Taysha Gene Therapies Reports 2024 Financial Results, Updates TSHA-102 Program

Dallas, Feb. 26, 2025 – Taysha Gene Therapies, Inc. (NASDAQ: TSHA) today announced its financial results for the full year ended December 31, 2024, along with a corporate update on its clinical-stage TSHA-102 program for Rett syndrome. The results were detailed in a press release furnished as Exhibit 99.1 to the company’s Form 8-K filing with the U.S. Securities and Exchange Commission.

The company reported a net loss of $89.3 million, or $0.36 per share, for the full year 2024 – an improvement over the $111.6 million, or $0.96 per share, net loss recorded in 2023. Research and development expenses increased to $66.0 million in 2024 from $56.8 million the prior year, driven primarily by expenses related to Good Manufacturing Practices batch activities for TSHA-102 and enhanced clinical trial efforts across the two REVEAL Phase 1/2 trials. General and administrative expenses declined by approximately $1.0 million for the same period. As of December 31, 2024, the company held $139.0 million in cash and cash equivalents, which it expects will fund operating expenses and capital requirements into the fourth quarter of 2026.

On the clinical front, Taysha Gene Therapies provided an update regarding its TSHA-102 program, a gene transfer therapy developed for Rett syndrome. The company announced that dosing of the 10 patients in Part A of its dual REVEAL trials has been completed. The clinical dataset comprises six patients in the high dose cohort (1×10^15 total vector genomes) and four patients in the low dose cohort (5.7×10^14 total vector genomes). Notably, TSHA-102 was generally well tolerated across pediatric, adolescent, and adult patients, with no treatment-related serious adverse events or dose-limiting toxicities reported in either cohort as of the February 2025 data cutoff.

Taysha’s executives noted that the maturing data from the completed dosing phase continues to support advancement toward the pivotal Part B trial. The company is engaged in ongoing discussions with the U.S. Food and Drug Administration to solidify the regulatory pathway for TSHA-102, with an update on pivotal trial design expected in the first half of 2025. Additional clinical data, including safety and efficacy updates from both the adolescent/adult and pediatric trials, are also anticipated during the first half of 2025.

Taysha Gene Therapies, a clinical-stage biotechnology company focused on AAV-based gene therapies targeting severe monogenic diseases of the central nervous system, is advancing TSHA-102 as a one-time treatment aimed at addressing the genetic root cause of Rett syndrome. The company’s strategy includes leveraging regulatory designations and a proven gene therapy platform to potentially provide meaningful therapeutic benefits to a broad patient population affected by this rare neurodevelopmental disorder.

This financial and clinical update underscores Taysha’s continued progress in its mission to develop transformative therapies for severe unmet medical needs.

This article was generated by an automated content engine and was reviewed by a human editor prior to publication. For additional information, read Taysha Gene Therapies’s 8K filing here.

About Taysha Gene Therapies

Taysha Gene Therapies, Inc, a gene therapy company, focuses on developing and commercializing adeno-associated virus-based gene therapies for the treatment of monogenic diseases of the central nervous system. It primarily develops TSHA-120 for the treatment of giant axonal neuropathy; TSHA-102 for the treatment of Rett syndrome; TSHA-121 for the treatment of CLN7 disease; TSHA-118 for the treatment of CLN1 disease; TSHA-105 for the treatment of for SLC13A5 deficiency; TSHA-113 for the treatment of tauopathies; TSHA-106 for the treatment of angelman syndrome; TSHA-114 for the treatment of fragile X syndrome; and TSHA-101 for the treatment of GM2 gangliosidosis.