First patient dosed in REVEAL pivotal trial evaluating TSHA-102 (N=15, aged 6 to <22 years) in Q4 2025, with enrollment advancing across multiple sites
Taysha Gene Therapies, Inc., a clinical-stage biotechnology company focused on advancing adeno-associated virus based gene therapies for severe monogenic diseases of the central nervous system announced progress across the TSHA-102 pivotal program, an intrathecally delivered AAV9 gene therapy with disease modifying potential, in clinical evaluation for the treatment of Rett syndrome.
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“Dosing the first patient in our REVEAL pivotal trial last quarter represents a significant milestone in the development of TSHA-102 for Rett syndrome, and enrollment is further advancing across multiple sites. As we progress pivotal development, we continue to maintain consistent and productive dialogue with the FDA,” said Sean P. Nolan, Chairman and Chief Executive Officer of Taysha. “Importantly, we recently reached written alignment with the FDA that our planned BLA submission will include at least three months of safety data from three patients aged two to under four years in the ASPIRE trial to support a broad label for patients aged two years and older with Rett syndrome. We believe this recent alignment on ASPIRE, together with the alignment on a six-month interim analysis for our REVEAL pivotal trial, streamlines our path toward BLA submission for TSHA-102. We expect to complete dosing across our REVEAL and ASPIRE trials in the second quarter of 2026. With an estimated 15,000 to 20,000 patients affected by Rett syndrome across the U.S., EU and U.K. and compelling REVEAL Part A clinical data, we see a significant opportunity to redefine the treatment paradigm for this devastating disease with high unmet need.”
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TSHA-102 Program Updates and Overview
- Dosed First Patient in REVEAL Pivotal Trial in the Fourth Quarter of 2025. Additional enrollment continues to advance across multiple clinical trial sites. The single-arm, open-label trial is evaluating a single IT administration of high dose TSHA-102 (1×1015 total vector genomes (vg)) in 15 females between the ages of 6 and <22 years in the developmental plateau population of Rett syndrome. The primary endpoint will assess response rate, defined as the percentage of patients who gain or regain ≥one of the 28 natural history-defined developmental milestones, with each patient serving as their own control. The study includes a six-month interim analysis that may serve as the basis for Biologics License Application (BLA) submission.
- Reached Written FDA Alignment on ASPIRE Trial and Data for Inclusion in BLA Submission to Enable Broad Label Following Type D Meeting. The Company confirmed prior alignment and obtained further written alignment with the U.S. Food and Drug Administration (FDA) on the ASPIRE safety-focused trial and the data for inclusion in the planned BLA submission to enable broad labeling of TSHA-102 for patients aged ≥2 years with Rett syndrome, including:
- ASPIRE will enroll three females aged 2 to <4 years with Rett syndrome to evaluate the safety and preliminary efficacy of a single IT administration of high dose TSHA-102 (1×1015 total vg), scaled to account for the lower brain volume in 2 to <4‑year‑olds
- A minimum of three months of ASPIRE safety data will be included in the planned BLA submission, while efficacy in the 2 to <6-year-old population will be extrapolated from data collected in the REVEAL pivotal trial, to support a broad label for TSHA-102 in patients aged ≥2 years with Rett syndrome
Anticipated Milestones
- Completion of dosing in the REVEAL pivotal trial is expected in the second quarter of 2026
- Completion of dosing in the ASPIRE trial is expected in the second quarter of 2026
- Update on longer-term safety and efficacy data from Part A of the REVEAL Phase 1/2 trials is expected in the first half of 2026
About TSHA-102
TSHA-102 is a self-complementary intrathecally delivered AAV9 investigational gene transfer therapy in clinical evaluation for Rett syndrome. Designed as a one-time treatment, TSHA-102 aims to address the genetic root cause of the disease by delivering a functional form of MECP2 to cells in the CNS. TSHA-102 utilizes a novel miRNA-Responsive Auto-Regulatory Element (miRARE) technology designed to mediate levels of MECP2 in the CNS on a cell-by-cell basis without risk of overexpression. TSHA-102 has received Breakthrough Therapy, Regenerative Medicine Advanced Therapy, Fast Track and Orphan Drug and Rare Pediatric Disease designations from the FDA, Orphan Drug designation from the European Commission and Innovative Licensing and Access Pathway designation from the Medicines and Healthcare products Regulatory Agency.
About Rett Syndrome
Rett syndrome is a rare neurodevelopmental disorder caused by mutations in the X-linked MECP2 gene encoding methyl CpG-binding protein 2 (MeCP2), which is essential for regulating neuronal and synaptic function in the brain. The disorder is characterized by loss of communication and hand function, slowing and/or regression of development, motor and respiratory impairment, seizures, intellectual disabilities and shortened life expectancy. Rett syndrome progression is divided into four key stages, beginning with early onset stagnation at 6 to 18 months of age followed by rapid regression, plateau and late motor deterioration. Rett syndrome primarily occurs in females and is one of the most common genetic causes of severe intellectual disability. Currently, there are no approved disease-modifying therapies that treat the genetic root cause of the disease. Rett syndrome caused by a pathogenic/likely pathogenic MECP2 mutation is estimated to affect between 15,000 and 20,000 patients in the U.S., EU, and U.K.
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Source- GlobeNewswire
