Taysha Gene Therapies
Taysha Gene Therapies focuses on developing gene therapies for severe monogenic diseases of the central nervous system, with its lead program TSHA-102 targeting Rett syndrome using an AAV9 capsid and intrathecal delivery.
Services
Taysha Gene Therapies focuses on advancing gene therapies for severe monogenic diseases of the central nervous system. The company aims to improve the lives of patients suffering from these debilitating conditions by developing innovative gene therapy treatments.
Lead Program: TSHA-102
TSHA-102 is Taysha Gene Therapies' lead clinical program, targeting Rett syndrome. Rett syndrome is a severe neurological disorder that primarily affects females. TSHA-102 utilizes a clinically and commercially proven AAV9 capsid for gene delivery, providing a promising avenue for advanced treatment options.
Innovative Gene Delivery Methods
Taysha Gene Therapies employs innovative delivery methods for their gene therapies, including intrathecal administration. This technique delivers the therapeutic agents directly to the cerebrospinal fluid, ensuring targeted delivery and potentially enhancing treatment efficacy.
Manufacturing Process
Taysha Gene Therapies operates a flexible manufacturing process using a HEK293 triple transfection suspension process. This process allows for efficient production of gene therapy vectors, ensuring scalability and consistency in the manufacturing pipeline.
Regulatory Designations for TSHA-102
TSHA-102 has secured multiple regulatory designations, highlighting its potential impact on treating Rett syndrome. These designations include U.S. RMAT, Fast Track, Orphan Drug, and Rare Pediatric Disease designations, EMA Orphan Drug designation, and U.K. MHRA Innovative Licensing and Access Pathway designation.
Corporate and Laboratory Locations
Taysha Gene Therapies' corporate headquarters is located at 3000 Pegasus Park Drive, Suite 1430, Dallas, Texas 75247. The company also operates a laboratory facility at 5 Laboratory Drive, Durham, North Carolina 27709.