gene therapy

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Despite trial miss, Sarepta plans new filing for DMD therapy

Sarepta Therapeutics’ gene therapy for Duchenne muscular dystrophy has failed to meet its primary objective in a phase 3 trial, but the company says it will file to extend

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Early result backs Regeneron’s deafness gene therapy

Regeneron’s gene therapy for a profound form of deafness affecting children, DB-OTO, has shown the first signs that it can improve hearing in a clinical trial.

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Roche builds gene therapy portfolio with SpliceBio deal

Roche’s gene therapy unit Spark has added to its pipeline in inherited retinal diseases (IRDs) with a $216 million alliance with Spanish biotech SpliceBio.

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uniQure sheds a fifth of its staff and half its R&D

Netherlands gene therapy company uniQure has said it will shed 20% of its workforce, around 114 jobs, and slash its R&D projects in an effort to extend its cash reserv

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Orchard will help Kyowa Kirin grow in gene therapy

Japan’s Kyowa Kirin has reached an agreement to buy Orchard Therapeutics of the UK in a deal valued at up to $478 million, ahead of an FDA decision on the biotech’s lead g

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Gene Therapy Process Development Summit

Gene therapy developers are striving to platform their process development to more quickly and cost-effectively bring their drugs to market, meaning optimizing upstream process development

Despite trial miss, Sarepta plans new filing for DMD therapy

Early result backs Regeneron’s deafness gene therapy

Roche builds gene therapy portfolio with SpliceBio deal

uniQure sheds a fifth of its staff and half its R&D

Orchard will help Kyowa Kirin grow in gene therapy

Gene Therapy Process Development Summit

Lilly bags FDA okay for Wegovy pill rival orforglipron

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