FDA releases draft guidance for approving gene therapies — STAT News

In the United States, in Washington, the FDA on Tuesday released draft guidance on streamlining the approval of certain individualized gene therapies. The initiative was supported by representatives of communities of patients with rare diseases, who had criticized the regulator’s approach to such treatments, STAT News reports.

Reaction of patient communities

Paul Scribner, vice president of public programs and education at the PKD Foundation, said at an event dedicated to rare diseases that the draft guidance underscores the importance of this area. According to the publication, advocates for patients with rare diseases welcomed the FDA’s move.

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Individualized treatments

Gene therapy involves treating or curing diseases by changing defective genes in human cells. The development of such methods has interested researchers and companies, which believe that personalized medicines may be useful for many patients with rare mutations. The FDA’s draft guidance applies only to some individualized gene therapies.