Gene Therapy Guidances Will Focus On Specific Diseases, Gottlieb Says

US FDA's first such guidance will focus on hemophilia, commissioner tells Congress.

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The US FDA appears to be adding onto its planned slew of disease-specific guidances, with Commissioner Scott Gottlieb announcing that the agency will be releasing "a suite" of such guidances on the development of specific gene therapy documents.

Testifying before the Senate Committee on Health, Education, Labor and Pensions Dec

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