Taysha Gene Therapies to unveil 30-fold MeCP2 expression boost with TSHA-102 at ASGCT
In vitro tests show self-complementary AAV9 drives approximately 30-fold higher MeCP2 expression than single-stranded AAV9 in neuronal cells, supporting TSHA-102’s delivery potential via lumbar intrathecal administration. MiniMeCP2 demonstrated molecular and biochemical functionality and stable neuronal expression comparable to full-length MeCP2, reinforcing construct design ahead of Phase 1/2 results.
1. Preclinical Data Comparison
In vitro experiments comparing self-complementary AAV9 (scAAV9) and single-stranded AAV9 (ssAAV9) in neuronal cell models revealed approximately 30-fold higher MeCP2 protein expression with scAAV9, validating its enhanced transduction efficiency.
2. miniMeCP2 Functional Profile
The miniaturized MECP2 transgene exhibited molecular and biochemical functions equivalent to the full-length protein and maintained stable expression in neuronal cells, supporting its use in TSHA-102.
3. TSHA-102 Construct and Administration
TSHA-102 combines scAAV9 vector with miRARE-regulated miniMeCP2 in a one-time intrathecal gene therapy designed to deliver functional MeCP2 to the CNS, leveraging a minimally invasive lumbar IT route.
4. ASGCT Presentation and Next Steps
New data will be presented at the ASGCT Annual Meeting May 11-15, 2026, in Boston, with longer-term safety and efficacy findings from Part A of the REVEAL Phase 1/2 trial expected later this quarter.