The Advocate

Volume 1, Issue 9

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November 2021

In This Issue:

FDA Announces OTAT Growth Program at ASGCT Liaison Meeting
ASGCT Weighs in on Regulatory Issues
National Partnership Will Accelerate Gene Therapy Development for Rare Diseases
Save the Date for the 25th Annual Meeting
Policy News


ASGCT Advocacy

FDA Announces OTAT Growth Program at ASGCT Liaison Meeting

At the fourth annual liaison meeting last week between ASGCT and the Office of Tissues and Advanced Therapies (OTAT), OTAT Director Wilson Bryan, MD, announced the OTAT Growth Program, a three-phase program to expedite advances in cell and gene therapy and improve Center for Biologics Evaluation and Research (CBER) staff satisfaction and sustainability in the face of rapid increases in clinical development. Arising from ideas gathered from stakeholders in the field during the first phase of the program, OTAT will pilot solutions to these challenges. Efforts will include increasing collaborative educational efforts with organizations such as ASGCT; revising its website to provide centralized information, especially on sponsor/agency meetings; and investigating ways to increase communication on the status of submissions. The proposed commitment goals in the Prescription Drug User Fee Act reauthorization (PDUFA VII) would contribute to the program’s objective to create more staff and management capacity and sustainability.

To read more about the meeting and view presentation slides from ASGCT and FDA, check out this blog post.

ASGCT Weighs in on Regulatory Issues

ASGCT submitted comments last week on FDA’s draft guidance document on the International Council for Harmonisation (ICH) S12 Nonclinical Biodistribution Considerations for Gene Therapy Products. The Society recommended changes, including:

  • Utilizing the same (or at least a similar material) medical device throughout trials to inform product labeling, regardless of whether the delivery system is a novel device.

  • Not requiring nonclinical shedding studies in studies using a non-replicative virus (e.g., AAV); adoption of this recommendation would harmonize EMA requirements with current FDA requirements.

ASGCT also submitted comments last month in support of FDA’s commitment letter outlining its proposed performance goals for the upcoming reauthorization of the Prescription Drug User Fee Act for FYs 2023-2027 (PDUFA VII). In its comments, the Society highlighted its support for the inclusion of proposals it had recommended including: 

  • Allocation of additional user fees to CBER: the letter establishes a goal to hire 228 new FTEs in CBER, nearly 65 percent of the total new FTEs supported by PDUFA VII. 

  • Improved engagement of FDA with sponsors: FDA proposes the creation of Type D meetings to provide sponsors with feedback within 50 days, on up to two issues; the agency also clarifies the scope, intent, and timeline for INTERACT meetings which, combined with the creation of Type D meetings, ASGCT believes will better utilize FDA resources in a clear manner for sponsors. 

  • Provision of guidance to clarify post-market study requirements: the letter indicates the agency will create draft guidance on methods and approaches for capturing post-approval safety and efficacy data for cell and gene therapy products. 

In its comments, ASGCT also expressed support for the development of several guidance documents on topics relevant to the field, such as updating the Expedited Programs for Regenerative Medicine Therapies for Serious Conditions guidance to include additional thinking on approaches and processes relating to chemistry, manufacturing, and controls (CMC). In addition, FDA’s plan to create a guidance document on common questions and answers regarding gene and cell therapy development would be welcome, and ASGCT requested the agency to consider including in that guidance document requirements for immunogenicity testing, CMC requirements prior to a phase 3 trial and in later stages of development, and comparability requirements for manufacturing changes for gene and cell therapy products.

National Partnership Will Accelerate Gene Therapy Development for Rare Diseases

ASGCT has joined forces with FDA, 11 NIH institutes and centers, and more than a dozen other nonprofit groups and private companies to help accelerate the delivery of gene therapies for rare disease patients through the newly launched Bespoke Gene Therapy Consortium (BGTC). This public-private collaboration, managed by the Foundation for the National Institutes of Health (FNIH), aims to optimize and streamline the gene therapy development process to help fill the unmet medical needs of people with many rare diseases, in particular those diseases that are so rare they challenge the typical commercial development model. Read more on our blog.


Save the Date for the 25th Annual Meeting

Save the date for ASGCT's 25th Annual Meeting, to be held for the first time as a hybrid event! Pre-Meeting Workshops will be held on May 15, while the Annual Meeting will run May 16-19. Also for the first time, ASGCT will host this milestone meeting at a convention center—the Walter E. Washington Convention Center in downtown Washington, DC. You can sign up for email updates about the meeting to stay updated on registration, abstracts, programming, and more; registration will open in mid-December. We hope to see you in DC or virtually!

Policy News

  • Check out the new issue of our Gene, Cell, & RNA Therapy Landscape Report, created in partnership with Informa Pharma Intelligence, that covers the therapeutics pipeline, clinical targets, developer progress, and more. In the third quarter, highlights include approvals of two new gene therapies in the European Union (EU) and China, growth in the RNA therapeutic space, and a spotlight on non-oncology indications.

  • In a recent article, CBER director Peter Marks, MD, PhD addresses the lessons FDA has learned from the COVID-19 pandemic, such as the usefulness of greater global regulatory collaboration. Marks notes that refocusing resources on COVID-19 has delayed the development of other crucial medical products, such as gene therapies. Therefore, as the world slowly moves into the post-pandemic period, regulators will need to select which aspects of regulatory process enhancements should be maintained.

  • The Working Group on Pediatric Gene Therapy & Medical Ethics (PGTME) is hosting a free virtual series of multi-stakeholder discussions later this month on five ethical topics in pediatric gene therapy research.

  • The Pathway Development Consortium, a public-private partnership to facilitate gene therapy development in rare diseases, released a draft white paper this month that proposes a framework for using accelerated approval for AAV gene therapies.

  • Sharif Tabebordbar, recipient of a 2019 ASGCT Career Development Award, has developed capsid variants of AAVs that deliver gene therapy directly to muscles of mice with muscular dystrophy.

  • Beam Therapeutics has been given the green light by FDA to proceed with first-in-human trials of base editing technology to treat sickle cell disease. Beam’s President/CSO presented an overview of this technology at ASGCT’s Policy Summit in September.

  • FDA has delayed the PDUFA date by three months for an anti-BCMA CAR T-cell therapy candidate developed by Legend Biotech and Johnson & Johnson. The first and so far only CAR T-cell therapy to treat multiple myeloma is Abecma, which was granted FDA approval earlier this year.


ASGCT 25th Annual Meeting

May 16-19, 2022

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