Gene editing companies to gain from FDA’s platform designation update

FDA’s platform technology designation is getting an update that should make it more accessible to gene editing platforms moving into the clinic, but leaves unanswered questions about how, if at all, the guidance may apply to AAV-based technologies.

During the Alliance for Regeneration Medicine (ARM) State of the Industry briefing last month, Peter Marks, director of FDA’s Center for Biologics Evaluation and Research, told attendees that the agency will be relaxing a requirement in the designation’s draft guidance that restricts its use to platforms that have already produced an approved product. ...