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Posts Tagged ‘Gene Therapy for Inherited Retinal Diseases’


Real Time Coverage @BIOConvention #BIO2019: Gene Therapy 2.0: No Longer Science Fiction 1:00-2:15 pm June 3 Philadelphia PA

Reporter: Stephen J. Williams Ph.D. @StephenJWillia2

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Other Articles on Gene Therapy on this Open Access Journal Include:

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Real Time Coverage @BIOConvention #BIO2019: Chat with @FDA Commissioner, & Challenges in Biotech & Gene Therapy June 4 Philadelphia

Reporter: Stephen J. Williams, PhD @StephenJWillia2

 

  • taking patient concerns and voices from anecdotal to data driven system
  • talked about patient accrual hearing patient voice not only in ease of access but reporting toxicities
  • at FDA he wants to remove barriers to trial access and accrual; also talk earlier to co’s on how they should conduct a trial

Digital tech

  • software as medical device
  • regulatory path is mixed like next gen sequencing
  • wearables are concern for FDA (they need to recruit scientists who know this tech

Opioids

  • must address the crisis but in a way that does not harm cancer pain patients
  • smaller pain packs “blister packs” would be good idea

Clinical trial modernization

  • for Alzheimers disease problem is science
  • for diabetes problem is regulatory
  • different diseases calls for different trial design
  • have regulatory problems with rare diseases as can’t form control or placebo group, inhumane. for example ras tumors trials for MEK inhibitors were narrowly focused on certain ras mutants
Realizing the Promise of Gene Therapies for Patients Around the World

103ABC, Level 100

Speakers
Lots of promise, timeline is progressing faster but we need more education on use of the gene therapy
Regulatory issues: Cell and directly delivered gene based therapies have been now approved. Some challenges will be the ultrarare disease trials and how we address manufacturing issues.  Manufacturing is a big issue at CBER and scalability.  If we want to have global impact of these products we need to address the manufacturing issues
 of scalability.
Pfizer – clinical grade and scale is important.
Aventis – he knew manufacturing of biologics however gene therapy manufacturing has its separate issues and is more complicated especially for regulatory purposes for clinical grade as well as scalability.  Strategic decision: focusing on the QC on manufacturing was so important.  Had a major issue in manufacturing had to shut down and redesign the system.
Albert:  Manufacturing is the most important topic even to the investors.  Investors were really conservative especially seeing early problems but when academic centers figured out good efficacy then they investors felt better and market has exploded.  Now you can see investment into preclinical and startups but still want mature companies to focus on manufacturing.  About $10 billion investment in last 4 years.

How Early is Too Early? Valuing and De-Risking Preclinical Opportunities

109AB, Level 100

Speakers
Valuing early-stage opportunities is challenging. Modeling will often provide a false sense of accuracy but relying on comparable transactions is more art than science. With a long lead time to launch, even the most robust estimates can ultimately prove inaccurate. This interactive panel will feature venture capital investors and senior pharma and biotech executives who lead early-stage transactions as they discuss their approaches to valuing opportunities, and offer key learnings from both successful and not-so-successful experiences.
Dr. Schoenbeck, Pfizer:
  • global network of liaisons who are a dedicated team to research potential global startup partners or investments.  Pfizer has a separate team to evaluate academic laboratories.  In Most cases Pfizer does not initiate contact.  It is important to initiate the first discussion with them in order to get noticed.  Could be just a short chat or discussion on what their needs are for their portfolio.

Question: How early is too early?

Luc Marengere, TVM:  His company has early stage focus, on 1st in class molecules.  The sweet spot for their investment is a candidate selected compound, which should be 12-18 months from IND.  They will want to bring to phase II in less than 4 years for $15-17 million.  Their development model is bad for academic labs.  During this process free to talk to other partners.

Dr. Chaudhary, Biogen:  Never too early to initiate a conversation and sometimes that conversation has lasted 3+ years before a decision.  They like build to buy models, will do convertible note deals, candidate compound selection should be entering in GLP/Tox phase (sweet spot)

Merck: have MRL Venture Fund for pre series A funding.  Also reiterated it is never too early to have that initial discussion.  It will not put you in a throw away bin.  They will have suggestions and never like to throw out good ideas.

Michael Hostetler: Set expectations carefully ; data should be validated by a CRO.  If have a platform, they will look at the team first to see if strong then will look at the platform to see how robust it is.

All noted that you should be completely honest at this phase.  Do not overstate your results or data or overhype your compound(s).  Show them everything and don’t have a bias toward compounds you think are the best in your portfolio.  Sometimes the least developed are the ones they are interested in.  Also one firm may reject you however you may fit in others portfolios better so have a broad range of conversations with multiple players.

