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author：comprehensive 2025-09-30 23:06:39 - browse：62643

Peter Marks. -- health coverage from STAT — Peter Marks, Director of the Center for Biologics Evaluation and Research at the Food and Drug Administration. Susan Walsh-Pool/Getty Images

Peter Marks wants drug developers to ask more stupid questions.

It’s part of the top Food and Drug Administration official’s plan to reinvigorate gene therapy, a field that has struggled despite significant technological advances. Some companies are shelving programs or going out of business, even when they have promising data.

The problems are numerous: The diseases are often exceptionally rare, limiting the potential market. Manufacturing at commercial quality is complex and expensive. Proving a drug works can be difficult, because there may be too few patients to run a traditional randomized study.

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