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Three Gene Therapies, Moderna Metabolic Drug In Inaugural START Class; CBER Oversubscribes

 
• By Kate Rawson

Seven rare disease programs make up the first round of participants in the US FDA’s ‘Operation Warp Speed’-style program. Sponsors will benefit from ‘rapid, ad hoc’ communication with the agency.

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FDA to seven rare disease sponsors: Let's Go. • Source: Shutterstock

The US Food & Drug Administration’s inaugural class of applications for its START rare disease pilot program includes three early-stage gene therapy programs intended to treat pediatric diseases, including Rett syndrome (Neurogene), NGLY1 deficiency (Grace Science), and Canavan disease (Myrtelle).

The FDA announced the “Support for clinical Trials Advancing Rare disease Treatment” (START) pilot last fall as offering an “Operation Warp Speed”-style of communication. Center for Biologics Evaluation and Research Director Peter Marks described the idea as applying the COVID vaccine response model to rare diseases where there is unmet medical need

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