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Taysha Gene Therapies Announces Publication of Natural History Data for TSHA-120 in Giant Axonal Neuropathy in the Journal, Brain
Largest cohort of genetically confirmed patients with GAN, including patients with the classic (early-onset) and milder (late-onset) forms of GAN Largest
About this update from Taysha Gene Therapies, Inc.
[{"type":"text","content":"\nLargest cohort of genetically confirmed patients with GAN, including patients with the classic (early-onset) and milder (late-onset) forms of GAN\n\nLargest cross-sectional analysis highlighted clinical differences in patients with early-onset GAN versus late-onset GAN based on MFM32 performance as well as other functional motor scales and disease markers \n\nRobust assessment across clinical outcomes for GAN, including motor, sensory, respiratory, neurophysiologic, MRI and biopsy data\n\nFirst clinical study to evaluate a cohort of individuals with GAN for autonomic impairment\n\nOn track to report clinical data for TSHA-120 from the 3.5x1014 total vg dose cohort in the second half of 2021\n\nPlanning to engage with major regulatory agencies to discuss the approval pathway and expect to provide a regulatory update by year-end 2021\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 publication of new analyses of natural history data for TSHA-120 in giant axonal neuropathy, or GAN. The data were published online and will be included in the June edition of Brain, a highly esteemed neurological science peer-reviewed journal.\n\nGAN is a progressive neurodegenerative disease that affects both the central and peripheral nervous systems. The disease is caused by loss-of-function mutations in the gene coding for gigaxonin, which results in dysregulation of intermediate filament turnover, an important structural component of the cell. Although no symptoms are present in the first few months of life, many children with GAN do show early symptoms and features before the age of five, including unsteady gait, frequent falls, and motor weakness. Symptoms worsen over time and children develop scoliosis, contractures, atrophy of the spinal cord and abnormalities of the white matter in the brain. Currently, there are no approved treatments for GAN, which results in death for patients in their late teens or early twenties.\n\nIn this natural history study, 45 patients, age 3 years to 21 years old, with genetically confirmed GAN were enrolled at NIH and evaluated a...