Government Relations Publications

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. 

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