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Genome Editing: US FDA Eyes Platform-Based Approach To Development And Approval

 
• By Sue Sutter

CBER Director Peter Marks outlines a streamlined process to approval for treating different mutations of the same gene. NCATS’ Philip Brooks tells the Pink Sheet the approach avoids the need to “start from scratch for every new mutation.”

Diving platform
Genome editing poses special considerations for the platform approach to development and regulatory review. • Source: Shutterstock

The US Food and Drug Administration’s biologics center is considering ways to streamline the development and regulatory review of genome editing technology targeted at different mutations of the same gene.

The Center for Biologics Evaluation and Research “is working on developing a guidance on genome editing technology under the platform framework,” Julie Tierney, deputy center director for strategy, policy and

Key Takeaways

  • CBER is developing guidance on how genome editing technology can leverage the platform framework.

  • The FDA could grant approval for treating all mutations in a single gene after the sponsor has demonstrated an effect on a few mutations, CBER’s Peter Marks says.

  • NCATS’ Philip “P.J

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