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Gene Therapy

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Sarepta Reports 3-Year EMBARK Data Showing Elevidys Slows Duchenne Progression

The readout lands under heightened FDA scrutiny following two liver‑failure deaths in non‑ambulatory patients.

Vertex Projects Casgevy Sales Above $100 Million in 2025 as CRISPR Therapeutics Waits for Payoff

FDA Adds Boxed Warning to Elevidys, Narrows Use to Ambulatory Patients 4 and Older

NEJM Study Finds 95% Long-Term Success for ADA-SCID Gene Therapy

Gene Therapy Shows 75% Slower Huntington’s Decline in Early Trial

FDA Lifts Hold on Elevidys Shipments to Ambulatory Duchenne Patients

AAV-OTOF Gene Therapy Restores Hearing in Teens and Adults

Sarepta Halts Elevidys Shipments for Non-Ambulatory Patients After Second Fatal Liver Failure

Personalized CRISPR Therapy Sends Baby KJ Home from CHOP