Jack Allen

Kristen Kluska

Gil Blum

Joon Lee

Maurice Raycroft

Silvan Tuerkcan

Yanan Zhu

Biren Amin

Christopher Raymond

Salveen Richter

Whitney Ijem

Elizabeth Webster

Ethan Markowski

Evan Seigerman

Keith Tapper

Lydia Erdman

Tazeen Ahmad

- Taysha Gene Therapies, Inc. has **regained full rights to its lead TSHA-102 program** for the treatment of Rett syndrome, following the expiration of the 2022 Option Agreement with Astellas.  **[1806310_0001193125-25-241415_d35488dex991.htm:0]** 
- The TSHA-102 program has demonstrated **compelling Part A REVEAL Phase 1/2 safety and efficacy data** (May 2025 data cutoff), including a 100% response rate for the pivotal trial primary endpoint.  **[1806310_0001193125-25-241415_d35488dex991.htm:0]** 
- TSHA-102 has received **FDA Breakthrough Therapy designation**, and Taysha has finalized the REVEAL pivotal trial protocol and statistical analysis plan with the FDA.  **[1806310_0001193125-25-241415_d35488dex991.htm:0]** **[1806310_0001193125-25-241415_d35488dex991.htm:1]** 
- **Dosing of the first patient in the REVEAL pivotal trial is scheduled for the current quarter** (Q3 2025), evaluating TSHA-102 in 15 females with Rett syndrome.  **[1806310_0001193125-25-241415_d35488dex991.htm:0]** **[1806310_0001193125-25-241415_d35488dex991.htm:1]**

Taysha Gene Therapies Regains Full Rights to TSHA-102 Program

- Taysha Gene Therapies (TSHA) announced new supplemental data analysis from Part A of the REVEAL Phase 1/2 trials for **TSHA-102 in Rett Syndrome** on **October 9, 2025**.  **[1806310_0001193125-25-235212_d949053dex991.htm:0]** 
- The analysis revealed a **100% response rate** across all 10 patients in Part A for the pivotal trial primary endpoint, which involved gaining or regaining at least one natural history defined developmental milestone.  **[1806310_0001193125-25-235212_d949053dex991.htm:0]** **[1806310_0001193125-25-235212_d949053dex991.htm:1]** 
- A new supplemental analysis showed that **100% of patients** also demonstrated multiple skill gains/improvements outside of the natural history defined developmental milestones.  **[1806310_0001193125-25-235212_d949053dex991.htm:0]** **[1806310_0001193125-25-235212_d949053dex991.htm:1]** 
- In total, **22 developmental milestones** and **165 additional skills/improvements** were achieved across the 10 patients treated with TSHA-102.  **[1806310_0001193125-25-235212_d949053dex991.htm:0]** **[1806310_0001193125-25-235212_d949053dex991.htm:1]** 
- TSHA-102 is a one-time intrathecally delivered AAV9 investigational gene transfer therapy for Rett syndrome and has received several FDA designations, including **Breakthrough Therapy**.  **[1806310_0001193125-25-235212_d949053dex991.htm:1]**

Taysha Gene Therapies Presents Positive Supplemental Data for TSHA-102 in Rett Syndrome

- Taysha Gene Therapies' stock surged, reaching as high as **$4.89** during trading, with volume exceeding **101 million shares**, following the U.S. FDA's granting of **Breakthrough Therapy designation** to its lead candidate, TSHA-102, for Rett syndrome.  **[1806310_1707e2042c0f4bd98236d01cb8ebbe94]** 
- The FDA's designation, coupled with alignment on the pivotal REVEAL trial protocol, positions Taysha to potentially **expedite its Biologics License Application submission** for TSHA-102, with patient enrollment in the trial expected to begin by **late 2025**.  **[1806310_1707e2042c0f4bd98236d01cb8ebbe94]** 
- Analysts, including Needham's Gil Blum, have responded positively by raising the price target for Taysha shares from **$8 to $10** while maintaining Buy ratings.  **[1806310_1707e2042c0f4bd98236d01cb8ebbe94]** 
- Despite a strong liquidity position, highlighted by a current ratio of **12.48**, Taysha faces financial challenges including **revenue stagnation** and **negative operating and net margins**.  **[1806310_1707e2042c0f4bd98236d01cb8ebbe94]**

Taysha Gene Therapies Receives FDA Breakthrough Therapy Designation for Rett Syndrome Candidate

- Taysha Gene Therapies, Inc. announced on **October 2, 2025**, that the **FDA granted Breakthrough Therapy designation to TSHA-102** for the treatment of Rett syndrome.  **[1806310_0001193125-25-227505_d61846dex991.htm:0]**  This designation was based on the FDA's review of clinical evidence from Part A of the REVEAL Phase 1/2 trials, which demonstrated a **100% response rate** for the primary endpoint of gaining or regaining developmental milestones (May 2025 data cutoff).  **[1806310_0001193125-25-227505_d61846dex991.htm:0]** 
- The company has **finalized alignment with the FDA** on the REVEAL pivotal trial protocol and statistical analysis plan (SAP) for TSHA-102, following the resolution of clinical and statistical queries.  **[1806310_0001193125-25-227505_d61846dex991.htm:0]** **[1806310_0001193125-25-227505_d61846dex991.htm:1]** 
- Patient enrollment for the REVEAL pivotal trial is **on track to begin in Q4 2025**, with key design elements including a **6-month interim analysis** that could potentially expedite the Biologics License Application (BLA) submission.  **[1806310_0001193125-25-227505_d61846dex991.htm:0]** **[1806310_0001193125-25-227505_d61846dex991.htm:1]**

Taysha Gene Therapies Announces FDA Breakthrough Therapy Designation for TSHA-102 and Regulatory Alignment