the deaths were a result of the rapid expansion of T cells in the patient and not from a toxicodynamic issue.
What is Rapid Manufacturing Technology?
Rapid manufacturing of CAR-T therapy: strategies and impact
The accessibility of autologous chimeric antigen receptor T cell (CAR-T) therapies is challenged by the complex processes and capacity constraints of manufacturing. Rapid manufacturing capable of shortening manufacturing timelines could transform the CAR-T field. Here, we outline approaches to rapid CAR-T manufacturing, highlighting its impact on various stakeholders in the landscape.
Main textOver the past decade, autologous CAR-T therapies have shown remarkable success in treating hematological malignancies. However, their accessibility remains limited, which can be attributed to the complex manufacturing process involving the patient’s cells and the limited capacity of manufacturing sites, both of which contribute to the high cost of the therapy. These factors have resulted in prolonged and uncertain turnaround times for the production of these therapies, rendering a sizable population of patients ineligible for treatment due to the deterioration of their medical condition while they wait for treatment.
To resolve this issue, the field has delved into rapid manufacturing platforms. Here, we discuss approaches toward rapid CAR-T manufacturing, examine its impact on different stakeholders in the landscape, and provide insights into future directions for the field.
Approaches toward rapid manufacturing of CAR-T therapiesThe conventional clinical CAR-T manufacturing workflow involves leukapheresis, enrichment, activation (2–3 days), CAR gene insertion (1–2 days, typically performed through viral transduction with <50% efficiency), expansion (>7 days), formulation, and cryopreservation. Rapid CAR-T manufacturing have been approached via acceleration and/or simplification of the above workflow ( Figure 1 ).
Workflow accelerationWorkflow acceleration seeks to obtain CAR-T products with similar cell yields but within a shorter timeframe by refining unit operations of conventional workflows. The critical quality attributes (CQAs) and release criteria of the manufactured products are projected to remain unchanged or similar to those established in conventional workflows.
Enhancing the efficiency of CAR gene insertion and expediting cell expansion are pivotal factors for this approach. In particular, CAR gene insertion could benefit from advanced gene integration technologies, such as CRISPR/Cas9 [ 1] or transposon systems [ 2], and cell expansion could be hastened by optimizing various culture parameters, such as media formulation, feeding strategy, and cell density. Machine learning could aid the optimization processes, as demonstrated in a study in which a base media formulation optimized for cell viability and expansion in a 6-day culture was generated through a model with a training data set obtained from 104 cultures [ 3].
One example of this rapid manufacturing approach is evident in Kite Pharma’s amended manufacturing protocol for its CAR-T product, Yescarta, announced earlier in 2024. Kite Pharma successfully shortened its manufacturing time by 2 days and proved comparability between the original and amended protocols i.
Workflow simplificationWorkflow simplification aims to enrich highly efficacious CAR-T stem cells (depending on the protocol and product, this population can comprise central memory T cells, naïve T cells, stem cell memory T cells, or a combination thereof) in the final product with short-term culture. This can dramatically reduce the manufacturing turnaround time from 10–12 days to 1–3 days, or even the next day. Given the emphasis on CAR-T stem cells, the CQAs and release criteria of the manufactured products are expected to differ from those established in conventional workflows.
This strategy arose from findings that CAR-T products containing cells of greater stemness demonstrated improved persistence and efficacy in clinical trials [ 4]. Therapies manufactured by this approach rely on a much lower dosage (10 6–10 7) of T stem cells, which have superior in vivo proliferative potential and are abundant in the early stages of ex vivo culture. Consequently, this approach drastically reduces or even eliminates the need for ex vivo expansion, allowing for the entire omission of cell expansion and (possibly) cell activation operations [ 5, 6] from the conventional workflow.
Establishing novel and suitable CQAs and release criteria (including cell phenotype, yield, and function) and aligning them with advanced process analytical technologies are crucial in this approach. Notably, cells manufactured by this approach may not have completed the CAR gene integration process by the end of the ex vivo manufacturing. New methods to examine the presence of edited genes, such as using quantification of reverse transcription intermediates, may be useful in this regard [ 6].
An example of this approach was explored in a clinical study sponsored by Novartis. The study compared the conventional workflow-produced CAR-T product, Kymriah, with a T stem-enriched variant produced through the T-Charge platform, in which T cells were cultured for <2 days. The study demonstrated that the T stem-enriched version had comparable efficacy and a favorable safety profile despite being administered at 10–100 times lower dosage [ 7].
Impact of rapid CAR-T manufacturingThe direct impact of rapid CAR-T manufacturing is evident in the cell therapy manufacturing sector due to its ability to overcome current hurdles, such as workforce shortages, and in implementing decentralized and point-of-care (POC) models of manufacturing. Additionally, rapid CAR-T manufacturing also aids shortening the vein-to-vein time and improving predictability in treatment start times, delivering substantial benefits to patients and other key stakeholders in the healthcare system ( Figure 2 ).
Alleviating workforce shortagesConventional manufacturing of autologous CAR-T therapies involves a predominantly manual scale-out process, which requires highly skilled workforce who can perform consistently in a high-stress current good manufacturing practice (cGMP) environment. As a result, a skilled workforce remains a pressing need in the industry [ 8] and the launch of training programs worldwide is clear evidence for the need to develop this workforce [ 9]. Rapid manufacturing reduces cleanroom turnover time, allowing facilities to produce more treatments with a smaller workforce. Together with automated platforms, such as advanced bioreactors [ 10], rapid CAR-T manufacturing has the potential to drastically reduce human intervention and to address the workforce shortage in the industry.
Towards decentralized and POC models of manufacturingDecentralized and POC models of manufacturing have often been proposed as a key solution to improve CAR-T treatment accessibility, but hurdles, such as cross-site process variability and high infrastructure costs, have hindered their implementation [ 11]. Rapid manufacturing has the potential to effectively address these hurdles. By streamlining the workflow and shortening the manufacturing cycle, it minimizes overall process variabilities to enable a smoother technology transfer between manufacturing sites. With reduced culture capacity (for the approach of workflow simplification), it also diminishes the footprint of the process, and subsequently lowers the infrastructure cost of the cGMP facility. Together with the development of compact closed cell manufacturing systems, it can reduce the need for high-grade clean rooms by allowing manufacturing in GMP-in-a-box. The implementation of a decentralized/POC manufacturing model may also eliminate additional steps in the conventional manufacturing workflow, such as cryopreservation and packaging, further reducing time and cost ( Figure 1).
Concluding remarks and future perspectivesRapid manufacturing of CAR-T products via workflow acceleration takes advantage of the clinical and regulatory experience of conventionally manufactured CAR-T products, although its workflow complexity remains a limiting factor. Workflow simplification presents a streamlined approach due to its potential to significantly reduce manufacturing turnaround time and minimize the number of process steps. However, it necessitates significant efforts to establish process and product knowledge. If comparable clinical evidence can be collected to confirm the safety and effectiveness of products, workflow simplification can outpace workflow acceleration and emerge as the preferred manufacturing trend. With workflow simplification, the bottleneck in the CAR-T cell manufacturing cycle would be shifted to release testing, which conventionally includes a 14-day sterility test following US Pharmacopeia <71> guidelines. Rapid sterility testing utilizing highly sensitive cytometry systems [ 12], or non-growth-based methods [ 13], such as sequencing, can reduce the test time significantly to a few days or even hours. Their advancement will be the next disruptive step that can perfectly complement the transition of CAR-T manufacturing toward the decentralized/POC model ( Figure 1) and enable further shortened vein-to-vein time.
Given that the number of patients receiving CAR-T is expected to increase due to expanded therapy indications, there is an mounting need for treatment accessibility. Rapid manufacturing appears poised to address this need by enabling decentralized models of manufacturing; however, its implementation is not straightforward and would face challenges, including workforce training, facility availability, and supply chain logistics [ 11]. In addition, we must remember that other activities in the CAR-T treatment value chain, such as patient selection, bridging therapy, and patient management, also significantly impact the patient journey. Thus, enhancing the efficiency of these activities alongside rapid manufacturing is crucial for maximizing the potential of rapid CAR-T manufacturing and the broader adoption of CAR-T therapy.
Summary: Rapid manufacturing of CAR-T therapies, through workflow acceleration and simplification, can significantly shorten production times, improve treatment accessibility, and enable decentralized manufacturing models while addressing workforce and infrastructure challenges.
Source: Chen, S., & Liu, D. (2025). Rapid manufacturing of CAR-T therapy: strategies and impact. Trends in Biotechnology, 43(4), 745-748. https://doi.org/10.1016/j.tibtech.2024.09.013
Now the first CAR-T therapies were performed at university hospitals for leukemias and were all produced in small batch at the university site. However in attempts to scale up there have been difficulties including
interuptions in funding and capital flow to build these manufacturing facilities
quality control issues
supplier issues related to manufacturing
potential issues in modeling of scale up
In another update we will further examine these issues related to each of these points as news on these topics emerges
Please read these additional article on Cell Based Manufacturing on this Open Access Scientific Journal
Companies Actively Engaging Artificial Intellegence for Drug Design: 2026
Curator: Stephen J. Williams, Ph.D.
Work-in-Progress
This is not an exhaustive list of all AI companies involved in the drug discovery process. Some like Chai Discovery will be featured in separate posts and this will be constantly updated as companies are acquired and formed.
UPDATED 8/31/2026
First Bankruptcy Casualty for AI-Drug Discovery Firm: BioXcel Files for Chapter 11 Bankruptcy and sells Alzheimers Pipeline to Teva Pharmaceuticals
Aug 27 (Reuters) – AI-driven biopharma firm BioXcel Therapeutics filed for Chapter 11 bankruptcy protection in the U.S. on Thursday.
Here are more details:
• BioXcel listed estimated liabilities in the range of $100 million to $500 million and assets of $10 million to $50 million in its petition filed with U.S. Bankruptcy Court for the District of Delaware.
• The filing comes after BioXcel entered into an amended credit agreement on Monday, under which creditors would provide an additional $1.25 million in loans to the company.
• BioXcel Therapeutics reported a quarterly adjusted loss of 49 cents per share for the three months ended June 30.
• The firm, whose shares have fallen about 55% so far this year, said in May it was exploring strategic options including a sale, merger or licensing agreement of its assets, but did not provide a timeline.
• BioXcel is focused on developing neurological medication. Its experimental drug BXCL501 is marketed as an acute treatment for agitation associated with Alzheimer’s dementia.
(Reporting by Anusha Shah in Bengaluru; Editing by Eileen Soreng)
The traditional paradigm in the pharmaceutical industry of selecting new chemical entities that are put through lead identification and selection in drug discovery and development is marred by decade-long timelines, high costs that increase every year, and low success rates. Consequently, many diseases and medical conditions remain without treatments. Yet, an underutilized and expansive universe of potential drug candidates exists that consists of approved products that could be effective for additional indications as well as compounds that have already demonstrated safety in prior clinical trials but may have been discontinued by their original developers for various reasons.
BioXcel Therapeutics is focused on AI-based drug re-innovation in neuroscience. We expedite the discovery and development of new indications for existing late-stage drug candidates and/or approved drugs by leveraging NovareAI: a composite of AI tools and approaches. NovareAI enables us to reduce therapeutic development costs, potentially accelerate timelines, while improving the success rate of bringing new treatment options to patients.
Original Article Starts Here
I want to start with a Great interview and white paper by McKinsey where they interview key opinion leaders on the business intelligence on AI firms in the drug discovery world and what to look for in companies that are in this space.
Artificial intelligence could help scientists develop better medicines faster—and thus improve millions of people’s lives. But for that to happen, companies will need to change the way they work.
Below is a Mckinsey report on How AI will Change the Future of Biotech
The AI-driven drug discovery industry continues to grow, fueled by new entrants in the market, significant capital investment, and technology maturation. We’ve identified more than 250 companies working in the industry. More than half of them are based in the United States, but key hubs are emerging in Western Europe and Southeast Asia as well. The best of these companies will fully integrate AI into research workflows, as the exhibit shows. By putting AI at the center of the research engine, companies can transform research at scale—and bring about dramatic improvements in patient outcomes. For the full article, see:
Parts of a high-throughput screening (HTS) process embedded with AI technology
1. High-throughput screen commenced with diverse compound sets Scientist selects diverse compound sets (a set of chemical compounds with a wide range of chemical structures) as first high-throughput screen
In silico/ on-the-chip simulations
In-vitro/ ‘wet lab’ experiments
2. Automated compound selection and transfer Using HTS machinery, individual compounds are transferred to individual wells of cells under experimental conditions
3. Computer-vision-based hit selection Cell response to each compound is measured using microscope analysis (eg, through computer vision techniques); promising compounds are labeled “hits”
4. Automated machine learning (ML) model training from screen outcomes Information from HTS for first few plates is automatically transferred into an ML pipeline, which “learns” how cells respond to each kind of chemical structure
5. Compound library inferencing and prioritization ML algorithm then scans the remainder of the library compounds and predicts which plates should be prioritized to identify the highest number of hits in the next screen
6. Automated compound selection based on ML recommendations ML recommendations are automatically queued and used in the next round of HTS. The cycle continues, with the algorithm continuously learningfrom “real world” outputs. Recommendations trigger scientists to explore new chemical space and begin downstream screening processes more quickly. These recommendations feed into the selection of chemical compounds in step 1
Human bodies are incredibly complex. It takes many years to discover even just one new medicine to successfully treat a disease. Could artificial intelligence help speed up that process? McKinsey experts believe so. (The following transcript has been edited for clarity.)
Lydia The: What excites me about AI and drug discovery is the convergence between technology, drug development, and biology, which is going to lead to better drugs being developed faster—using all of the capabilities that Silicon Valley and the tech ecosystem have developed—to help us have even greater impact on patients.
Christoph Sandler: Today, to discover and develop a drug takes more than ten years.
Alex Devereson: We might be able to have drugs in one-tenth of the time, from being discovered to being able to treat patients. Today, many diseases simply have no treatments whatsoever. I think, and I hope, we’re going to see a world where we can generate therapies that can treat those patients very effectively. Fundamentally, we will have life-changing, game-changing drugs—on a scale and at a pace that we’ve never seen before—getting to the right patient at the right time.
The promise of personalized medicine
Christoph Sandler: In the not-so-distant future, we might collect health data across different inputs: from wearables, from our electronic medical records, or from clinical or academic research. And we will have the opportunity, on a voluntary basis, to upload these data into a central, secure, trusted data storage system.
Lydia The: You could imagine using the data to figure out not just what drug might work for you but exactly what drug would work for you at what time, in what sequence, in what dose—really personalized to you.
Will AI replace scientists?
Lydia The: What we’ve found is that technology doesn’t supplant the people. Rather, it will enable scientists to do things faster and better—and potentially develop insights that humans would not be able to develop at all.
Alex Devereson: Scientists will be able to discover things with machine learning that they could never have thought of by themselves, generate entirely new ideas, and move at a pace at which one person can do what it would have taken 100 to do before.
Lydia The: While in previous generations, scientists would spend a lot of their time—maybe even the majority of their time—on manual efforts such as pipetting from one tray to another tray or manually curating and cleaning data, I think AI will help us do all of those things in a more automated and quick way, and develop hypotheses that can then lead a scientist to think through, “What would the next experiment be? What are the implications of the data?”
What companies should do today
Alex Devereson: I think the challenge a lot of companies have is that they want to explore new ideas but, for good reason, they are reluctant to commit until they’ve seen some results and some tangible impact. So they explore a lot of pilots.
Lydia The: We have a term for that. We call it “pilot purgatory”: companies focus on one pilot, and they see the returns in a single pilot, but they don’t establish that approach and way of operating across their organization.
Define a ‘North Star’
Lydia The: One of the most important things for companies to escape pilot purgatory is a real mindset shift, from the top end all the way through the rest of the organization.
