2021 Virtual World Medical Innovation Forum, Mass General Brigham, Gene and Cell Therapy, VIRTUAL May 19–21, 2021
About the World Medical Innovation Forum
Mass General Brigham is pleased to present the World Medical Innovation Forum (WMIF) virtual event Wednesday, May 19 – Friday, May 21. This interactive web event features expert discussions of gene and cell therapy (GCT) and its potential to change the future of medicine through its disease-treating and potentially curative properties. The agenda features 150+ executive speakers from the healthcare industry, venture, startups, life sciences manufacturing, consumer health and the front lines of care, including many Harvard Medical School-affiliated researchers and clinicians. The annual in-person Forum will resume live in Boston in 2022. The World Medical Innovation Forum is presented by Mass General Brigham Innovation, the global business development unit supporting the research requirements of 7,200 Harvard Medical School faculty and research hospitals including Massachusetts General, Brigham and Women’s, Massachusetts Eye and Ear, Spaulding Rehab and McLean Hospital. Follow us on Twitter: twitter.com/@MGBInnovation
Accelerating the Future of Medicine with Gene and Cell Therapy What Comes Next
https://worldmedicalinnovation.org/wp-content/uploads/2021/05/2021-WMIF-White-Paper-1.0.pdf
https://worldmedicalinnovation.org/agenda/
Virtual | May 19–21, 2021
#WMIF2021
@MGBInnovation
Leaders in Pharmaceutical Business Intelligence (LPBI) Group
will cover the event in Real Time
Aviva Lev-Ari, PhD, RN
Founder LPBI 1.0 & LPBI 2.0
will be in virtual attendance producing the e-Proceedings
and the Tweet Collection of this Global event expecting +15,000 attendees
@pharma_BI
@AVIVA1950
LPBI’s Eighteen Books in Medicine
https://lnkd.in/ekWGNqA
Among them, books on Gene and Cell Therapy include the following:
Topics for May 19 – 21 include:
Impact on Patient Care – Therapeutic and Potentially Curative GCT Developments
GCT Delivery, Manufacturing – What’s Next
GCT Platform Development
Oncolytic Viruses – Cancer applications, start-ups
Regenerative Medicine/Stem Cells
Future of CAR-T
M&A Shaping GCT’s Future
Market Priorities
Venture Investing in GCT
China’s GCT Juggernaut
Disease and Patient Focus: Benign blood disorders, diabetes, neurodegenerative diseases
Click here for the current WMIF agenda
Plus:
Fireside Chats: 1:1 interviews with industry CEOs/C-Suite leaders including Novartis Gene Therapies, ThermoFisher, Bayer AG, FDA
First Look: 18 briefings on emerging GCT research from Mass General Brigham scientists
Virtual Poster Session: 40 research posters and presenters on potential GCT discoveries from Mass General Brigham
Announcement of the Disruptive Dozen, 12 GCT technologies likely to break through in the next few years
AGENDA
Wednesday, May 19, 2021
Opening Remarks
Welcome and the vision for Gene and Cell Therapy and why it is a top Mass General Brigham priority.
- Introducer:
- Presenter:
-
May 22,23,24, 2022 – in Boston, in-person 2022 WMIF on CGT
The Grand Challenge of Widespread GCT Patient Benefits
Co-Chairs identify the key themes of the Forum – set the stage for top GCT opportunities, challenges, and where the field might take medicine in the future.
- Moderator:
- Speakers:
-
Oncolytic virus triple threat: Toxic, immunological, combine with anti cancer therapies
Polygenic therapy – multiple genes involved, plug-play,
Gene and Cell Therapy 2.0 – What’s Next as We Realize their Potential for Patients
- Moderator:
- Speaker:
-
Q&A
8:55 AM – 9:10 AM
The Patient and GCT
GCT development for rare diseases is driven by patient and patient-advocate communities. Understanding their needs and perspectives enables biomarker research, the development of value-driving clinical trial endpoints and successful clinical trials. Industry works with patient communities that help identify unmet needs and collaborate with researchers to conduct disease natural history studies that inform the development of biomarkers and trial endpoints. This panel includes patients who have received cutting-edge GCT therapy as well as caregivers and patient advocates.
- Moderator:
- Speakers:
-
Q&A
9:25 AM – 9:40 AM
Building a GCT Platform for Mainstream Success
This panel of GCT executives, innovators and investors explore how to best shape a successful GCT strategy. Among the questions to be addressed:
- How are GCT approaches set around defining and building a platform?
- Is AAV the leading modality and what are the remaining challenges?
- What are the alternatives?
- Is it just a matter of matching modalities to the right indications?
- Moderator:
- Speakers:
-
Q&A
10:20 AM – 10:35 AM
AAV Success Studies | Retinal Dystrophy | Spinal Muscular Atrophy
Recent AAV gene therapy product approvals have catalyzed the field. This new class of therapies has shown the potential to bring transformative benefit to patients. With dozens of AAV treatments in clinical studies, all eyes are on the field to gauge its disruptive impact.
The panel assesses the largest challenges of the first two products, the lessons learned for the broader CGT field, and the extent to which they serve as a precedent to broaden the AAV modality.
- Is AAV gene therapy restricted to genetically defined disorders, or will it be able to address common diseases in the near term?
- Lessons learned from these first-in-class approvals.
- Challenges to broaden this modality to similar indications.
- Reflections on safety signals in the clinical studies?
- Moderator:
- Speakers:
-
Q&A
10:50 AM – 11:05 AM
AAV Delivery
This panel will address the advances in the area of AAV gene therapy delivery looking out the next five years. Questions that loom large are: How can biodistribution of AAV be improved? What solutions are in the wings to address immunogenicity of AAV? Will patients be able to receive systemic redosing of AAV-based gene therapies in the future? What technical advances are there for payload size? Will the cost of manufacturing ever become affordable for ultra-rare conditions? Will non-viral delivery completely supplant viral delivery within the next five years?What are the safety concerns and how will they be addressed?
- Moderators:
- Speakers:
-
Q&A
11:50 AM – 12:05 PM
M&A | Shaping GCT Innovation
The GCT M&A market is booming – many large pharmas have made at least one significant acquisition. How should we view the current GCT M&A market? What is its impact of the current M&A market on technology development? Are these M&A trends new are just another cycle? Has pharma strategy shifted and, if so, what does it mean for GCT companies? What does it mean for patients? What are the long-term prospects – can valuations hold up?
- Moderator:
- Speakers:
Debby Baron,
- Worldwide Business Development, Pfizer
CGT is an important area Pfizer is active looking for innovators, advancing forward programs of innovation with the experience Pfizer has internally
Scalability and manufacturing regulatory conversations, clinical programs safety in parallel to planning getting drug to patients
- Vice President, Business Development and Lilly New Ventures, Eli Lilly and Company
Head of Strategy, Business Development & Licensing, and Member of the Executive Committee, Bayer
Absolute Leadership in Gene editing, gene therapy, via acquisition and strategic alliance
Operating model of the acquired company discussed , company continue independence
- Board Chairman, Encoded Therapeutics & Affinia
Executive Chairman, Jaguar Gene Therapy & Istari Oncology
As acquiree multiple M&A: How the acquirer looks at integration and cultures of the two companies
Traditional integration vs jump start by external acquisition
AAV – epilepsy, next generation of vectors
-
Q&A
12:20 PM – 12:35 PM
Oncolytic Viruses in Cancer | Curing Melanoma and Beyond
Oncolytic viruses represent a powerful new technology, but so far an FDA-approved oncolytic (Imlygic) has only occurred in one area – melanoma and that what is in 2015. This panel involves some of the protagonists of this early success story. They will explore why and how Imlygic became approved and its path to commercialization. Yet, no other cancer indications exist for Imlygic, unlike the expansion of FDA-approved indication for immune checkpoint inhibitors to multiple cancers. Why? Is there a limitation to what and which cancers can target? Is the mode of administration a problem?
No other oncolytic virus therapy has been approved since 2015. Where will the next success story come from and why? Will these therapies only be beneficial for skin cancers or other easily accessible cancers based on intratumoral delivery?
The panel will examine whether the preclinical models that have been developed for other cancer treatment modalities will be useful for oncolytic viruses. It will also assess the extent pre-clinical development challenges have slowed the development of OVs.
- Moderator:
- Speakers:
-
Q&A
1:45 PM – 2:00 PM
Market Interest in Oncolytic Viruses | Calibrating
There are currently two oncolytic virus products on the market, one in the USA and one in China. As of late 2020, there were 86 clinical trials 60 of which were in phase I with just 2 in Phase III the rest in Phase I/II or Phase II. Although global sales of OVs are still in the ramp-up phase, some projections forecast OVs will be a $700 million market by 2026. This panel will address some of the major questions in this area:
What regulatory challenges will keep OVs from realizing their potential? Despite the promise of OVs for treating cancer only one has been approved in the US. Why has this been the case? Reasons such have viral tropism, viral species selection and delivery challenges have all been cited. However, these are also true of other modalities. Why then have oncolytic virus approaches not advanced faster and what are the primary challenges to be overcome?
- Will these need to be combined with other agents to realize their full efficacy and how will that impact the market?
- Why are these companies pursuing OVs while several others are taking a pass?
- Moderators:
- Speakers:
-
- Anlong Li, MD, PhD
- Jeffrey Infante, MD
- Loic Vincent, PhD
-
Q&A
2:15 PM – 2:30 PM
Entrepreneurial Growth | Oncolytic Virus
In 2020 there were a total of 60 phase I trials for Oncolytic Viruses. There are now dozens of companies pursuing some aspect of OV technology. This panel will address:
- How are small companies equipped to address the challenges of developing OV therapies better than large pharma or biotech?
- Will the success of COVID vaccines based on Adenovirus help the regulatory environment for small companies developing OV products in Europe and the USA?
- Is there a place for non-viral delivery and other immunotherapy companies to engage in the OV space? Would they bring any real advantages?
- Moderator:
- Speakers:
-
Q&A
2:50 PM – 3:05 PM
CAR-T | Lessons Learned | What’s Next
Few areas of potential cancer therapy have had the attention and excitement of CAR-T. This panel of leading executives, developers, and clinician-scientists will explore the current state of CAR-T and its future prospects. Among the questions to be addressed are:
- Is CAR-T still an industry priority – i.e. are new investments being made by large companies? Are new companies being financed? What are the trends?
- What have we learned from first-generation products, what can we expect from CAR-T going forward in novel targets, combinations, armored CAR’s and allogeneic treatment adoption?
- Early trials showed remarkable overall survival and progression-free survival. What has been observed regarding how enduring these responses are?
- Most of the approvals to date have targeted CD19, and most recently BCMA. What are the most common forms of relapses that have been observed?
- Is there a consensus about what comes after these CD19 and BCMA trials as to additional targets in liquid tumors? How have dual-targeted approaches fared?
- Moderator:
-
Is CAR-T Industry priority
- Speakers:
- Head of R&D, Atara BioTherapeutics
- Phyno-type of the cells for hematologic cancers
- solid tumor
- inventory of Therapeutics for treating patients in the future
- Progressive MS program
- EBBT platform B-Cells and T-Cells
-
- Stefan Hendriks
- Gobal Head, Cell & Gene, Novartis
- yes, CGT is a strategy in the present and future
- Journey started years ago
- Confirmation the effectiveness of CAR-T therapies, 1 year response prolonged to 5 years 26 months
- Patient not responding – a lot to learn
- Patient after 8 months of chemo can be helped by CAR-T
- Christi Shaw
- CEO, Kite
- CAR-T is priority 120 companies in the space
- Manufacturing consistency
- Patients respond with better quality of life
- Blood cancer – more work to be done
- Stefan Hendriks
-
3:30 PM – 3:45 PM
CAR-T | Solid Tumors Success | When?
The potential application of CAR-T in solid tumors will be a game-changer if it occurs. The panel explores the prospects of solid tumor success and what the barriers have been. Questions include:
- How would industry and investor strategy for CAR-T and solid tumors be characterized? Has it changed in the last couple of years?
- Does the lack of tumor antigen specificity in solid tumors mean that lessons from liquid tumor CAR-T constructs will not translate well and we have to start over?
- Whether due to antigen heterogeneity, a hostile tumor micro-environment, or other factors are some specific solid tumors more attractive opportunities than others for CAR-T therapy development?
