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

29 May 2024

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...