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Taysha Gene Therapies Acquires Exclusive Worldwide Rights to Clinical-Stage AAV9 Gene Therapy Program, Now Known as TSHA-120, for the Treatment of Giant Axonal Neuropathy, a Rare and Severe Neurodegenerative Disease
Program invented in the lab of Dr. Steven Gray, Taysha’s Chief Scientific Advisor, immediately transforms Taysha into a sustainable pivotal-stage gene
About this update from Taysha Gene Therapies, Inc.
[{"type":"text","content":"\nProgram invented in the lab of Dr. Steven Gray, Taysha’s Chief Scientific Advisor, immediately transforms Taysha into a sustainable pivotal-stage gene therapy company\n\nClinical and preclinical data package validates the scientific approach of Dr. Steven Gray, UT Southwestern, and Taysha, with readthrough to existing portfolio\n\nGroundbreaking clinical trial run by the NIH is the first intrathecally dosed gene therapy program in history\n\nHuman proof-of-concept data for TSHA-120 demonstrated clear arrest of disease progression and long-term durability at therapeutic dose levels in patients with giant axonal neuropathy\n\nPlans to engage with regulatory agencies in the United States, Europe and Japan as soon as possible\n\nEstimated 2,400 patients in U.S. and Europe represent potentially greater than $2 billion near-term commercial opportunity\n\nProgram provides basis for accelerating build-out of commercial infrastructure to support patient identification, payor engagement and product distribution\n\nConference call and webcast today at 8:00 AM Eastern Time\n\n DALLAS--(BUSINESS WIRE)--\nTaysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced the acquisition of exclusive worldwide rights to a clinical-stage AAV9 gene therapy program, now known as TSHA-120, for the treatment of giant axonal neuropathy (GAN). TSHA-120 is an intrathecally dosed AAV9 gene therapy currently being evaluated in a clinical trial for the treatment of GAN. The trial is being conducted by the National Institutes of Health (NIH) in close collaboration with a leading patient advocacy group focused on finding treatments and cures for GAN. TSHA-120 has received rare pediatric disease and orphan drug designations from the U.S. Food and Drug Administration (FDA) for the treatment of GAN.\n\nGAN is a rare inherited genetic disorder that affects both the central and peripheral nervous systems and is caused by loss-of-function mutations in the gene coding for gigaxonin. Many children with GAN show symptoms and features before the age of five, including progressive scoliosis, contractures, atrophy of the spinal cord, gi...