- Given the many challenges that CAR-T faces in solid tumors, does the use of combination therapies from the start, for example, to mitigate TME effects, offer a more compelling opportunity.
- Moderator:
- Speakers:
-
Q&A
4:00 PM – 4:15 PM
GCT Manufacturing | Vector Production | Autologous and Allogeneic | Stem Cells | Supply Chain | Scalability & Management
The modes of GCT manufacturing have the potential of fundamentally reordering long-established roles and pathways. While complexity goes up the distance from discovery to deployment shrinks. With the likelihood of a total market for cell therapies to be over $48 billion by 2027, groups of products are emerging. Stem cell therapies are projected to be $28 billion by 2027 and non-stem cell therapies such as CAR-T are projected be $20 billion by 2027. The manufacturing challenges for these two large buckets are very different. Within the CAR-T realm there are diverging trends of autologous and allogeneic therapies and the demands on manufacturing infrastructure are very different. Questions for the panelists are:
- Help us all understand the different manufacturing challenges for cell therapies. What are the trade-offs among storage cost, batch size, line changes in terms of production cost and what is the current state of scaling naïve and stem cell therapy treatment vs engineered cell therapies?
- For cell and gene therapy what is the cost of Quality Assurance/Quality Control vs. production and how do you think this will trend over time based on your perspective on learning curves today?
- Will point of care production become a reality? How will that change product development strategy for pharma and venture investors? What would be the regulatory implications for such products?
- How close are allogeneic CAR-T cell therapies? If successful what are the market implications of allogenic CAR-T? What are the cost implications and rewards for developing allogeneic cell therapy treatments?
- Moderator:
-
- Michael Paglia
- Speakers:
-
- Dannielle Appelhans
- SVP TechOps and Chief Technical Officer, Novartis Gene Therapies
- Thomas Page, PhD
- VP, Engineering and Asset Development, FUJIFILM Diosynth Biotechnologies
- Rahul Singhvi, ScD
- CEO and Co-Founder, National Resilience, Inc.
- Thomas VanCott, PhD
- Global Head of Product Development, Gene & Cell Therapy, Catalent
- 2/3 autologous 1/3 allogeneic CAR-T high doses and high populations scale up is not done today quality maintain required the timing logistics issues centralized vs decentralized allogeneic are health donors innovations in cell types in use improvements in manufacturing
Ropa Pike, Director, Enterprise Science & Partnerships, Thermo Fisher Scientific
Centralized biopharma industry is moving to decentralized models site specific license
- Dannielle Appelhans
-
Q&A
4:30 PM – 4:45 PM
Thursday, May 20, 2021
GCT | The China Juggernaut
China embraced gene and cell therapies early. The first China gene therapy clinical trial was in 1991. China approved the world’s first gene therapy product in 2003—Gendicine—an oncolytic adenovirus for the treatment of advanced head and neck cancer. Driven by broad national strategy, China has become a hotbed of GCT development, ranking second in the world with more than 1,000 clinical trials either conducted or underway and thousands of related patents. It has a booming GCT biotech sector, led by more than 45 local companies with growing IND pipelines.
In late 1990, a T cell-based immunotherapy, cytokine-induced killer (CIK) therapy became a popular modality in the clinic in China for tumor treatment. In early 2010, Chinese researchers started to carry out domestic CAR T trials inspired by several important reports suggested the great antitumor function of CAR T cells. Now, China became the country with the most registered CAR T trials, CAR T therapy is flourishing in China.
The Chinese GCT ecosystem has increasingly rich local innovation and growing complement of development and investment partnerships – and also many subtleties.
This panel, consisting of leaders from the China GCT corporate, investor, research and entrepreneurial communities, will consider strategic questions on the growth of the gene and cell therapy industry in China, areas of greatest strength, evolving regulatory framework, early successes and products expected to reach the US and world market.
- Moderator:
- Speakers:
-
Q&A
8:30 AM – 8:45 AM
Impact of mRNA Vaccines | Global Success Lessons
The COVID vaccine race has propelled mRNA to the forefront of biomedicine. Long considered as a compelling modality for therapeutic gene transfer, the technology may have found its most impactful application as a vaccine platform. Given the transformative industrialization, the massive human experience, and the fast development that has taken place in this industry, where is the horizon? Does the success of the vaccine application, benefit or limit its use as a therapeutic for CGT?
- How will the COVID success impact the rest of the industry both in therapeutic and prophylactic vaccines and broader mRNA lessons?
- How will the COVID success impact the rest of the industry both on therapeutic and prophylactic vaccines and broader mRNA lessons?
- Beyond from speed of development, what aspects make mRNA so well suited as a vaccine platform?
- Will cost-of-goods be reduced as the industry matures?
- How does mRNA technology seek to compete with AAV and other gene therapy approaches?
- Moderator:
- Speakers:
-
- Melissa Moore
- Chief Scientific Officer, Moderna
Many years of mRNA pivoting for new diseases, DARPA, nucleic Acids global deployment of a manufacturing unit on site where the need arise Elan Musk funds new directions at Moderna
How many mRNA can be put in one vaccine: Dose and tolerance to achieve efficacy
45 days for Personalized cancer vaccine one per patient
- Ron Renaud
- CEO, Translate Bio
1.6 Billion doses produced rare disease monogenic correct mRNA like CF multiple mutation infection disease and oncology applications
Platform allowing to swap cargo reusing same nanoparticles address disease beyond Big Pharma options for biotech
WHat strain of Flu vaccine will come back in the future when people do not use masks
- Kate Bingham, UK Vaccine Taskforce
July 2020, AAV vs mRNA delivery across UK local centers administered both types supply and delivery uplift
- Melissa Moore
-
Q&A
9:00 AM – 9:15 AM
Benign Blood Disorders
Hemophilia has been and remains a hallmark indication for the CGT. Given its well-defined biology, larger market, and limited need for gene transfer to provide therapeutic benefit, it has been at the forefront of clinical development for years, however, product approval remains elusive. What are the main hurdles to this success? Contrary to many indications that CGT pursues no therapeutic options are available to patients, hemophiliacs have an increasing number of highly efficacious treatment options. How does the competitive landscape impact this field differently than other CGT fields? With many different players pursuing a gene therapy option for hemophilia, what are the main differentiators? Gene therapy for hemophilia seems compelling for low and middle-income countries, given the cost of currently available treatments; does your company see opportunities in this market?
- Moderator:
- Speakers:
-
Q&A
9:30 AM – 9:45 AM
Diabetes | Grand Challenge
The American Diabetes Association estimates 30 million Americans have diabetes and 1.5 million are diagnosed annually. GCT offers the prospect of long-sought treatment for this enormous cohort and their chronic requirements. The complexity of the disease and its management constitute a grand challenge and highlight both the potential of GCT and its current limitations.
- Islet transplantation for type 1 diabetes has been attempted for decades. Problems like loss of transplanted islet cells due to autoimmunity and graft site factors have been difficult to address. Is there anything different on the horizon for gene and cell therapies to help this be successful?
- How is the durability of response for gene or cell therapies for diabetes being addressed? For example, what would the profile of an acceptable (vs. optimal) cell therapy look like?
- Moderator:
- Speakers:
-
Q&A
10:20 AM – 10:35 AM
Getting to the Heart of the Matter: Curing Genetic Cardiomyopathy
- Christine Seidman, MD
-
- Director, Cardiovascular Genetics Center, BWH
- Smith Professor of Medicine & Genetics, HMS
2021 Virtual World Medical Innovation Forum, Mass General Brigham, Gene and Cell Therapy, VIRTUAL May 19–21, 2021
The 2021 Virtual World Medical Innovation Forum will focus on the growing impact of gene and cell therapy.Senior healthcare leaders from all over look to shape and debate the area of gene and cell therapy. Our shared belief: no matter the magnitude of change, responsible healthcare is centered on a shared commitment to collaborative innovation–industry, academia, and practitioners working together to improve patients’ lives.About the World Medical Innovation Forum
Mass General Brigham is pleased to present the World Medical Innovation Forum (WMIF) virtual event Wednesday, May 19 – Friday, May 21. This interactive web event features expert discussions of gene and cell therapy (GCT) and its potential to change the future of medicine through its disease-treating and potentially curative properties. The agenda features 150+ executive speakers from the healthcare industry, venture, startups, life sciences manufacturing, consumer health and the front lines of care, including many Harvard Medical School-affiliated researchers and clinicians. The annual in-person Forum will resume live in Boston in 2022. The World Medical Innovation Forum is presented by Mass General Brigham Innovation, the global business development unit supporting the research requirements of 7,200 Harvard Medical School faculty and research hospitals including Massachusetts General, Brigham and Women’s, Massachusetts Eye and Ear, Spaulding Rehab and McLean Hospital. Follow us on Twitter: twitter.com/@MGBInnovation
Accelerating the Future of Medicine with Gene and Cell Therapy What Comes Next
https://worldmedicalinnovation.org/wp-content/uploads/2021/05/2021-WMIF-White-Paper-1.0.pdf
https://worldmedicalinnovation.org/agenda/
Virtual | May 19–21, 2021
#WMIF2021
@MGBInnovation
Leaders in Pharmaceutical Business Intelligence (LPBI) Group
will cover the event in Real Time
Aviva Lev-Ari, PhD, RN
Founder LPBI 1.0 & LPBI 2.0
will be in virtual attendance producing the e-Proceedings
and the Tweet Collection of this Global event expecting +15,000 attendees
@pharma_BI
@AVIVA1950
LPBI’s Eighteen Books in Medicine
https://lnkd.in/ekWGNqA
Among them, books on Gene and Cell Therapy include the following:
Topics for May 19 – 21 include:
Impact on Patient Care – Therapeutic and Potentially Curative GCT Developments
GCT Delivery, Manufacturing – What’s Next
GCT Platform Development
Oncolytic Viruses – Cancer applications, start-ups
Regenerative Medicine/Stem Cells
Future of CAR-T
M&A Shaping GCT’s Future
Market Priorities
Venture Investing in GCT
China’s GCT Juggernaut
Disease and Patient Focus: Benign blood disorders, diabetes, neurodegenerative diseases
Click here for the current WMIF agenda
Plus:
Fireside Chats: 1:1 interviews with industry CEOs/C-Suite leaders including Novartis Gene Therapies, ThermoFisher, Bayer AG, FDA
First Look: 18 briefings on emerging GCT research from Mass General Brigham scientists
Virtual Poster Session: 40 research posters and presenters on potential GCT discoveries from Mass General Brigham
Announcement of the Disruptive Dozen, 12 GCT technologies likely to break through in the next few years
AGENDA
Wednesday, May 19, 2021
8:00 AM – 8:10 AMOpening Remarks
Welcome and the vision for Gene and Cell Therapy and why it is a top Mass General Brigham priority.
- Introducer:
- Presenter:
-
May 22,23,24, 2022 – in Boston, in-person 2022 WMIF on CGT
8:10 AM – 8:30 AMThe Grand Challenge of Widespread GCT Patient Benefits
Co-Chairs identify the key themes of the Forum – set the stage for top GCT opportunities, challenges, and where the field might take medicine in the future.
- Moderator:
- Speakers:
-
Oncolytic virus triple threat: Toxic, immunological, combine with anti cancer therapies
Polygenic therapy – multiple genes involved, plug-play,
8:35 AM – 8:50 AMFIRESIDEGene and Cell Therapy 2.0 – What’s Next as We Realize their Potential for Patients
- Moderator:
- Speaker:
-
Q&A
8:55 AM – 9:10 AM
8:55 AM – 9:20 AMThe Patient and GCT
GCT development for rare diseases is driven by patient and patient-advocate communities. Understanding their needs and perspectives enables biomarker research, the development of value-driving clinical trial endpoints and successful clinical trials. Industry works with patient communities that help identify unmet needs and collaborate with researchers to conduct disease natural history studies that inform the development of biomarkers and trial endpoints. This panel includes patients who have received cutting-edge GCT therapy as well as caregivers and patient advocates.
- Moderator:
- Speakers:
-
Q&A
9:25 AM – 9:40 AM
9:25 AM – 9:45 AMFIRESIDE9:50 AM – 10:15 AMBuilding a GCT Platform for Mainstream Success
This panel of GCT executives, innovators and investors explore how to best shape a successful GCT strategy. Among the questions to be addressed:
- How are GCT approaches set around defining and building a platform?
- Is AAV the leading modality and what are the remaining challenges?
- What are the alternatives?
