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Taysha Gene Therapies Receives Orphan Drug Designation from the European Commission for TSHA-120 for the Treatment of Giant Axon Neuropathy (GAN)
Clinical efficacy data for TSHA-120 provide quantitative evidence of long-term durability across all therapeutic dose cohorts with a 10-point improvement in
About this update from Taysha Gene Therapies, Inc.
[{"type":"text","content":"Clinical efficacy data for TSHA-120 provide quantitative evidence of long-term durability across all therapeutic dose cohorts with a 10-point improvement in mean change in MFM32 by Year 3 compared to estimated natural history decline of 24 points Biopsy data in five of six patient samples analyzed to date confirmed active regeneration of nerve fibers following treatment with TSHA-120 53 patient-years of clinical data support favorable safety and tolerability profile of TSHA-120 Estimated addressable patient population of 5,000 worldwide represents a multi-billion dollar commercial opportunity No approved treatments for the underlying cause of the disease DALLAS, May 03, 2022 (GLOBE NEWSWIRE) -- Taysha 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 that it has been granted orphan drug designation from the European Commission for TSHA-120, an intrathecally dosed AAV9 gene therapy currently in ongoing clinical evaluation for the treatment of giant axonal neuropathy (GAN). “GAN is a progressive and devastating neurodegenerative disease that has an estimated addressable patient population of 5,000 worldwide. The disease impacts a broad range of patients, with an early onset form of the disease affecting young infants, while a late onset can affect patients into adulthood,” said Suyash Prasad, MBBS, M.SC., MRCP, MRCPCH, FFPM, Chief Medical Officer and Head of Research and Development of Taysha. “In January we announced promising data for TSHA-120, our most advanced program, demonstrating long-term durability for all three therapeutic dose cohorts and clinically significant improvements in MFM32 over time compared to decline in patients observed in natural history studies. The long-term safety and tolerability of TSHA-120 was supported by 53-patient years of data, and importantly, biopsy data confirmed active nerve fiber regeneration following treatment with TSHA-120. We are pleased to receive orphan drug designation by the European commission which can help facilitate rapid clinical advancement and subsequent access to patients as we further approach regulatory approval.” GAN is a rare inheri...