USA - CBER chief on remote work, CRISPR-Cas9, and rare disease gene therapy accelerated approval guidance

The use of CRISPR-Cas9 gene editing technology has potential to transform the field of gene therapy through its simple and cost-effective delivery mechanism, said Peter Marks, director of the US Food and Drug Administration’s (FDA) Center for Biologics Evaluation and Research (CBER), during a 23 April webinar sponsored by the Alliance for a Stronger FDA. He also addressed FDA’s forthcoming guidance on accelerated approval for gene therapies for treating rare diseases. Marks also said that FD...