TSHA Profile
Taysha Gene Therapies, Inc. is a leading gene therapy company dedicated to advancing the treatment of monogenic diseases affecting the central nervous system. The company specializes in developing adeno-associated virus (AAV)-based gene therapies, leveraging this technology to address genetic disorders with high unmet medical needs. Taysha’s innovative approach aims to provide transformative treatments by directly targeting the genetic mutations underlying these diseases.
Among its pipeline of therapeutic candidates, Taysha is developing TSHA-120, which is targeted at treating giant axonal neuropathy (GAN), a rare genetic disorder characterized by progressive nerve degeneration. Another key program, TSHA-102, is focused on Rett syndrome, a neurodevelopmental disorder that primarily affects females and leads to severe cognitive and motor impairments. Additionally, TSHA-121 and TSHA-118 are being developed for CLN1 disease, a form of neuronal ceroid lipofuscinosis (NCL) that causes severe neurodegeneration in early childhood.
The company is also advancing TSHA-105, which targets SLC13A5 deficiency, a rare genetic condition that affects brain development and function, and TSHA-101 for GM2 gangliosidosis, a group of inherited neurodegenerative disorders. These programs reflect Taysha’s commitment to addressing a broad spectrum of severe genetic disorders through its gene therapy platform.
Founded in 2019 and headquartered in Dallas, Texas, Taysha Gene Therapies has established a strategic partnership with The University of Texas Southwestern Medical Center to further develop and commercialize its gene therapy treatments. This collaboration enhances the company’s capabilities in research and clinical development, positioning Taysha at the forefront of gene therapy innovation. By focusing on transformative gene therapies, Taysha aims to significantly improve the lives of patients with rare and debilitating central nervous system diseases.
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