Taysha Gene Therapies is a clinical-stage biotechnology company dedicated to developing single-dose, adeno-associated virus (AAV)–based gene therapies for patients with rare monogenic diseases affecting the central nervous system and retina. The company’s research platform is designed to enable rapid progression of multiple assets through preclinical and clinical development by leveraging optimized AAV capsids and proprietary regulatory elements tailored to specific disease targets.
The company’s pipeline includes several late preclinical and early clinical programs. Lead candidate TSHA-102 is being developed for the treatment of Rett syndrome and related MECP2-associated disorders. Additional programs include TSHA-104 for giant axonal neuropathy, a severe inherited neurological disorder, and TSHA-120 for GM2 gangliosidosis (Tay-Sachs and Sandhoff diseases). Each program employs AAV-mediated gene replacement or augmentation to address the underlying genetic defect with a single administration.
Headquartered in Houston, Texas, Taysha has established in-house manufacturing capabilities for AAV vector production to support its clinical supply requirements and advance multiple assets in parallel. The company’s operations include research, process development and cGMP manufacturing under one roof, allowing tight integration of preclinical development and clinical manufacturing activities. Taysha’s team combines expertise in neurology, ophthalmology and vector engineering to drive its gene therapy platform forward.
In addition to its internal R&D efforts, Taysha has licensed several programs from leading academic institutions, including Nationwide Children’s Hospital, where foundational work on Rett syndrome gene therapy was conducted. The company is conducting clinical and preclinical studies primarily in the United States, with plans to expand trial sites globally. Taysha is led by a management team with deep experience in gene therapy development, manufacturing scale-up and regulatory strategy, positioning it to address critical unmet needs in rare neurological and ophthalmic diseases.
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