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 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

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
.

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. 

2019
22nd Annual Meeting
April 29 – May 2 | Washington D.C.
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