gene therapies

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Touchdown for Rocket as its first gene therapy gets US okay

Rocket Pharma has won FDA approval for the first gene therapy for LAD-I, a devastating genetic disorder causing severe vulnerability to infections.

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Boy in a wheelchair looking out to the forest

Regenxbio, Solid report data with Duchenne gene therapies

Regenxbio and Solid Bio say their gene therapies for Duchenne muscular dystrophy show no sign so far of the liver issues seen with Sarepta's Elevidys.

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New FDA knockback hits uniQure and Huntington patients hard

uniQure looks like it may have to run another trial of its Huntington's disease gene therapy AMT-130, delaying the highly anticipated programme.

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Black and white image of the side of a baby's face, focused on its ear

Lilly allies with gene-editing firm Seamless on hearing loss

Gene-editing firm Seamless Therapeutics has attracted its first major pharma partner, Eli Lilly, with a $1.12bn alliance focused on hearing loss.

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A woman hides her face behind her hands, revealing her right eye

Ocugen shares fall, despite positive eye disorder trial

A trial of Ocugen's gene therapy for sight-robbing disease geographic atrophy (GA) showed efficacy – but still sparked a selloff in its shares.

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FDA clears first gene therapy for rare genetic disease WAS

Fondazione Telethon's Waskyra has become the first gene therapy in the US for Wiskott-Aldrich syndrome, and the first taken to market by a non-profit.

Touchdown for Rocket as its first gene therapy gets US okay

Regenxbio, Solid report data with Duchenne gene therapies

New FDA knockback hits uniQure and Huntington patients hard

Lilly allies with gene-editing firm Seamless on hearing loss

Ocugen shares fall, despite positive eye disorder trial

FDA clears first gene therapy for rare genetic disease WAS

LSX2026: On promise and patience, with Hans Schambye

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