Christoph Sandler: It is important for the organization to define what a North Star for them should be, so that the data and analytics transformation of the R&D function can be targeted toward that North Star.
Identify—and solve—the biggest pain points
Alex Devereson: Truly understand what your biggest scientific and operational pain points are. For the lab scientists, for the patients who are in your clinical trials, for the patients who might get the drug: What is the biggest unsolved problem today?
Embed analytics into decision making
Christoph Sandler: Bring the organization on board and help them understand the potential of data and analytics.
Alex Devereson: You need to really, truly redesign a process that embeds analytics, where it’s not something on the side; it’s truly a part of the decision making.
Christoph Sandler: And you need to establish trust in the data and in these models. Equally important as the technical part is the human part.
Deliver value quickly
Alex Devereson: Don’t set up a project that delivers only after five years. Have a view on how you can deliver value in three months—and on what it takes in terms of analytics, data, and technology—with a relentless laser focus on value for the patient and for the scientific process.
The inventor of some of AI’s technical underpinnings is shifting focus to an RNA startup. Also, Rick Pazdur has some ideas on how to determine whether Revolution Medicines’ pancreatic cancer drug can work in a first-line setting. And an oncologist explains why she believes the failed Grail trial matters.
artificial intelligence
The AI architect taking aim at RNAi
Jakob Uszkoreit helped create the transformer architecture — the “T” in ChatGPT — that sparked the generative AI boom. Now he’s trying to do something just as ambitious in drug development, STAT’s Brittany Trang writes. His startup, Inceptive Nucleics, is building biological foundation models that can be applied across a wide range of sequence-based medicines, from RNA interference therapies to mRNA and antisense drugs.
That vision caught the attention of Alnylam, which yesterday announced that it had struck a three-year partnership worth up to $2 billion in potential milestone payments and royalties, along with a $30 million upfront investment. The idea is that AI could perhaps do more than analyze biological data — and instead design the molecules themselves.
Top 10 Leading AI Drug Discovery Companies Transforming the Market: Trends, Technologies, Growth Outlook & Competitive Landscape (2026-2034)
Artificial Intelligence (AI) is redefining the future of pharmaceutical research, making drug development faster, smarter, and more cost-effective than ever before. What once took more than a decade and billions of dollars can now be significantly accelerated through advanced machine learning algorithms, generative AI, predictive analytics, and computational biology.
AI Drug Discovery Market is projected to grow from USD 3.41 billion in 2026 to USD 10.45 billion by 2034, registering a robust 15.7% CAGR. This remarkable expansion reflects the pharmaceutical industry’s increasing reliance on AI-powered platforms to improve research efficiency, reduce clinical failures, and identify breakthrough therapies for complex diseases.
As healthcare systems worldwide demand faster innovation and precision medicine continues to evolve, AI is becoming an essential component of modern drug discovery rather than an experimental technology.
Key Technologies Powering AI Drug Discovery
The industry’s rapid growth is supported by several advanced technologies that work together throughout the drug development pipeline.
Machine Learning helps identify disease targets, recognize biological patterns, and predict therapeutic outcomes using historical datasets.
Deep Learning improves molecular analysis by recognizing highly complex biological relationships that conventional computational methods may overlook.
Generative AI creates entirely new molecular structures designed for specific therapeutic applications, enabling scientists to explore millions of virtual compounds before physical synthesis.
Predictive Modeling estimates toxicity, efficacy, pharmacokinetics, and safety profiles early in development, reducing unnecessary experimentation.
Molecular Docking Simulations evaluate how drug molecules interact with proteins, helping researchers prioritize candidates with the highest therapeutic potential.
AI-Driven Clinical Trial Optimization assists pharmaceutical companies in selecting suitable patient populations, improving trial design, and increasing the probability of successful outcomes.
Top 10 Leading AI Drug Discovery Companies Driving Innovation
The competitive landscape is rapidly evolving, with established technology providers and specialized biotechnology companies competing to develop next-generation AI platforms.
1. Insilico Medicine
Insilico Medicine has become one of the industry’s most recognized innovators through its end-to-end AI drug discovery platform. The company combines generative AI with biological research to accelerate target identification and therapeutic development.
2. Exscientia
Exscientia focuses on AI-designed precision medicines and has established multiple collaborations with leading pharmaceutical organizations to improve drug candidate selection and optimization. Exscientia is a pioneering artificial intelligence-driven pharmatech company that was acquired by Recursion Pharmaceuticals in November 2024
3. Atomwise
Known for its deep learning platform, Atomwise uses artificial intelligence to identify promising small-molecule therapies across oncology, infectious diseases, and rare disorders.
4. Recursion Pharmaceuticals
Recursion Pharmaceuticals integrates high-content imaging, automation, and AI to analyze millions of biological experiments, enabling large-scale phenotypic drug discovery.
5. BERG
BERG (now operating or known as BPGbio) specializes in biology-driven AI by combining multi-omics datasets with artificial intelligence to identify biomarkers and novel therapeutic opportunities.
6. Cyclica
Cyclica’s AI platform focuses on polypharmacology, helping researchers understand complex drug-protein interactions while improving drug design efficiency. , focusing on MatchMaker and POEM technology platforms before being acquired by Recursion for $40 million in May 2023.
7. GNS Healthcare
GNS Healthcare applies causal AI and patient-level analytics to support personalized medicine and identify drug repurposing opportunities.
8. DeepCure
DeepCure develops generative chemistry platforms capable of designing optimized molecules while reducing early-stage research complexity.
9. Schrödinger
Schrödinger combines computational chemistry, molecular simulation, and AI technologies to improve molecular modeling and accelerate pharmaceutical innovation.
10. IBM Watson Health & DeepMind Health
These technology pioneers continue expanding AI capabilities across healthcare by leveraging advanced analytics, large-scale biological data processing, and intelligent research platforms that support drug development initiatives. https://www.ibm.com/products/watsonx
There are many other companies that are using AI in Innovative ways to support drug discovery. Here is another list from
11 Innovative Companies Using AI for Drug Discovery
A look at 11 companies using AI-driven platforms to reshape drug discovery, including generative models, computational chemistry, and data-centric biology.
AI-Native Drug Discovery Landscape 2026 – How LPBI Group Differentiates in the New Era of Foundation Models
Curators: Aviva Lev-Ari, PhD, RN with Grok Assistence
As the race to build powerful biology foundation models intensifies, several well-funded AI-native companies have emerged with ambitious platforms for protein design, small-molecule generation, and multimodal drug discovery. While these companies bring strong technical capabilities, LPBI Group occupies a distinct and complementary position in the ecosystem.
Key AI-Native Players (2026)
Competitive Landscape Table for AI-Native in Drug Discovery
Company
Focus
Stage
LPBI Differentiation
Isomorphic Labs (DeepMind)
Protein/small molecule design using AI
Advanced (AlphaFold3 based)
LPBI offers curated multimodal training data + COM methodology (they need high-quality data to train/validate)
EvolutionaryScale
Protein design (ESM models)
Early commercial
LPBI’s strength is in clinical/therapeutic context + mechanism-of-action curation across full disease spectrum
Chai Discovery
Multimodal AI for drug discovery
Early
LPBI provides the upstream high-provenance corpus + ontology they would need for better results
Recursion Pharma
Phenotypic screening + AI
Clinical stage
LPBI’s expert-curated literature + images + COM complements their wet-lab focus
Generative AI for protein design & therapeutic optimization
Early-stage
LPBI supplies the high-provenance, expert-curated multimodal corpus and COM Tool Factory that significantly enhances model accuracy and reduces failure rates in downstream development
How LPBI Group Differentiates
LPBI Group does not compete directly in building foundation models. Instead, we provide the critical upstream layer these companies and hyperscalers urgently need:
A 9 GB private multimodal corpus of expert-curated scientific content (6,290+ articles, 48 e-Books, 7,500+ images, 300+ podcasts)
The 17-part Composition of Methods (COM) Tool Factory, including AJAUS (autonomous 24/7 refresh) and Rosetta Stone Ontology (causal mapping)
15 Subject Matter Small Language Models (SLMs) ready for concatenation into proprietary LLMs and MFMH
Proven track record of 4–5×+ uplift in novel causal relationship extraction when combined with frontier models
Strategic Positioning
While AI-native startups excel at model architecture and computation, they still face the persistent bottleneck of high-quality, causally structured, provenance-rich training data. LPBI Group’s vertically integrated assets and methodology offer a defensible moat and a true “own-both” advantage when partnered with hyperscalers or pharma companies.
This complementary role positions LPBI Group as the ideal upstream partner for the next generation of domain-aware AI in Health.
AlignedHQ.ai as Partner vs. Insilico Medicine
AlignedHQ.ai is a strong potential partner.
Why?
They are AI-native focused on protein design and generative models — directly complementary to LPBI’s strengths in curated biomedical literature, mechanism-of-action, and multimodal data.
They would benefit enormously from access to LPBI’s high-quality training data and COM methodology.
Partnership model: They use LPBI data (licensed) + Grok 5 / SpaceXAI compute and frontier models → Co-develop specific therapeutic pipelines.
Insilico Medicine is also a good candidate but slightly less ideal than AlignedHQ for early partnership because:
Insilico is more advanced clinically (has candidates in trials) and may want more control.
AlignedHQ appears earlier-stage and more open to collaboration.
Recommendation: Start with AlignedHQ.ai as a proof-of-concept partner (easier entry, high complementarity). Use success there to approach Insilico and others from a position of strength.
Where does AlignedHQ.ai’s Domain Knowledge in Medicine Come From?
From public information:
Primarily from public + licensed datasets (PDB, UniProt, scientific literature, clinical trial data, etc.).
They rely heavily on large-scale public biomedical databases and pre-trained models (e.g., AlphaFold derivatives).
Like most AI-native drug discovery companies, they have limited proprietary clinical/therapeutic context compared to LPBI’s expert-curated, mechanism-rich corpus.
Their domain knowledge is model-derived rather than expert-curated at source.
This is LPBI’s Core Delta: AlignedHQ (and similar companies) excel at model architecture and generation but lack the deep, traceable, expert-validated biomedical knowledge that LPBI has built over 14+ years. This is why access to LPBI’s portfolio would be highly valuable to them.
AI Initiatives in Big Pharma @Grok prompt & Proprietary Training Data and Inference by LPBI Group’s IP Asset Class III: 100 e-Proceedings and 50 Tweet Collections of Top Biotech and Medical Global Conferences, 2013-2025
Curator: Aviva Lev-Ari, PhD, RN
We had researched the topic of AI Initiatives in Big Pharma in the following article:
Authentic Relevance of LPBI Group’s Portfolio of IP as Proprietary Training Data Corpus for AI Initiatives at Big Pharma
We are publishing a Series of Five articles that demonstrate the Authentic Relevance of Five of the Ten Digital IP Asset Classes in LPBI Group’s Portfolio of IP for AI Initiatives at Big Pharma.
For the Ten IP Asset Classes in LPBI Group’s Portfolio, See
This Corpus comprises of Live Repository of Domain Knowledge Expert-Written Clinical Interpretationsof Scientific Findings codified in the following five Digital IP ASSETS CLASSES:
LPBI Group’s IP Asset Class III assets are “rare, defensible” for Big Pharma AI, powering from R&D acceleration to equitable care. Technical Implications: Enables theme-specific models (e.g., oncology conferences) for diagnostics/trials. Business Implications: Boosts ROI on $500M investments; licensing for symposia/webinars. Unique Insight: As the sole record of speaker insights, these outpace public data for “frontier” inference—key in series for holistic pharma AI moats.Promotional with resource links (e.g., IP portfolio, biotech conference lists). Complements prior pieces by adding temporal/event depth.
• IP Asset Class V: 7,500 Biological Images in our Digital Art Media Gallery, as prior art. The Media Gallery resides in WordPress.com Cloud of LPBI Group’s Web site
BECAUSE THE ABOVE ASSETS ARE DIGITAL ASSETS they are ready for use as Proprietary TRAINING DATA and INFERENCE for AI Foundation Models in HealthCare.
Expert‑curated healthcare corpus mapped to a living ontology, already packaged for immediate model ingestion and suitable for safe pre-training, evals, fine‑tuning and inference. If healthcare domain data is on your roadmap, this is a rare, defensible asset.
The article TITLE of each of the five Digital IP Asset Classes matched to AI Initiatives in Big Pharma, an article per IP Asset Class are:
AI Initiatives in Big Pharma @Grok prompt & Proprietary Training Data and Inference by LPBI Group’s IP Asset Class I: PharmaceuticalIntelligence.com Journal, 2.5MM Views, 6,250 Scientific articles and Live Ontology
AI Initiatives in Big Pharma @Grok prompt & Proprietary Training Data and Inference by LPBI Group’s IP Asset Class II: 48 e-Books: English Edition & Spanish Edition. 152,000 pages downloaded under pay-per-view
AI Initiatives in Big Pharma @Grok prompt & Proprietary Training Data and Inference by LPBI Group’s IP Asset Class III: 100 e-Proceedings and 50 Tweet Collections of Top Biotech and Medical Global Conferences, 2013-2025
AI Initiatives in Big Pharma @Grok prompt & Proprietary Training Data and Inference by LPBI Group’s IP Asset Class V: 7,500 Biological Images in LPBI Group’s Digital Art Media Gallery, as Prior Art
AI Initiatives in Big Pharma @Grok prompt & Proprietary Training Data and Inference by LPBI Group’s IP Asset Class X: +300 Audio Podcasts Library: Interviews with Scientific Leaders
In the series of five articles, as above, we are presenting the key AI Initiatives in Big Pharma as it was created by our prompt to @Grok on 11/18/2025:
Generative AI tools that save scientists up to 16,000 hours annually in literature searches and data analysis.
Drug Discovery and Development Acceleration Pfizer uses AI, supercomputing, and ML to streamline R&D timelines
Clinical Trials and Regulatory Efficiency AI:
-Predictive Regulatory Tools
-Decentralize Trials
-inventory management
Disease Detection and Diagnostics:
– ATTR-CM Initiative
– Rare diseases
Generative AI and Operational Tools:
– Charlie Platform
– Scientific Data Cloud AWS powered ML on centralized data
– Amazon’s SageMaker /Bedrock for Manufacturing efficiency
– Global Health Grants:
Pfizer Foundation’s AI Learning Lab for equitable access to care and tools for community care
Partnerships and Education
– Collaborations: IMI Big Picture for 3M – sample disease database
– AI in Pharma AIPM Symposium: Drug discovery and Precision Medicine
– Webinars of AI for biomedical data integration
– Webinar on AI in Manufacturing
Strategic Focus:
– $500M R&D reinvestment by 2026 targets AI for Productivity
– Part of $7.7B cost savings
– Ethical AI, diverse DBs
– Global biotech advances: China’s AI in CRISPR
AI Initiatives in Big Pharma @Grok prompt & Proprietary Training Data and Inference by LPBI Group’s IP Asset Class III: 100 e-Proceedings and 50 Tweet Collections of Top Biotech and Medical Global Conferences, 2013-2025
AI Initiatives in Big Pharma @Grok prompt & Proprietary Training Data and Inference by LPBI Group’s IP Asset Class III: 100 e-Proceedings and 50 Tweet Collections of Top Biotech and Medical Global Conferences, 2013-2025
Overview: Third in LPBI Group’s five-article series on AI-ready digital IP assets for Pharma companies. This installment highlights IP Asset Class III—100 e-proceedings and 50 tweet collections from top global biotech/medical conferences (2013-2025)—as a proprietary, expert-curated corpus of frontier science insights. Using a November 18, 2025, Grok prompt on Pfizer’s AI efforts, it maps these assets to pharma applications, stressing their role in training/inference for foundation models. Unlike prior classes (journal articles, e-books), this focuses on real-time event captures (e.g., speaker quotes, agendas) as unique, non-replicable data for efficiency, education, and branding in AI-driven R&D.
