NCT07480564 TSHA-102 Rett Syndrome Clinical Landscape Report 2026: Design, Endpoints, Sponsor and Readout Outlook
Move from a broad disease map to a decision-ready trial dossier. This focused report examines NCT07480564—Safety and Preliminary Efficacy of TSHA-102 Gene Therapy in Pediatric Females Aged >2 to <4 Years With Rett Syndrome (ASPIRE)—using PatSnap Clinical Trials, Drug & Asset, and Company & Deal Intelligence MCP evidence. Explore PatSnap MCP Servers to reproduce the workflow inside an AI research process.
MCP evidence snapshot: 16 July 2026; publication date: 17 July 2026. Trial records can change after the snapshot and should be rechecked before operational decisions.
Why NCT07480564 is a hot trial to watch
Rett Syndrome is no longer one homogeneous development market. The most consequential programs increasingly compete through a specific mechanism, biomarker, treatment line, delivery strategy or endpoint architecture. NCT07480564 is notable because it tests TSHA-102 in a Phase 3 design with Primary Safety as a primary decision variable. The wider PatSnap topic query returned 38 trial records and 32 result records, so differentiation depends on evidence quality rather than activity alone.
PatSnap Clinical Trials MCP makes the protocol fields machine-readable, while the companion asset and organization servers add mechanism and sponsor context.
Trial landscape snapshot
| Field | Indexed detail |
|---|---|
| Registration | NCT07480564 |
| Official title | Safety and Preliminary Efficacy of TSHA-102 Gene Therapy in Pediatric Females Aged >2 to <4 Years With Rett Syndrome (ASPIRE) |
| Phase / status | Phase 3 / Recruiting |
| Intervention | TSHA-102 |
| Sponsor | Taysha Gene Therapies, Inc. |
| Geography | United States |
| Enrollment | 3 |
| Primary endpoint | Primary Safety |
| Endpoint time frame | Baseline through Week 25 |
| Primary completion | 2031-06-01 |
| Study completion | 2031-06-01 |
Design and endpoint interpretation
The design should be read as an evidence architecture, not just a phase label. The primary endpoint—Primary Safety—determines what uncertainty this study can resolve. The reported time frame is Baseline through Week 25. Enrollment of 3 participants and geography in United States shape statistical precision, operational risk and external validity. A strong readout will need to be interpreted against baseline risk, prior treatment, assessment schedule, missing-data handling and the clinical relevance of the observed effect.
Benchmark readouts in the same clinical field
- Safety and effectiveness of diazepam nasal spray in patients with Rett syndrome and seizure clusters: post hoc analyses from a long-term safety study and survey of severity and burden (Phase 2): SEIVAL = Increase in SEIVAL was also similar (P1 = 7.6, P4 = 25.0 days and P1 = 13.0, P4 = 25.9 days). ; SEIVAL = Increase in SEIVAL was also similar (P1 = 7.6, P4 = 25.0 days and P1 = 13.0, P4 = 25.9 days). .
- Associations Between Alzheimer’s Disease and Rett Syndrome: Clinical Trials of NA‐831 for the Treatment Alzheimer’s and NA‐921 for Rett Syndrome (Phase 2/3): CGI-I(12 week) = 3.83 Point ; CGI-I(12 week) = 3.6 Point .
- Biomed Industries Presents Four Breakthrough Studies on Alzheimer’s, Rett Syndrome, and Obesity Therapies at AAIC 2025 (Phase 2/3): CGI-I = 3.83 Point ; CGI-I = 3.6 Point ; CGI-I: P-Value = 0.002.
These result records are contextual benchmarks rather than direct head-to-head evidence. Cross-trial comparisons can be distorted by population, line of therapy, endpoint definition, follow-up and analysis set. Their value is to clarify what magnitude and type of evidence the market already recognizes.
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Asset and sponsor context
Drug & Asset context: TSHA-102 (Phase 3; MECP2).
Company & Deal Intelligence context: Taysha Gene Therapies, Inc. (TSHA) — http://www.tayshagtx.com.
The sponsor profile matters because a trial's strategic value depends on more than scientific rationale. Manufacturing readiness, portfolio fit, geographic reach, partnering capacity and the ability to fund confirmatory development can determine whether a positive signal becomes a competitive asset.
White space around this program
- Sharper patient selection: prospective biomarker definitions that identify who is most likely to benefit.
- Clinically interpretable endpoints: outcomes that connect biological activity with function, symptoms, survival or treatment burden.
- Sequencing evidence: randomized data after the most relevant contemporary standard of care.
- Broader external validity: evidence across additional geographies, demographic groups and real-world care settings.
- Operational differentiation: a development path that closes the readout gap without sacrificing safety monitoring or durability.
What to monitor next
Monitor recruitment status, enrollment changes, protocol amendments, endpoint hierarchy, primary-completion timing, first result indexing, asset ownership and sponsor partnerships. The most important inflection point is not always the headline data release; a change in endpoint, population or ownership can alter probability of success months earlier.
Bottom line
NCT07480564 is a focused lens on Rett Syndrome development. Its value will be determined by whether TSHA-102 can convert the current design into evidence that is clinically meaningful, operationally credible and differentiated from topic-level benchmark readouts.
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