FDA Committed to Advancing Progress in Gene Therapy

The FDA announcement on June 6 of its new efforts to advance biotechnology innovation is the most recent indication of its support of the development of novel technology, including genome editing. The FDA’s new Biotech Working Group aims to ensure a flexible regulatory framework for evaluating the safety of products with indirect public benefit, namely plant and animal biotechnology innovation, including genome editing for uses such as improving the health of food-producing animals by reducing their susceptibility to diseases, and developing disease-resistant plants, which can positively affect consumers through better yields and healthier nutrient profiles.

As reported previously on ASGCT’s Twitter feed, FDA Commissioner Scott Gottlieb reported on the FDA’s intent to advance progress of gene therapy that directly affects human disease in a speech on May 22 at the Legislative Fly-In hosted by the Alliance for Regenerative Medicine in collaboration with ASGCT. The FDA intends to release a suite of draft guidance documents articulating its framework for the manufacturing and clinical development of gene therapy products beginning with a guidance on gene therapy for hemophilia, expected to be released the middle to end of this month.

The guidance documents will identify potential accelerated approval endpoints, such as the potential for factor production to be sufficient in some cases as a surrogate measure of benefit for hemophilia, with the ability to identify reduction in bleeding rates in post-market research. Other highlights of the speech include that the FDA has an internal initiative to improve the yield of cell lines used to produce gene therapy vectors, and the agency is taking steps to investigate the application of advanced manufacturing technologies, such as continuous manufacturing. These efforts are an attempt by the FDA to provide tools to expedite the development process for potentially life-altering gene therapies to treat multiple diseases with unmet medical need.

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