• Lysogene Announces First Patient in the United States Dosed with LYS-GM101 Investigational Gene Therapy for the Treatment of GM1 Gangliosidosis

    22 days ago - By Gene Therapy

    Posted on: 30 August 2021,
    Lysogene , a phase 3 gene therapy platform company targeting central nervous system diseases, today announced dosing of the first patient in the United States with LYS-GM101 investigational gene therapy at CHOC Hospital in a global adaptative-design clinical trial for the treatment of GM1 gangliosidosis. This trial is an interventional, multi-center, single-arm, two-stage adaptive-design study evaluating the intracisternal delivery of a recombinant adeno-associated virus vector serotype rh.10 carrying the human β-galactosidase gene. The clinical trial includes a...
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  • Amicus spins off gene therapy unit in $600M SPAC deal as CEO becomes chief at Caritas

    22 days ago - By Fierce Biotech

    Amicus spins off gene therapy unit in $600M SPAC deal as CEO becomes chief at Caritas badams
    Wed, 09/29/2021 - 08:26
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