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gene-therapy: Archive

Zacks Equity Research

SRPT Slides on Voluntary US Elevidys Shipment Pause Amid Scrutiny

Sarepta stock down as it halts U.S. Elevidys shipments after FDA pressure and safety concerns tied to patient deaths due to liver failure.

RHHBYNegative Net Change BAYRYPositive Net Change SRPTNegative Net Change VRNANegative Net Change

Zacks Equity Research

SRPT Down After Third Death in Muscular Dystrophy Gene Therapy Program

Sarepta plummets after a third death in its muscular dystrophy program for investigational gene therapies, prompts FDA action and intensifies safety scrutiny.

RHHBYNegative Net Change BAYRYPositive Net Change SRPTNegative Net Change VRNANegative Net Change

Zacks Equity Research

SRPT Stock Soars on Unveiling New Restructuring Plan, Pipeline Pivots

Sarepta's shares jump after unveiling a major restructuring plan to cut costs. The company shifts focus to siRNA and reprioritizes its pipeline.

RHHBYNegative Net Change SRPTNegative Net Change ARWRPositive Net Change

Zacks Equity Research

Ultragenyx Pharmaceuticals Gets CRL for UX111 Gene Therapy for MPS IIIA

RARE faces regulatory setback as FDA issues a CRL for UX111, delaying approval of the MPS IIIA gene therapy until 2026.

BAYRYPositive Net Change AGENNegative Net Change RAREPositive Net Change VRNANegative Net Change