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Taysha Gene Therapies Announces Positive Preliminary Clinical Safety Data For First-Generation Construct In CLN7 Disease and Completion of Next-Generation CLN7 Construct Design
Preliminary clinical data for first-generation CLN7 construct support favorable tolerability and safety profile with no major adverse events across doses
About this update from Taysha Gene Therapies, Inc.
[{"type":"text","content":"\nPreliminary clinical data for first-generation CLN7 construct support favorable tolerability and safety profile with no major adverse events across doses delivered\n\nData Safety Monitoring Board (DSMB) supported dose escalation from 5.0x1014 (n=1) to 1.0x1015(n=2), which is the highest dose delivered intrathecally in humans to date\n\nPreliminary clinical efficacy and safety data to be presented at the 18th Annual WORLDSymposium in February 2022 by Dr. Ben Greenberg, Vice Chair of Clinical and Translational Research and Professor in the departments of Neurology and Pediatrics at UT Southwestern\n\nDesign of next-generation construct to improve potency, packaging efficiency and manufacturability as well as reduce risk of immunogenicity over first-generation construct completed\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 preliminary clinical safety data for the first-generation construct in CLN7 disease. Preliminary clinical efficacy and safety data will be presented at the 18th Annual WORLDSymposium in February 2022 by Dr. Ben Greenberg, Vice Chair of Clinical and Translational Research and Professor in the departments of Neurology ad Pediatrics at UT Southwestern (UTSW). In addition, UTSW has completed the design of a next-generation construct, which is expected to further improve potency, packaging efficiency and manufacturability as well as reduce risk of immunogenicity over the first-generation construct.\n\n“We are very pleased that the first-generation construct has been well tolerated and preliminary data from the ongoing clinical trial support a favorable safety profile. To date, three patients with CLN7 disease have been treated, including two patients dosed at 1.0x1015 total vg, which is the highest dose ever safely administered intrathecally in humans for a gene therapy. The DSMB supported dose escalation from the 5.0x1014 initial dose to the 1.0x1015 high dose. Importantly, there have been no major adverse events observed. We look forward to Dr. Ben Greenberg’s presentation of the preliminary clinical efficacy and safety data at the...