gene therapy

News

Uncertainty ends Sarepta's Duchenne gene therapy Elevidys ends after an FDA label update, with the company's shares on the up despite restrictions.

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enGene is hoping to file its gene for a form of bladder cancer before the end of 2026, based on phase 2 data revealing a 62% complete response rate.

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uniQure has revealed that the FDA has said it is not prepared to review its Huntington's gene therapy in what seems to be a change in policy.

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Rocket Pharma has withdrawn a filing in the US for its experimental gene therapy for Fanconi anaemia, but may consider partnering the programme.

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uniQure's gene therapy AMT-130 has cleared a trial in Huntington's, setting up filings and raising the prospect of a first treatment for the disease.

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The FDA has added a warning label to bluebird bio's Skysona, after launching a probe into cases of blood cancer linked to the gene therapy.