genetic diseases

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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.

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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

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Ionis plans phase 3 for Biogen-rejected Angelman drug

Ionis has committed to a phase 3 programme for its antisense therapy for rare genetic disorder Angelman syndrome, just weeks after Biogen decided against exercising an option to license the

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Prime editing comes of age as FDA clears human trials

Prime Medicine has been given the go-ahead by the FDA for the first human trial of its prime editing technology, which promises to deliver one-shot therapies for a range o

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Nanoscope preps filing for retinitis pigmentosa gene therapy

Nanoscope Therapeutics is on the brink of filing for FDA approval of what could be the first gene therapy for incurable eye disease retinitis pigmentosa (RP) that can be u

News

Regulus cues up $100m placement on ADPKD drug data

Buoyed by encouraging data for its lead drug for autosomal dominant polycystic kidney disease, Regulus Therapeutics has moved swiftly to file a financing that will give it

Neurogene falls again on Rett gene therapy side effects

Neurogene shares fall on Rett syndrome gene therapy data

Ionis plans phase 3 for Biogen-rejected Angelman drug

Prime editing comes of age as FDA clears human trials

Nanoscope preps filing for retinitis pigmentosa gene therapy

Regulus cues up $100m placement on ADPKD drug data

Pfizer elevates Chris Boshoff to top R&D role

Coming soon: The Life Sciences Industry Report 2025 – insigh...

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