Main Thesis and Key Arguments
Core Idea: LPBI’s IP Asset Class III assets provide the “only written record” of +100 top conferences, with tweet collections as verbatim speaker quotes/affiliations—ideal for ingesting into AI platforms to amplify human expertise in combinatorial predictions. This supports Pfizer’s goals like 16,000-hour savings via generative AI, enabling subject-specific training (e.g., immunotherapy) and future agenda building.
Value Proposition: 150 total assets (100 e-proceedings + 50 tweet collections) form a live repository of domain knowledge, mapped to ontology for immediate AI use. Equivalent to $50MM value (aligned with series benchmarks); unique for branding (“make or break”) as no other source offers such curated event intel. Part of five AI-ready classes (I, II, III, V, X) for healthcare models.
Broader Context: Builds on series by emphasizing event-based data for partnerships/education; contrasts generic datasets with defensible, ethical expert interpretations for global equity (e.g., Pfizer’s AI Learning Lab).
AI Initiatives in Big Pharma (Focus on Pfizer)Reuses Grok prompt highlights, presented in a verbatim table:
Initiative Category
Description
Generative AI Tools
Save scientists up to 16,000 hours annually in literature searches and data analysis.
Drug Discovery Acceleration
Uses AI, supercomputing, and ML to streamline R&D timelines.
Charlie Platform; Scientific Data Cloud (AWS-powered ML on centralized data); Amazon’s SageMaker/Bedrock for Manufacturing efficiency; Pfizer Foundation’s AI Learning Lab for equitable access to care and community tools.
Partnerships & Education
IMI Big Picture (3M-sample disease database); AI in Pharma AIPM Symposium (Drug discovery and Precision Medicine); Webinars on AI for biomedical data integration; Webinar on AI in Manufacturing.
Strategic Focus
$500M R&D reinvestment by 2026 for AI productivity; Part of $7.7B cost savings; Ethical AI with diverse DBs; Global biotech advances (e.g., China’s AI in CRISPR).
Mapping to LPBI’s Proprietary DataCore alignment table (verbatim extraction, linking Pfizer initiatives to Class III assets):
Pfizer AI Initiative
Class III Alignment (100 e-Proceedings + 50 Tweet Collections)
Generative AI Tools (16,000 hours saved)
(No specific mapping.)
Drug Discovery Acceleration
e-Proceedings of +100 TOP Conferences in Biotech, Medicine, Genomics, Precision Medicine (2013-2025). Frontier of Science presented; ONLY written record of events. Tweet Collections: Speaker QUOTES on record (not elsewhere available by name/affiliation).
Generative AI & Operational Tools (Charlie, AWS, etc.)
Ingest ALL e-Proceedings into Charlie Platform. Apply GPT: Training Data—one conference at a time; OR All Conferences on ONE subject (e.g., Immunotherapy, Oncolytic Virus Immunotherapy, Immune Oncology).
Partnerships & Education (IMI, AIPM, webinars)
Use Past Agendas/Speakers Lists/Topics for: Employee Training & Leadership Development; Build Future Conference Agendas.
Strategic Focus ($500M reinvestment, ethics)
Access to +100 e-Proceedings vs. None = Make or Break in Branding.
Examples: Subject clusters like Immunotherapy; resources include conference lists (2013-present) and e-proceedings deliverables.Methodologies and Frameworks
AI Training Pipeline: Ingest proceedings/tweets into Charlie/AWS (e.g., SageMaker); GPT processing per conference or theme for pre-training/fine-tuning/inference. Use ontology for semantic mapping; tweets for quote-based evals.
Productivity Model: Enhances Pfizer’s savings ($7.7B total) via event intel for education/partnerships; ethical diverse data for global grants (e.g., CRISPR AI).
Insights: Quote from Dr. Stephen J. Williams, PhD: Emphasizes strategic branding via access. Predicts revolution in AI education/leadership from historical agendas.
Conclusions and Implications
LPBI Group’s IP Asset Class III assets are “rare, defensible” for Big Pharma AI, powering from R&D acceleration to equitable care. Technical Implications: Enables theme-specific models (e.g., oncology conferences) for diagnostics/trials. Business Implications: Boosts ROI on $500M investments; licensing for symposia/webinars. Unique Insight: As the sole record of speaker insights, these outpace public data for “frontier” inference—key in series for holistic pharma AI moats.Promotional with resource links (e.g., IP portfolio, biotech conference lists). Complements prior pieces by adding temporal/event depth.
Let me know if you want series comparisons or dives into specific conferences!
China is Making Large Inroads into Biotech: Is Investment Money Following? Is US Investment Money Following the China Biotech Boom?
Curator:Stephen J. Williams, Ph.D.
UPDATED: 6/02/2026
From ASCO 2026, a Chinese biotech, Akeso, shows amazing clinical results for lung cancer with its bispecific PD-L1-VEGF bispecific antibody. As more and more US companies are making deals with Chinese biotechs is drug discovery and the biotech world heading far East?
In 2017, a little-known company called Nanjing Legend Biotech walked into the ASCO annual meeting at the 11th hour, dropped a 100% objective response rate for a cell therapy that would become Carvykti, and single-handedly put Chinese biotech on the global map. Nearly a decade later, taking the center stage at ASCO 2026, Akeso’s ivonescimab made history by offering the first-ever Chinese data set to command a coveted spot on the plenary session.
“We see a lot of sophistication and skill in Chinese companies,” Marjorie Green, M.D., head of oncology global clinical development at Merck Research Laboratories, said in an interview.
The maturity of the Chinese biopharma industry is evidenced by the deals it has signed. Through a potential $3.3 billion pact in 2024, Merck secured global rights to a PD-1xVEGF competitor to ivonescimab from China’s LaNova Medicines. The following year, LaNova was acquired by Sino Biopharm in the first full acquisition of an innovative Chinese biotech by a domestic large pharma on record, a milestone widely viewed as evidence of an increasingly mature ecosystem. At ASCO, ivonescimab’s landmark overall survival (OS) win against a PD-1 inhibitor in their respective combinations with chemotherapy in first-line squamous non-small cell lung cancer (NSCLC) was just one of numerous studies of Chinese assets that reshaped the conference’s narrative. In what Natalie Vokes, M.D., of the MD Anderson Cancer Center said “could well be a practice-changing study” if validated in a global trial, Kelun-Biotech’s TROP2 antibody-drug conjugate, sac-TMT, paired with Keytruda, slashed the risk of progression or death by 65% versus Keytruda alone in Chinese patients with previously untreated PD-L1-positive NSCLC. That drug, too, also has been swept into the Merck universe.
Bristol Myers Squibb this week announced a partnership with Hengrui Pharma to develop drugs together.
American and European biopharmaceutical companies are increasingly looking to China for their next blockbusters.
Bristol’s deal is unique because the U.S. drugmaker will send several experimental drugs to China for early testing.
Bristol Myers Squibb on Tuesday announced the potential multibillion-dollar partnership with one of China’s top drugmakers, Hengrui Pharma. The companies will work together to develop about a dozen drugs, including four that Bristol discovered and will send to China for Hengrui to run the early-stage clinical trials. The pair of companies will also collaborate to discover new drugs.
“It’s a huge signal,” said Michael Baran, head of private investments at healthcare-focused hedge fund Affinity Asset Advisors and a former partner at Pfizer Ventures.
He said U.S. drugmakers have partnered with Chinese companies to develop drugs before, including Amgen’s 2019 collaboration with BeOne. But Bristol’s deal is significant because it is more reciprocal, he said. It raises the prospect that more U.S. drugmakers could increasingly carry out early drug development in China as they try to bring treatments to market more quickly, and that Chinese companies could start to become global powerhouses.
By John George – Senior Reporter, Philadelphia Business Journal
Updated
Conshohocken firm enters into potential $4.5B deal to expand drug pipeline
Madrigal Pharmaceuticals broadened its pipeline of drug candidates this week by entering into a global licensing agreement, potentially valued at more than $4 billion, for six experimental therapy programs. Under the terms of the deal, Madrigal (NASDAQ: MDGL) agreed to pay Suzhou Ribo Life Science Co. Ltd. of China and its subsidiary Ribocure Pharmaceuticals AB $60 million upfront. Ribo could also receive up to $4.4 billion in development, regulatory and commercial milestone payments based on the programs achieving a series of unspecified goals. Conshohocken-based Madrigal has one product in the market, Rezdiffra, a treatment for the serious liver disease metabolic dysfunction-associated steatohepatitis (MASH). A common route for raising capital or exit strategy for many US biotechs has been strategic transfer or sale of intellectual property (IP) or strategic partnership with large pharmaceutical companies looking to acquire new biotechnologies or expand their own pipelines. Most US based biotechs had enjoyed a favorable (although not fully exclusive) deal-making environment with US pharmaceutical companies with some competition from international biotech companies. US government agencies such as FINRA, CFIUS, and the SEC closely monitored such international deals and the regulatory environment for such international deal making in the biotechnology space was tight. The company last July entered into a licensing agreement, valued at up to $2.1 billion, for an MASH drug being developed by CSPC Pharmaceutical Group Ltd of China.
Smaller Chinese biotechs have operated in the United States (at various biotech hubs around the country) and have usually set up as either service entities to the biotech industry as contract research organizations (Wuxi AppTech), developing research reagents for biotech (Sino Biological) or conducting research for purposes of transferring IP to a parent company in China. Most likely Chinese biotechs set up research operations because of the overabundance of biotech hubs in the United States, with a dearth of these innovation hubs in the China mainland.
However, as highlighted in the Next in Health Podcast Series from PriceWaterHouseCoopers (PwC), China has been rapidly been developing innovation hubs as well as biotech hubs. And Chinese biotech companies are staying home in mainly China and exporting their IP to major US pharmaceutical companies. As PwC notes this deal making between Chinese biotech in China and US pharmaceutical companies have rapidly expanded recently.
The following are notes from PriceWaterHouseCoopers (PwC) podcast entitled: Strategic Shifts: Navigating China’s Biotech Boom and Its Impact on US Pharma:
Tune in as Glenn Hunzinger, PwC’s Health Industries Leader and Roel van den Akker, PwC’s Pharma and Life Sciences Deals Leader discuss the rapid rise of China’s biotech industry and what it means for U.S. pharmaceutical companies. They discuss the evolving role of Chinese biotech in the global innovation landscape and share perspectives on how U.S. pharmaceutical companies can thoughtfully assess opportunities, manage cross-border complexities, and build effective partnering and diligence strategies.
Discussion highlights:
China’s biotech industry is growing fast and becoming a global player, with U.S. companies increasingly looking to partner with Chinese firms on cutting-edge science
U.S. pharma leaders are encouraged to move beyond skepticism and stay curious by building relationships, learning from local innovation, and exploring new partnership opportunities
Successfully partnering with Chinese biotech firms requires a careful and well-structured approach that accounts for global complexity, protects data and IP, and uses creative deal structures like new company formations to manage risk and stay flexible
U.S. companies need to be proactive in order to stay competitive by actively exploring global innovation, understanding the risks, and having a clear strategy to bring high-potential science to U.S. patients
In 2019 there were zero in licensing deals from China to US pharma…. Today one in five come from China.
China evolved into a expanding economy because China invested in biotech companies
Lots of skilled people
Built centers that rivaled biotech innovation centers in places like Boston, California Bay Area, and Philadelphia
China has gone from low cost manufacturing country to an innovative economy with great science coming out of it. US pharma boardrooms need to understand this
The analysts at PWC suggest to look at Data integrity, IP protection and risks before bringing China biotech IP in US. It is imperative that companies do ample due diligence.
China’s rise as a biotech innovation hub: 4 key strategic questions for US biopharma executives
May 08, 2025
Roel van den Akker; Partner, Pharmaceutical & Life Science Deals Leader, PwC
China’s biotech sector is evolving at breakneck speed — and the implications for US pharma are too significant to ignore. Over the past five years, China has transitioned from being a nice to watch market to a central pillar of global biopharma innovation. Today, one-third of in-licensed molecules at US pharma multinationals originate from China, up from virtually zero in 2019.
China’s biotech sector, however, is not monolithic or uniform. The ecosystem spans high-quality, globally competitive biotech hubs in cities like Hangzhou and Suzhou — home to companies producing first-in-class and novel innovations in ophthalmology, cardiovascular, and immunology — as well as a long tail of undercapitalized players where execution and capability gaps remain profound.
And now, Washington is paying attention, too. A recent report from the US National Security Commission on Emerging Biotechnology (NSCEB) highlighted China’s ambitions to dominate biotech as a “strategic priority” with dual-use implications across health and security. The report urges the US government and private sector to reassess dependencies and increase scrutiny of biotechnology partnerships abroad. For the US biopharma industry, this isn’t just a supply chain concern — it is a boardroom issue.
With the licensing market still skewed toward buyers, venture funding remaining depressed in China and IPO windows in Hong Kong slowly reopening, there is a compelling window for US companies to secure differentiated assets at relatively attractive terms. Speedy deal execution is increasingly important as the highest quality assets are being quickly scooped up. But navigating this terrain can require more than opportunism. It calls for deliberate strategy, structured governance and a nuanced geopolitical risk framework.
Here are four questions every US biopharma executive should be asking:
1. What is our posture toward preclinical and clinical science from China?
Are we approaching Chinese innovation with a default posture of skepticism or strategic curiosity? Many top-tier Chinese biotechs are now generating US-caliber data at the speed of light, particularly in therapeutic modalities such as mAbs, ADCs and T-cell engagers, but plenty still have execution gaps. Those that elect to lean in will likely need a deliberate eco-system approach geared towards being the partner of choice and local brand building.
2. What does our China diligence playbook look like?
In light of national security concerns, companies need a China-specific diligence framework — one that goes beyond the science. This includes scrutiny around data integrity, IP protection, export controls, and cross border data sharing.
3. What is our plan post-licensing or acquisition?
Ownership is just the start. US companies need a clear strategy for globalizing China-origin assets — from IND transfers to FDA filing to commercial launch. In some cases, that may require reworking the preclinical package or rebuilding the CMC infrastructure entirely. Increasingly, US (or Europe)-based “Newcos” may serve as geopolitical firewalls.
4. How can we preserve agility amid regulatory and political volatility?
With rising US-China tensions and new export control proposals under review, companies must future-proof deal structures. This could include regional carveouts, US-only development rights, or milestone-gated commitments. The NSCEB report makes clear: passive engagement is no longer tenable.
Innovation strategy meets national interest
The trendlines are clear: China is not just a manufacturing hub — it is an increasingly important source of global biotech innovation. But sourcing innovation from China now sits at the intersection of science, strategy and security. US pharma and biopharma companies can no longer afford to treat China engagement as tactical. Those who adopt a deliberate, resilient and agile China strategy — grounded in scientific rigor and geopolitical realism — likely lead in tomorrow’s innovation race.
A researcher prepares medicine at a laboratory in Nanjing University in Nanjing, Jiangsu province, April 29, 2011. REUTERS/Aly Song/File Photo Purchase Licensing Rights
U.S. drugmakers turn to Chinese companies as they face patent expirations
Licensing deals accelerate while traditional mergers decline
Chinese biotechs are challenging Western peers, analysts say
June 16 (Reuters) – U.S. drugmakers are licensing molecules from China for potential new medicines at an accelerating pace, according to new data, betting they can turn upfront payments of as little as $80 million into multibillion-dollar treatments.
Through June, U.S. drugmakers have signed 14 deals potentially worth $18.3 billion to license drugs from China-based companies. That compares with just two such deals in the year-earlier period, according to data from GlobalData provided exclusively to Reuters.
How to stop the shift of drug discovery from the U.S. to China. The FDA must make it easier to do such work in the U.S.
