genetic diseases

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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

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SparingVision raises €75m for eye disease gene therapies

SparingVision has raised €75 million in second-round funding that will be used to fund clinical trials of gene therapies for ocular diseases retinitis pigmentosa (RP) and dry age-related ma

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Young lonely man in a wheelchair on a balcony looking at the nature in the spring

Sarepta says early filing for DMD gene therapy is back on

Sarepta is pressing forward with a bold plan to file with the FDA for accelerated approval of its gene therapy SRP-9001 for Duchenne muscular dystrophy (DMD) in the next few months, with a

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

SparingVision raises €75m for eye disease gene therapies

Sarepta says early filing for DMD gene therapy is back on

Novartis raises mid-term sales outlook on pipeline promise

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

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