Government Relations Publications

ASGCT often posts presentations, white papers, and other materials designed to educate and inform regulatory bodies in the United States government.

ASGCT Leads Congressional Briefing on Gene Therapy for Sickle Cell Disease Alongside Sens. Tim Scott and Cory Booker

ASGCT co-hosted a congressional briefing on sickle cell disease (SCD) with Senators Tim Scott and Cory Booker alongside additional stakeholder organizations.

See Sen. Scott's Opening Remarks
on the ASGCT Twitter Account

Taking place before a standing-room only audience of nearly 100 congressional staff members and other stakeholders, the briefing addressed progress in gene therapy for sickle cell disease and related policy implications. At the briefing, ASGCT echoed Sen. Scott’s call for use of novel payment models to address the unique, one-time nature of gene therapies—payment over time and outcomes-based payments. ASGCT speakers addressed the importance of the additional policies of robust funding to the NIH; appropriating funds for surveillance of SCD authorized by a law passed in December; and reauthorizing the newborn screening program this year. ASGCT researchers Punam Malik, M.D. (Cincinnati Children’s Hospital), Matthew Porteus, M.D., Ph.D. (Stanford University), and David Williams, M.D. (Dana-Farber Cancer Institute, Boston Children’s Hospital, and Harvard University) presented the progress they and their peers have contributed to through a variety of gene therapy and gene editing approaches for SCD.

Download the Entire SCD Briefing Slide Deck:

ASGCT Holds First-Ever FDA Liaison Meeting on Gene Therapy

ASGCT held its first-ever liaison meeting with the FDA’s Center for Biologics Evaluation and Research (CBER) on September 13. Liaison meetings provide an opportunity to discuss topics of mutual interest in the field, and ASGCT members addressed testing methods and depth recommendation for off-target analysis of gene editing technologies, as well as three topics that relate to the gene therapy guidance documents the FDA recently issued: RCR and RCL testing of drug product; manufacturing considerations; and data from long-term follow-up for persistent vs. transient gene therapy product classes on critical safety parameters.

Full presentation materials from the FDA liaison meeting are available for download:

Full FDA Liaison Meeting Agenda

Replication competent lentivirus (RCL) and
replication competent retrovirus (RCR) testing
of drug product
Kenneth Cornetta, M.D.

Testing methods and recommendations for
off-target analysis of gene editing technologies
J. Keith Joung, M.D., Ph.D.

Regenerative Medicine Advanced Therapy
(RMAT) Designation
Wilson W. Bryan, M.D.

Manufacturing Considerations
John T. Gray, Ph.D.

Long term follow up in recipients of HSCs and
Immune Effector Cells modified by Retroviral or
Lentiviral Vectors
Helen Heslop, M.D.

In Vivo Gene Therapy with DNA Vectors
Ronald Crystal, M.D.

ASGCT Releases White Paper Addressing the Value of Gene Therapy (UPDATED)

The value of gene therapy has become an increasingly prominent topic following the 2017 FDA approval of three gene therapies (including gene-modified cell therapies). Any discussion of the value of gene therapy must include the vast potential of these therapies to treat a variety of diseases, many of them rare disorders with great unmet need. ASGCT has therefore created a white paper:

Addressing the Value of Gene Therapy:
Enhancing Patient Access to Transformative Treatments
UPDATED 2-8-2019

The paper highlights the substantial value of gene therapy and the need to lower barriers between patients and access to approved therapies. ASGCT identifies priority solutions and recommends further exploration of additional means for stakeholders in the field to work toward maximizing the potential value of gene therapy through enhanced patient access.

Gene editing is a process that repairs or changes a gene. Medical researchers are exploring ways to use gene editing to treat or prevent human diseases. This report provides a foundation for understanding the science of gene editing, its potential therapeutic applications and its current U.S. regulatory framework.

Download the White Paper 

The National Center for Advancing Translational Sciences (NCATS) issued a Request for Information on opportunities to increase the efficiency of human gene therapy trials in rare diseases, with particular interest in input regarding the use of viral vectors as platforms for therapeutic gene delivery to specific organs or cell types, and how to most efficiently develop such platforms in clinical trials for the treatment of multiple diseases. The potential impact of a platform approach is great for rare genetic diseases, given the large number of rare diseases that may be impacted by gene therapy and the limited financial resources available for gene therapy trials for each rare disease. ASGCT submitted this response to NCATS regarding rare disease trial efficiencies.

Download the NCATS Response

FDA Guidance Documents

Read about regulatory changes that effect the field of gene and cell therapy. 


ASGCT 24th Annual Meeting

May 12-15, 2021