- ByInvesting.com
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Taysha Gene Therapies, Inc. is a patient-centric gene therapy company. The Company is focused on developing and commercializing adeno-associated virus (AAV)-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS). Its portfolio of gene therapy candidates targets a range of neurological indications across three distinct therapeutic categories: neurodegenerative diseases, neurodevelopmental disorders, and genetic epilepsies. Its product candidates include TSHA-120, a clinical-stage, intrathecally dosed AAV9 gene therapy program for the treatment of giant axonal neuropathy (GAN); TSHA-102, a self-complementary intrathecally delivered AAV9 gene transfer therapy product candidate in clinical evaluation for Rett syndrome; TSHA-118, a self-complementary AAV9 viral vector that expresses human codon-optimized Ceroid Lipofuscinosis Neuronal 1 (CLN1) complementary deoxyribonucleic acid under control of the chicken b-actin hybrid promoter, and others.