Taysha Gene Therapies Inc
NASDAQ:TSHA
Taysha Gene Therapies Inc
Taysha Gene Therapies, Inc.is a patient-centric, clinical-stage gene therapy company, which engages in the development and commercialization of adeno-associated viruses (AAV) based gene therapies for the treatment of monogenic diseases of the central nervous system. The company is headquartered in Dallas, Texas and currently employs 178 full-time employees. The company went IPO on 2020-09-24. The firm 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-101, which is being developed for the treatment of giant axonal neuropathy (GAN); TSHA-102, a neurodevelopmental disorder product candidate, which is being developed for the treatment of Rett syndrome; TSHA-118, which is a AAV9 viral vector that expresses human codon-optimized Ceroid Lipofuscinosis Neuronal 1 complementary deoxyribonucleic acid under control of the chicken b-actin hybrid promoter, and TSHA-105, a gene replacement therapy for the treatment of SLC13A5 deficiency.
Taysha Gene Therapies, Inc.is a patient-centric, clinical-stage gene therapy company, which engages in the development and commercialization of adeno-associated viruses (AAV) based gene therapies for the treatment of monogenic diseases of the central nervous system. The company is headquartered in Dallas, Texas and currently employs 178 full-time employees. The company went IPO on 2020-09-24. The firm 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-101, which is being developed for the treatment of giant axonal neuropathy (GAN); TSHA-102, a neurodevelopmental disorder product candidate, which is being developed for the treatment of Rett syndrome; TSHA-118, which is a AAV9 viral vector that expresses human codon-optimized Ceroid Lipofuscinosis Neuronal 1 complementary deoxyribonucleic acid under control of the chicken b-actin hybrid promoter, and TSHA-105, a gene replacement therapy for the treatment of SLC13A5 deficiency.
Breakthrough Therapy: TSHA-102 received FDA Breakthrough Therapy designation in September, expediting development and review for Rett syndrome.
Pivotal Trial Progress: Enrollment for the REVEAL pivotal trial is on track to begin this quarter, with high demand and swift expected enrollment.
Regulatory Alignment: The company has finalized alignment with the FDA on the trial protocol and statistical plan, with a clear path to BLA submission.
Strong Efficacy Data: Part A of the REVEAL trials showed 100% response rate at 9 months for high-dose patients and 83% at 6 months.
Safety Profile: TSHA-102 continues to show a favorable safety profile with no treatment-related serious adverse events or dose-limiting toxicities.
Financial Position: Taysha ended Q3 with $297.3 million in cash and expects this to fund operations into 2028.
Commercial Rights & Buildout: The company regained full global rights to TSHA-102 and has strengthened its commercial leadership team.
Upcoming Data: Longer-term clinical data from Part A is expected in the first half of 2026.
