Full Press Release Details
TSHA-102 granted Breakthrough Therapy designation by FDA
Finalized FDA alignment on REVEAL pivotal trial protocol and SAP, including 6-month interim analysis that may expedite BLA submission, which was enabled by the rigorous developmental milestone evaluation in Part A REVEAL Phase 1/2 trials showing an unprecedented response rate
Dosing of first patient in REVEAL pivotal trial scheduled for Q4 2025, with enrollment of additional patients expected to continue at multiple sites this quarter
Presented new supplemental analysis of Part A REVEAL data reinforcing the broad and consistent, multi-domain impact of TSHA-102on activities of daily living at the CNS Annual Meeting
TSHA-102 continues to be generally well tolerated with no treatment-related SAEs or DLTs in the 12 patients treated in the Part A REVEAL Phase 1/2 trials as of October 2025 data cutoff
Regained full unencumbered rights to TSHA-102 Rett syndrome program, enabling Taysha to focus on driving long-term value with full strategic flexibility and optionality
Conference call and webcast today at 8:30 AM Eastern Time
DALLAS, Nov. 04, 2025 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA) (Taysha or the Company), a clinical-stage biotechnology company focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system (CNS), today reported financial results for the third quarter ended September 30, 2025, and provided a corporate update.
“The progress we’ve made in the third quarter of 2025 sets the stage for a potentially transformative period ahead for Taysha. We recently received FDA Breakthrough Therapy designation, which reflects the FDA’s recognition of the therapeutic potential of TSHA-102 for individuals with Rett syndrome, who face a profound unmet need. Additionally, we’re pleased to have finalized alignment with the FDA on our pivotal trial protocol and SAP, including a six-month interim analysis, which we believe provides a clear opportunity to expedite our BLA submission by at least two quarters,” said Sean P. Nolan, Chairman and Chief Executive Officer of Taysha. “With Breakthrough Therapy designation and finalized FDA alignment, together with our strong balance sheet and regained global rights to TSHA-102, we believe we are strongly positioned to initiate our REVEAL pivotal trial and accelerate execution toward BLA submission. We remain on track to dose the first patient in the REVEAL pivotal trial this quarter and expect additional enrollment to continue at multiple sites this quarter. With an estimated 15,000 to 20,000 patients affected by Rett syndrome across the U.S., EU and U.K. and compelling clinical data from Part A of our REVEAL trials, we see a significant opportunity to bring an innovative therapy with disease-modifying potential to patients.”
Recent Corporate and TSHA-102 Program Highlights
Anticipated Milestones
Third Quarter 2025 Financial Highlights
Conference Call and Webcast InformationTaysha management will hold a conference call and webcast today at 8:30 a.m. ET to review its financial and operating results and provide a corporate update. The dial-in number for the conference call is 800-245-3047 (U.S./Canada) or 203-518-9765 (international). The conference ID for all callers is TAYSHA. The live webcast and replay may be accessed by visiting Taysha’s website.
About TSHA-102TSHA-102 is a self-complementary intrathecally delivered AAV9 investigational gene transfer therapy in clinical evaluation for Rett syndrome. Designed as a one-time treatment, TSHA-102 aims to address the genetic root cause of the disease by delivering a functional form ofMECP2to cells in the CNS. TSHA-102 utilizes a novel miRNA-Responsive Auto-Regulatory Element (miRARE) technology designed to mediate levels ofMECP2in the CNS on a cell-by-cell basis without risk of overexpression. TSHA-102 has received Breakthrough Therapy, Regenerative Medicine Advanced Therapy, Fast Track and Orphan Drug and Rare Pediatric Disease designations from the FDA, Orphan Drug designation from the European Commission and Innovative Licensing and Access Pathway designation from the Medicines and Healthcare products Regulatory Agency.
