Neurogene reported a patient experienced systemic hyperinflammatory syndrome during their Rett syndrome gene therapy trial. CEO Rachel McMinn confirmed the female patient is in critical condition. The ...
Syndax Pharmaceuticals won the first FDA approval last week in a new class of medicines called menin inhibitors.
The FDA on Monday offered new draft guidance to help drugmakers understand the nuts and bolts of FDA processes for developing and submitting an application for a new cell or gene therapy, from pre-IND ...
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