Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
TSHA-102 · 3 trials · 1 indication
Safety and Tolerability of TSHA-102 Proportions of participants experiencing any treatment-emergent adverse events (AEs) and serious adverse events (SAEs)
Proportions of participants experiencing any treatment-emergent adverse events (AEs) and serious adverse events (SAEs)
Change from baseline in percentage of participants who gain or regain any one or more of the 28 items from the Developmental Milestones Assessment (DMA), which are video recorded and scored by independent, blinded central raters.
| Arm | Type | Description |
|---|---|---|
| Treatment | EXPERIMENTAL | Participants receive a single intrathecal (IT) administration of TSHA-102 at 1.0 × 10¹⁵ total vector genomes (vg) adjusted for the participant's brain volume. |
| Part A Cohort 1 | EXPERIMENTAL | TSHA-102 Dose Level 1: 5.7×10¹⁴ total vector genomes (vg). Participants receive a single intrathecal (IT) administration of TSHA-102 at Dose Level 1 (fully enrolled, 2 participants). |
| Part A Cohort 2 | EXPERIMENTAL | TSHA-102 Dose Level 2: 1.0×10¹⁵ total vector genomes (vg) Participants receive a single intrathecal (IT) administration of TSHA-102 at Dose Level 2 (fully enrolled, 4 participants). |
| Part B Pivotal Cohort | EXPERIMENTAL | TSHA-102 at Selected Dose (Dose Level 2): 1.0 × 10¹⁵ total vector genomes (vg) Participants receive a single intrathecal (IT) administration of TSHA-102 at Dose Level 2 (1.0 × 10¹⁵) (fully enrolled, 17 participants). |
| Cohort 1 | EXPERIMENTAL | Dose Level 1 |
| Cohort 2 | EXPERIMENTAL | Dose Level 2 |
| Name | Type | Description |
|---|---|---|
| TSHA-102 | GENETIC | TSHA-102 is a recombinant, non-replicating, self-complementary adeno-associated virus serotype 9 (scAAV9) vector encoding for the miniMECP2 gene. TSHA-102 is a one-time intrathecal (IT) administration. |
Inclusion Criteria: * Pediatric females between the ages of 2 and less than 4 years old. * Participant has a clinical diagnosis of classic/typical Rett syndrome with a documented pathogenic mutation of the methyl-CpG-binding protein 2 (MECP2) gene that results in loss of gene function. * Participan...
TSHA-102 is an investigational gene therapy being developed for the treatment of Rett Syndrome, a rare neurological disorder that primarily affects females. It is designed to address the underlying genetic cause of the condition. TSHA-102 is currently in clinical development and has not yet been approved by regulatory authorities.
TSHA-102 is being developed by Taysha Gene Therapies, Inc., a biopharmaceutical company focused on gene therapies for rare diseases. The company is publicly traded under the ticker symbol TSHA. Taysha is conducting clinical trials to evaluate the safety and efficacy of TSHA-102 in patients with Rett Syndrome.
TSHA-102 is in Phase 3 clinical development for Rett Syndrome. It is being evaluated in multiple trials, including a pivotal Phase 3 study and a Phase 3 study in younger pediatric patients. The drug is investigational and has not received regulatory approval. It has been granted several FDA designations, including Breakthrough Therapy and RMAT.
TSHA-102 is being studied in three active clinical trials. The REVEAL Pivotal Study (NCT05606614) is a Phase 3 trial in females aged 6 years and older. The REVEAL Pediatric Study (NCT06152237) is a Phase 1 trial in females aged 5 years and older. A third trial (NCT07480564) is a Phase 3 study in females aged 2 to 4 years.
TSHA-102 is not FDA approved. It is an investigational gene therapy currently in clinical development for Rett Syndrome. The FDA has granted it Breakthrough Therapy, RMAT, Fast Track, Orphan Drug, and Rare Pediatric Disease designations, which are intended to expedite its development and review, but approval has not yet been granted.
The TSHA-102 clinical program includes three active trials with a total planned enrollment of 27 female participants. The trials are randomized and controlled, but not double-blind. They are conducted in the United States, Canada, and the United Kingdom, and enroll females as young as 2 years of age with Rett Syndrome.