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gene-therapy: Archive

Zacks Equity Research

SRPT Finishes Enrolment in Late-Stage Study for Rare Muscular Disorder

The phase III EMERGENE study evaluates Sarepta's experimental gene therapy for an ultra-rare form of muscle disorder for which there is no treatment beyond symptom management.

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Ekta Bagri

3 Biotech Stocks With Promising Gene Therapies to Watch Out in 2025

We have picked three biotech stocks with promising gene therapies in their portfolios/pipelines that are likely to perform well in 2025.

SRPTPositive Net Change VYGRPositive Net Change CRSPPositive Net Change

Zacks Equity Research

EDIT Stock Falls on Decision to End Reni-Cel Studies, Cut Workforce

Editas stock falls as it ends development of reni-cel for SCD and TDT following failed attempts to find a commercial partner. It plans to cut workforce by 65%.

EDITPositive Net Change CTMXPositive Net Change SPRONegative Net Change CSTLNegative Net Change

Zacks Equity Research

QURE Soars on FDA Alignment for Speedy Approval of Huntington's Drug

uniQure stock rallies 110% on reaching alignment with the FDA on key elements of an accelerated approval pathway for AMT-130 to treat Huntington's disease.

ALNYPositive Net Change PFENegative Net Change GILDNegative Net Change QUREPositive Net Change

Sundeep Ganoria

Sarepta Therapeutics Stock Surges 43% YTD: How to Play the Stock?

The rise in SRPT's share price can be attributed to the encouraging sales performance of its recently approved one-shot gene therapy for DMD.

RHHBYNegative Net Change SRPTPositive Net Change ARWRPositive Net Change

Zacks Equity Research

SRPT Inks $11B Licensing Deal With Arrowhead for Rare Gene Therapies

Sarepta in-licenses rights to four clinical-stage and three preclinical-stage programs targeting muscle, central nervous system and rare pulmonary disorders.

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