- Is it just a matter of matching modalities to the right indications?
- Moderator:
- Speakers:
-
Q&A
10:20 AM – 10:35 AM
10:20 AM – 10:45 AMAAV Success Studies | Retinal Dystrophy | Spinal Muscular Atrophy
Recent AAV gene therapy product approvals have catalyzed the field. This new class of therapies has shown the potential to bring transformative benefit to patients. With dozens of AAV treatments in clinical studies, all eyes are on the field to gauge its disruptive impact.
The panel assesses the largest challenges of the first two products, the lessons learned for the broader CGT field, and the extent to which they serve as a precedent to broaden the AAV modality.
- Is AAV gene therapy restricted to genetically defined disorders, or will it be able to address common diseases in the near term?
- Lessons learned from these first-in-class approvals.
- Challenges to broaden this modality to similar indications.
- Reflections on safety signals in the clinical studies?
- Moderator:
- Speakers:
-
Q&A
10:50 AM – 11:05 AM
10:45 AM – 10:55 AM10:55 AM – 11:05 AMFIRST LOOK11:05 AM – 11:15 AMFIRST LOOK11:20 AM – 11:45 AMHOT TOPICSAAV Delivery
This panel will address the advances in the area of AAV gene therapy delivery looking out the next five years. Questions that loom large are: How can biodistribution of AAV be improved? What solutions are in the wings to address immunogenicity of AAV? Will patients be able to receive systemic redosing of AAV-based gene therapies in the future? What technical advances are there for payload size? Will the cost of manufacturing ever become affordable for ultra-rare conditions? Will non-viral delivery completely supplant viral delivery within the next five years?What are the safety concerns and how will they be addressed?
- Moderators:
- Speakers:
-
Q&A
11:50 AM – 12:05 PM
11:50 AM – 12:15 PMM&A | Shaping GCT Innovation
The GCT M&A market is booming – many large pharmas have made at least one significant acquisition. How should we view the current GCT M&A market? What is its impact of the current M&A market on technology development? Are these M&A trends new are just another cycle? Has pharma strategy shifted and, if so, what does it mean for GCT companies? What does it mean for patients? What are the long-term prospects – can valuations hold up?
- Moderator:
- Speakers:
Debby Baron,
- Worldwide Business Development, Pfizer
CGT is an important area Pfizer is active looking for innovators, advancing forward programs of innovation with the experience Pfizer has internally
Scalability and manufacturing regulatory conversations, clinical programs safety in parallel to planning getting drug to patients
- Vice President, Business Development and Lilly New Ventures, Eli Lilly and Company
Head of Strategy, Business Development & Licensing, and Member of the Executive Committee, Bayer
Absolute Leadership in Gene editing, gene therapy, via acquisition and strategic alliance
Operating model of the acquired company discussed , company continue independence
- Board Chairman, Encoded Therapeutics & Affinia
Executive Chairman, Jaguar Gene Therapy & Istari Oncology
As acquiree multiple M&A: How the acquirer looks at integration and cultures of the two companies
Traditional integration vs jump start by external acquisition
AAV – epilepsy, next generation of vectors
-
Q&A
12:20 PM – 12:35 PM
12:15 PM – 12:25 PMFIRST LOOK12:25 PM – 12:35 PMFIRST LOOK12:35 PM – 1:15 PM1:15 PM – 1:40 PMOncolytic Viruses in Cancer | Curing Melanoma and Beyond
Oncolytic viruses represent a powerful new technology, but so far an FDA-approved oncolytic (Imlygic) has only occurred in one area – melanoma and that what is in 2015. This panel involves some of the protagonists of this early success story. They will explore why and how Imlygic became approved and its path to commercialization. Yet, no other cancer indications exist for Imlygic, unlike the expansion of FDA-approved indication for immune checkpoint inhibitors to multiple cancers. Why? Is there a limitation to what and which cancers can target? Is the mode of administration a problem?
No other oncolytic virus therapy has been approved since 2015. Where will the next success story come from and why? Will these therapies only be beneficial for skin cancers or other easily accessible cancers based on intratumoral delivery?
The panel will examine whether the preclinical models that have been developed for other cancer treatment modalities will be useful for oncolytic viruses. It will also assess the extent pre-clinical development challenges have slowed the development of OVs.
- Moderator:
- Speakers:
-
Q&A
1:45 PM – 2:00 PM
1:45 PM – 2:10 PMMarket Interest in Oncolytic Viruses | Calibrating
There are currently two oncolytic virus products on the market, one in the USA and one in China. As of late 2020, there were 86 clinical trials 60 of which were in phase I with just 2 in Phase III the rest in Phase I/II or Phase II. Although global sales of OVs are still in the ramp-up phase, some projections forecast OVs will be a $700 million market by 2026. This panel will address some of the major questions in this area:
What regulatory challenges will keep OVs from realizing their potential? Despite the promise of OVs for treating cancer only one has been approved in the US. Why has this been the case? Reasons such have viral tropism, viral species selection and delivery challenges have all been cited. However, these are also true of other modalities. Why then have oncolytic virus approaches not advanced faster and what are the primary challenges to be overcome?
- Will these need to be combined with other agents to realize their full efficacy and how will that impact the market?
- Why are these companies pursuing OVs while several others are taking a pass?
- Moderators:
- Speakers:
-
- Anlong Li, MD, PhD
- Jeffrey Infante, MD
- Loic Vincent, PhD
-
Q&A
2:15 PM – 2:30 PM
2:10 PM – 2:20 PMFIRST LOOK2:20 PM – 2:45 PMEntrepreneurial Growth | Oncolytic Virus
In 2020 there were a total of 60 phase I trials for Oncolytic Viruses. There are now dozens of companies pursuing some aspect of OV technology. This panel will address:
- How are small companies equipped to address the challenges of developing OV therapies better than large pharma or biotech?
- Will the success of COVID vaccines based on Adenovirus help the regulatory environment for small companies developing OV products in Europe and the USA?
- Is there a place for non-viral delivery and other immunotherapy companies to engage in the OV space? Would they bring any real advantages?
- Moderator:
- Speakers:
-
Q&A
2:50 PM – 3:05 PM
2:45 PM – 3:00 PM3:00 PM – 3:25 PMCAR-T | Lessons Learned | What’s Next
Few areas of potential cancer therapy have had the attention and excitement of CAR-T. This panel of leading executives, developers, and clinician-scientists will explore the current state of CAR-T and its future prospects. Among the questions to be addressed are:
- Is CAR-T still an industry priority – i.e. are new investments being made by large companies? Are new companies being financed? What are the trends?
- What have we learned from first-generation products, what can we expect from CAR-T going forward in novel targets, combinations, armored CAR’s and allogeneic treatment adoption?
- Early trials showed remarkable overall survival and progression-free survival. What has been observed regarding how enduring these responses are?
- Most of the approvals to date have targeted CD19, and most recently BCMA. What are the most common forms of relapses that have been observed?
- Is there a consensus about what comes after these CD19 and BCMA trials as to additional targets in liquid tumors? How have dual-targeted approaches fared?
- Moderator:
-
Is CAR-T Industry priority
- Speakers:
- Head of R&D, Atara BioTherapeutics
- Phyno-type of the cells for hematologic cancers
- solid tumor
- inventory of Therapeutics for treating patients in the future
- Progressive MS program
- EBBT platform B-Cells and T-Cells
-
- Stefan Hendriks
- Gobal Head, Cell & Gene, Novartis
- yes, CGT is a strategy in the present and future
- Journey started years ago
- Confirmation the effectiveness of CAR-T therapies, 1 year response prolonged to 5 years 26 months
- Patient not responding – a lot to learn
- Patient after 8 months of chemo can be helped by CAR-T
- Christi Shaw
- CEO, Kite
- CAR-T is priority 120 companies in the space
- Manufacturing consistency
- Patients respond with better quality of life
- Blood cancer – more work to be done
- Stefan Hendriks
-
3:30 PM – 3:45 PM
3:30 PM – 3:55 PMHOT TOPICSCAR-T | Solid Tumors Success | When?
The potential application of CAR-T in solid tumors will be a game-changer if it occurs. The panel explores the prospects of solid tumor success and what the barriers have been. Questions include:
- How would industry and investor strategy for CAR-T and solid tumors be characterized? Has it changed in the last couple of years?
- Does the lack of tumor antigen specificity in solid tumors mean that lessons from liquid tumor CAR-T constructs will not translate well and we have to start over?
- Whether due to antigen heterogeneity, a hostile tumor micro-environment, or other factors are some specific solid tumors more attractive opportunities than others for CAR-T therapy development?
- Given the many challenges that CAR-T faces in solid tumors, does the use of combination therapies from the start, for example, to mitigate TME effects, offer a more compelling opportunity.
- Moderator:
- Speakers:
-
Q&A
4:00 PM – 4:15 PM
4:00 PM – 4:25 PMGCT Manufacturing | Vector Production | Autologous and Allogeneic | Stem Cells | Supply Chain | Scalability & Management
The modes of GCT manufacturing have the potential of fundamentally reordering long-established roles and pathways. While complexity goes up the distance from discovery to deployment shrinks. With the likelihood of a total market for cell therapies to be over $48 billion by 2027, groups of products are emerging. Stem cell therapies are projected to be $28 billion by 2027 and non-stem cell therapies such as CAR-T are projected be $20 billion by 2027. The manufacturing challenges for these two large buckets are very different. Within the CAR-T realm there are diverging trends of autologous and allogeneic therapies and the demands on manufacturing infrastructure are very different. Questions for the panelists are:
- Help us all understand the different manufacturing challenges for cell therapies. What are the trade-offs among storage cost, batch size, line changes in terms of production cost and what is the current state of scaling naïve and stem cell therapy treatment vs engineered cell therapies?
- For cell and gene therapy what is the cost of Quality Assurance/Quality Control vs. production and how do you think this will trend over time based on your perspective on learning curves today?
- Will point of care production become a reality? How will that change product development strategy for pharma and venture investors? What would be the regulatory implications for such products?
- How close are allogeneic CAR-T cell therapies? If successful what are the market implications of allogenic CAR-T? What are the cost implications and rewards for developing allogeneic cell therapy treatments?
- Moderator:
-
- Michael Paglia
- Speakers:
-
- Dannielle Appelhans
- SVP TechOps and Chief Technical Officer, Novartis Gene Therapies
- Thomas Page, PhD
- VP, Engineering and Asset Development, FUJIFILM Diosynth Biotechnologies
- Rahul Singhvi, ScD
- CEO and Co-Founder, National Resilience, Inc.
- Thomas VanCott, PhD
- Global Head of Product Development, Gene & Cell Therapy, Catalent
- 2/3 autologous 1/3 allogeneic CAR-T high doses and high populations scale up is not done today quality maintain required the timing logistics issues centralized vs decentralized allogeneic are health donors innovations in cell types in use improvements in manufacturing
Ropa Pike, Director, Enterprise Science & Partnerships, Thermo Fisher Scientific
Centralized biopharma industry is moving to decentralized models site specific license
- Dannielle Appelhans
-
Q&A
4:30 PM – 4:45 PM
4:30 PM – 4:40 PMFIRST LOOK4:40 PM – 4:50 PMFIRST LOOK4:50 PM – 5:00 PMFIRST LOOK5:00 PM – 5:20 PMFIRESIDE5:20 PM – 5:30 PMThursday, May 20, 2021
8:00 AM – 8:25 AMGCT | The China Juggernaut
China embraced gene and cell therapies early. The first China gene therapy clinical trial was in 1991. China approved the world’s first gene therapy product in 2003—Gendicine—an oncolytic adenovirus for the treatment of advanced head and neck cancer. Driven by broad national strategy, China has become a hotbed of GCT development, ranking second in the world with more than 1,000 clinical trials either conducted or underway and thousands of related patents. It has a booming GCT biotech sector, led by more than 45 local companies with growing IND pipelines.
In late 1990, a T cell-based immunotherapy, cytokine-induced killer (CIK) therapy became a popular modality in the clinic in China for tumor treatment. In early 2010, Chinese researchers started to carry out domestic CAR T trials inspired by several important reports suggested the great antitumor function of CAR T cells. Now, China became the country with the most registered CAR T trials, CAR T therapy is flourishing in China.
The Chinese GCT ecosystem has increasingly rich local innovation and growing complement of development and investment partnerships – and also many subtleties.
