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...
Top 2 of 7 competitors
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Akebia Therapeutics, Inc. | AKBA | 1 | PHASE2 | Vadadustat |
| Rigel Pharmaceuticals, Inc. | RIGL | 1 | PHASE2 | Fostamatinib |
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. The therapy is currently in clinical development and has not yet been approved by regulatory authorities.
TSHA-102 targets the MECP2 gene, which is the gene that is mutated in Rett Syndrome. The therapy is designed to deliver a functional copy of this gene to cells. By targeting MECP2, TSHA-102 aims to correct the genetic defect that leads to the symptoms of Rett Syndrome.
TSHA-102 is being developed by Taysha Gene Therapies, Inc., a biopharmaceutical company. Taysha Gene Therapies is publicly traded on the NASDAQ under the ticker symbol TSHA. The company is focused on developing gene therapies for rare diseases, with TSHA-102 being one of its lead candidates.
TSHA-102 is currently in Phase 3 clinical development for Rett Syndrome. It is an investigational therapy, meaning it has not yet been approved by the FDA or other regulatory agencies. The drug has received several FDA designations, including Breakthrough Therapy, RMAT, Fast Track, Orphan Drug, and Rare Pediatric Disease designations.
TSHA-102 is being studied in three active clinical trials. NCT05606614 is a Phase 1/2/3 pivotal study in females with Rett Syndrome. NCT06152237 is a Phase 1 pediatric study. NCT07480564 is a Phase 3 study in pediatric females aged 2 to 4 years. All trials are active but not recruiting participants.
TSHA-102 is a unique gene therapy specifically designed for Rett Syndrome. It is not the same as other treatments for the condition, as it targets the MECP2 gene directly. No alternative names for TSHA-102 have been reported in clinical trial registries.