ASGCT Submits Comments on FDA Draft Guidance on Regenerative Medicine Expedited Programs

ASGCT leadership submitted comments to the FDA last week on the draft guidance titled, “Expedited Programs for Regenerative Medicine Therapies for Serious Conditions.” ASGCT expressed appreciation for guidance on these programs, especially on the Regenerative Medicine Advanced Therapy (RMAT) designation and the inclusion of certain gene therapies in the eligibility for consideration for RMAT designation.

The guidance identifies gene therapies, including genetically modified cells, which lead to a durable modification of cells or tissues, as eligible for RMAT designation.

A primary recommendation from ASGCT is to expand the eligibility for RMAT designation to all gene therapies to encourage the development of such therapies, which hold the potential to provide treatment for significant unmet need for multiple diseases.

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