This panel, consisting of leaders from the China GCT corporate, investor, research and entrepreneurial communities, will consider strategic questions on the growth of the gene and cell therapy industry in China, areas of greatest strength, evolving regulatory framework, early successes and products expected to reach the US and world market.
- Moderator:
- Speakers:
-
Q&A
8:30 AM – 8:45 AM
8:30 AM – 8:55 AMImpact of mRNA Vaccines | Global Success Lessons
The COVID vaccine race has propelled mRNA to the forefront of biomedicine. Long considered as a compelling modality for therapeutic gene transfer, the technology may have found its most impactful application as a vaccine platform. Given the transformative industrialization, the massive human experience, and the fast development that has taken place in this industry, where is the horizon? Does the success of the vaccine application, benefit or limit its use as a therapeutic for CGT?
- How will the COVID success impact the rest of the industry both in therapeutic and prophylactic vaccines and broader mRNA lessons?
- How will the COVID success impact the rest of the industry both on therapeutic and prophylactic vaccines and broader mRNA lessons?
- Beyond from speed of development, what aspects make mRNA so well suited as a vaccine platform?
- Will cost-of-goods be reduced as the industry matures?
- How does mRNA technology seek to compete with AAV and other gene therapy approaches?
- Moderator:
- Speakers:
-
- Melissa Moore
- Chief Scientific Officer, Moderna
Many years of mRNA pivoting for new diseases, DARPA, nucleic Acids global deployment of a manufacturing unit on site where the need arise Elan Musk funds new directions at Moderna
How many mRNA can be put in one vaccine: Dose and tolerance to achieve efficacy
45 days for Personalized cancer vaccine one per patient
- Ron Renaud
- CEO, Translate Bio
1.6 Billion doses produced rare disease monogenic correct mRNA like CF multiple mutation infection disease and oncology applications
Platform allowing to swap cargo reusing same nanoparticles address disease beyond Big Pharma options for biotech
WHat strain of Flu vaccine will come back in the future when people do not use masks
- Kate Bingham, UK Vaccine Taskforce
July 2020, AAV vs mRNA delivery across UK local centers administered both types supply and delivery uplift
- Melissa Moore
-
Q&A
9:00 AM – 9:15 AM
9:00 AM – 9:25 AMHOT TOPICSBenign Blood Disorders
Hemophilia has been and remains a hallmark indication for the CGT. Given its well-defined biology, larger market, and limited need for gene transfer to provide therapeutic benefit, it has been at the forefront of clinical development for years, however, product approval remains elusive. What are the main hurdles to this success? Contrary to many indications that CGT pursues no therapeutic options are available to patients, hemophiliacs have an increasing number of highly efficacious treatment options. How does the competitive landscape impact this field differently than other CGT fields? With many different players pursuing a gene therapy option for hemophilia, what are the main differentiators? Gene therapy for hemophilia seems compelling for low and middle-income countries, given the cost of currently available treatments; does your company see opportunities in this market?
- Moderator:
- Speakers:
-
Q&A
9:30 AM – 9:45 AM
9:25 AM – 9:35 AMFIRST LOOK9:35 AM – 9:45 AMFIRST LOOK9:50 AM – 10:15 AMHOT TOPICSDiabetes | Grand Challenge
The American Diabetes Association estimates 30 million Americans have diabetes and 1.5 million are diagnosed annually. GCT offers the prospect of long-sought treatment for this enormous cohort and their chronic requirements. The complexity of the disease and its management constitute a grand challenge and highlight both the potential of GCT and its current limitations.
- Islet transplantation for type 1 diabetes has been attempted for decades. Problems like loss of transplanted islet cells due to autoimmunity and graft site factors have been difficult to address. Is there anything different on the horizon for gene and cell therapies to help this be successful?
- How is the durability of response for gene or cell therapies for diabetes being addressed? For example, what would the profile of an acceptable (vs. optimal) cell therapy look like?
- Moderator:
- Speakers:
-
Q&A
10:20 AM – 10:35 AM
10:20 AM – 10:40 AMFIRESIDE10:40 AM – 10:50 AM10:50 AM – 11:00 AMFIRST LOOK11:00 AM – 11:10 AMFIRST LOOK11:10 AM – 11:35 AMRare and Ultra Rare Diseases | GCT Breaks Through
One of the most innovative segments in all of healthcare is the development of GCT driven therapies for rare and ultra-rare diseases. Driven by a series of insights and tools and funded in part by disease focused foundations, philanthropists and abundant venture funding disease after disease is yielding to new GCT technology. These often become platforms to address more prevalent diseases. The goal of making these breakthroughs routine and affordable is challenged by a range of issues including clinical trial design and pricing.
- What is driving the interest in rare diseases?
- What are the biggest barriers to making breakthroughs ‘routine and affordable?’
- What is the role of retrospective and prospective natural history studies in rare disease? When does the expected value of retrospective disease history studies justify the cost?
- Related to the first question, what is the FDA expecting as far as controls in clinical trials for rare diseases? How does this impact the collection of natural history data?
- Moderator:
- Speakers:
-
Do 100 patient study then have information on natural history to develop a clinical trial
-
Q&A
11:40 AM – 11:55 AM
11:40 AM – 12:00 PMFIRESIDE12:05 PM – 12:30 PMCEO Panel | Anticipating Disruption | Planning for Widespread GCT
The power of GCT to cure disease has the prospect of profoundly improving the lives of patients who respond. Planning for a disruption of this magnitude is complex and challenging as it will change care across the spectrum. Leading chief executives shares perspectives on how the industry will change and how this change should be anticipated.
- Moderator:
- Speakers:
-
Q&A
12:35 PM – 12:50 PM
12:35 PM – 12:55 PMFIRESIDEBuilding a GCT Portfolio
GCT represents a large and growing market for novel therapeutics that has several segments. These include Cardiovascular Disease, Cancer, Neurological Diseases, Infectious Disease, Ophthalmology, Benign Blood Disorders, and many others; Manufacturing and Supply Chain including CDMO’s and CMO’s; Stem Cells and Regenerative Medicine; Tools and Platforms (viral vectors, nano delivery, gene editing, etc.). Bayer’s pharma business participates in virtually all of these segments. How does a Company like Bayer approach the development of a portfolio in a space as large and as diverse as this one? How does Bayer approach the support of the production infrastructure with unique demands and significant differences from its historical requirements?
- Moderator:
-
- Speaker:
-
Q&A
1:00 PM – 1:15 PM
12:55 PM – 1:35 PM1:40 PM – 2:05 PMGCT Delivery | Perfecting the Technology
Gene delivery uses physical, chemical, or viral means to introduce genetic material into cells. As more genetically modified therapies move closer to the market, challenges involving safety, efficacy, and manufacturing have emerged. Optimizing lipidic and polymer nanoparticles and exosomal delivery is a short-term priority. This panel will examine how the short-term and long-term challenges are being tackled particularly for non-viral delivery modalities.
- Moderator:
- Speakers:
-
- Matthew Stanton, PhD
- Sonya Montgomery
- Laura Sepp-Lorenzino, PhD
- Doug Williams, PhD
-
Q&A
2:10 PM – 2:25 PM
2:05 PM – 2:10 PMInvention Discovery Grant Announcement
IDG Announcement
Tool for translation research at MGB
Commercialization of Lab to Clinic
$70MM was invested by VC minority equity investments in early stage for 1.2MM internal funding by MGB – Academia and Industry – Bayer as Investor
Six Winners
Lydia Lynche, PhDPeter Page, PhDPietr from MEE
-
2:10 PM – 2:20 PMFIRST LOOK2:20 PM – 2:30 PMFIRST LOOK2:55 PM – 3:20 PMHOT TOPICS
Gene Editing | Achieving Therapeutic Mainstream
Gene editing was recognized by the Nobel Committee as “one of gene technology’s sharpest tools, having a revolutionary impact on life sciences.” Introduced in 2011, gene editing is used to modify DNA. It has applications across almost all categories of disease and is also being used in agriculture and public health.
Today’s panel is made up of pioneers who represent foundational aspects of gene editing. They will discuss the movement of the technology into the therapeutic mainstream.
- Successes in gene editing – lessons learned from late-stage assets (sickle cell, ophthalmology)
- When to use what editing tool – pros and cons of traditional gene-editing v. base editing. Is prime editing the future? Specific use cases for epigenetic editing.
- When we reach widespread clinical use – role of off-target editing – is the risk real? How will we mitigate? How practical is patient-specific off-target evaluation?
- Moderator:
- Speakers:
-
Rachel Haurwitz, PhD
Caribou BioSciences, Off UC, Berkeley, CA
Innovation to delivery large quantities of DNA
-
Q&A
3:25 PM – 3:50 PM
3:25 PM – 3:50 PMHOT TOPICSCommon Blood Disorders | Gene Therapy
There are several dozen companies working to develop gene or cell therapies for Sickle Cell Disease, Beta Thalassemia, and Fanconi Anemia. In some cases, there are enzyme replacement therapies that are deemed effective and safe. In other cases, the disease is only managed at best. This panel will address a number of questions that are particular to this class of genetic diseases:
- What are the pros and cons of various strategies for treatment? There are AAV-based editing, non-viral delivery even oligonucleotide recruitment of endogenous editing/repair mechanisms. Which approaches are most appropriate for which disease?
- How can companies increase the speed of recruitment for clinical trials when other treatments are available? What is the best approach to educate patients on a novel therapeutic?
- How do we best address ethnic and socio-economic diversity to be more representative of the target patient population?
- How long do we have to follow up with the patients from the scientific, patient’s community, and payer points of view? What are the current FDA and EMA guidelines for long-term follow-up?
- Where are we with regards to surrogate endpoints and their application to clinically meaningful endpoints?
- What are the emerging ethical dilemmas in pediatric gene therapy research? Are there challenges with informed consent and pediatric assent for trial participation?
- Are there differences in reimbursement policies for these different blood disorders? Clearly durability of response is a big factor. Are there other considerations?
- Moderator:
- Speakers:
-
Q&A
3:55 PM – 4:15 PM
3:50 PM – 4:00 PMFIRST LOOKGene Editing
FIRST LOOK
RNA Therapy for Brain Cancer
Pierpaolo Peruzzi, MD, PhD
Neurosurgery, BWH; Assistant Professor of Neurosurgery, HMS
Targeting with RNA clusters enhances chemotheraphy in GBM
AAV delivery micro RNA – viral mediated and by exosomes (non viral)
Therapeutic impact in Brain Tumors 2-3 readiness
-
Q&A
4:00 PM – 4:20 PM
4:20 PM – 4:45 PMHOT TOPICSGene Expression | Modulating with Oligonucleotide-Based Therapies
Oligonucleotide drugs have recently come into their own with approvals from companies such as Biogen, Alnylam, Novartis and others. This panel will address several questions:
How important is the delivery challenge for oligonucleotides? Are technological advancements emerging that will improve the delivery of oligonucleotides to the CNS or skeletal muscle after systemic administration?
- Will oligonucleotides improve as a class that will make them even more effective? Are further advancements in backbone chemistry anticipated, for example.
- Will oligonucleotide based therapies blaze trails for follow-on gene therapy products?
- Are small molecules a threat to oligonucleotide-based therapies?
- Beyond exon skipping and knock-down mechanisms, what other roles will oligonucleotide-based therapies take mechanistically — can genes be activating oligonucleotides? Is there a place for multiple mechanism oligonucleotide medicines?
- Are there any advantages of RNAi-based oligonucleotides over ASOs, and if so for what use?
