Thousands of patients are affected by monogenic CNS diseases.

At Taysha, we aim to address this devastating need through our pipeline of AAV-based gene therapies.

Our gene therapy candidates are designed to target the unique, underlying biology of diseases of the CNS. We are focused on advancing our lead clinical program in Rett syndrome.





Phase 1/2


Rett Syndrome


Regulated GRT

REVEAL Adult Study Canada

Regulated GRT

REVEAL Pediatric Study U.S.
Phase 1/2

GRT: Gene replacement therapy

Other Programs

Taysha has a pipeline of early-stage gene therapy programs targeting CNS diseases that we may progress in the future or advance through potential partnerships. We have deprioritized the company sponsored evaluation of certain clinical-stage programs, including TSHA-105 for SLC13A5 and TSHA-118 for CLN1, and are seeking external strategic options to potentially enable further development of these programs.

Please contact us to discuss potential opportunities for partnering and collaborating at BD@tayshagtx.com.


TSHA-120 is a clinical-stage AAV9 gene therapy program being developed for the treatment of giant axonal neuropathy, or GAN, which is an ultra-rare autosomal recessive, progressive neurodegenerative disease of the central, peripheral and autonomic nervous systems caused by deficiency or complete loss-of-function of gigaxonin and the accumulation of intermediate filaments. TSHA-120 is a fully optimized, AAV9 self-complementary viral vector that encodes the full length human gigaxonin protein.


TSHA-102 is a self-complementary intrathecally delivered AAV9 gene transfer therapy in clinical evaluation for Rett syndrome, a neurodevelopmental disorder and one of the most common genetic causes of severe intellectual disability, characterized by rapid developmental regression and in many cases caused by heterozygous loss of function mutations in MECP2, a gene essential for neuronal and synaptic function in the brain. TSHA-102 is constructed from a neuronal specific promoter, MeP426, coupled with the miniMECP2 transgene, a truncated version of MECP2, and miRNA-Responsive Auto-Regulatory Element, or miRARE, our novel miRNA target panel, packaged in self-complementary AAV9, which enables cellular regulation of both endogenous and exogenous MECP2 expression.


TSHA-118 is being developed for the treatment of CLN1 disease, also known as infantile Batten disease, a rapidly progressing rare lysosomal storage disease with no approved treatment. TSHA-118 is a self-complementary AAV9 viral vector that expresses human codon-optimized CLN1 complementary deoxyribonucleic acid under control of the chicken ß-actin hybrid promoter.


Prasad, S. et al. Immune Responses and Immunosuppressive Strategies for Adeno-Associated Virus–Based Gene Therapy for Treatment of Central Nervous System Disorders: Current Knowledge and Approaches. Human Gene Therapy. DOI: 10.1089/hum.2022.138

Goodspeed, K, et al. Gene Therapy: Novel Approaches to Targeting Monogenic Epilepsies. Frontiers in Neurology. 2022 Jun; https://doi.org/10.3389/fneur.2022.805007

Prasad, S, et al. Accepting the Challenge: Innovative Approaches and Translational Strategies in Gene Therapy Development. Abstract/Presentation. American Society of Gene and Cell Therapy Annual Meeting 2022.

Gray, SJ. Timing of Gene Therapy Interventions: The Earlier, the Better. Mol. Ther. 2016 Jun; 24(6): 1017-1018