Scott GottliebMay 6, 2025
Five years ago, U.S. pharmaceutical companies didn’t license any new drugs from China. By 2024, one-third of their new compounds were coming from Chinese biotechnology firms.
Why are U.S. drugmakers sending their business to China? As in many other industries, it’s so much cheaper to synthesize new compounds inside Chinese biotechnology firms once a novel biological target has been discovered in American laboratories.
Yet the costs of developing new drugs in the U.S. needn’t be so high. They are driven up, in part, by increasing regulatory requirements that burden early-stage drug discovery in America. That’s especially true for Phase I clinical trials, in which drugs are tested in people for the first time.
Newsletter
The smartest thinkers in life sciences on what’s happening — and what’s to come
This shift of discovery work to China is going to accelerate if we don’t take deliberate steps to make it easier to do such work here in America. Yet the imperative to modernize early-stage drug development — to ensure that groundbreaking drug discovery remains in the U.S. rather than migrating to China — is colliding head-on with an impulse to slash the very government workforce capable of spearheading these reforms. These conflicting impulses have created a paradoxical tension: on one hand, the desire to stay competitive with China in biotechnology innovation, and on the other, a parallel campaign to reduce and in some cases dismantle the investments and institutions essential to achieving that goal.
In most cases, Chinese firms are not discovering new biological targets, nor are they crafting genuinely novel compounds to engage these targets through homegrown Chinese research. Instead, they piggyback on Western innovations by scouring U.S. patents, zeroing in on biological targets that are initially uncovered in American labs, and then developing “me too” drugs that replicate American-made compounds with only superficial tweaks, or producing “fast follower” drugs that capitalize on the original breakthroughs while refining key features to try to surpass U.S. innovation. Facing fewer regulations, the Chinese drugmakers can move more quickly than U.S. biotechnology companies — synthesizing copy-cat drugs based on our biological advances and then promptly moving these Chinese-made compounds into early-stage clinical trials, outpacing their American counterparts.
According to the investment bank Jefferies, large American drug companies spent more than $4.2 billion over the past year licensing or acquiring new compounds originally synthesized by Chinese firms. Many comprised advanced compounds such as antibody drugs and cell therapies — underscoring Chinese companies’ growing sophistication in adopting the latest American technologies. The cost of licensing these compounds from China, rather than synthesizing them in American labs, can be significantly lower. At a time when research funding in the U.S. is being cut, and research budgets are becoming painfully stretched, companies are looking to lower the cost of building their pipelines. In a fast-moving field such as oncology, this shift toward Chinese-synthesized compounds is particularly striking: I am told by someone inside the FDA process that nearly three-quarters of new small molecule cancer drugs submitted to the Food and Drug Administration for permission to begin U.S.-based clinical trials are initially made in China.
Usually, only a few months elapse between the moment a U.S. research team publishes a patent identifying a new biological target and when a biotechnology firm in China creates the corresponding drug that capitalizes on these findings. Because Chinese firms can synthesize new molecules at a fraction of the cost incurred by U.S. biotechnology companies — owing to a large and skilled but much cheaper workforce — they find the most intriguing biological targets pursued by Western researchers, rapidly churning out potent yet less expensive copycat molecules that they then market to Western companies.
A major challenge for U.S. firms is the long and costly process of obtaining FDA approval for Phase I studies, in which drugmakers test a new drug’s safety and tolerability in a small group of human volunteers. In China, launching this initial phase of clinical trials is far simpler, giving Chinese biotechnology companies a competitive advantage: By swiftly advancing their molecules into early-stage patient testing, Chinese firms can more readily determine which compounds hit their biological targets and show the greatest therapeutic promise. This allows the Chinese firms to quickly refine their molecules and then leapfrog their American counterparts, who are slowed by more cautious regulatory processes. While China’s regulatory process doesn’t uphold the patient safeguards that Americans rightly insist upon, the U.S. FDA could still streamline its path into early-stage drug development, bolstering America’s competitive edge without compromising patient safety.
In the U.S., one of the costliest early hurdles is the exhaustive animal testing that the FDA requires before a drug can be advanced into Phase I studies. These “pre-clinical” studies help safeguard patients, but the agency also uses this testing to weed out potential failures before a drug requires more intensive FDA scrutiny in later trials.
Over time, this regulatory framework has frontloaded a significant share of costs to the earliest phases of drug development, when biotechnology startups are often running on shoestring budgets, lack clinical data to attract investors, and can least afford delays. One measure of the increasing difficulty in securing the FDA’s permission for Phase I trials is the growing number of U.S. drugmakers who take compounds discovered on American soil and conduct these clinical trials in other Western markets, where they can obtain data more quickly and inexpensively before bringing it back to the FDA. One popular locale is Australia, where costs run about 60% lower than U.S.-based clinical trials, largely because the Australian government offers tax incentives to attract this kind of biomedical investment.
Many animal studies address esoteric questions about a drug’s long-term effects on parameters that may not be relevant to its eventual use — for example, at doses and durations of use that may be far beyond how patients will ultimately use the drug. The FDA’s preclinical testing protocols sometimes require American researchers to administer new compounds to animals at levels up to 500 times higher than any intended dose for patients, aiming for maximum animal exposure before human trials can begin. Where the FDA needs to screen for certain remote risks, many animal studies could be safely deferred until human trials confirm that a drug may benefit patients. At that point, it becomes easier for biotechnology companies to raise capital to fund these pro forma testing efforts.
To modernize the process, the FDA could tap into the wealth of data from existing drugs to establish a more phased approach to these requirements, where the amount of initial animal testing is more closely matched to a drug’s novelty and a better estimation of its perceived risks. It’s a prime opportunity to employ artificial intelligence — mining current data and extrapolating known information to newly discovered molecules. For new molecules that share structural similarities with established drugs, where a robust body of safety information already exists (and the likelihood of uncovering novel risks is judged to be minimal), some animal studies might simply be unnecessary. To establish a graduated approach to the scope of pre-clinical toxicology studies that the FDA requires for new molecules, Congress could revise the agency’s statutory framework, explicitly empowering it to adopt such flexible standards. It would also require targeted investments, enabling the FDA to craft the necessary tools and protocols to implement these refined methodologies.
Mice and even primates are often poor proxies for many of the remote toxicities the FDA is trying to test for, anyway. The agency can also make a more concerted effort to adopt advanced technologies, like pieces of human organs embedded in chips that can be used to test for remote dangers a drug may pose to specific organs like the heart and liver. These tools can reliably screen for risks at a fraction of the time and cost. FDA Commissioner Marty Makary recently announced his intention to pursue a plan that would phase out animal studies in the preclinical evaluation of antibody drugs, shifting instead toward innovative technologies that assess toxicology without relying on live animals. This positive step requires the FDA to invest in new capabilities, and scientific staff that possess expertise in these novel domains.
But right now, that investment seems unlikely. The size and scientific scope of the FDA staff responsible for reviewing early-stage drug development — and evaluating data collected from animal studies — has failed to keep up with the increasing complexity and sheer volume of applications flooding into the agency to launch Phase I clinical trials. Now, the FDA has made deep staffing cuts, prompted by DOGE, that have specifically targeted scientific teams that would lead these essential reforms.
Adding to these woes, morale at the FDA has declined so markedly that many foresee a wave of voluntary resignations among clinical reviewers. By thinning the ranks of experts who tackle novel scientific questions and resolve issues that span across different drug development programs — especially the elimination of the policy office within the FDA’s Office of New Drugs, which adjudicated these kinds of cross-cutting scientific questions — the government has impeded the early dialogue with drug developers that often results in streamlining requirements for Phase I studies. Even more challenging, it weakens the staff’s ability to develop new guidance documents and put better review practices into place — reforms essential for lasting improvements to the preclinical review process.
Instead of strengthening America’s biotechnology ecosystem, such measures risk accelerating the migration of discovery activities to China, undermining innovation at home. When U.S. drugmakers license compounds from China, they divert funds that might otherwise bolster innovation hubs such as Boston’s Kendall Square or North Carolina’s Research Triangle. The U.S. biotechnology industry was the world’s envy, but if we’re not careful, every drug could be made in China.
Scott Gottlieb, M.D., is a senior fellow at the American Enterprise Institute and served as commissioner of the Food and Drug Administration from 2017 to 2019. He is a partner at the venture capital firm New Enterprise Associates and serves on the boards of directors of Pfizer Inc. and Illumina.
From FierceBiotech: US Biotech Companies are finding that foreign investments may put them in a precarious position for government funding
The Department of Health and Human Services is allegedly denying clinical trial funding for biotechs based on their ties to certain foreign investors, Fierce Biotech has learned.
At the BIO conference in Boston this week, Fierce spoke with a biotech executive who had their grant pulled, as well as an industry thought leader who backed up the claims about a change in the HHS’ funding approach.
“We’re in a situation where some of the companies are confused about their ability to take foreign investment,” said John Stanford, founder and executive director of Incubate, a nonprofit organization of biotech venture capital firms and patient advocacy groups designed to educate policymakers on life science investment and innovation.
“We’ve been hearing about SBIR grants canceled,” Stanford told Fierce in a separate interview at BIO. “Anecdotally, we’ve also heard it’s a lot more than China and it’s countries—Canada, Norway, the EU—that traditionally we think of as allies.”
“Again, that’s anecdotal,” he stressed. “But we would be very concerned [about] the idea that we won’t take Canadian investments or Japanese investments or EU-based investments.”
“We want foreign investors coming to U.S.-based companies to develop drugs for the world,” Stanford said. “That is a win-win-win.”
Back in February, President Donald Trump issued a memorandum titled the “America First Investment Policy” that aims to restrict both inbound and outbound investments related to “foreign adversaries” in certain strategic industries. The document lacks specifics but puts China front and center while mentioning both healthcare and biotech among the sectors it will regulate.
And the investment analysis firm Jeffries noted that
Looking at financial data from FactSet, Jefferies analysts found biotech funding in May 2025 was down 57%, to just over $2.7 billion, compared to the same time last year. That sum was only slightly better than the nearly $2.6 billion raised in April — the worst haul in three years — and was also 44% lower than the average seen across the past 12 months.
Today, total capital remains relatively steady, but it’s flowing differently.
Fewer companies are commanding a greater share of investment, and a new global map of biotech leadership is emerging—one where Israel, Italy, Korea, Saudi Arabia, and NAME are not just participants but strategic innovators and investors in the space.
While some correction was inevitable after the pandemic’s urgency subsided, the sector’s foundation had already changed.
CROs didn’t scale down; they doubled down, offering sponsors the flexibility to develop therapies without taking on the full weight of manufacturing and trials in-house.
This shift underpinned a new era of capital efficiency and strategic outsourcing, which is strongly influenced by new smart technologies that generate code and content at a blink of an eye and refine research protocols.
Selective but Strong: The New Capital Math
After the surge of 2020–2021, a funding correction began in late 2022.
According to Jefferies, biotech funding in May 2025 was down 57 per cent year-over-year, dropping to roughly $2.7 billion.
Public markets also cooled. In 2023, biotech IPOs hit their lowest numbers in a decade, and follow-on offerings became increasingly rare.
This deceleration prompted talk of a “biotech winter.” Yet key indicators suggest a market in transition rather than decline. Private equity and venture capital remain active but are more selective.
While early-stage companies face greater hurdles, late-stage biotechs and those with de-risked clinical programs continue to attract significant funding.
Follow the Late-Stage Money
A recent GlobalData report underscores this trend: late-stage biotech companies now receive nearly double the capital of their earlier-stage counterparts.
Median venture rounds for Phase III companies have climbed to $62.5 million, as investors increasingly prioritise assets with regulatory clarity and near-term commercialisation potential.
The post-COVID period has revealed an important funding shift: fewer biotech companies are securing a larger percentage of available capital.
In an environment of macroeconomic uncertainty, geopolitical risk, and rising interest rates, investors are retreating from speculative bets and doubling down on known quantities.
From Gemini: Is US biotech investment going overseas in 2025? Plot in a bar graph the US biotech investment versus worldwide biotech investment by country
Is US biotech investment going overseas in 2025? Plot in a bar graph the US biotech investment versus worldwide biotech investment by country
Yes the US has many more venture capital firms focused on Biotech investment but it is appearing that investment is not staying in the US.
The global biotech funding landscape in 2023: U.S. leads while Europe and China make strides
In 2023, the U.S. continued to demonstrate its position as the biotech funding leader, commanding over one-third, 35%, of the global investment in the sector. Overall, U.S. biotech firms attracted $56.79 billion in funding, according to a survey of Crunchbase data. Next in line was China, which contributed about 12.7% to the global funding pool, or $20.61 billion. Up next was Europe, which secured more than $11.46 billion and representing more than 7% of the worldwide funding.
While U.S. leads in total biotech funding, Chinese biotech companies, on average, saw larger funding rounds than either Europe or the U.S. The average funding size per company in China was roughly three times larger than that in the U.S. and six times larger than the average in Europe.
But while China-based companies had larger hauls, they were comparatively few. Chinese biotech secured in cumulative $20.61 billion among just 69 firms, with roughly $299 million in funding per company on average. Meanwhile, the 229 European biotech firms that won funding in the past year attracted $11.46 billion in funding, averaging $50 million each. In comparison, the 583 U.S. biotech companies with recent funding attracted $56.79 billion, averaging $97 million per company.
The map below represents the total biotech funding amount in USD across the globe. Funding amounts are shaded based on companies’ cumulative funding totals and density. Darker shades indicate higher funding amounts and density, with the U.S. hubs on the East and West Coasts showing the darkest shade, reflecting the combination of total funding and density.
Size of the US Biotech Market is expected to grow from half a billion in 2023 to 1.7 trillion in 2033. Source novaoneadvisor.com
Biotech investment trends in the US for 2025 present a complex picture. While some reports indicate a general slowdown in venture funding for biotech startups and concerns about tariff impacts, other sources suggest resilience within the sector, with strong revenue growth for public biotech companies in both the US and Europe in 2024, expected to continue into 2025. Additionally, there are calls for significant investment within the US to maintain its leadership in biotechnology. Therefore, it is not definitively clear that US biotech investment is predominantly going overseas in 2025, but rather navigating a challenging and evolving landscape.
Regarding US biotech investment versus worldwide biotech investment by country, here is a bar graph of key biotech investment moves in 2025 based on available data. Please note that this data reflects “key moves” in biotech funding for 2025 as reported by Labiotech, and a comprehensive worldwide investment breakdown for all countries was not available.
From Franklin Templeton: China is Emerging as a Global Biotechnology Player
The combined value of China’s outside licensing deals reached around US$46 billion in 2024, up from US$38 billion in 2023 and US$28 billion in 2022, according to data provider NextPharma. Meanwhile, the number of global companies licensing into China has decreased across the same period. These tailwinds have helped China expand its share of global drug development to nearly 30% compared to 48% for the United States, according to data provider Citeline. Strong IP protection has positioned China to receive global investment, with a 2024 policy encouraging more IP collaboration between global and Chinese companies. US investment bank Stifel projects that molecules licensed by large pharmaceutical firms from China will increase to 37% in 2025. This shift has been largely driven by US companies seeking cheaper drug development alternatives and has led to R&D spending in China outpacing that of the United States.
A Closer Look at the Financials and Comparison between China and US Biotech Investment Trends
This rapid growth of Chinese biopharma was predictable back in 2018 as this article from an investment newsletter suggests:
November 10, 2022Posted by China BriefingWritten by Yi WuReading Time: 5 minutes
Biopharma, short for biopharmaceuticals, are medical products produced using biotechnology (or biotech). Typical biopharma products include pharmaceuticals generated from living organisms, vaccines, gene therapy, etc.
An important subsector of biotech, China’s biopharma industry has much attention home and abroad, especially after Chinese companies developed multiple COVID-19 vaccines now in wide circulation. Market capitalization of Chinese biopharma companies grew to over US$200 billion in 2020 from US$1 billion in 2016.
