RESEARCH TRIANGLE PARK, N.C.--(BUSINESS WIRE)--Asklepios Biopharmaceutical Inc. (“AskBio”), a private development-stage biotechnology company, has entered into a cross license agreement with GlaxoSmithKline (“GSK”) to develop and commercialize gene therapy based treatments. Through the agreement, AskBio will gain exclusive access to selected recombinant adeno-associated virus vector (“rAAV”) serotypes that GSK licensed from the University of Pennsylvania for the treatment of three orphan drug indications, including Muscular Dystrophy, heart failure suffered by Muscular Dystrophy patients, and Hemophilia B. Other terms of the agreement, including the technology licensed to GSK and the monetary terms related thereto, were not disclosed.
"We are looking forward to obtaining this license to address two complex diseases,” said Sheila Mikhail, Chief Executive Officer of AskBio. This license will bring a combined platform of technologies to the Muscular Dystrophy and Hemophilia communities that will enhance patient care in these two important orphan drug diseases.”
AskBio is currently conducting a Phase I clinical trial, which has been financially supported by the Muscular Dystrophy Association (MDA), using BiostrophinTM for the treatment of Duchenne Muscular Dystrophy (“DMD”). The most common form of muscular dystrophy, DMD results from a mutation in the dystrophin gene, and is characterized by rapidly progressive muscle weakness. Young boys suffering from this disease lose the ability to walk usually by age twelve and due to largely to heart failure and respiratory disorders resulting from the disease, die in their early twenties. There is currently no life sustaining treatment for this genetically inherited disease. BiostrophinTM uses AskBio’s Biological NanoParticle (“BNP”) technology, which is engineered from rAAV and other parvoviruses, to deliver a genetically re-engineered mini-gene to express dystrophin and treat DMD at the molecular level. BNPs are designed to deliver therapeutic genetic materials to the patient’s muscle cells become bioreactors which produce long-term therapeutic benefit.
About Asklepios BioPharmaceutical, Inc.
AskBio is a private development-stage biotechnology company, which was spun out of the University of North Carolina – Chapel Hill. AskBio is engaged in the development of novel, intracellular protein therapeutics using its BNPTM technology platform and Self-Complementary vector technology. BNPsTM may be used to target delivery of a broad variety of biological material, including therapeutic genes, RNAi, and vaccines, among others, to specific tissue. The Self-Complementary technology reduces the amount of virus required for therapeutic treatment, thereby decreasing the likelihood of any immune response. The company is developing therapeutics for the treatment of muscular dystrophy (in collaboration with the Muscular Dystrophy Association), bone regrowth (in collaboration with the Musculoskeletal Tissue Foundation), and hemophilia. AskBio’s affiliate, NanoCor Therapeutics, Inc., in conjunction with researchers at Mass General Hospital, the University of Cincinnati and the University of North Carolina, is developing a therapeutic for congestive heart failure. For additional information, visit AskBio's web site at www.askbio.com.