Axovant Licenses Investigational Gene Therapies for GM1 Gangliosidosis, Tay-Sachs and Sandhoff Diseases from University of Massachusetts Medical School
Axon Enterprise, Inc. (AXON)
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Source: GlobeNewswire
Two novel programs for fatal pediatric diseases deepen Axovant’s neurological gene therapy pipelineAXO-AAV-GM1 expected to enter the clinic in first half 2019, with initial data expected in second half 2019First patient has been dosed with AXO-AAV-GM2, with initial data expected in first quarter 2019 BASEL, Switzerland, Dec. 13, 2018 (GLOBE NEWSWIRE) -- Axovant Sciences (NASDAQ: AXON), a clinical-stage company focused on innovative gene therapies for neurological and neuromuscular diseases, today announced that it has licensed exclusive worldwide rights for the development and commercialization of two novel gene therapy programs to address GM1 gangliosidosis and GM2 gangliosidosis (also known as Tay-Sachs and Sandhoff diseases) from the University of Massachusetts (UMass) Medical School. GM1 gangliosidosis, Tay-Sachs and Sandhoff diseases are rapidly progressive and fatal pediatric lysosomal storage disorders that reduce life expectancy to less than two to four years of age in the
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