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Taysha Gene Therapies Announces Publication of Positive Preclinical Data for TSHA-104 Demonstrating Therapeutic Potential in SURF1-associated Leigh Syndrome in Journal Molecular Therapy: Methods & Clinical Development
TSHA-104 restored to normal levels elevation of blood lactate on exhaustive exercise in dose-dependent manner in SURF1 knockout mice TSHA-104 increased COX1
About this update from Taysha Gene Therapies, Inc.
[{"type":"text","content":"\nTSHA-104 restored to normal levels elevation of blood lactate on exhaustive exercise in dose-dependent manner in SURF1 knockout mice\n\nTSHA-104 increased COX1 activity in brain and muscle in dose-dependent manner in SURF1 knockout mice\n\nCombination of intrathecal and intravenous delivery did not prove benefit over intrathecal alone\n\nNo associated toxicity risks or severe tissue damage were seen\n\nThere are no approved treatments for the underlying cause of the disease and the burden of disease and risk of mortality are high\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 publication of new preclinical data for TSHA-104 in SURF1-associated Leigh syndrome in Molecular Therapy: Methods & Clinical Development.\n\nSURF1 deficiency is a monogenic mitochondrial disorder that is the most common cause of cytochrome c oxidase deficient Leigh syndrome, a rapidly progressive neurological disorder that leads to degeneration of the CNS. Leigh syndrome typically presents in the first year of life and is characterized by progressive loss of mental and movement abilities that can result in death in early childhood. Approximately 10-15% of people with Leigh syndrome have a SURF1 mutation, and the estimated prevalence of SURF1 deficiency is 300 to 400 patients in the United States and European Union. There are currently no approved therapies to treat SURF1-associated Leigh syndrome.\n\n“SURF1 is part of cytochrome c oxidase, a mitochondrial enzyme known as COX involved in the metabolic production of ATP. Children with SURF1 deficiency have severely impaired COX activity and cannot generate ATP by aerobic respiration appropriately. This disruption in overall energy metabolism increases anaerobic respiration, leading to elevated levels of lactate and the clinical phenotype of Leigh syndrome,” said Suyash Prasad, MBBS, M.Sc., MRCP, MRCPCH, FFPM, Chief Medical Officer and Head of Research and Development of Taysha. “We are pleased that the published preclinical data for TSHA-104 demonstrate that the gene replacement therapy can address the hallmark characteristics ...