With China’s rapidly aging population and a growing affluent middle-class, the country’s biopharma industry presents challenging but compelling opportunities to investors.
In this article, we discuss the market size, growth drivers, and global competition facing China’s biopharma industry and suggest potential investment paths.
How big is China’s biopharma market?
Biopharmaceuticals in China is a lucrative business, with significant domestic demand due to an aging population and expanding household budgets for quality products and services as people’s living standards improve.
China’s healthcare market is predicted to expand from around US$900 billion (RMB 6.47 trillion) in 2019 to US$2.3 trillion (RMB 16.53 trillion) in 2030, and its market size is second to only the US. China’s total expenditure on healthcare as a component of its GDP increased to 5.35 percent in 2019 from 4.23 percent in 2010.
What are the growth drivers for China’s biopharma industry?
The broader biotech sector is a main focus of the Chinese government’s “Made in China 2025” strategy. The country needs a steady biopharmaceutical industry to address its healthcare needs and to build an internationally competitive and innovative pharmaceutical industry as part of wider economic restructuring. Under the same momentum, on January 30, 2022, nine agencies jointly issued the “14th Five-year Plan for the Development of the Pharmaceuticals Industry” as a guiding document that clarifies the goals and directions for China’s pharmaceutical industry development in the next five years.
Now let’s compare the size of the US biotech market: You can see the US biotech valuation is now similar to the estimated market capitalization of the China market.
The U.S. biotechnology market size was valued at USD 621.55 billion in 2024 and is projected to reach USD 1,794.11 billion by 2033, registering a CAGR of 12.5% from 2024 to 2033. Ongoing government initiatives are the key factors driving the growth of the market. Also, improving approval processes coupled with the favorable reimbursement policies can fuel market growth further.
Key Takeaways:
DNA sequencing dominated this market and held the highest revenue market share of 18% in 2023
The others’ segment is anticipated to grow at the fastest CAGR of 28.1% during the forecast period.
The health segment dominated the market and accounted for the largest revenue market share of 44.13% in 2023.
Bioinformatics is expected to witness the fastest growth, with a CAGR of 17.2% during the forecast period.
The U.S. biotechnology market is witnessing major growth contributed by the increasing adoption and applications of biotechnology in many industries like pharmaceuticals, agriculture, food production, environmental conservation, and energy. In addition, market players in the industry are increasingly focusing on innovations across many fields such as energy, medicine, and materials science using biological processes to overcome challenges and fuel technological advancements. Also, in recent years there has been a notable surge in the utilization of biotechnological methods including DNA fingerprinting, stem cell technology, and genetic engineering propelling the market expansion soon.
‘The bar has risen’: China’s biotech gains push US companies to adapt
A fast-improving pipeline of drugs invented in China is attracting pharma dealmakers, putting pressure on U.S. biotechs and the VC firms that back them.
Soon after starting a new biotechnology company, David Li realized he needed to rethink his strategy.
Li had been conducting the competitive research biotech entrepreneurs typically undertake before soliciting investment. He drew up a list of drug targets that his startup, Meliora Therapeutics, could pursue and checked them against the potential competition.
Li quickly found that biotechs in China were already working on many of the targets he had on his list. Curious, he visited Shanghai and Suzhou and witnessed a buzzing scene of startups set frenetically to task.
“They’re not really thinking about the U.S. at all. They’re just trying to create more value and stay alive to differentiate themselves from the next guy in China,” he said. “They’re moving quick. There are a lot of them and they’re just quite competitive.”
Li’s experience is illustrative of a trend that could pressure biotech companies in the U.S. and alter their drug development strategies. More and more, large pharmaceutical companies are licensing experimental drugs from China. Venture companies are testing similar tactics by launching new U.S. startups around compounds sourced from China’s laboratories. This shift has been sudden, with licensing deals ramping rapidly over the past two years. And it is occurring even as the shadow of U.S.-China competition within biotech grows longer.
Executives and investors interviewed by BioPharma Dive at the J.P. Morgan Healthcare Conference this week share Li’s outlook. They expect such deals will accelerate and, in the process, force U.S. biotechs to work harder to stand out.
“We’ve been warning people for a while, we’re losing our edge,” said Paul Hastings, CEO of cell therapy maker Nkarta and former chair of the U.S. lobbying group the Biotechnology Innovation Organization. “Innovation is now showing up on our doorstep.”
There’s perhaps no clearer example of this than ivonescimab, a drug developed by China-based Akeso Therapeutics and licensed by U.S.-based Summit Therapeutics. Recent results from a lung cancer study run in China showed ivonescimab outperformed Keytruda, Merck’s dominant immunotherapy and currently the pharmaceutical industry’s most lucrative single product.
The finding “put a huge focus on what’s happening in China,” said Boris Zaïtra, head of business development at Roche, which sells a rival to Keytruda.
Fast-moving research
Today’s deal boom has roots in efforts by the Chinese government to upgrade the country’s biotech capabilities by upping investment in technological innovation. In the life sciences, the initiative provided funding, discounted or even free laboratory space and grants to support what Li described as a “robust ecosystem” of biotechs.
The results are clear. Places like Shanghai and Suzhou are home to a skilled workforce of scientists and hundreds of homegrown companies that employ them. Science parks akin to the U.S. biotech hubs of Cambridge, Massachusetts and San Francisco have sprouted up.
Chinese companies generally can move faster, and at a lower cost, than their U.S. counterparts. Startups can go from launch to clinical trials in 18 months or less, compared to a few years in the U.S., Li estimated. Clinical trial enrollment is speedy, while staffing and supply chain costs are lower, helping companies move drugs along more cost effectively.
“If you’re a national company within China running a trial, just by virtue of the networks that you work within, you pay a fraction of what we pay, and the access to patients is enough that you can go really fast,” said Andy Plump, head of research at Takeda Pharmaceutical. “All of those are enablers.”
And what they’ve enabled is a large and growing stockpile of drug prospects, many of which are designed as “me too better” versions of existing medicines, analysts at the investment bank Jefferies wrote in a December report. Initially focused in oncology, China-based companies are now churning out high-quality compounds across multiple therapeutic areas, including autoimmune conditions and obesity.
“There was a huge boom of investment in China, cost of capital was very low, and all these companies blew out huge pipelines,” said Alexis Borisy, a biotech investor and founder of venture capital firm Curie.Bio. ”Anything that anybody was doing in the biotech and pharmaceutical industry, you could probably find 10 to 50 versions of it across the China ecosystem.”
Me-toos become me-betters
For years now, Western biopharma executives have scouted the pipelines of China’s biotech laboratories — exploration that yielded a smattering of licensing deals and research collaborations. Borisy was among them, starting in 2020 a company called EQRx that sought to bring Chinese versions of already-approved drugs to the U.S. and sell them for less. EQRx’s plan backfired amid scrutiny by the U.S. Food and Drug Administration of medicines tested only in people from a single country.
Now, however, the pace of deals has accelerated rapidly. There are a few reasons for this. According to Plump, one is the improving quality of the drug compounds being developed. The “me toos” are becoming “me betters” that could surpass available therapies and earn significant revenue for companies — like BeiGene’s blood cancer drug Brukinsa, which, in new prescriptions for the treatment of leukemia, overtook two established medicines of the same type last year.
Another reason, Plump said, is that China-based companies are becoming more innovative, studying drug targets that might not have yet yielded marketed medicines, or for which the most advanced competition is in early testing. Li notes how Chinese companies are going after harder “engineering problems,” like making complex, multifunctional antibody drugs, or antibody-drug conjugates.
“There are so many [companies] that the new assets are going to keep coming,” Li said.
Much as in the U.S., China-based biotechs are also fighting for funding, pushing them to consider licensing deals with multinational pharma companies. At the same time, these pharmas are hunting for cheap medicines they can plug into their pipelines ahead of looming patent cliffs. The two trends are “colliding,” said Kristina Burow, a managing director with Arch Venture Partners. “I don’t see an end to that.”
The statistics bear Burow’s view out. According to Jefferies, the number and average value of deals for China-developed drugs reached record levels last year. Another report, from Stifel’s Tim Opler, showed that pharma companies now source about one-third of their in-licensed molecules from China, up from around 10% to 12% between 2020 and 2022.
“I see huge opportunities for us to partner and work together with Chinese companies,” said Plump, of Takeda.
“There’s been a lot of really good, high quality molecules and data that have emerged from China over the last couple of years,” said Robert Plenge, the head of research at Bristol Myers Squibb. “It’s also no longer just simply repeating what’s been done with the exact same type of molecule.”
Geopolitical risks
These deals are happening against an uncertain backdrop. The U.S. Congress has spent the last year or so kicking around iterations of the Biosecure Act, a bill that would restrict U.S. biotechs from working with certain China-based drug contractors. A committee in the House of Representatives is calling for new limits on clinical trials that involve Chinese military hospitals. And the incoming Trump administration has threatened tariffs that could ripple across industrial sectors.
“We don’t know what this new administration is going to do,” said Jon Norris, a managing director at HSBC Innovation Banking.
The Biosecure Act “keeps going sideways,” added Hastings, who believes that any impact from the legislation, if passed, would be minimal. Instead, Hastings wonders if future tariffs may be more problematic. “There will be tariffs on other goods coming from China. Does that include raw materials and innovation? It’s hard to imagine that it won’t,” he said.
But executives and investors expect deals to continue, meaning U.S. biotechs will have to do more to compete.
“U.S. companies will need to figure out what it is they’re able to bring to the table that others can’t,” said Burow, of Arch.
Borisy said startups working on first-of-their-kind drugs need to be more secretive than ever. “Do not publish. Do not present at a scientific meeting. Do not put out a poster. Try to make your initial patent filing as obtuse as possible,” he cautioned.
“The second that paper comes out, or poster at any scientific meeting, or talk or patent, assume it has launched a thousand ships.”
Those that are further along should assume companies in China will be quick on their heels with potentially superior drugs. “The day when you could come out with a bad molecule and open up a field is over,” he said.
Greater competition isn’t necessarily a bad thing, according to Neil Kumar, CEO of BridgeBio Pharma. Drug development could become more efficient as pharmas acquire medicines from a “cheaper” starting point and advance them more quickly.
Venture dollars could be directed towards newer ideas, rather than standing up a host of similar companies.“If all of a sudden this makes us less ‘lemming-like,’” Kumar said, “I have no problem with that.”
Li similarly argues that, going forward, U.S. companies need to focus on “novelty and innovation.” At his own company, Li is now working on things “we felt others were not able to access.”
“The game has always been the same. Bring something super differentiated to market,” he said. But “the bar has risen.”
Gwendolyn Wu and Jacob Bell contributed reporting.
Is Chinese Biotechs just Producing Me-Too Drugs or are they Innovating New Molecular Entities?
The following articles explain the areas in which Chinese Biotech is expanding and focused on.
However the sort answer and summary to the aforementioned question is: Definately Chinese Biotechs are innovating at a rapid pace, and new molecular entities and new classes of drugs are outpacing any copycat or mee-too generic drug development.
This article by Joe Renny on LinkedIn focuses on the degree of innovation in Chinese biotech companies. I put the article in mostly its entirety because Joe did an excellent analysis of China’s biotech industry.
Joe Renny: Strategic Growth Leader | Driving M&A, Pharma Partnerships & Innovation | Unlocking the Commercial Potential of Science | Biotech & Pharmaceuticals
China’s biotech sector is in the midst of a stunning surge – its stocks have skyrocketed over 60% this year (outpacing even China’s high-flying tech sector), and the country now has over 1,250 innovative drugs in development, nearly catching up with the U.S. pipeline of ~1,440. Once known mainly for generic manufacturing, China is rapidly emerging as a source of differentiated innovation. Global pharma giants have taken notice: major licensing deals are proliferating as Western drugmakers snap up Chinese-born therapies in fields like oncology, metabolic diseases (obesity/diabetes), and immunology. The excitement is palpable – but a critical question looms beneath the optimism: Can this wave of innovation meaningfully improve the pharmaceutical industry’s return on investment (ROI)? In other words, will China’s biotech boom fix the underlying economics of drug development, or are the same old ROI challenges here to stay?
From Copycats to Cutting-Edge: China’s Rapid Ascent in Biotech
In the past decade, China’s pharma landscape has transformed from copycat chemistry to cutting-edge biotech. The sheer scale of innovation is unprecedented. A recent analysis found China had over 1,250 novel drug candidates enter development in 2024, far surpassing the EU and nearly reaching U.S. levels. This is a remarkable jump from just a few years ago – back in 2015, China contributed only ~160 compounds globally. Reforms to streamline drug approvals and massive R&D investments (spurred by initiatives like Made in China 2025) have unleashed a boom led by returnee scientists and ambitious startups.
Importantly, the quality of Chinese innovation has leapt upward alongside quantity. Drugs originating in China are increasingly clearing high bars of efficacy and safety. The world’s strictest regulators, including the U.S. FDA and European EMA, have begun fast-tracking more Chinese-developed drugs with priority reviews and “breakthrough” designations. For example, a cell therapy for blood cancer developed by China’s Legend Biotech won FDA approval (marketed by Johnson & Johnson) and is considered superior to a rival U.S. therapy. Another China-origin drug – Akeso Inc.’s novel cancer antibody that outperformed Merck’s Keytruda in trials – triggered a global wave of interest and a $500 million licensing deal in 2022. In short, China is no longer just a low-cost manufacturing base; it’s producing world-class treatments that Big Pharma is eager to get its hands on.
This trend is also evident in the stock markets. After a four-year slump, Chinese biotech stocks have roared back, becoming one of Asia’s best-performing sectors in 2025. The Hang Seng Biotech Index in Hong Kong is up over 60% since January, vastly outperforming broader tech indices. Investors are excited by signals that China is becoming a true global hub for biopharma innovation. According to one analyst, “China biotech is now a disruptive force reshaping global drug innovation… The science is real, the economics are compelling, and the pipeline is starting to deliver”. All of this represents a fundamental shift in the industry’s centre of gravity – and perhaps a new source of competitive pressure on Western incumbents.
Western Pharma’s Response: Licensing Deals and Partnerships Accelerate
Global pharmaceutical companies aren’t standing on the sidelines – they’re rushing to collaborate with and invest in Chinese biotechs. In fact, U.S. and European drugmakers have dramatically stepped up licensing deals to tap China’s innovations. Through the first half of 2025 alone, U.S. companies signed 14 licensing agreements worth up to $18.3 billion for Chinese-origin drugs, a huge jump from just 2 such deals in the same period a year earlier. Many of these partnerships involve potential blockbusters in cancer, metabolic disorders, and other areas where Chinese R&D is making leaps.
Oncology: China has become a hotbed for cancer drug innovation, especially with advanced biologics like bispecific antibodies. In May 2025, Pfizer paid a record $1.25 billion upfront to license a PD-1/VEGF bispecific antibody from China’s 3SBio (a deal worth up to $6 billion with milestones). Weeks later, Bristol Myers Squibb struck an $11.5 billion alliance for a similar immunotherapy developed in China. Virtually every active clinical trial for certain cutting-edge cancer combos (like PD-1/VEGF drugs) now originates in China, making it a goldmine for Western firms seeking the next breakthrough. AstraZeneca, Merck, Novartis, and others have all scooped up Chinese cancer therapies in recent years as they cast their nets wider for innovation.
Metabolic & Obesity Drugs: Western pharma is also eyeing China’s contributions in metabolic diseases. Notably, Merck licensed a Chinese-developed GLP-1 oral drug (for diabetes/obesity) from Hansoh Pharma in late 2022 for up to $1.7 billion. And in 2025, Regeneron paid $80 million upfront (in a deal worth up to $2 billion) for rights to an experimental obesity drug from Hansoh. These deals underscore that Chinese labs are producing competitive candidates in the red-hot obesity/diabetes arena – an area of huge global market potential.
