genetic diseases

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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

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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

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Dr Andrew Anzalone, Prime Medicine co-founder and head of platform development

Gene-editing firm Prime Medicine files $140m public offer

Gene-editing biotech Prime Medicine has priced a follow-on public offering, hoping to raise $140 million to advance its pipeline of one-shot therapies for severe genetic d

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Free access to Zolgensma curbed, says Novartis

Novartis' programme providing free access to its spinal muscular atrophy (SMA) gene therapy Zolgensma is being scaled back to a dozen countries worldwide, according to the company.

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Astellas makes another gene therapy play, takes stake in Tay...

Astellas has been having some safety-related issues with its gene therapy programmes of late, but it shows no signs of lessened enthusiasm for the category – as a new alliance with US biote

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Sanofi wagers $400m on miRecule muscular dystrophy therapy

Sanofi has added to its rare disease pipeline by licensing an antibody-RNA conjugate (ARC) for facioscapulohumeral muscular dystrophy (FSHD), a genetic muscle disorder, from US biotech miRe

Nanoscope preps filing for retinitis pigmentosa gene therapy

Regulus cues up $100m placement on ADPKD drug data

Gene-editing firm Prime Medicine files $140m public offer

Free access to Zolgensma curbed, says Novartis

Astellas makes another gene therapy play, takes stake in Tay...

Sanofi wagers $400m on miRecule muscular dystrophy therapy

Heart disease risk ‘calculator’ could save lives

Fixing clinical trial data management

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