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Gene Therapy

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FDA Agrees Three‑Year Phase I/II Data Can Support uniQure’s AMT‑130 Accelerated BLA

It opens a route for accelerated approval with negotiations underway over the design of a required post‑approval confirmatory trial.

Sarepta Reports Early siRNA Data Showing Muscle Delivery as Shares Surge

UK Trial’s Base-Edited Off-the-Shelf CAR T Cells Deliver Durable Remissions in T-Cell Leukemia

Vinay Prasad Resigns as FDA Biologics Director After Politicized Tenure