Autoimmune & Other Areas: While oncology leads, Chinese biotechs are also advancing novel therapies in immunology and autoimmune diseases. For example, multiple deals in 2024–25 have focused on inflammatory conditions and neurology, indicating breadth in China’s pipeline. As one industry banker observed, roughly one-third of all new assets licensed by large pharmas in 2024 originated from China, and this could rise to 40–50% in coming years. In other words, nearly half of Big Pharma’s in-licensed pipeline may soon be sourced from China – a radical change from a decade ago.
Underpinning this deal frenzy is a stark reversal of roles: China has shifted from mostly importing therapies to now exporting its homegrown innovations. Back in 2015, Chinese companies mainly signed “license-in” deals to bring foreign drugs to China. But by 2024, nearly half of China’s transactions were license-out deals, with Chinese firms granting global rights to their own drugs. In 2024 alone, Chinese biotechs out-licensed 94 novel projects to overseas partners, often at early clinical stages. This boom in outbound deals – especially for high-value cancer therapies (like ADCs and bispecific antibodies) – highlights China’s maturation as an innovation engine.
In a scientific paper published by Yan et al, the authors provided a comparative analysis between the US, EU, and China of new approved drugs from the years 2019- 2023.
Yan Y, Guo X, Li Z, Shi W, Long M, Yue X, Kong F, Zhao Z. New Drug Approvals in China: An International Comparative Analysis, 2019-2023. Drug Des Devel Ther. 2025 Apr 3;19:2629-2639. doi: 10.2147/DDDT.S514132.
In the paper, the authors retrieved approval data from from the National Medical Products Administration (NMPA), Food and Drug Administration (FDA), European Medicines Agency (EMA), and Pharmaceuticals and Medical Devices Agency (PMDA), including information on the generic name, trade name, applicants, target, approval date, drug type, approved indications, therapeutic area, the highest R&D status in China, and special approval status. The approval time gaps between China and other regions were calculated.
Results: Interestingly, China led with 256 new drug approvals, followed by the US (243 approvals), the EU (191 approvals), and Japan (187 approvals). Oncology, hematology, and infectiology were identified as the leading therapeutic areas globally and in China. Notably, PD-1 and EGFR inhibitors saw substantial approval, with 8 drugs each approved by the NMPA. China significantly reduced the approval timeline gap with the US and the EU since 2021, approving 15 first-in-class drugs during the study period.
The authors concluded, that despite the COVID-19 years, Chinese biotech has rapidly innovated in the biotech space and made up for the time gaps with increased research productivity.
Number of drug approvals by regulatory agency. Source: Yan Y, Guo X, Li Z, Shi W, Long M, Yue X, Kong F, Zhao Z. New Drug Approvals in China: An International Comparative Analysis, 2019-2023. Drug Des Devel Ther. 2025 Apr 3;19:2629-2639. doi: 10.2147/DDDT.S514132.
A comparison of drug approvals in US and China, as percentage of clinical use in various disease states. Source: Yan Y, Guo X, Li Z, Shi W, Long M, Yue X, Kong F, Zhao Z. New Drug Approvals in China: An International Comparative Analysis, 2019-2023. Drug Des Devel Ther. 2025 Apr 3;19:2629-2639. doi: 10.2147/DDDT.S514132.
China Biotech Innovation Hubs
The following was generated by Google AI
China has several prominent biotech innovation hubs, with the Yangtze River Delta region (including Shanghai, Suzhou, and Hangzhou) and Beijing being particularly strong. These regions leverage strong academic and research institutions, high R&D expenditures, and significant investment to foster a vibrant biotech ecosystem.
Here’s a closer look at some key hubs:
Yangtze River Delta:
Shanghai: A major hub with a focus on oncology, cell and gene therapy, and a strong track record of biotech IPOs. It’s home to the Zhangjiang Biotech and Pharmaceutical Base, known as China’s “Medicine Valley”.
Suzhou: Known for the BioBay industrial park, which houses numerous biotechnology and technology companies.
Hangzhou: Features a growing biotech sector, with companies like Hangzhou DAC Biotech.
Wuhan: Wuhan Biolake is a key player in areas like biomedicine, bio-agriculture, and bio-manufacturing.
Shenzhen: Features an innovation hub that supports synthetic biology startups and accelerates the commercialization of new biotech materials.
Key Factors Driving Growth:
Strong government support and investment: China has been actively promoting the growth of its biotech sector through various initiatives and funding programs.
High R&D expenditures: China is investing heavily in research and development, particularly in the tech, manufacturing, and biotech sectors.
Increasingly strong talent pool: China is producing a growing number of STEM graduates and globally recognized researchers.
AI and technology integration: AI is being applied to drug design and discovery, accelerating innovation.
Focus on specific areas: Different hubs are specializing in areas like oncology, regenerative medicine, and medical devices.
Overall, China’s biotech sector is experiencing rapid growth and is becoming a significant player in the global landscape, with these hubs leading the way.
Articles of Interest on International Biotech Venture Investment on the Open Access Scientific Journal Include:
Some Recent Challenging News from Gene Therapy Companies: Sarepta’s Gene Therapy Halted by FDA, Spark Therapeutics Program Gets a Realignment and Review from Roche
Curator: Stephen J.Williams, Ph.D.
Sarepta Therapeutics has received a order from the FDA to halt clinical trials on its Duchenne Muscular Dystrophy gene therapy Elevidys on July 18, 2025 following three deaths.
FDA Requests Sarepta Therapeutics Suspend Distribution of Elevidys and Places Clinical Trials on Hold for Multiple Gene Therapy Products Following 3 Deaths
For Immediate Release:
July 18, 2025
The U.S. Food and Drug Administration today announced it has placed Sarepta Therapeutics investigational gene therapy clinical trials for limb girdle muscular dystrophy on clinical hold following three deaths potentially related to these products and new safety concerns that the study participants are or would be exposed to an unreasonable and significant risk of illness or injury. The FDA has also revoked Sarepta’s platform technology designation.
The FDA leadership also met with Sarepta Therapeutics and requested it voluntarily stop all shipments of Elevidys today. The company refused to do so.
“Today, we’ve shown that this FDA takes swift action when patient safety is at risk.” said FDA Commissioner Marty Makary, M.D., M.P.H. “We believe in access to drugs for unmet medical needs but are not afraid to take immediate action when a serious safety signal emerges.”
The three deaths appear to have been a result of acute liver failure in individuals treated with Elevidys or investigational gene therapy using the same AAVrh74 serotype that is used in Elevidys. One of the fatalities occurred during a clinical trial conducted under an investigational new drug application for the treatment of Limb Girdle Muscular Dystrophy.
“Protecting patient safety is our highest priority, and the FDA will not allow products whose harms are greater than benefits. The FDA will halt any clinical trial of an investigational product if clinical trial participants would be exposed to an unreasonable and significant risk of illness or injury,” said Director of the FDA’s Center for Biologics Evaluation and Research Vinay Prasad, M.D., M.P.H.
Elevidys is an adeno-associated virus vector-based gene therapy using Sarepta Therapeutics, Inc.’s AAVrh74 Platform Technology for the treatment of Duchenne muscular dystrophy (DMD). It is designed to deliver into the body a gene that leads to production of Elevidys micro-dystrophin, a shortened protein (138 kDa, compared to the 427 kDa dystrophin protein of normal muscle cells) that contains selected domains of the dystrophin protein present in normal muscle cells. The product is administered as a single intravenous dose.
Duchenne muscular dystrophy is a rare and serious genetic condition which worsens over time, leading to weakness and wasting away of the body’s muscles. The disease occurs due to a defective gene that results in abnormalities in, or absence of, dystrophin, a protein that helps keep the body’s muscle cells intact.
Further, today, the FDA revoked the platform technology designation for Sarepta’s AAVrh74 Platform Technology because, among other things, given the new safety information, the preliminary evidence is insufficient to demonstrate that AAVrh74 Platform Technology has the potential to be incorporated in, or utilized by, more than one drug without an adverse effect on safety.
Elevidys received traditional approval for use in ambulatory DMD patients 4 years of age and older with a confirmed mutation in the DMD gene on June 20, 2024. It was approved for non-ambulatory patients on June 22, 2023 under the accelerated approval pathway. This pathway can allow earlier approval based on an effect on a surrogate endpoint or intermediate clinical endpoint that is reasonably likely to predict clinical benefit, while the company conducts confirmatory studies to verify the predicted clinical benefit. Continued approval for non-ambulatory patients is contingent upon verification and description of clinical benefit in a confirmatory trial. Given the new safety information, The FDA has notified the company that the indication should be restricted to use in ambulatory patients. The FDA is committed to further investigating the safety of the product in ambulatory patients and will take additional steps to protect patients as needed.
On July 18 Sarepta appeared to be disregarding the FDA release (according to the New York Times)
In a remarkable public dispute between drugmaker and regulator, the biotech company Sarepta Therapeutics is defying the Food and Drug Administration’s request that it halt distribution of its treatment for a deadly muscle-wasting disease.
In a news release on Friday evening, the agency said that it requested that the company voluntarily stop all shipments of the therapy, known as Elevidys, citing the deaths of three patients from liver failure who had taken the product or a similar therapy.
In its own news release later on Friday evening, Sarepta, which is based in Cambridge, Mass., said that it would continue to ship the treatment for patients who do not use wheelchairs. The company said its analysis showed no new safety problems in those patients and that it was committed to patient safety.
Dr. Marty Makary, the F.D.A. commissioner, said in the agency’s statement that its request to Sarepta demonstrated that the F.D.A. “takes swift action when patient safety is at risk.”
“We believe in access to drugs for unmet medical needs but are not afraid to take immediate action when a serious safety signal emerges,” he said.
In the past, the F.D.A. has sometimes asked companies to pause distribution of a drug until a new problem is better understood and mitigated. However, it can also press its case, and begin a process to revoke the drug’s license, which would begin with a formal notification and opportunity to respond and participate in a public hearing.
On July 21, 2025 Sarepta announces on their website in press release
Sarepta Therapeutics Announces Voluntary Pause of ELEVIDYS Shipments in the U.S.
07/21/25 7:40 PM EDT
CAMBRIDGE, Mass.–(BUSINESS WIRE)–Jul. 21, 2025– Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today issued the following statement:
Today, Sarepta Therapeutics notified the U.S. Food and Drug Administration (FDA) of its decision to voluntarily and temporarily pause all shipments of ELEVIDYS (delandistrogene moxeparvovec) for Duchenne muscular dystrophy in the United States, effective close of business Tuesday, July 22, 2025.
This proactive step will allow Sarepta the necessary time to respond toany requests for information and allow Sarepta and FDA to complete the ELEVIDYS safety labeling supplement process. The Company looks forward to a collaborative, science-driven review process and dialogue with the FDA.
“As a patient-centric organization, the decision to voluntarily and temporarily pause shipments of ELEVIDYS was a painful one, as individuals with Duchenne are losing muscle daily and in need of disease-modifying options,” said Doug Ingram, chief executive officer, Sarepta. “It is important for the patients we serve that Sarepta maintains a productive and positive working relationship with FDA, and it became obvious that maintaining that productive working relationship required this temporary suspension while we address any questions that FDA may have and complete the ELEVIDYS label supplement process.”
Sarepta remains committed to transparency and patient safety and will continue to provide timely updates to patients, families, healthcare providers, and the broader Duchenne community as additional information becomes available.
About ELEVIDYS (delandistrogene moxeparvovec-rokl) ELEVIDYS (delandistrogene moxeparvovec-rokl) is a single-dose, adeno-associated virus (AAV)-based gene transfer therapy for intravenous infusion designed to address the underlying genetic cause of Duchenne muscular dystrophy – mutations or changes in the DMD gene that result in the lack of dystrophin protein – through the delivery of a transgene that codes for the targeted production of ELEVIDYS micro-dystrophin in skeletal muscle.
ELEVIDYS is indicated for the treatment of Duchenne muscular dystrophy (DMD) in individuals at least 4 years of age.
For patients who are ambulatory and have a confirmed mutation in the DMD gene
For patients who are non-ambulatory and have a confirmed mutation in the DMD gene.
However this is not the first time Sarepta has been in the hot seat…
Read this interesting article from Derrick Lowe of Science. I will put it in its entirety as Derick Lowe really writes some great articles in his blog.
I really, really wish that I were not writing about Sarepta again. But here we are. Perhaps a quick review will explain my reluctance.
Back in 2013, the company was trying to get approval for an unusual “exon skipping” molecule (eteplirsen) as a therapy for Duchenne muscular dystropy. Nothing wrong with that – in fact, there’s a lot that’s right with that, since Duchenne is a perfect “unmet medical need” situation, and the exon-skipping idea was an innovative approach ten years ago (and it’s still not exactly a standard-issue therapy). Attacking very hard-to-treat diseases with new mechanisms of action is just what we’re supposed to be doing in this business.
The approval, though, was having trouble for some very good reasons. Sarepta’s trial was very, very small and the FDA later found that their trial design was very, very flawed. But in 2016 eteplirsen was suddenly approved, to the surprise of many observers (including me). A few years later, a follow-up drug (golodirsen) from the company (golodirsen) was also rejected by the FDA (with a Complete Response Letter) but then was later suddenly approved, although no new data had been presented. That was particularly mystifying since the eventually-published CRL detailed a number of real problems with eteplirsen since its approval, problems that looked to be possibly even greater with the follow-up drug. To the best of my knowledge, the confirmatory Phase III trial that was required at the time of golodirsen’s approval is still going on and is expected to read out next year. In 2021, another Sarepta exon-skipping drug (different exon this time) was approved (casimirsen) on the basis of biomarker levels that were expected to show eventual clinical benefit, and I believe that its confirmatory trial is part of the golodirsen one. That one at least did not go through the first-rejected-then-approved pathway.
More recently the company has been working on an outright gene therapy (elevidys) for Duchenne, and the initial results were quite promising. The company got accelerated FDA approval for that one last June for 4- and 5-year-old patients, even though actual clinical benefit had not yet been established. But gene therapy is a winding road, and last October the Phase III results for Elevidys were a complete miss in the primary endpoint. Arguing commenced, with the company saying that the results in the secondary endpoints showed that the drug was “modifying the trajectory” of the disease, and the CEO called the results a “massive win” and said that the company would use them to ask for a much wider label approval from the FDA. Apparently during the conference call, when he was asked about why he was so confident, he said that the FDA’s CBER head Peter Marks was “very supportive”. (It should be noted that since then another Duchenne gene therapy effort, this one from Pfizer, also failed its Phase III, so it’s not like this is a straightforward area).
Boy, was that the truth. The agency has just granted that use expansion, and it turns out that it was all due to Peter Marks, who completely overruled three review teams and two of his highest-level staffers (all of whom said that Sarepta had not proven its case). Honestly, I’m starting to wonder why any of us go to all this trouble. It appears that all you need is a friend high up in the agency and your clinical failures just aren’t an issue any more. Review committees aren’t convinced? Statisticians don’t buy your arguments? Who cares! Peter Marks is here to deliver hot, steaming takeout containers full of Hope.
Back in 2016, when eteplirsen first came up for its advisory committee vote, I wrote that there was a matrix of possible votes and interpretations, which I summed up this way:
(1) A negative vote, which is a rejection of the potential of the drug, the suffering of DMD patients, and their right to try a therapy which apparently does no harm, for a disease that has no other options.
(2) A negative vote, which is the only possible one, considering that the company’s trial data are far too sparse and unconvincing to allow a recommendation to approve the drug. If this gets recommended, what doesn’t? Why do we require new drugs to show efficacy at all?
(3) A positive vote, which is a victory for patient advocates everywhere, and in particular for the extremely ill boys who suffer from this disease, or. . .