- Moderator:
- Jeannie Lee, MD, PhD
-
- Molecular Biologist, MGH
- Professor of Genetics, HMS
2021 Virtual World Medical Innovation Forum, Mass General Brigham, Gene and Cell Therapy, VIRTUAL May 19–21, 2021
The 2021 Virtual World Medical Innovation Forum will focus on the growing impact of gene and cell therapy.Senior healthcare leaders from all over look to shape and debate the area of gene and cell therapy. Our shared belief: no matter the magnitude of change, responsible healthcare is centered on a shared commitment to collaborative innovation–industry, academia, and practitioners working together to improve patients’ lives.About the World Medical Innovation Forum
Mass General Brigham is pleased to present the World Medical Innovation Forum (WMIF) virtual event Wednesday, May 19 – Friday, May 21. This interactive web event features expert discussions of gene and cell therapy (GCT) and its potential to change the future of medicine through its disease-treating and potentially curative properties. The agenda features 150+ executive speakers from the healthcare industry, venture, startups, life sciences manufacturing, consumer health and the front lines of care, including many Harvard Medical School-affiliated researchers and clinicians. The annual in-person Forum will resume live in Boston in 2022. The World Medical Innovation Forum is presented by Mass General Brigham Innovation, the global business development unit supporting the research requirements of 7,200 Harvard Medical School faculty and research hospitals including Massachusetts General, Brigham and Women’s, Massachusetts Eye and Ear, Spaulding Rehab and McLean Hospital. Follow us on Twitter: twitter.com/@MGBInnovation
Accelerating the Future of Medicine with Gene and Cell Therapy What Comes Next
https://worldmedicalinnovation.org/wp-content/uploads/2021/05/2021-WMIF-White-Paper-1.0.pdf
https://worldmedicalinnovation.org/agenda/
Virtual | May 19–21, 2021
#WMIF2021
@MGBInnovation
Leaders in Pharmaceutical Business Intelligence (LPBI) Group
will cover the event in Real Time
Aviva Lev-Ari, PhD, RN
Founder LPBI 1.0 & LPBI 2.0
will be in virtual attendance producing the e-Proceedings
and the Tweet Collection of this Global event expecting +15,000 attendees
@pharma_BI
@AVIVA1950
LPBI’s Eighteen Books in Medicine
https://lnkd.in/ekWGNqA
Among them, books on Gene and Cell Therapy include the following:
Topics for May 19 – 21 include:
Impact on Patient Care – Therapeutic and Potentially Curative GCT Developments
GCT Delivery, Manufacturing – What’s Next
GCT Platform Development
Oncolytic Viruses – Cancer applications, start-ups
Regenerative Medicine/Stem Cells
Future of CAR-T
M&A Shaping GCT’s Future
Market Priorities
Venture Investing in GCT
China’s GCT Juggernaut
Disease and Patient Focus: Benign blood disorders, diabetes, neurodegenerative diseases
Click here for the current WMIF agenda
Plus:
Fireside Chats: 1:1 interviews with industry CEOs/C-Suite leaders including Novartis Gene Therapies, ThermoFisher, Bayer AG, FDA
First Look: 18 briefings on emerging GCT research from Mass General Brigham scientists
Virtual Poster Session: 40 research posters and presenters on potential GCT discoveries from Mass General Brigham
Announcement of the Disruptive Dozen, 12 GCT technologies likely to break through in the next few years
AGENDA
Wednesday, May 19, 2021
8:00 AM – 8:10 AMOpening Remarks
Welcome and the vision for Gene and Cell Therapy and why it is a top Mass General Brigham priority.
- Introducer:
- Presenter:
-
May 22,23,24, 2022 – in Boston, in-person 2022 WMIF on CGT
8:10 AM – 8:30 AMThe Grand Challenge of Widespread GCT Patient Benefits
Co-Chairs identify the key themes of the Forum – set the stage for top GCT opportunities, challenges, and where the field might take medicine in the future.
- Moderator:
- Speakers:
-
Oncolytic virus triple threat: Toxic, immunological, combine with anti cancer therapies
Polygenic therapy – multiple genes involved, plug-play,
8:35 AM – 8:50 AMFIRESIDEGene and Cell Therapy 2.0 – What’s Next as We Realize their Potential for Patients
- Moderator:
- Speaker:
-
Q&A
8:55 AM – 9:10 AM
8:55 AM – 9:20 AMThe Patient and GCT
GCT development for rare diseases is driven by patient and patient-advocate communities. Understanding their needs and perspectives enables biomarker research, the development of value-driving clinical trial endpoints and successful clinical trials. Industry works with patient communities that help identify unmet needs and collaborate with researchers to conduct disease natural history studies that inform the development of biomarkers and trial endpoints. This panel includes patients who have received cutting-edge GCT therapy as well as caregivers and patient advocates.
- Moderator:
- Speakers:
-
Q&A
9:25 AM – 9:40 AM
9:25 AM – 9:45 AMFIRESIDE9:50 AM – 10:15 AMBuilding a GCT Platform for Mainstream Success
This panel of GCT executives, innovators and investors explore how to best shape a successful GCT strategy. Among the questions to be addressed:
- How are GCT approaches set around defining and building a platform?
- Is AAV the leading modality and what are the remaining challenges?
- What are the alternatives?
- Is it just a matter of matching modalities to the right indications?
- Moderator:
- Speakers:
-
Q&A
10:20 AM – 10:35 AM
10:20 AM – 10:45 AMAAV Success Studies | Retinal Dystrophy | Spinal Muscular Atrophy
Recent AAV gene therapy product approvals have catalyzed the field. This new class of therapies has shown the potential to bring transformative benefit to patients. With dozens of AAV treatments in clinical studies, all eyes are on the field to gauge its disruptive impact.
The panel assesses the largest challenges of the first two products, the lessons learned for the broader CGT field, and the extent to which they serve as a precedent to broaden the AAV modality.
- Is AAV gene therapy restricted to genetically defined disorders, or will it be able to address common diseases in the near term?
- Lessons learned from these first-in-class approvals.
- Challenges to broaden this modality to similar indications.
- Reflections on safety signals in the clinical studies?
- Moderator:
- Speakers:
-
Q&A
10:50 AM – 11:05 AM
10:45 AM – 10:55 AM10:55 AM – 11:05 AMFIRST LOOK11:05 AM – 11:15 AMFIRST LOOK11:20 AM – 11:45 AMHOT TOPICSAAV Delivery
This panel will address the advances in the area of AAV gene therapy delivery looking out the next five years. Questions that loom large are: How can biodistribution of AAV be improved? What solutions are in the wings to address immunogenicity of AAV? Will patients be able to receive systemic redosing of AAV-based gene therapies in the future? What technical advances are there for payload size? Will the cost of manufacturing ever become affordable for ultra-rare conditions? Will non-viral delivery completely supplant viral delivery within the next five years?What are the safety concerns and how will they be addressed?
- Moderators:
- Speakers:
-
Q&A
11:50 AM – 12:05 PM
11:50 AM – 12:15 PMM&A | Shaping GCT Innovation
The GCT M&A market is booming – many large pharmas have made at least one significant acquisition. How should we view the current GCT M&A market? What is its impact of the current M&A market on technology development? Are these M&A trends new are just another cycle? Has pharma strategy shifted and, if so, what does it mean for GCT companies? What does it mean for patients? What are the long-term prospects – can valuations hold up?
- Moderator:
- Speakers:
Debby Baron,
- Worldwide Business Development, Pfizer
CGT is an important area Pfizer is active looking for innovators, advancing forward programs of innovation with the experience Pfizer has internally
Scalability and manufacturing regulatory conversations, clinical programs safety in parallel to planning getting drug to patients
- Vice President, Business Development and Lilly New Ventures, Eli Lilly and Company
Head of Strategy, Business Development & Licensing, and Member of the Executive Committee, Bayer
Absolute Leadership in Gene editing, gene therapy, via acquisition and strategic alliance
Operating model of the acquired company discussed , company continue independence
- Board Chairman, Encoded Therapeutics & Affinia
Executive Chairman, Jaguar Gene Therapy & Istari Oncology
As acquiree multiple M&A: How the acquirer looks at integration and cultures of the two companies
Traditional integration vs jump start by external acquisition
AAV – epilepsy, next generation of vectors
-
Q&A
12:20 PM – 12:35 PM
12:15 PM – 12:25 PMFIRST LOOK12:25 PM – 12:35 PMFIRST LOOK12:35 PM – 1:15 PM1:15 PM – 1:40 PMOncolytic Viruses in Cancer | Curing Melanoma and Beyond
Oncolytic viruses represent a powerful new technology, but so far an FDA-approved oncolytic (Imlygic) has only occurred in one area – melanoma and that what is in 2015. This panel involves some of the protagonists of this early success story. They will explore why and how Imlygic became approved and its path to commercialization. Yet, no other cancer indications exist for Imlygic, unlike the expansion of FDA-approved indication for immune checkpoint inhibitors to multiple cancers. Why? Is there a limitation to what and which cancers can target? Is the mode of administration a problem?
No other oncolytic virus therapy has been approved since 2015. Where will the next success story come from and why? Will these therapies only be beneficial for skin cancers or other easily accessible cancers based on intratumoral delivery?
The panel will examine whether the preclinical models that have been developed for other cancer treatment modalities will be useful for oncolytic viruses. It will also assess the extent pre-clinical development challenges have slowed the development of OVs.
- Moderator:
- Speakers:
-
Q&A
1:45 PM – 2:00 PM
1:45 PM – 2:10 PMMarket Interest in Oncolytic Viruses | Calibrating
There are currently two oncolytic virus products on the market, one in the USA and one in China. As of late 2020, there were 86 clinical trials 60 of which were in phase I with just 2 in Phase III the rest in Phase I/II or Phase II. Although global sales of OVs are still in the ramp-up phase, some projections forecast OVs will be a $700 million market by 2026. This panel will address some of the major questions in this area:
What regulatory challenges will keep OVs from realizing their potential? Despite the promise of OVs for treating cancer only one has been approved in the US. Why has this been the case? Reasons such have viral tropism, viral species selection and delivery challenges have all been cited. However, these are also true of other modalities. Why then have oncolytic virus approaches not advanced faster and what are the primary challenges to be overcome?
- Will these need to be combined with other agents to realize their full efficacy and how will that impact the market?
- Why are these companies pursuing OVs while several others are taking a pass?
- Moderators:
- Speakers:
-
- Anlong Li, MD, PhD
- Jeffrey Infante, MD
- Loic Vincent, PhD
-
Q&A
2:15 PM – 2:30 PM
2:10 PM – 2:20 PMFIRST LOOK2:20 PM – 2:45 PMEntrepreneurial Growth | Oncolytic Virus
In 2020 there were a total of 60 phase I trials for Oncolytic Viruses. There are now dozens of companies pursuing some aspect of OV technology. This panel will address:
- How are small companies equipped to address the challenges of developing OV therapies better than large pharma or biotech?
- Will the success of COVID vaccines based on Adenovirus help the regulatory environment for small companies developing OV products in Europe and the USA?
- Is there a place for non-viral delivery and other immunotherapy companies to engage in the OV space? Would they bring any real advantages?
- Moderator:
- Speakers:
-
Q&A
2:50 PM – 3:05 PM
2:45 PM – 3:00 PM3:00 PM – 3:25 PMCAR-T | Lessons Learned | What’s Next
Few areas of potential cancer therapy have had the attention and excitement of CAR-T. This panel of leading executives, developers, and clinician-scientists will explore the current state of CAR-T and its future prospects. Among the questions to be addressed are:
- Is CAR-T still an industry priority – i.e. are new investments being made by large companies? Are new companies being financed? What are the trends?
- What have we learned from first-generation products, what can we expect from CAR-T going forward in novel targets, combinations, armored CAR’s and allogeneic treatment adoption?
- Early trials showed remarkable overall survival and progression-free survival. What has been observed regarding how enduring these responses are?
- Most of the approvals to date have targeted CD19, and most recently BCMA. What are the most common forms of relapses that have been observed?
- Is there a consensus about what comes after these CD19 and BCMA trials as to additional targets in liquid tumors? How have dual-targeted approaches fared?
- Moderator:
-
Is CAR-T Industry priority
- Speakers:
- Head of R&D, Atara BioTherapeutics
- Phyno-type of the cells for hematologic cancers
- solid tumor
- inventory of Therapeutics for treating patients in the future
- Progressive MS program
- EBBT platform B-Cells and T-Cells
-
- Stefan Hendriks
- Gobal Head, Cell & Gene, Novartis
- yes, CGT is a strategy in the present and future
- Journey started years ago
- Confirmation the effectiveness of CAR-T therapies, 1 year response prolonged to 5 years 26 months
- Patient not responding – a lot to learn
- Patient after 8 months of chemo can be helped by CAR-T
- Christi Shaw
- CEO, Kite
- CAR-T is priority 120 companies in the space
- Manufacturing consistency
- Patients respond with better quality of life
- Blood cancer – more work to be done
- Stefan Hendriks
-
3:30 PM – 3:45 PM
3:30 PM – 3:55 PMHOT TOPICSCAR-T | Solid Tumors Success | When?
The potential application of CAR-T in solid tumors will be a game-changer if it occurs. The panel explores the prospects of solid tumor success and what the barriers have been. Questions include:
- How would industry and investor strategy for CAR-T and solid tumors be characterized? Has it changed in the last couple of years?
