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The drug will evaluate the safety, tolerability and efficacy of a single IV infusion of gene therapy in adult subjects.
February 27, 2023
By: Anthony Vecchione
Asklepios BioPharmaceutical, Inc., a wholly owned and independently operated subsidiary of Bayer AG, announced that the European Commission (EC) has granted orphan drug designation for AB-1003 (also known as LION-101) for the treatment of limb-girdle muscular dystrophy (LGMD). AB-1003 is a novel investigational recombinant adeno-associated virus (AAV) based gene therapy currently being developed as a one-time intravenous (IV) infusion for the treatment of patients with LGMD type 2I/R9 (LGMD2I...
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