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Taysha Gene Therapies Announces Sponsored Genetic Testing for Giant Axonal Neuropathy (GAN) in Partnership with GeneDx as well as a Collaboration with Hereditary Neuropathy Foundation and Charcot-Marie-Tooth Association Centers of Excellence
GeneDx’s Hereditary Neuropathy Panel will be available free of charge to individuals at risk for or suspected of having GAN Taysha will collaborate with
About this update from Taysha Gene Therapies, Inc.
[{"type":"text","content":"\nGeneDx’s Hereditary Neuropathy Panel will be available free of charge to individuals at risk for or suspected of having GAN\n\nTaysha will collaborate with Hereditary Neuropathy Foundation and Charcot-Marie-Tooth Association Centers of Excellence to increase disease awareness and access to testing\n\nTSHA-120 is the first and only gene therapy to demonstrate clinical-stage arrest of disease progression at the 1.2x1014 and 1.8x1014 total vg dose\n\nClinical data for high dose 3.5x1014 total vg cohort expected in the second half of 2021 with regulatory feedback by year-end 2021\n\nEstimated prevalence of patients with GAN is 2,400 in United States and Europe\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 sponsored genetic testing for giant axonal neuropathy (GAN) in partnership with GeneDx, Inc., a leader in genomic analysis and a wholly owned subsidiary of BioReference Laboratories, Inc., an OPKO Health company (NASDAQ:OPK). Under the partnership, Taysha will sponsor the inclusion of a genetic marker to test for GAN in the GeneDx hereditary neuropathy panel free of charge to individuals at risk for or suspected of having GAN.\n\nGAN is a progressive neurodegenerative disease that affects both the central and peripheral nervous systems leading to motor weakness, sensory impairment, and cognitive dysfunction. Currently, there are no approved treatments for GAN, which results in death for patients often in their late teens or early twenties. Although no symptoms are present in the first few months of life, many children with early onset GAN develop symptoms and features before the age of three. A more recently identified later onset phenotype of GAN is often mischaracterized as Charcot-Marie-Tooth. The estimated prevalence for GAN is 2,400 patients, but the GAN population is anticipated to be larger than previously appreciated.\n\nIn the natural history study, Motor Function Measure 32 (MFM32), a validated and well-known scale to measure strength and motor function, is the primary endpoint. A four-point change on the MFM32 score is considered clinical...