- Does the lack of tumor antigen specificity in solid tumors mean that lessons from liquid tumor CAR-T constructs will not translate well and we have to start over?
- Whether due to antigen heterogeneity, a hostile tumor micro-environment, or other factors are some specific solid tumors more attractive opportunities than others for CAR-T therapy development?
- Given the many challenges that CAR-T faces in solid tumors, does the use of combination therapies from the start, for example, to mitigate TME effects, offer a more compelling opportunity.
- Moderator:
- Speakers:
-
Q&A
4:00 PM – 4:15 PM
4:00 PM – 4:25 PMGCT Manufacturing | Vector Production | Autologous and Allogeneic | Stem Cells | Supply Chain | Scalability & Management
The modes of GCT manufacturing have the potential of fundamentally reordering long-established roles and pathways. While complexity goes up the distance from discovery to deployment shrinks. With the likelihood of a total market for cell therapies to be over $48 billion by 2027, groups of products are emerging. Stem cell therapies are projected to be $28 billion by 2027 and non-stem cell therapies such as CAR-T are projected be $20 billion by 2027. The manufacturing challenges for these two large buckets are very different. Within the CAR-T realm there are diverging trends of autologous and allogeneic therapies and the demands on manufacturing infrastructure are very different. Questions for the panelists are:
- Help us all understand the different manufacturing challenges for cell therapies. What are the trade-offs among storage cost, batch size, line changes in terms of production cost and what is the current state of scaling naïve and stem cell therapy treatment vs engineered cell therapies?
- For cell and gene therapy what is the cost of Quality Assurance/Quality Control vs. production and how do you think this will trend over time based on your perspective on learning curves today?
- Will point of care production become a reality? How will that change product development strategy for pharma and venture investors? What would be the regulatory implications for such products?
- How close are allogeneic CAR-T cell therapies? If successful what are the market implications of allogenic CAR-T? What are the cost implications and rewards for developing allogeneic cell therapy treatments?
- Moderator:
-
- Michael Paglia
- Speakers:
-
- Dannielle Appelhans
- SVP TechOps and Chief Technical Officer, Novartis Gene Therapies
- Thomas Page, PhD
- VP, Engineering and Asset Development, FUJIFILM Diosynth Biotechnologies
- Rahul Singhvi, ScD
- CEO and Co-Founder, National Resilience, Inc.
- Thomas VanCott, PhD
- Global Head of Product Development, Gene & Cell Therapy, Catalent
- 2/3 autologous 1/3 allogeneic CAR-T high doses and high populations scale up is not done today quality maintain required the timing logistics issues centralized vs decentralized allogeneic are health donors innovations in cell types in use improvements in manufacturing
Ropa Pike, Director, Enterprise Science & Partnerships, Thermo Fisher Scientific
Centralized biopharma industry is moving to decentralized models site specific license
- Dannielle Appelhans
-
Q&A
4:30 PM – 4:45 PM
4:30 PM – 4:40 PMFIRST LOOK4:40 PM – 4:50 PMFIRST LOOK4:50 PM – 5:00 PMFIRST LOOK5:00 PM – 5:20 PMFIRESIDE5:20 PM – 5:30 PMThursday, May 20, 2021
8:00 AM – 8:25 AMGCT | The China Juggernaut
China embraced gene and cell therapies early. The first China gene therapy clinical trial was in 1991. China approved the world’s first gene therapy product in 2003—Gendicine—an oncolytic adenovirus for the treatment of advanced head and neck cancer. Driven by broad national strategy, China has become a hotbed of GCT development, ranking second in the world with more than 1,000 clinical trials either conducted or underway and thousands of related patents. It has a booming GCT biotech sector, led by more than 45 local companies with growing IND pipelines.
In late 1990, a T cell-based immunotherapy, cytokine-induced killer (CIK) therapy became a popular modality in the clinic in China for tumor treatment. In early 2010, Chinese researchers started to carry out domestic CAR T trials inspired by several important reports suggested the great antitumor function of CAR T cells. Now, China became the country with the most registered CAR T trials, CAR T therapy is flourishing in China.
The Chinese GCT ecosystem has increasingly rich local innovation and growing complement of development and investment partnerships – and also many subtleties.
This panel, consisting of leaders from the China GCT corporate, investor, research and entrepreneurial communities, will consider strategic questions on the growth of the gene and cell therapy industry in China, areas of greatest strength, evolving regulatory framework, early successes and products expected to reach the US and world market.
- Moderator:
- Speakers:
-
Q&A
8:30 AM – 8:45 AM
8:30 AM – 8:55 AMImpact of mRNA Vaccines | Global Success Lessons
The COVID vaccine race has propelled mRNA to the forefront of biomedicine. Long considered as a compelling modality for therapeutic gene transfer, the technology may have found its most impactful application as a vaccine platform. Given the transformative industrialization, the massive human experience, and the fast development that has taken place in this industry, where is the horizon? Does the success of the vaccine application, benefit or limit its use as a therapeutic for CGT?
- How will the COVID success impact the rest of the industry both in therapeutic and prophylactic vaccines and broader mRNA lessons?
- How will the COVID success impact the rest of the industry both on therapeutic and prophylactic vaccines and broader mRNA lessons?
- Beyond from speed of development, what aspects make mRNA so well suited as a vaccine platform?
- Will cost-of-goods be reduced as the industry matures?
- How does mRNA technology seek to compete with AAV and other gene therapy approaches?
- Moderator:
- Speakers:
-
- Melissa Moore
- Chief Scientific Officer, Moderna
Many years of mRNA pivoting for new diseases, DARPA, Nucleic Acids global deployment of a manufacturing unit on site where the need arise. Elan Musk funds new directions at Moderna
How many mRNA can be put in one vaccine: Dose and tolerance to achieve efficacy
45 days for Personalized cancer vaccine one per patient
- Ron Renaud
- CEO, Translate Bio
1.6 Billion doses produced rare disease monogenic correct mRNA like CF multiple mutation infection disease and oncology applications
Platform allowing to swap cargo reusing same nanoparticles address disease beyond Big Pharma options for biotech
WHat strain of Flu vaccine will come back in the future when people do not use masks
- Kate Bingham, UK Vaccine Taskforce
July 2020, AAV vs mRNA delivery across UK local centers administered both types supply and delivery uplift
- Melissa Moore
-
Q&A
9:00 AM – 9:15 AM
9:00 AM – 9:25 AMHOT TOPICSBenign Blood Disorders
Hemophilia has been and remains a hallmark indication for the CGT. Given its well-defined biology, larger market, and limited need for gene transfer to provide therapeutic benefit, it has been at the forefront of clinical development for years, however, product approval remains elusive. What are the main hurdles to this success? Contrary to many indications that CGT pursues no therapeutic options are available to patients, hemophiliacs have an increasing number of highly efficacious treatment options. How does the competitive landscape impact this field differently than other CGT fields? With many different players pursuing a gene therapy option for hemophilia, what are the main differentiators? Gene therapy for hemophilia seems compelling for low and middle-income countries, given the cost of currently available treatments; does your company see opportunities in this market?
- Moderator:
- Speakers:
-
Q&A
9:30 AM – 9:45 AM
9:25 AM – 9:35 AMFIRST LOOK9:35 AM – 9:45 AMFIRST LOOK9:50 AM – 10:15 AMHOT TOPICSDiabetes | Grand Challenge
The American Diabetes Association estimates 30 million Americans have diabetes and 1.5 million are diagnosed annually. GCT offers the prospect of long-sought treatment for this enormous cohort and their chronic requirements. The complexity of the disease and its management constitute a grand challenge and highlight both the potential of GCT and its current limitations.
- Islet transplantation for type 1 diabetes has been attempted for decades. Problems like loss of transplanted islet cells due to autoimmunity and graft site factors have been difficult to address. Is there anything different on the horizon for gene and cell therapies to help this be successful?
- How is the durability of response for gene or cell therapies for diabetes being addressed? For example, what would the profile of an acceptable (vs. optimal) cell therapy look like?
- Moderator:
- Speakers:
-
Q&A
10:20 AM – 10:35 AM
10:20 AM – 10:40 AMFIRESIDE10:40 AM – 10:50 AM10:50 AM – 11:00 AMFIRST LOOKGetting to the Heart of the Matter: Curing Genetic Cardiomyopathy
- Christine Seidman, MD
- Director, Cardiovascular Genetics Center, BWH
- Smith Professor of Medicine & Genetics, HMS
The Voice of Dr. Seidman – Her abstract is cited below
The ultimate opportunity presented by discovering the genetic basis of human disease is accurate prediction and disease prevention. To enable this achievement, genetic insights must enable the identification of at-risk
individuals prior to end-stage disease manifestations and strategies that delay or prevent clinical expression. Genetic cardiomyopathies provide a paradigm for fulfilling these opportunities. Hypertrophic cardiomyopathy (HCM) is characterized by left ventricular hypertrophy, diastolic dysfunction with normal or enhanced systolic performance and a unique histopathology: myocyte hypertrophy, disarray and fibrosis. Dilated cardiomyopathy (DCM) exhibits enlarged ventricular volumes with depressed systolic performance and nonspecific histopathology. Both HCM and DCM are prevalent clinical conditions that increase risk for arrhythmias, sudden death, and heart failure. Today treatments for HCM and DCM focus on symptoms, but none prevent disease progression. Human molecular genetic studies demonstrated that these pathologies often result from dominant mutations in genes that encode protein components of the sarcomere, the contractile unit in striated muscles. These data combined with the emergence of molecular strategies to specifically modulate gene expression provide unparalleled opportunities to silence or correct mutant genes and to boost healthy gene expression in patients with genetic HCM and DCM. Many challenges remain, but the active and vital efforts of physicians, researchers, and patients are poised to ensure success.
-
Q&A
11:00 AM – 11:20 AM
11:00 AM – 11:10 AMFIRST LOOK11:10 AM – 11:35 AMRare and Ultra Rare Diseases | GCT Breaks Through
One of the most innovative segments in all of healthcare is the development of GCT driven therapies for rare and ultra-rare diseases. Driven by a series of insights and tools and funded in part by disease focused foundations, philanthropists and abundant venture funding disease after disease is yielding to new GCT technology. These often become platforms to address more prevalent diseases. The goal of making these breakthroughs routine and affordable is challenged by a range of issues including clinical trial design and pricing.
- What is driving the interest in rare diseases?
- What are the biggest barriers to making breakthroughs ‘routine and affordable?’
- What is the role of retrospective and prospective natural history studies in rare disease? When does the expected value of retrospective disease history studies justify the cost?
- Related to the first question, what is the FDA expecting as far as controls in clinical trials for rare diseases? How does this impact the collection of natural history data?
- Moderator:
- Speakers:
-
Do 100 patient study then have information on natural history to develop a clinical trial
-
Q&A
11:40 AM – 11:55 AM
11:40 AM – 12:00 PMFIRESIDE12:05 PM – 12:30 PMCEO Panel | Anticipating Disruption | Planning for Widespread GCT
The power of GCT to cure disease has the prospect of profoundly improving the lives of patients who respond. Planning for a disruption of this magnitude is complex and challenging as it will change care across the spectrum. Leading chief executives shares perspectives on how the industry will change and how this change should be anticipated.
- Moderator:
- Speakers:
-
Q&A
12:35 PM – 12:50 PM
12:35 PM – 12:55 PMFIRESIDEBuilding a GCT Portfolio
GCT represents a large and growing market for novel therapeutics that has several segments. These include Cardiovascular Disease, Cancer, Neurological Diseases, Infectious Disease, Ophthalmology, Benign Blood Disorders, and many others; Manufacturing and Supply Chain including CDMO’s and CMO’s; Stem Cells and Regenerative Medicine; Tools and Platforms (viral vectors, nano delivery, gene editing, etc.). Bayer’s pharma business participates in virtually all of these segments. How does a Company like Bayer approach the development of a portfolio in a space as large and as diverse as this one? How does Bayer approach the support of the production infrastructure with unique demands and significant differences from its historical requirements?
