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' 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

Taysha Gene Therapies (TSHA)