Taysha Gene Therapies

TSHA-118 Clin1/Clin2 CLN1 disease; TSHA-102 Clin1/Clin2 Rett syndrome; TSHA-105 Clin0 genetic epilepsy

Taysha Gene Therapies is developing novel therapies for the treatment of monogenic diseases of the central nervous system (CNS).. Monogenic diseases are caused by mutations in single genes. Gene therapy can be employed to replace, change or inactivate the disease-causing gene. Taysha is using adeno-associated viruses (AAVs) to deliver gene therapies - these AAVs are clinically and commercially proven vectors to efficiently deliver the new gene into a cell. Taysha’s lead program, TSHA-102, is in development for patients with Rett syndrome, one of the most common genetic causes of severe intellectual disability in females. The therapy is directly injected into the cerebral spinal fluid to facilitate uptake into the CNS.

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