- Moderator:
-
- Speaker:
-
Q&A
1:00 PM – 1:15 PM
12:55 PM – 1:35 PM1:40 PM – 2:05 PMGCT Delivery | Perfecting the Technology
Gene delivery uses physical, chemical, or viral means to introduce genetic material into cells. As more genetically modified therapies move closer to the market, challenges involving safety, efficacy, and manufacturing have emerged. Optimizing lipidic and polymer nanoparticles and exosomal delivery is a short-term priority. This panel will examine how the short-term and long-term challenges are being tackled particularly for non-viral delivery modalities.
- Moderator:
- Speakers:
-
Q&A
2:10 PM – 2:25 PM
2:05 PM – 2:10 PM2:10 PM – 2:20 PMFIRST LOOK2:20 PM – 2:30 PMFIRST LOOK2:55 PM – 3:20 PMHOT TOPICSGene Editing | Achieving Therapeutic Mainstream
Gene editing was recognized by the Nobel Committee as “one of gene technology’s sharpest tools, having a revolutionary impact on life sciences.” Introduced in 2011, gene editing is used to modify DNA. It has applications across almost all categories of disease and is also being used in agriculture and public health.
Today’s panel is made up of pioneers who represent foundational aspects of gene editing. They will discuss the movement of the technology into the therapeutic mainstream.
- Successes in gene editing – lessons learned from late-stage assets (sickle cell, ophthalmology)
- When to use what editing tool – pros and cons of traditional gene-editing v. base editing. Is prime editing the future? Specific use cases for epigenetic editing.
- When we reach widespread clinical use – role of off-target editing – is the risk real? How will we mitigate? How practical is patient-specific off-target evaluation?
- Moderator:
- Speakers:
-
Q&A
3:25 PM – 3:50 PM
3:25 PM – 3:50 PMHOT TOPICSCommon Blood Disorders | Gene Therapy
There are several dozen companies working to develop gene or cell therapies for Sickle Cell Disease, Beta Thalassemia, and Fanconi Anemia. In some cases, there are enzyme replacement therapies that are deemed effective and safe. In other cases, the disease is only managed at best. This panel will address a number of questions that are particular to this class of genetic diseases:
- What are the pros and cons of various strategies for treatment? There are AAV-based editing, non-viral delivery even oligonucleotide recruitment of endogenous editing/repair mechanisms. Which approaches are most appropriate for which disease?
- How can companies increase the speed of recruitment for clinical trials when other treatments are available? What is the best approach to educate patients on a novel therapeutic?
- How do we best address ethnic and socio-economic diversity to be more representative of the target patient population?
- How long do we have to follow up with the patients from the scientific, patient’s community, and payer points of view? What are the current FDA and EMA guidelines for long-term follow-up?
- Where are we with regards to surrogate endpoints and their application to clinically meaningful endpoints?
- What are the emerging ethical dilemmas in pediatric gene therapy research? Are there challenges with informed consent and pediatric assent for trial participation?
- Are there differences in reimbursement policies for these different blood disorders? Clearly durability of response is a big factor. Are there other considerations?
- Moderator:
- Speakers:
-
Q&A
3:55 PM – 4:15 PM
3:50 PM – 4:00 PMFIRST LOOK4:20 PM – 4:45 PMHOT TOPICSGene Expression | Modulating with Oligonucleotide-Based Therapies
Oligonucleotide drugs have recently come into their own with approvals from companies such as Biogen, Alnylam, Novartis and others. This panel will address several questions:
How important is the delivery challenge for oligonucleotides? Are technological advancements emerging that will improve the delivery of oligonucleotides to the CNS or skeletal muscle after systemic administration?
- Will oligonucleotides improve as a class that will make them even more effective? Are further advancements in backbone chemistry anticipated, for example.
- Will oligonucleotide based therapies blaze trails for follow-on gene therapy products?
- Are small molecules a threat to oligonucleotide-based therapies?
- Beyond exon skipping and knock-down mechanisms, what other roles will oligonucleotide-based therapies take mechanistically — can genes be activating oligonucleotides? Is there a place for multiple mechanism oligonucleotide medicines?
- Are there any advantages of RNAi-based oligonucleotides over ASOs, and if so for what use?
- Moderator:
- Speakers:
-
Q&A
4:50 PM – 5:05 PM
4:45 PM – 4:55 PMFIRST LOOKFriday, May 21, 2021
8:30 AM – 8:55 AMVenture Investing | Shaping GCT Translation
What is occurring in the GCT venture capital segment? Which elements are seeing the most activity? Which areas have cooled? How is the investment market segmented between gene therapy, cell therapy and gene editing? What makes a hot GCT company? How long will the market stay frothy? Some review of demographics — # of investments, sizes, etc. Why is the market hot and how long do we expect it to stay that way? Rank the top 5 geographic markets for GCT company creation and investing? Are there academic centers that have been especially adept at accelerating GCT outcomes? Do the business models for the rapid development of coronavirus vaccine have any lessons for how GCT technology can be brought to market more quickly?
- Moderator:
- Speakers:
-
- David Berry, MD, PhD
- CEO, Valo Health
- General Partner, Flagship Pioneering
Bring disruptive frontier as a platform with reliable delivery CGT double knock out disease cure all change efficiency and scope human centric vs mice centered right scale of data converted into therapeutics acceleratetion
Innovation in drugs 60% fails in trial because of Toxicology system of the future deal with big diseases
Moderna is an example in unlocking what is inside us Microbiome and beyond discover new drugs epigenetics
- Robert Nelsen
- Managing Director, Co-founder, ARCH Venture Partners
Manufacturing change is not a new clinical trial FDA need to be presented with new rethinking for big innovations Drug pricing cheaper requires systematization How to systematically scaling up systematize the discovery and the production regulatory innovations
- Kush Parmar, MD, PhD
- Managing Partner, 5AM Ventures
Responsibility mismatch should be and what is “are”
Long term diseases Stack holders and modalities risk benefir for populations
- David Berry, MD, PhD
- Speakers:
-
Q&A
4:50 PM – 5:05 PM
4:45 PM – 4:55 PMFIRST LOOKFriday, May 21, 2021
8:30 AM – 8:55 AMVenture Investing | Shaping GCT Translation
What is occurring in the GCT venture capital segment? Which elements are seeing the most activity? Which areas have cooled? How is the investment market segmented between gene therapy, cell therapy and gene editing? What makes a hot GCT company? How long will the market stay frothy? Some review of demographics — # of investments, sizes, etc. Why is the market hot and how long do we expect it to stay that way? Rank the top 5 geographic markets for GCT company creation and investing? Are there academic centers that have been especially adept at accelerating GCT outcomes? Do the business models for the rapid development of coronavirus vaccine have any lessons for how GCT technology can be brought to market more quickly?
- Moderator:
- Speakers:
-
Q&A
9:00 AM – 9:15 AM
9:00 AM – 9:25 AMRegenerative Medicine | Stem Cells
The promise of stem cells has been a highlight in the realm of regenerative medicine. Unfortunately, that promise remains largely in the future. Recent breakthroughs have accelerated these potential interventions in particular for treating neurological disease. Among the topics the panel will consider are:
- Stem cell sourcing
- Therapeutic indication growth
- Genetic and other modification in cell production
- Cell production to final product optimization and challenges
- How to optimize the final product
- Moderator:
- Speakers:
-
Q&A
9:30 AM – 9:45 AM
9:25 AM – 9:35 AMFIRST LOOK9:35 AM – 10:00 AMCapital Formation ’21-30 | Investing Modes Driving GCT Technology and Timing
The dynamics of venture/PE investing and IPOs are fast evolving. What are the drivers – will the number of investors grow will the size of early rounds continue to grow? How is this reflected in GCT target areas, company design, and biotech overall? Do patients benefit from these trends? Is crossover investing a distinct class or a little of both? Why did it emerge and what are the characteristics of the players? Will SPACs play a role in the growth of the gene and cell therapy industry. What is the role of corporate investment arms eg NVS, Bayer, GV, etc. – has a category killer emerged? Are we nearing the limit of what the GCT market can absorb or will investment capital continue to grow unabated?
- Moderator:
- Speakers:
-
Q&A
10:05 AM – 10:20 AM
10:00 AM – 10:10 AMFIRST LOOK10:10 AM – 10:35 AMHOT TOPICSNeurodegenerative Clinical Outcomes | Achieving GCT Success
Can stem cell-based platforms become successful treatments for neurodegenerative diseases?
- What are the commonalities driving GCT success in neurodegenerative disease and non-neurologic disease, what are the key differences?
- Overcoming treatment administration challenges
- GCT impact on degenerative stage of disease
- How difficult will it be to titrate the size of the cell therapy effect in different neurological disorders and for different patients?
- Demonstrating clinical value to patients and payers
- Revised clinical trial models to address issues and concerns specific to GCT
- Moderator:
- Speakers:
-
Q&A
10:40 AM – 10:55 AM
10:35 AM – 11:35 AMDisruptive Dozen: 12 Technologies that Will Reinvent GCT
Nearly one hundred senior Mass General Brigham Harvard faculty contributed to the creation of this group of twelve GCT technologies that they believe will breakthrough in the next two years. The Disruptive Dozen identifies and ranks the GCT technologies that will be available on at least an experimental basis to have the chance of significantly improving health care.
11:35 AM – 11:45 AMConcluding Remarks
The co-chairs convene to reflect on the insights shared over the three days. They will discuss what to expect at the in-person GCT focused May 2-4, 2022 World Medical Innovation Forum.
Hypertrophic and Dilated Cardiomyopaies ‘
10% receive heart transplant 12 years survival
Mutation puterb function
TTN: contribute 20% of dilated cardiomyopaty
Silence gene
pleuripotential cells deliver therapies
-
Q&A
11:00 AM – 11:20 AM
Rare and Ultra Rare Diseases | GCT Breaks Through
One of the most innovative segments in all of healthcare is the development of GCT driven therapies for rare and ultra-rare diseases. Driven by a series of insights and tools and funded in part by disease focused foundations, philanthropists and abundant venture funding disease after disease is yielding to new GCT technology. These often become platforms to address more prevalent diseases. The goal of making these breakthroughs routine and affordable is challenged by a range of issues including clinical trial design and pricing.
- What is driving the interest in rare diseases?
- What are the biggest barriers to making breakthroughs ‘routine and affordable?’
- What is the role of retrospective and prospective natural history studies in rare disease? When does the expected value of retrospective disease history studies justify the cost?
- Related to the first question, what is the FDA expecting as far as controls in clinical trials for rare diseases? How does this impact the collection of natural history data?
- Moderator:
- Speakers:
-
Do 100 patient study then have information on natural history to develop a clinical trial
-
Q&A
11:40 AM – 11:55 AM
CEO Panel | Anticipating Disruption | Planning for Widespread GCT
The power of GCT to cure disease has the prospect of profoundly improving the lives of patients who respond. Planning for a disruption of this magnitude is complex and challenging as it will change care across the spectrum. Leading chief executives shares perspectives on how the industry will change and how this change should be anticipated.
- Moderator:
- Speakers:
-
Q&A
12:35 PM – 12:50 PM
Building a GCT Portfolio
GCT represents a large and growing market for novel therapeutics that has several segments. These include Cardiovascular Disease, Cancer, Neurological Diseases, Infectious Disease, Ophthalmology, Benign Blood Disorders, and many others; Manufacturing and Supply Chain including CDMO’s and CMO’s; Stem Cells and Regenerative Medicine; Tools and Platforms (viral vectors, nano delivery, gene editing, etc.). Bayer’s pharma business participates in virtually all of these segments. How does a Company like Bayer approach the development of a portfolio in a space as large and as diverse as this one? How does Bayer approach the support of the production infrastructure with unique demands and significant differences from its historical requirements?
- Moderator:
-
- Speaker:
-
Q&A
1:00 PM – 1:15 PM
GCT Delivery | Perfecting the Technology
Gene delivery uses physical, chemical, or viral means to introduce genetic material into cells. As more genetically modified therapies move closer to the market, challenges involving safety, efficacy, and manufacturing have emerged. Optimizing lipidic and polymer nanoparticles and exosomal delivery is a short-term priority. This panel will examine how the short-term and long-term challenges are being tackled particularly for non-viral delivery modalities.
- Moderator:
- Speakers:
-
Q&A
2:10 PM – 2:25 PM
Gene Editing | Achieving Therapeutic Mainstream
Gene editing was recognized by the Nobel Committee as “one of gene technology’s sharpest tools, having a revolutionary impact on life sciences.” Introduced in 2011, gene editing is used to modify DNA. It has applications across almost all categories of disease and is also being used in agriculture and public health.
