gene therapies

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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.

News

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.

News

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.

News

Sarepta backs down and pauses Elevidys shipments

The FDA seems to have won its staring contest with Sarepta over shipments of Elevidys, as the company blinks and agrees to a pause from today.

News

A woman holds her hands up in a signal of 'No'

Sarepta resists as FDA seeks pause on Elevidys use

Sarepta has defied an FDA request to halt all use of its gene therapy for Duchenne muscular dystrophy, creating an intriguing regulatory impasse.

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

Sarepta backs down and pauses Elevidys shipments

Sarepta resists as FDA seeks pause on Elevidys use

Lily gets FDA nod for SERD-CDK 4/6 combo in breast cancer

Discover a new era of healthcare panel engagement: Grounded ...

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