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Gene Therapy

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Taysha Advances Rett Gene Therapy Program With Pivotal Dosing, FDA Alignment and Q2 Data Timeline

FDA feedback supports a streamlined route to a potential BLA.

uniQure Investors Face April 13 Deadline in Securities Class Action Over FDA Setback

uniQure Soars 36% After FDA Biologics Chief Vinay Prasad Announces April Exit

FDA Rejects Regenxbio’s Hunter Syndrome Gene Therapy Application

Lilly Strikes Up to $1.12 Billion Pact With Seamless Therapeutics for Hearing‑Loss Gene Editing

Lilly Licenses MeiraGTx Eye Gene Therapy in Deal Worth Up to $475 Million

Sarepta Shares Plunge After ESSENCE Phase 3 Miss in Duchenne Trial

FDA Setback Puts uniQure’s Huntington’s Gene Therapy Filing in Doubt

Kriya Nets $320 Million Series D as Odyssey Raises $213 Million and Amgen Retools Research Leadership

Regulators Halt Elevidys Gene Therapy Worldwide After Safety Failures

Sarepta Defies FDA, Keeps Duchenne Gene Therapy on the Market

Sarepta Shares Plummet on FDA Review of Elevidys Following Third Patient Death

Eli Lilly to Acquire Verve Therapeutics as Gene-Editing Trial Begins

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