Founded in 2001, Asklepios BioPharmaceutical, Inc. (AskBio) is a privately held, clinical-stage gene therapy company dedicated to improving the lives of children and adults with genetic disorders. AskBio’s gene therapy platform includes an industry-leading proprietary cell line manufacturing process called Pro10™ and an extensive AAV capsid and promoter library. With global headquarters in Research Triangle Park, North Carolina, and European headquarters in Edinburgh, UK, the company has generated hundreds of proprietary AAV capsids and promoters, several of which have entered clinical testing. An early innovator in the space, the company holds more than 500 gene therapy patents. AskBio has a portfolio of clinical programs across a range of neurodegenerative and neuromuscular indications including therapeutics for Pompe disease, limb-girdle muscular dystrophy 2i/R9 and congestive heart failure, and out-licensed clinical indications for hemophilia and Duchenne Muscular Dystrophy.
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Pompe gene therapy
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