Today’s panel is made up of pioneers who represent foundational aspects of gene editing. They will discuss the movement of the technology into the therapeutic mainstream.
- Successes in gene editing – lessons learned from late-stage assets (sickle cell, ophthalmology)
- When to use what editing tool – pros and cons of traditional gene-editing v. base editing. Is prime editing the future? Specific use cases for epigenetic editing.
- When we reach widespread clinical use – role of off-target editing – is the risk real? How will we mitigate? How practical is patient-specific off-target evaluation?
- Moderator:
- Speakers:
-
Q&A
3:25 PM – 3:50 PM
Common Blood Disorders | Gene Therapy
There are several dozen companies working to develop gene or cell therapies for Sickle Cell Disease, Beta Thalassemia, and Fanconi Anemia. In some cases, there are enzyme replacement therapies that are deemed effective and safe. In other cases, the disease is only managed at best. This panel will address a number of questions that are particular to this class of genetic diseases:
- What are the pros and cons of various strategies for treatment? There are AAV-based editing, non-viral delivery even oligonucleotide recruitment of endogenous editing/repair mechanisms. Which approaches are most appropriate for which disease?
- How can companies increase the speed of recruitment for clinical trials when other treatments are available? What is the best approach to educate patients on a novel therapeutic?
- How do we best address ethnic and socio-economic diversity to be more representative of the target patient population?
- How long do we have to follow up with the patients from the scientific, patient’s community, and payer points of view? What are the current FDA and EMA guidelines for long-term follow-up?
- Where are we with regards to surrogate endpoints and their application to clinically meaningful endpoints?
- What are the emerging ethical dilemmas in pediatric gene therapy research? Are there challenges with informed consent and pediatric assent for trial participation?
- Are there differences in reimbursement policies for these different blood disorders? Clearly durability of response is a big factor. Are there other considerations?
- Moderator:
- Speakers:
-
Q&A
3:55 PM – 4:15 PM
Gene Expression | Modulating with Oligonucleotide-Based Therapies
Oligonucleotide drugs have recently come into their own with approvals from companies such as Biogen, Alnylam, Novartis and others. This panel will address several questions:
How important is the delivery challenge for oligonucleotides? Are technological advancements emerging that will improve the delivery of oligonucleotides to the CNS or skeletal muscle after systemic administration?
- Will oligonucleotides improve as a class that will make them even more effective? Are further advancements in backbone chemistry anticipated, for example.
- Will oligonucleotide based therapies blaze trails for follow-on gene therapy products?
- Are small molecules a threat to oligonucleotide-based therapies?
- Beyond exon skipping and knock-down mechanisms, what other roles will oligonucleotide-based therapies take mechanistically — can genes be activating oligonucleotides? Is there a place for multiple mechanism oligonucleotide medicines?
- Are there any advantages of RNAi-based oligonucleotides over ASOs, and if so for what use?
- Moderator:
- Speakers:
-
Q&A
4:50 PM – 5:05 PM
Friday, May 21, 2021
Computer connection to the iCloud of WordPress.com FROZE completely at 10:30AM EST and no file update was possible. COVERAGE OF MAY 21, 2021 IS RECORDED BELOW FOLLOWING THE AGENDA BY COPY AN DPASTE OF ALL THE TWEETS I PRODUCED ON MAY 21, 2021
Venture Investing | Shaping GCT Translation
What is occurring in the GCT venture capital segment? Which elements are seeing the most activity? Which areas have cooled? How is the investment market segmented between gene therapy, cell therapy and gene editing? What makes a hot GCT company? How long will the market stay frothy? Some review of demographics — # of investments, sizes, etc. Why is the market hot and how long do we expect it to stay that way? Rank the top 5 geographic markets for GCT company creation and investing? Are there academic centers that have been especially adept at accelerating GCT outcomes? Do the business models for the rapid development of coronavirus vaccine have any lessons for how GCT technology can be brought to market more quickly?
- Moderator:
- Speakers:
-
Q&A
9:00 AM – 9:15 AM
Regenerative Medicine | Stem Cells
The promise of stem cells has been a highlight in the realm of regenerative medicine. Unfortunately, that promise remains largely in the future. Recent breakthroughs have accelerated these potential interventions in particular for treating neurological disease. Among the topics the panel will consider are:
- Stem cell sourcing
- Therapeutic indication growth
- Genetic and other modification in cell production
- Cell production to final product optimization and challenges
- How to optimize the final product
- Moderator:
- Speakers:
-
Q&A
9:30 AM – 9:45 AM
Capital Formation ’21-30 | Investing Modes Driving GCT Technology and Timing
The dynamics of venture/PE investing and IPOs are fast evolving. What are the drivers – will the number of investors grow will the size of early rounds continue to grow? How is this reflected in GCT target areas, company design, and biotech overall? Do patients benefit from these trends? Is crossover investing a distinct class or a little of both? Why did it emerge and what are the characteristics of the players? Will SPACs play a role in the growth of the gene and cell therapy industry. What is the role of corporate investment arms eg NVS, Bayer, GV, etc. – has a category killer emerged? Are we nearing the limit of what the GCT market can absorb or will investment capital continue to grow unabated?
- Moderator:
- Speakers:
-
Q&A
10:05 AM – 10:20 AM
Neurodegenerative Clinical Outcomes | Achieving GCT Success
Can stem cell-based platforms become successful treatments for neurodegenerative diseases?
- What are the commonalities driving GCT success in neurodegenerative disease and non-neurologic disease, what are the key differences?
- Overcoming treatment administration challenges
- GCT impact on degenerative stage of disease
- How difficult will it be to titrate the size of the cell therapy effect in different neurological disorders and for different patients?
- Demonstrating clinical value to patients and payers
- Revised clinical trial models to address issues and concerns specific to GCT
- Moderator:
- Speakers:
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Q&A
10:40 AM – 10:55 AM
Disruptive Dozen: 12 Technologies that Will Reinvent GCT
Nearly one hundred senior Mass General Brigham Harvard faculty contributed to the creation of this group of twelve GCT technologies that they believe will breakthrough in the next two years. The Disruptive Dozen identifies and ranks the GCT technologies that will be available on at least an experimental basis to have the chance of significantly improving health care.
Concluding Remarks
The co-chairs convene to reflect on the insights shared over the three days. They will discuss what to expect at the in-person GCT focused May 2-4, 2022 World Medical Innovation Forum.
ALL THE TWEETS PRODUCED ON MAY 21, 2021 INCLUDE THE FOLLOWING:
Erwan Bezard, PhD INSERM Research Director, Institute of Neurodegenerative Diseases Cautious on reversal
Nikola Kojic, PhD CEO and Co-Founder, Oryon Cell Therapies Autologus cell therapy placed focal replacing missing synapses reestablishment of neural circutary
Bob Carter, MD, PhD Chairman, Department of Neurosurgery, MGH William and Elizabeth Sweet, Professor of Neurosurgery, HMS Neurogeneration REVERSAL or slowing down?
Penelope Hallett, PhD NRL, McLean Assistant Professor Psychiatry, HMS efficacy Autologous cell therapy transplantation approach program T cells into dopamine genetating cells greater than Allogeneic cell transplantation
Penelope Hallett, PhD NRL, McLean Assistant Professor Psychiatry, HMS Pharmacologic agent in existing cause another disorders locomo-movement related
Roger Kitterman VP, Venture, Mass General Brigham Saturation reached or more investment is coming in CGT Multi OMICS and academia originated innovations are the most attractive areas
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Roger Kitterman VP, Venture, Mass General Brigham Saturation reached or more investment is coming in CGT
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Oleg Nodelman Founder & Managing Partner, EcoR1 Capital Invest in company next round of investment will be IPO 20% discount
Peter Kolchinsky, PhD Founder and Managing Partner, RA Capital Management Future proof for new comers disruptors Ex Vivo gene therapy to improve funding products what tool kit belongs to
Deep Nishar Senior Managing Partner, SoftBank Investment Advisors Young field vs CGT started in the 80s high payloads is a challenge
Bob Carter, MD, PhD MGH, HMS cells producing dopamine transplantation fibroblast cells metabolic driven process lower mutation burden Quercetin inhibition elimination undifferentiated cells graft survival oxygenation increased
Chairman, Department of Neurosurgery, MGH, Professor of Neurosurgery, HMS Cell therapy for Parkinson to replace dopamine producing cells lost ability to produce dopamine skin cell to become autologous cells reprogramed
Kapil Bharti, PhD Senior Investigator, Ocular and Stem Cell Translational Research Section, NIH Off-th-shelf one time treatment becoming cure Intact tissue in a dish is fragile to maintain metabolism to become like semiconductors
Ole Isacson, MD, PhD Director, Neuroregeneration Research Institute, McLean Professor, Neurology and Neuroscience, MGH, HMS Opportunities in the next generation of the tactical level Welcome the oprimism and energy level of all
Erin Kimbrel, PhD Executive Director, Regenerative Medicine, Astellas In the ocular space immunogenecity regulatory communication use gene editing for immunogenecity Cas1 and Cas2 autologous cells
Nabiha Saklayen, PhD CEO and Co-Founder, Cellino scale production of autologous cells foundry using semiconductor process in building cassettes by optic physicists
Joe Burns, PhD VP, Head of Biology, Decibel Therapeutics Ear inside the scall compartments and receptors responsible for hearing highly differentiated tall ask to identify cell for anticipated differentiation control by genomics
Kapil Bharti, PhD Senior Investigator, Ocular and Stem Cell Translational Research Section, NIH first drug required to establish the process for that innovations design of animal studies not done before
Meredith Fisher, PhD Partner, Mass General Brigham Innovation Fund Strategies, success what changes are needed in the drug discovery process@pharma_BI
Robert Nelsen Managing Director, Co-founder, ARCH Venture Partners Manufacturing change is not a new clinical trial FDA need to be presented with new rethinking for big innovations Drug pricing cheaper requires systematization
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Kush Parmar, MD, PhD Managing Partner, 5AM Ventures Responsibility mismatch should be and what is “are”
David Berry, MD, PhD CEO, Valo Health GP, Flagship Pioneering Bring disruptive frontier platform reliable delivery CGT double knockout disease cure all change efficiency scope human centric vs mice centered right scale acceleration
Kush Parmar, MD, PhD Managing Partner, 5AM Ventures build it yourself, benefit for patients FIrst Look at MGB shows MEE innovation on inner ear worthy investment
Robert Nelsen Managing Director, Co-founder, ARCH Venture Partners Frustration with supply chain during the Pandemic, GMC anticipation in advance CGT rapidly prototype rethink and invest proactive investor .edu and Pharma
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Amandeep Kaur
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Adina Hazan
Alan F. Kaul, PharmD., MS, MBA, FCCP
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David Orchard-Webb, PhD
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Demet Sag, Ph.D., CRA, GCP
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Rosalind Codrington, PhD
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Rick Mandahl
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Srinivas Sriram
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Dr. Sudipta Saha
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This is very insightful. There is no doubt that there is the bias you refer to. 42 years ago, when I was postdocing in biochemistry/enzymology before completing my residency in pathology, I knew that there were very influential mambers of the faculty, who also had large programs, and attracted exceptional students. My mentor, it was said (although he was a great writer), could draft a project on toilet paper and call the NIH. It can’t be true, but it was a time in our history preceding a great explosion. It is bizarre for me to read now about eNOS and iNOS, and about CaMKII-á, â, ã, ä – isoenzymes. They were overlooked during the search for the genome, so intermediary metabolism took a back seat. But the work on protein conformation, and on the mechanism of action of enzymes and ligand and coenzyme was just out there, and became more important with the research on signaling pathways. The work on the mechanism of pyridine nucleotide isoenzymes preceded the work by Burton Sobel on the MB isoenzyme in heart. The Vietnam War cut into the funding, and it has actually declined linearly since.
A few years later, I was an Associate Professor at a new Medical School and I submitted a proposal that was reviewed by the Chairman of Pharmacology, who was a former Director of NSF. He thought it was good enough. I was a pathologist and it went to a Biochemistry Review Committee. It was approved, but not funded. The verdict was that I would not be able to carry out the studies needed, and they would have approached it differently. A thousand young investigators are out there now with similar letters. I was told that the Department Chairmen have to build up their faculty. It’s harder now than then. So I filed for and received 3 patents based on my work at the suggestion of my brother-in-law. When I took it to Boehringer-Mannheim, they were actually clueless.