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Taysha Gene Therapies Announces Positive Safety Data from UT Southwestern-Sponsored Clinical Trial for the Treatment of CLN7 Batten Disease at 18th Annual WORLDSymposium
Data demonstrated favorable safety and tolerability profile at 1.0x1015 total vg, the highest dose ever delivered intrathecally in humans Stabilization in
About this update from Taysha Gene Therapies, Inc.
[{"type":"text","content":"\nData demonstrated favorable safety and tolerability profile at 1.0x1015 total vg, the highest dose ever delivered intrathecally in humans\n\nStabilization in nerve conduction demonstrated absence of dorsal root ganglia inflammation\n\nTaysha holds exclusive option to license CLN7 Batten disease program from UT Southwestern\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 presentation of clinical safety data supporting the first-generation construct at the highest dose of 1.0x1015 total vg for the treatment of CLN7 Batten disease at the 18th Annual WORLD Symposium, held February 7-11, 2022, in San Diego, CA by investigators from UT Southwestern.\n\n“This is the first ever gene therapy trial for the treatment of CLN7 Batten disease, and we are encouraged that the first-generation construct was well tolerated by all patients treated at the highest dose ever delivered intrathecally in humans,” said Suyash Prasad, MBBS, M.Sc., MRCP, MRCPCH, FFPM, Chief Medical Officer and Head of Research and Development of Taysha. “Importantly, the absence of evidence of dorsal root ganglia inflammation following gene transfer supports the therapy’s safety profile and provides positive readthrough across our platform.”\n\nSafety data for the first-generation construct for the treatment of CLN7 Batten disease from the ongoing clinical trial following intrathecal administration further demonstrated that the first-generation construct was well-tolerated at multiple doses including 1.0x1015 total vg, which is the highest dose administered in humans ever for a gene therapy product. No adverse immune responses have been noted, including no evidence of dorsal root ganglion toxicity or brain inflammation across all subjects. Moreover, stabilization in sural nerve conduction supported the absence of dorsal root ganglia inflammation. The ongoing trial includes three patients dosed to date, with two patients treated at the highest dose of 1.0x1015. Complete blood counts revealed no signs of bone marrow suppression or clinically significant bone marrow reactivity, and cerebral ...