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DALLAS - Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a biotech company specializing in central nervous system diseases with a market capitalization of $528 million, is advancing towards a pivotal trial for its gene therapy candidate TSHA-102 after aligning with the U.S. Food and Drug Administration (FDA). The therapy is designed for patients with Rett syndrome, a severe neurological disorder. According to InvestingPro data, the company maintains a strong liquidity position with a current ratio of 5.35, indicating robust short-term financial stability despite being in the pre-revenue development phase.
The decision follows an analysis of the International Rett Syndrome Foundation’s natural history study, which indicated a negligible chance of developmental progress in patients over six years old. In contrast, all participants in the early-stage REVEAL trial showed improvement after receiving TSHA-102. The stock has shown strong momentum, posting a year-to-date return of over 50%, though InvestingPro analysis indicates the company is currently trading above its Fair Value.
The upcoming single-arm, open-label pivotal trial will involve 15 patients, each serving as their own control. The primary endpoint will be the gain or regain of developmental milestones. The FDA has advised Taysha to submit the trial protocol and statistical analysis plan as an amendment to the Investigational New Drug (IND) application, expected in the current quarter. Trial initiation is anticipated in the third quarter of 2025.
Clinical data from the REVEAL trial, with a cutoff date of May 19, 2025, reported gains in developmental milestones in all 10 patients aged 6-21 years treated with TSHA-102. These milestones are unlikely to be achieved without treatment, based on the natural history study. The high dose of TSHA-102 showed more rapid and profound effects compared to the low dose.
Safety data, as of May 20, 2025, indicated that TSHA-102 was generally well-tolerated with no serious adverse events or dose-limiting toxicities related to treatment.
TSHA-102 is an adeno-associated virus (AAV)-based gene therapy that delivers a functional form of the MECP2 gene to the central nervous system. It includes a novel miRNA-Responsive Auto-Regulatory Element (miRARE) technology to control MECP2 levels.
Rett syndrome, primarily affecting females, is one of the most common genetic causes of severe intellectual disability. There are currently no approved treatments that address the genetic root cause of the disorder.
This report is based on a press release statement from Taysha Gene Therapies, Inc.
In other recent news, Taysha Gene Therapies reported its first-quarter 2025 financial results, surpassing analyst expectations. The company achieved an earnings per share (EPS) of -$0.08, beating the forecasted -$0.09, and reported revenue of $2.3 million, exceeding the expected $1.91 million. These results were accompanied by significant progress in their gene therapy trials for Rett syndrome, which remains a key focus for the company. Additionally, Taysha has secured a cash runway through the fourth quarter of 2026, ensuring continued support for their operational activities. In regulatory developments, Taysha reached an agreement with the FDA on critical elements of their pivotal study for the TSHA-102 gene therapy candidate, which is a notable step forward in their clinical development efforts. Analyst firm Baird maintained an Outperform rating on Taysha, reiterating a $7 price target, reflecting confidence in the company’s advancements and potential. Investors are keenly awaiting detailed study designs and further insights, which Taysha plans to disclose at the upcoming International Rett Syndrome Foundation conference. These recent developments underscore Taysha’s strategic progress in the biotechnology sector.
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