gene therapy

News

Gene therapy firm Kate Tx snapped up by Novartis for $1.1bn

Novartis buys gene therapy specialist Kate Therapeutics for $1.1 billion, adding an early-stage rival to Sarepta's muscular dystrophy therapy Elevidys

News

Neurogene falls again on Rett gene therapy side effects

Neurogene has halted patient enrolment in the high-dose arm of its trial of Rett syndrome gene therapy, sparking another run on its shares.

News

PTC gets FDA okay for first brain-delivered gene therapy

More than two years after its approval in Europe, PTC Therapeutics' gene therapy for rare genetic disorder AADC deficiency will soon be made available to patients in the US.

News

Rachel McMinn, Neurogene's founder and chief executive

Neurogene shares fall on Rett syndrome gene therapy data

Neurogene has reported encouraging efficacy results with its gene therapy for rare genetic disorder Rett syndrome, but investors' concerns about safety seem to be weighing on its share pric

News

Lexeo shares dip on early data with Alzheimer's gene therapy

Lexeo Therapeutics' gene therapy for Alzheimer's has shown that it can reduce biomarkers of the disease in a small study, including reductions in disease biomarkers.

News

MeiraGTx plans to take Parkinson's gene therapy into phase 3

Armed with data from a small phase 1/2 bridging study, MeiraGTx is preparing to take its gene therapy for Parkinson's disease into a pivotal trial.

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gene therapy

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