 

 

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From Technicall.y Philly.com

Reporter: Stephen J. Williams, PhD

Spark Therapeutics’ $4.8B deal confirmed as biggest-ever VC-backed exit in Philly

Quick update on this week’s news: The University City life sciences company’s acquisition by Swiss pharma giant Roche is the biggest acquisition ever of a VC-backed company within city limits, per PitchBook and PACT.

The eye-popping $4.8 billion sticker price on Spark Therapeutics’acquisition deal with Roche announced on Monday is shaping up to be the largest exit ever within city limits for a venture-backed company, according to data from financial data provider PitchBook and the Philadelphia Alliance for Capital and Technologies (PACT).

“Filtering down to just Philadelphia proper does reveal that Spark Therapeutics, once the deal closes, will be the biggest exit ever for Philly-based venture-backed exits,” the company said in an email, citing data from an upcoming report.

According to the Seattle-based company’s data, the current holder of the largest Philly-proper exit title goes to Avid Radiopharmaceuticals, which in 2010 announced its acquisition by Lilly in a deal valued at up to $800 million.

Founded in 2013, Spark is a publicly traded spinout of Children’s Hospital of Philadelphia (CHOP), which invested $33 million in the company. The Philadelphia Inquirer reports that CHOP stands to reap a total return of $430 million for its minority stake in Spark Therapeutics.

As part of the acquisition deal, the company will remain based out of 3711 Market St., and continue to do business as a standalone Roche company.

“This transaction demonstrates the enormous value that global biotech companies like Roche see in gene therapy, a field in which Philadelphia is the unquestioned leader,” said Saul Behar, senior VP of  advancement and strategic initiatives at the University City Science Center, the West Philly research park where Spark began and grew its operations. “[This] further validates Greater Philadelphia’s status as a biotech hub with a very bright future.”

Spark CEO Jeff Marrazzo said the deep pool of resources from Roche, the company plans to “accelerate the development of more gene therapies for more patients for more diseases and further expedite our vision of a world where no life is limited by genetic disease.”

Other articles on Gene Therapy and Retinal Disease on this Open Access Online Journal include:

Women Leaders in Cell and Gene Therapy

AGTC (AGTC) , An adenoviral gene therapy startup, expands in Florida with help from $1 billion deal with Biogen

Artificial Vision: Cornell and Stanford Researchers crack Retinal Code

D-Eye: a smartphone-based retinal imaging system

 

 

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Top 10 Medical Innovations for 2018 by Cleveland Clinic

Reporter: Aviva Lev-Ari, PhD, RN

 

Top 10 for 2018

#1 Hybrid Closed-Loop Insulin Delivery System

#1 Hybrid Closed-Loop Insulin Delivery System

This approach has not just made T1D management easier than ever, it is also getting praise for stabilizing blood glucose at an unprecedented level.


#2 Neuromodulation to Treat Obstructive Sleep Apnea

#2 Neuromodulation to Treat Obstructive Sleep Apnea

While C.P.A.P. is the gold standard treatment for OSA, the risk of misuse or discontinued use has created an opportunity for innovators to search for a less intrusive way to treat it. The result is ne


#3 Gene Therapy for Inherited Retinal Diseases

#3 Gene Therapy for Inherited Retinal Diseases

In 2018 gene therapy is expected to make its comeback with expected FDA approvals for a variety of inherited retinal diseases (“IRDs”).


#4 The Unprecedented Reduction of LDL Cholesterol

#4 The Unprecedented Reduction of LDL Cholesterol

These new drugs are taking cholesterol to low levels never seen before.


#5 The Emergence of Distance Health

#5 The Emergence of Distance Health

In 2018, the prevalence of connectivity enables distance health.


#6 Next Generation Vaccine Platforms

#6 Next Generation Vaccine Platforms

In 2018, innovators will be upgrading the entire vaccine infrastructure to support the rapid development of new vaccines (a concept that was #1 on the Top 10 Medical Innovations for 2015).


#7 Arsenal of Targeted Breast Cancer Therapies

#7 Arsenal of Targeted Breast Cancer Therapies

For breast cancer patients that are BRCA1 or BRCA2 positive, there is new hope for a targeted therapy that is already seeing success in the ovarian cancer market.


#8 Enhanced Recovery After Surgery

#8 Enhanced Recovery After Surgery

After seeing substantial growth in hospital readmissions and an opioid epidemic spiraling out of control, it is clear that physicians need to overhaul the post-surgery strategies currently in use.


#9 Centralized Monitoring of Hospital Patients

#9 Centralized Monitoring of Hospital Patients

Centralized monitoring has emerged as the answer, as part of a “mission control” operation in which off-site personnel use advanced equipment monitor patients.


#10 Scalp Cooling for Reducing Chemotherapy Induced Hair Loss

#10 Scalp Cooling for Reducing Chemotherapy Induced Hair Loss

The practice of “Scalp Cooling” has been shown to be highly effective for preserving hair in women receiving neoadjuvant or adjuvant chemotherapy for early-stage breast cancer.

SOURCE

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