Taysha Gene Therapies

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Taysha Gene Therapies is a publicly traded, clinical-stage biotechnology company singularly focused on eradicating monogenic diseases of the central nervous system. Founded in 2019, the company has rapidly advanced a pipeline of AAV9 gene therapies, with its lead candidate, TSHA-102, now in pivotal studies for Rett syndrome. Following a strategic restructuring in 2023 to extend its cash runway, Taysha's strategy centers on disciplined execution of its most promising clinical programs, supported by deep expertise from leaders who commercialized the first FDA-approved gene therapy for a monogenic CNS disorder.

Proprietary platform utilizing intrathecally delivered AAV9 vectors, featuring miRNA-responsive auto-regulatory elements (miRARE) for precise transgene expression control in CNS tissues.