Taysha Gene Therapies reported that the first patient in its REVEAL pivotal trial for TSHA‑102, a self‑complementary AAV9 gene therapy delivered intrathecally, received dosing in the fourth quarter of 2025. The therapy uses miRARE technology to regulate MECP2 expression, addressing the genetic root of Rett syndrome.
The company also confirmed that it has received written alignment from the U.S. Food and Drug Administration to include at least three months of safety data from its ASPIRE safety study—comprising three patients aged two to under four years—in the planned Biologics License Application. The alignment supports a broad label for patients two years and older and reflects the FDA’s recognition of the therapy’s potential to fill an unmet need.
Taysha projects completion of dosing for both the REVEAL pivotal trial and the ASPIRE safety study in the second quarter of 2026, with a longer‑term data update from REVEAL Part A expected in the first half of 2026. These milestones position the company to advance toward regulatory submission and potential market entry for a disease with no approved disease‑modifying therapies.
Rett syndrome is a rare neurodevelopmental disorder affecting an estimated 15,000 to 20,000 patients in the U.S., EU, and U.K. TSHA‑102 is one of several gene‑therapy candidates in development, with competitors such as Neurogene’s NGN‑401 also in clinical trials. The absence of approved treatments underscores the therapeutic opportunity and the importance of regulatory approvals for TSHA‑102.
Financially, Taysha reported a net loss of $32.7 million and earnings per share of –$0.09 for the third quarter of 2025, a figure that met analyst expectations. The company’s cash balance stood at $297.3 million as of September 30 2025, providing a runway through 2028 and supporting ongoing clinical development and regulatory activities.
CEO Sean P. Nolan emphasized the significance of the milestone, stating that “dosing the first patient in our REVEAL pivotal trial last quarter represents a significant milestone in the development of TSHA‑102 for Rett syndrome, and enrollment is further advancing across multiple sites. As we progress pivotal development, we continue to maintain consistent and productive dialogue with the FDA.” He added that the recent alignment on ASPIRE and the six‑month interim analysis for REVEAL streamline the path toward BLA submission.
Investors have historically sold after positive news for TSHA, a pattern that has led to a “sell the news” reaction. While the clinical progress is encouraging, market sentiment has not yet translated into sustained upside, reflecting broader concerns about cash burn and the inherent risks of clinical‑stage biotechnology companies.
The first‑patient dosing and FDA alignment are pivotal steps that could accelerate TSHA’s regulatory trajectory, but the company’s financial position and investor perception remain critical factors that will influence its path to approval and market entry.
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