(4) A positive vote, which marks an undeserved and potentially hazardous victory of emotional rhetoric and relentless patient advocacy over the scientific and medical evidence.
As I’ve said many times since, including just a few days ago, I believe that the FDA is tilting very, very noticeably towards #4 while proclaiming the wonderful new world of #3. And while I realize that this may make me sound like a heartless SOB, I think this is a huge mistake that we will be paying for for a long time.
Note that there has been reported deaths in 2024.
The following was from some data published in Nature in 2025 from Clinical Trial ClinicalTrials.gov: NCT05096221.
Mendell JR, Muntoni F, McDonald CM, Mercuri EM, Ciafaloni E, Komaki H, Leon-Astudillo C, Nascimento A, Proud C, Schara-Schmidt U, Veerapandiyan A, Zaidman CM, Guridi M, Murphy AP, Reid C, Wandel C, Asher DR, Darton E, Mason S, Potter RA, Singh T, Zhang W, Fontoura P, Elkins JS, Rodino-Klapac LR. AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial.Nat Med. 2025 Jan;31(1):332-341. doi: 10.1038/s41591-024-03304-z
Abstract
Duchenne muscular dystrophy (DMD) is a rare, X-linked neuromuscular disease caused by pathogenic variants in the DMD gene that result in the absence of functional dystrophin, beginning at birth and leading to progressive impaired motor function, loss of ambulation and life-threatening cardiorespiratory complications. Delandistrogene moxeparvovec, an adeno-associated rh74-viral vector-based gene therapy, addresses absent functional dystrophin in DMD. Here the phase 3 EMBARK study aimed to assess the efficacy and safety of delandistrogene moxeparvovec in patients with DMD. Ambulatory males with DMD, ≥4 years to <8 years of age, were randomized and stratified by age group and North Star Ambulatory Assessment (NSAA) score to single-administration intravenous delandistrogene moxeparvovec (1.33 × 1014 vector genomes per kilogram; n = 63) or placebo (n = 62). At week 52, the primary endpoint, change from baseline in NSAA score, was not met (least squares mean 2.57 (delandistrogene moxeparvovec) versus 1.92 (placebo) points; between-group difference, 0.65; 95% confidence interval (CI), -0.45, 1.74; P = 0.2441). Secondary efficacy endpoints included mean micro-dystrophin expression at week 12: 34.29% (treated) versus 0.00% (placebo). Other secondary efficacy endpoints at week 52 (between-group differences (95% CI)) included: Time to Rise (-0.64 (-1.06, -0.23)), 10-meter Walk/Run (-0.42 (-0.71, -0.13)), stride velocity 95th centile (0.10 (0.00, 0.19)), 100-meter Walk/Run (-3.29 (-8.28, 1.70)), time to ascend 4 steps (-0.36 (-0.71, -0.01)), PROMIS Mobility and Upper Extremity (0.05 (-0.08, 0.19); -0.04 (-0.24, 0.17)) and number of NSAA skills gained/improved (0.19 (-0.67, 1.06)). In total, 674 adverse events were recorded with delandistrogene moxeparvovec and 514 with placebo. There were no deaths, discontinuations or clinically significant complement-mediated adverse events; 7 patients (11.1%) experienced 10 treatment-related serious adverse events. Delandistrogene moxeparvovec did not lead to a significant improvement in NSAA score at week 52. Some of the secondary endpoints numerically favored treatment, although no statistical significance can be claimed. Safety was manageable and consistent with previous delandistrogene moxeparvovec trials.
As noted in the adobe abstract everything seemed to fine as reported in this trial.
However there was a report of an immunoloically related death in 2023:
For the first time, in June 2023, delandistrogene moxeparvovec (SRP-9001), a gene replacement therapy based on an adeno-associated virus (AAV) vector, was approved in the USA for children aged 4-5 years with DMD. Other promising gene therapies are in preclinical development or clinical trials, including CRISPR/Cas9-mediated strategies to restore dystrophin expression. Two deaths following DMD gene therapy with high-dose AAV vectors were attributed to AAV-mediated immune responses. The pre-existing disease underlying the therapy is most likely involved in the fatal AAV toxicity.
Now this may have been dose related as the patient was given a high dose.
DMD gene therapy death exposes risks of treating older patients
Cure Rare Disease plans to continue its programs with alternative vectors. (iStock / Getty Images Plus)
Cure Rare Disease has shared a deep dive into the death of the only participant in a gene therapy trial. The nonprofit and its collaborators tied the death of a patient with Duchenne muscular dystrophy (DMD) to an immune reaction to the viral vector, raising concerns about dosing older, more advanced people.
Commercial development of DMD gene therapies has focused on younger patients, with Sarepta Therapeutics limiting enrollment in its phase 3 trial to children aged 4 to 8 years old. The restrictive recruitment criteria have stopped many DMD patients from accessing gene therapies in clinical trials run by Sarepta and its rivals. The patient dosed in the Cure Rare Disease clinical trial was 27 years of age, and the therapy had been designed for him.
Last year, the nonprofit reported that the patient, who was the brother of its CEO, died after receiving the therapy. The death led to an investigation into what happened after the patient received the therapy, which was designed to use CRISPR transactivation to upregulate an alternate form of a key DMD protein.
Writing in preprint journal medRxiv (PDF), Cure Rare Disease described the findings of the investigation. A post-mortem showed injuries to the patient’s lungs, likely caused by a strong immune reaction to the high dose of the adeno-associated virus (AAV) vector that was given to try to ensure sufficient expression to achieve a therapeutic effect. There was minimal expression of the transgene in the liver.
At 1×1014 vg/kg, the studied dose was similar to that tested in other clinical trials but resulted in a higher vector genome load, a finding the researchers attributed to the patient’s lower lean muscle mass, 45%. The analysis suggests the patient had “a more severe innate immune reaction than others receiving similar or slightly higher doses of rAAV in microdystrophin gene therapy trials.”
Based on the finding, the researchers identified a need for more data on the characteristics that may predispose people to severe innate immune reactions and concluded “dose determination will remain a challenge for custom-designed AAV-mediated therapies, as by definition the precise therapeutic dose will not have been established.”
As for the application of CRISPR, the researchers said the toxicity and eventual death of the patient meant that an assessment of the safety and efficacy of the treatment was not possible.
AAV related clinical trials have been halted for drug-induced liver injury, predominantly due to severe immune reaction. In many cases it appears when high dose AAV therapy is used.
.10.015. Epub 2023 Oct 10. PMID: 37822079; PMCID: PMC10638066.
Abstract
High-dose systemic gene therapy with adeno-associated virus (AAV) is in clinical trials to treat various inherited diseases. Despite remarkable success in spinal muscular atrophy and promising results in other diseases, fatality has been observed due to liver, kidney, heart, or lung failure. Innate and adaptive immune responses to the vector play a critical role in the toxicity. Host factors also contribute to patient death. This mini-review summarizes clinical findings and calls for concerted efforts from all stakeholders to better understand the mechanisms underlying lethality in AAV gene therapy and to develop effective strategies to prevent/treat high-dose systemic AAV-gene-therapy-induced immunotoxicity.
Table 1.
Fatality cases following high-dose systemic AAV delivery
Roche Decides to Stop backing Sparks Therapeutics Hemophilia A Gene Therapy Program
In 2019, Roche acquired Children’s Hospital of Pennsylvania (CHOP) spinout Spark Therapeutics for $4.8 billion, one of the largest pharma acquisitions up to that time. It was reported on this site here
However as reported by Fierce Biotech (and updated above link) at https://www.fiercepharma.com/pharma/roche-overhauls-spark-gene-therapy-unit-recording-24b-full-impairment Roche will reorganize the company and deal, bringing in Spark into the corporate fold. However this meant massive layoffs and possibly either end of the gene therapy program in order to integrate it with Roche’s current programs. The Spark gene therapy has met with success so it will be interesting to see how Roche continues this program in the future.
However it has been a rough year for many gene therapies.
Other Articles in this Open Access Scientific Journol of Gene Therapy
Nearly half of the global population—and 80 percent of patients in therapeutic areas such as immunology—are women. Yet, treatments are frequently developed without tailored insights for female patients, often ignoring critical biological differences such as hormonal impacts, genetic factors, and cellular sex. Historically, women’s health has been narrowly defined through the lens of reproductive organs, while for non-reproductive conditions, women were treated as “small men.” This lack of focus on sex-specific biology has contributed to significant gaps in healthcare.
A recent analysis found that women spend 25 percent more of their lives in poor health compared with men due to the absence of sex-based treatments. Addressing this disparity could not only improve women’s quality of life but also unlock over $1 trillion in annual global GDP by 2040.
Four key factors contribute to the women’s health gap: limited understanding of sex-based biological differences, healthcare systems designed around male physiology, incomplete data that underestimates women’s disease burden, and chronic underfunding of female-focused research. For instance, despite women representing 78 percent of U.S. rheumatoid arthritis patients, only 7 percent of related NIH funding in 2019 targeted female-specific studies.
However, change is happening. Companies have demonstrated how targeted R&D can drive better outcomes for women. These therapies achieved expanded FDA approvals after clinical trials revealed their unique benefits for female patients. Similarly, addressing sex-based treatment gaps in asthma, atrial fibrillation, and tuberculosis could prevent millions of disability-adjusted life years.
By closing the women’s health gap, biopharma companies can drive innovation, improve therapeutic outcomes, and build high-growth markets while addressing long-standing inequities. This untapped opportunity holds the potential to transform global health outcomes for women and create a more equitable future.
Clarivate was formed in 2016, following the acquisition of Thomson Reuters‘ Intellectual Property and Science business by Onex Corporation and Baring Private Equity Asia. Clarivate has acquired various companies since then, including, notably, ProQuest in 2021.
Clarivate (formerly CPA Global) was formerly the Intellectual Property and Science division of Thomson Reuters. Before 2008, it was known as Thomson Scientific. In 2016, Thomson Reuters struck a $3.55 billion deal in which they spun it off as an independent company, and sold it to private-equity firms Onex Corporation and Baring Private Equity Asia.
June 1, 2017: Publons, a platform for researchers to share recognition for peer review.
April 10, 2018: Kopernio, AI-tech startup providing ability to search for full-text versions of selected scientific journal articles.
October 30, 2018: TrademarkVision, provider of Artificial Intelligence (AI) trademark research applications.
September 9, 2019: SequenceBase, provider of patent sequence information and search technology to the biotech, pharmaceutical and chemical industries.
December 2, 2019: Darts-ip, provider of case law data and analytics for intellectual property (IP) professionals.
January 17, 2020: Decision Resources Group (DRG), a leading healthcare research and consulting company, providing high-value healthcare industry analysis and insights.
June 22, 2020: CustomersFirst Now, in intellectual property (“IP”) software and tech-enabled services.
October 1, 2020: CPA Global, intellectual property (“IP”) software and tech-enabled services.
December 1, 2021: ProQuest, software, data and analytics provider to academic, research and national institutions.[27]It was acquired for $5.3 billion from Cambridge Information Group in what was described as a “huge deal in the library and information publishing world”. The company said that the operational concept behind the acquisition was integrating ProQuest’s products and applications with Web of Science. Chairman of ProQuest Andy Snyder became the vice chairman of Clarivate. The Scholarly Publishing and Academic Resources Coalition, an advocacy group for open access to scholarship, voiced antitrust concerns. The acquisition had been delayed mid-year due to a Federal Trade Commission antitrust probe.
We are a leading global information services and analytics company serving the scientific research, intellectual property and life sciences end-markets. We provide structured information and analytics to facilitate the discovery, protection and commercialization of scientific research, innovations and brands. Our product porfolio includes well-established market-leading brands such as Web of Science, Derwent Innovation, Life Sciences, CompuMark and MarkMonitor (which they later divested). We believe that the stron balue proposition of our content, user interfaces, visualization and analytical tools, combined with the integration of our products and services into customers’ daily workflows, leads to our substantial customer loyalty as evidenced by their willingness to renew subscriptions with us.
Our structure, enabling a sharp focus on cross-selling opportunities within markets, is comprised of two product groups:
Science Group: consists of Web of Science and Life Science Product Lines
Intellectual Property Group: consists of Derwent, CompuMark and MarkMonitor
Corporations, government agencies, universities, law firms depend on our high-value curated content, analytics and services. Unstructured data has grown exponentially over the last decade. The trend has resulted in a critical need for unstructured data to be meaningfully filtered, analyzed and curated into relvent information that facilitates key operational and strategic decision making. Our highly curated, proprietary information created through our sourcing, aggregation, verification, translation, and categorization (ONTOLOGY) of data has resulted in our solutions being embedded in our customers’ workflow and decision-making processes.
Overview of Clarivate PLC five year strategy in 2019. Note that in 2019 the Science Group accounted for 56.2% of revenue! This was driven by their product Cortellis!
Figure. Overview of Clarivate PLC five year strategy in 2019. Note that in 2019 the Science Group accounted for 56.2% of revenue! This was driven by their product Cortellis!
Also Note nowhere in the M&A Discussion in years before 2023 was anything mentioned concerning AI or Large Language Models.
The Clarivate of Today: Built for Life Sciences with Cortellis
Clarivate PLC has integrated multiple platforms into their offering Cortellis, which integrated AI and LLM into the structured knowledge bases (see more at https://clarivate.com/products/cortellis-family/)
“Life sciences organizations are tasked, now more than ever, to discover and develop treatments that challenge the status quo, increase ROI, and improve patient lives. However, its become increasingly difficult to find, integrate and analyze the key data your teams need to make critical decisions and get your Cortellis products to patients faster.
Cortellis Competitive Intelligence: maximize ROI and improve patient outcomes
Cortellis Deals Intelligence: Portfolio Strategy and Business Development (find best deal)
Cortellis Clinical Intelligence: Clinical Trial Support and Regulatory
Cortellis Digital Health Intelligence: understand digital health ecosystem
Cortellis Drug Discovery: improve drug development speed and efficiency
MetaBase and MetaCore: integrated omics knowledge bases for drug discovery
Cortellis Regulatory: help with filings
Cortellis HTA: health tech compliance (HIPAA)
CMC Intelligence: new drug marketing
Generics Intelligence
Drug Safety Intelligence: both preclinical safety and post marketing pharmacovigilence
Watch Videos on Cortellis for Drug Discovery
Watch Video on Qiagen Site to see how Cortellis Integrates with Qiagen Omics Platform IPA with Clarivate Meta Core to gain more insights into genomic and proteomic data
Understand complex ‘omics data to accelerate your research
Discover why QIAGEN Ingenuity Pathway Analysis (IPA) is the leading pathway analysis application among the life science research community and is cited in tens of thousands of articles for the analysis, integration and interpretation of data derived from ‘omics experiments. Such experiments include:
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With QIAGEN IPA you can predict downstream effects and identify new targets or candidate biomarkers. QIAGEN Ingenuity Pathway Analysis helps you perform insightful data analysis and interpretation to understand your experimental results within the context of various biological systems.
Articles Relevant to Drug Development, Natural Language Processing in Drug Development, and Clarivate on this Open Access Scientific Journal Include:
How to Create a Twitter Space for @pharma_BI for Live Broadcasts
Right now, Twitter Spaces are available on Android and iOS operating systems ONLY. For use on a PC desktop you must install an ANDROID EMULATOR. This means best to set up the Twitter Space using your PHONE APP not on the desktop or laptop computer. Right now, even though there is the ability to record a Twitter Space, that recording is not easily able to be embedded in WordPress as a tweet is (or chain of tweets). However you can download the recording (takes a day or two) and convert to mpeg using a program like Audacity to convert into an audio format conducible to WordPress.
A while ago I had put a post where I link to a Twitter Space I created for a class on Dissemination of Scientific Discoveries. The post
This online discussion was tweeted out and got a fair amount of impressions (60) as well as interactors (50).
