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Gene Therapies Without Randomized Clinical Trials: Marks Outlines Rare Disease Development Path

 
• By Derrick Gingery

A single-arm trial using a biomarker and animal model could be enough for an accelerated approval, especially in a pediatric rare disease where randomization and placebo controls may not be feasible, the CBER director said.

clinical trial and laboratory
Gene therapies in pediatric rare diseases may not need a randomized clinical trial to gain accelerated approval. • Source: Shutterstock

Peter Marks continues to sketch a pathway to market for pediatric gene therapies in rare diseases that could be faster than traditional product development and not require a randomized clinical trial.

Marks, who heads the US Food and Drug Administration’s Center for Biologics Evaluation and Research, said that the center will...

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Branch Chief Vijay Kumar Named Acting Head Of US FDA’s Cell, Gene Therapy Office

 
• By Derrick Gingery

Kumar takes over as acting director of the Center for Biologics Evaluation and Research’s Office of Therapeutic Products days after director Nicole Verdun was ousted.

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• By Eliza Slawther

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US FDA’s Top Cell and Gene Therapy Regulators Forced Out

 
• By Sarah Karlin-Smith

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More from Advanced Technologies

UK’s Decentralized Manufacturing Rules Designed To Allow For ‘Future Developments’

 
• By Eliza Slawther

The UK drug regulator acknowledges that technology will “move on” and, as such, has designed its decentralized manufacturing regulation to be as “enduring” as possible. Experts from the MHRA explain what products are covered by the new framework and how it has been future-proofed.

Profit-Sharing AI Model Proposed To Boost Korean Drug R&D

 
• By Jung Won Shin

A differentiated profit-sharing model to accelerate the use of AI in drug development using clinical and patient data is being proposed in South Korea, but the idea faces multiple practical challenges

US FDA’s Top Cell and Gene Therapy Regulators Forced Out

 
• By Sarah Karlin-Smith

Office of Therapeutic Products Director Nicole Verdun and her deputy Rachael Anatol were escorted out of FDA headquarters on June 18. Disagreements over CAR-T regulation and Capricor’s DMD treatment may be to blame.