About Twitter Spaces
Spaces is a way to have live audio conversations on Twitter. Anyone can join, listen, and speak in a Space on Twitter for iOS and Android. Currently you can listen in a Space on web.
The creator of a Space is the host. As a host on iOS, you can start a Space by long pressing on the Tweet Composer from your Home timeline and and then selecting the Spaces icon.
You can also start a Space by selecting the Spaces tab on the bottom of your timeline.
Step 2
Spaces are public, so anyone can join as a listener, including people who don’t follow you. Listeners can be directly invited into a Space by DMing them a link to the Space, Tweeting out a link, or sharing a link elsewhere.
Step 3
Up to 13 people (including the host and 2 co-hosts) can speak in a Space at any given time. When creating a new Space, you will see options to Name your Space and Start your Space.
Step 4
To schedule a Space, select Schedule for later. Choose the date and time you’d like your Space to go live.
Step 5
Once the Space has started, the host can send requests to listeners to become co-hosts or speakers by selecting the people icon and adding co-hosts or speakers, or selecting a person’s profile picture within a Space and adding them as a co-host or speaker. Listeners can request permission to speak from the host by selecting the Request icon below the microphone.
Step 6
When creating a Space, the host will join with their mic off and be the only speaker in the Space. When ready, select Start your Space.
Step 7
Allow mic access (speaking ability) to speakers by toggling Allow mic access to on.
Step 8
Get started chatting in your Space.
Step 9
As a host, make sure to Tweet out the link to your Space so other people can join. Select the icon to Share via a Tweet.
Spaces FAQ
Where is Spaces available?
Anyone can join, listen, and speak in a Space on Twitter for iOS and Android. Currently, starting a Space on web is not possible, but anyone can join and listen in a Space.
Who can start a Space?
People on Twitter for iOS and Android can start a Space.
Who can see my Space?
For now, all Spaces are public like Tweets, which means they can be accessed by anyone. They will automatically appear at the top of your Home timeline, and each Space has a link that can be shared publicly. Since Spaces are publicly accessible by anyone, it may be possible for people to listen to a Space without being listed as a guest in the Space.
We make certain information about Spaces available through the Twitter Developer Platform, such as the title of a Space, the hosts and speakers, and whether it is scheduled, in progress, or complete. For a more detailed list of the information about Spaces we make available via the Twitter API, check out our Spaces endpoints documentation.
Can other people see my presence while I am listening or speaking in a Space?
Since all Spaces are public, your presence and activity in a Space is also public. If you are logged into your Twitter account when you are in a Space, you will be visible to everyone in the Space as well as to others, including people who follow you, people who peek into the Space without entering, and developers accessing information about the Space using the Twitter API.
If you are listening in a Space, your profile icon will appear with a purple pill at the top of your followers’ Home timelines. You have the option to change this in your settings.
Instructions for:
Manage who can see your Spaces listening activity
Step 1
On the left nav menu, select the more icon and go to Settings and privacy.
Step 2
Under Settings, navigate to Privacy and safety.
Step 3
Under Your Twitter activity, go to Spaces.
Step 4
Choose if you want to Allow followers to see which Spaces you’re listening to by toggling this on or off.
Your followers will always see at the top of their Home timelines what Spaces you’re speaking in.
What does it mean that Spaces are public? Can anyone listen in a Space?
Spaces can be listened to by anyone on the Internet. This is part of a broader feature of Spaces that lets anyone listen to Spaces regardless of whether or not they are logged in to a Twitter account (or even have a Twitter account). Because of this, listener counts may not match the actual number of listeners, nor will the profile photos of all listeners necessarily be displayed in a Space.
How do I invite people to join a Space?
Invite people to join a Space by sending an invite via DM, Tweeting the link out to your Home timeline, or copying the invite link to send it out.
Who can join my Space?
For now, all Spaces are public and anyone can join any Space as a listener. If the listener has a user account, you can block their account. If you create a Space or are a speaker in a Space, your followers will see it at the top of their timeline.
Who can speak in my Space?
By default, your Space will always be set to Only people you invite to speak. You can also modify the Speaker permissions once your Space has been created. Select the icon, then select Adjust settings to see the options for speaker permissions, which include Everyone, People you follow, and the default Only people you invite to speak. These permissions are only saved for this particular Space, so any Space you create in the future will use the default setting.
Once your Space has started, you can send requests to listeners to become speakers or co-hosts by selecting the icon and adding speakers or selecting a person’s profile picture within a Space and adding them as a co-host or speaker. Listeners can request to speak from the host.
Hosts can also invite other people outside of the Space to speak via DM.
How does co-hosting work?
Up to 2 people can become co-hosts and speak in a Space in addition to the 11 speakers (including the primary host) at one time. Co-host status can be lost if the co-host leaves the Space. A co-host can remove their own co-host status to become a Listener again.
Hosts can transfer primary admin rights to another co-host. If the original host drops from Space, the first co-host added will become the primary admin. The admin is responsible for promoting and facilitating a healthy conversation in the Space in line with the Twitter Rules.
Once a co-host is added to a Space, any accounts they’ve blocked on Twitter who are in the Space will be removed from the Space.
Can I schedule a Space?
Hosts can schedule a Space up to 30 days in advance and up to 10 scheduled Spaces. Hosts can still create impromptu Spaces in the meantime, and those won’t count toward the maximum 10 scheduled Spaces.
Before you create your Space, select the scheduler icon and pick the date and time you’d like to schedule your Space to go live. As your scheduled start time approaches, you will receive push and in-app notifications reminding you to start your Space on time. If you don’t have notifications turned on, follow the in-app steps on About notifications on mobile devices to enable them for Spaces. Scheduled Spaces are public and people can set reminders to be notified when your scheduled Space begins.
How do I edit my scheduled Space(s)?
Follow the steps below to edit any of your scheduled Spaces.
Instructions for:
Manage your scheduled Spaces
Step 1
From your timeline, navigate to and long press on the . Or, navigate to the Spaces Tab at the bottom of your timeline.
Step 2
Select the Spaces icon.
Step 3
To manage your scheduled Spaces, select the scheduler icon at the top.
Step 4
You’ll see the Spaces that you have scheduled.
Step 5
Navigate to the more icon of the Space you want to manage. You can edit, share, or cancel the Space.
If you are editing your Space, make sure to select “Save changes” after making edits.
How do I get notified about a scheduled Space?
Guests can sign up for reminder notifications from a scheduled Space card in a Tweet. When the host starts the scheduled Space, the interested guests get notified via push and in-app notifications.
Can I record a Space?
Hosts can record Spaces they create for replay. When creating a Space, toggle on Record Space.
While recording, a recording symbol will appear at the top to indicate that the Space is being recorded by the host. Once the Space ends, you will see how many people attended the Space along with a link to share out via a Tweet. Under Notifications, you can also View details to Tweet the recording. Under host settings, you will have the option to choose where to start your recording with Edit start time. This allows you to cut out any dead air time that might occur at the beginning of a Space.
If you choose to record your Space, once the live Space ends, your recording will be immediately and publicly available for anyone to listen to whenever they want. You can always end a recording to make it no longer publicly available on Twitter by deleting your recording via the more icon on the recording itself. Unless you delete your recording, it will remain available for replay after the live Space has ended.* As with live Spaces, Twitter will retain audio copies for 30 after they end to review for violations of the Twitter Rules. If a violation is found, Twitter may retain audio copies for up to 120 days in total. For more information on downloading Spaces, please see below FAQ, “What happens after a Space ends and is the data retained anywhere?”
Co-hosts and speakers who enter a Space that is being recorded will see a recording symbol (REC). Listeners will also see the recording symbol, but they will not be visible in the recording.
Recordings will show the host, co-host(s), and speakers from the live Space.
*Note: Hosts on iOS 9.15+ and Android 9.46+ will be able to record Spaces that last indefinitely. For hosts on older app versions, recording will only be available for 30 days. For Spaces that are recorded indefinitely, Twitter will retain a copy for as long as the Space is replayable on Twitter, but for no less than 30 days after the live Space ended.
What is clipping?
Clipping is a new feature we’re currently testing and gradually rolling out that lets a limited group of hosts, speakers, and listeners capture 30 seconds of audio from any live or recorded Space and share it through a Tweet if the host has not disabled the clipping function. To start clipping a Space, follow the instructions below to capture the prior 30 seconds of audio from that Space. There is no limit to the number of clips that participants in a Space can create.
When you enter the Space as a co-host or speaker, you will be informed that the Space is clippable through a tool tip notification above the clipping icon.
Note: Currently, creating a clip is available only on iOS and Android, while playing a clip is available on all platforms to everyone.
Instructions for:
Host instructions: How to turn off clipping
When you start your Space, you’ll receive a notification about what a clip is and how to turn it off, as clipping is on by default. You can turn off clipping at any time. To turn it off, follow the instructions below.
Step 1
Navigate to the more icon.
Step 2
Select Adjust settings .
Step 3
Under Clips, toggle Allow clips off.
Instructions for:
Host and speaker instructions: How to create a clipping
Step 1
In a recorded or live Space that is recorded, navigate to the clipping icon. Please note that, for live Spaces, unless the clipping function is disabled, clips will be publicly available on your Twitter profile after your live Space has ended even though the Space itself will no longer be available.
Step 2
On the Create clip pop-up, go to Next.
Step 3
Preview the Tweet and add a comment if you’d like, similarly to a Quote Tweet.
Step 4
Select Tweet to post it to your timeline.
Why is my clip not displaying captions?
What controls do hosts have over existing clips?
What controls do clip creators have over clips they’ve created?
Other controls over clips: how to report, block, or mute
What controls do I have over my Space?
The host and co-host(s) of a Space have control over who can speak. They can mute any Speaker, but it is up to the individual to unmute themselves if they receive speaking privileges. Hosts and co-hosts can also remove, report, and block others in the Space.
Speakers and listeners can report and block others in the Space, or can report the Space. If you block a participant in the Space, you will also block that person’s account on Twitter. If the person you blocked joins as a listener, they will appear in the participant list with a Blocked label under their account name. If the person you blocked joins as a speaker, they will also appear in the participant list with a Blocked label under their account name and you will see an in-app notification stating, “An account you blocked has joined as a speaker.” If you are entering a Space that already has a blocked account as a speaker, you will also see a warning before joining the Space stating, “You have blocked 1 person who is speaking.”
If you are hosting or co-hosting a Space, people you’ve blocked can’t join and, if you’re added as a co-host during a Space, anyone in the Space who you blocked will be removed from the Space.
What are my responsibilities as a Host or Co-Host of a Space?
As a Host, you are responsible for promoting and supporting a healthy conversation in your Space and to use your tools to ensure that the Twitter Rules are followed. The following tools are available for you to use if a participant in the Space is being offensive or disruptive:
Revoke speaking privileges of other users if they are being offensive or disruptive to you or others
Block, remove or report the user.
Here are some guidelines to follow as a Host or Co-Host:
Always follow the Twitter Rulesin the Space you host or co-host. This also applies to the title of your Space which should not include abusive slurs, threats, or any other rule-violating content.
Do not encourage behavior or content that violates the Twitter Rules.
Do not abuse or misuse your hosting tools, such as arbitrarily revoking speaking privileges or removing users, or use Spaces to carry out activities that break our rules such as following schemes.
How can I block someone in a Space?
How can I mute a speaker in a Space?
How can I see people in my Space?
Hosts, speakers, and listeners can select the icon to see people in a Space. Since Spaces are publicly accessible by anyone, it may also be possible for an unknown number of logged-out people to listen to a Space’s audio without being listed as a guest in the Space.
How can I report a Space?
How can I report a person in a Space?
Can Twitter suspend my Space while it’s live?
How many people can speak in a Space?
How many people can listen in a Space?
What happens after a Space ends and is the data retained anywhere?
Hosts can choose to record a Space prior to starting it. Hosts may download copies of their recorded Spaces for as long as we have them by using the Your Twitter Data download tool.
For unrecorded Spaces, Twitter retains copies of audio from recorded Spaces for 30 days after a Space ends to review for violations of the Twitter Rules. If a Space is found to contain a violation, we extend the time we maintain a copy for an additional 90 days (a total of 120 days after a Space ends) to allow people to appeal if they believe there was a mistake. Twitter also uses Spaces content and data for analytics and research to improve the service.
Links to Spaces that are shared out (e.g., via Tweet or DM) also contain some information about the Space, including the description, the identity of the hosts and others in the Space, as well as the Space’s current state (e.g., scheduled, live, or ended). We make this and other information about Spaces available through the Twitter Developer Platform. For a detailed list of the information about Spaces we make available, check out our Spaces endpoints documentation.
For full details on what data we retain, visit our Privacy Policy.
Who can end a Space?
Does Spaces work for accounts with protected Tweets?
Following the Twitter Rules in Spaces
Spaces Feedback Community
We’re opening up the conversation and turning it over to the people who are participating in Spaces. This Community is a dedicated place for us to connect with you on all things Spaces, whether it’s feedback around features, ideas for improvement, or any general thoughts.
Who can join?
Anyone on Spaces can join, whether you are a host, speaker, or listener.
How do I join the Community?
You can request to join the Twitter Spaces Feedback Community here. By requesting to join, you are agreeing to our Community rules.
As a Community admin or moderator, you can create and host a Space for your Community members to join.
Note:
Currently, creating Community Spaces is only available to some admins and moderators using the Twitter for iOS and Twitter for Android apps.
Instructions for:
Admins & moderators: How to create a Space
Step 1
Navigate to the Community landing page.
Step 2
Long press on the Tweet Composer and select the Spaces icon.
Step 3
Select Spaces and begin creating your Space by adding in a title, toggling on record Space (optional), and adding relevant topics.
Step 4
Invite admins, moderators, and other people to be a part of your Space.
Members: How to find a Community Space
If a Community Space is live, you will see the Spacebar populate at the top of your Home timeline. To enter the Space and begin listening, select the live Space in the Spacebar.
Community Spaces FAQ
What are Community Spaces?
Spaces Social Narrative
A social narrative is a simple story that describes social situations and social behaviors for accessibility.
Twitter Spaces allows me to join or host live audio-only conversations with anyone.
Joining a Space
When I join a Twitter Space, that means I’ll be a listener. I can join any Space on Twitter, even those hosted by people I don’t know or follow.
I can join a Space by selecting a profile photo with a purple, pulsing outline at the top of my timeline, selecting a link from someone’s Tweet, or a link in a Direct Message (DM).
Once I’m in a Space, I can seethe profile photos and names of some people in the Space, including myself.
I can hearone or multiple people talking at the same time. If it’s too loud or overwhelming, I can turn down my volume.
As a listener, I am not able to speak. If I want to say something, I can send a request to the host. The host might not approve my request though.
If the host accepts my request, I will become a speaker. It may take a few seconds to connect my microphone, so I’ll have to wait.
Now I can unmute myself and speak. Everyone in the Space will be able to hear me.
When someone says something I want to react to, I can choosean emoji to show everyone how I feel. I will be able to see when other people react as well.
I can leave the Space at any time. After I leave, or when the host ends the Space, I’ll go back to my timeline.
Hosting a Space
When I start a Space, that means I’ll be the host. Anyone can join my Space, even people I don’t know and people I don’t follow.
Once I start my space, it may take a few seconds to be connected, so I’ll have to wait.
Now I’m in my Space and I can seemy profile photo. If other logged-in, people have joined, I will be able to see their profile photos, too.
I will start out muted, which is what the microphone with a slash through it means. I can mute and unmute myself, and anyone in my Space, at any time.
I can invitepeople to join my Space by sending them a Direct Message (DM), sharing the link in a Tweet, and by copying the link and sharing it somewhere else like in an email.
Up to 10 other people can have speaking privileges in my Space at the same time, and I can choosewho speaks and who doesn’t. People can also request to speak, and I can choose to approve their request or not.
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