About Rett SyndromeRett syndrome is a rare neurodevelopmental disorder caused by mutations in the X-linkedMECP2gene encoding methyl CpG-binding protein 2 (MeCP2), which is essential for regulating neuronal and synaptic function in the brain. The disorder is characterized by loss of communication and hand function, slowing and/or regression of development, motor and respiratory impairment, seizures, intellectual disabilities and shortened life expectancy. Rett syndrome progression is divided into four key stages, beginning with early onset stagnation at 6 to 18 months of age followed by rapid regression, plateau and late motor deterioration. Rett syndrome primarily occurs in females and is one of the most common genetic causes of severe intellectual disability. Currently, there are no approved disease-modifying therapies that treat the genetic root cause of the disease. Rett syndrome caused by a pathogenic/likely pathogenicMECP2mutation is estimated to affect between 15,000 and 20,000 patients in the U.S., EU, and U.K.
About Taysha Gene TherapiesTaysha Gene Therapies (Nasdaq: TSHA) is a clinical-stage biotechnology company focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system. Its lead clinical program TSHA-102 is in development for Rett syndrome, a rare neurodevelopmental disorder with no approved disease-modifying therapies that address the genetic root cause of the disease. With a singular focus on developing transformative medicines, Taysha aims to address severe unmet medical needs and dramatically improve the lives of patients and their caregivers. The Company’s management team has proven experience in gene therapy development and commercialization. Taysha leverages this experience, its manufacturing process and a clinically and commercially proven AAV9 capsid in an effort to rapidly translate treatments from bench to bedside. For more information, please visitwww.tayshagtx.com.
Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as “anticipates,” “believes,” “expects,” “intends,” “projects,” “plans,” and “future” or similar expressions are intended to identify forward-looking statements. Forward-looking statements include, but are not limited to, statements concerning: the potential of TSHA-102, including the reproducibility and durability of any favorable results initially seen in patients dosed to date in clinical trials, including with respect to functional milestones, to positively impact quality of life and alter the course of disease in the patients Taysha seeks to treat; Taysha’s research, development and regulatory plans for TSHA-102, including the timing of enrolling and dosing patients, initiating additional trials, reporting data from Taysha’s clinical trials and making regulatory submissions, communications with feedback from the FDA on the regulatory pathway for TSHA-102; the potential for TSHA-102 to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed; Taysha’s ability to realize the benefits of Breakthrough Therapy Designation; Taysha’s ability to drive long-term value for stockholders; and the potential market opportunity for Taysha’s product candidates and Taysha’s anticipated cash runway. Forward-looking statements are based on management’s current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding Taysha’s business are described in detail in Taysha’s Securities and Exchange Commission (“SEC”) filings, including in Taysha’s Annual Report on Form 10-K for the full-year ended December 31, 2024 and Quarterly Report on Form 10-Q for the quarter ended September 30, 2025, which are available on the SEC’s website atwww.sec.gov. Additional information will be made available in other filings that Taysha makes from time to time with the SEC. These forward-looking statements speak only as of the date hereof, and Taysha disclaims any obligation to update these statements except as may be required by law.
| Taysha Gene Therapies, Inc.Condensed Consolidated Statements of Operations(in thousands, except share and per share data) | ||||||||||||||||
| For the Three MonthsEnded September 30, | For the Nine MonthsEnded September 30, | |||||||||||||||
| 2025 | 2024 | 2025 | 2024 | |||||||||||||
| Revenue | $ | — | $ | 1,788 | $ | 4,288 | $ | 6,311 | ||||||||
| Operating expenses: | ||||||||||||||||
| Research and development | 25,745 | 14,946 | 61,451 | 50,676 | ||||||||||||
| General and administrative | 8,279 | 7,902 | 25,035 | 22,324 | ||||||||||||
| Impairment of long-lived assets | — | 4,838 | — | 4,838 | ||||||||||||
| Total operating expenses | 34,024 | 27,686 | 86,486 | 77,838 | ||||||||||||
| Loss from operations | (34,024 | ) | (25,898 | ) | (82,198 | ) | (71,527 | ) | ||||||||
| Other income (expense): | ||||||||||||||||
| Change in fair value of warrant liability | (292 | ) | 75 | (463 | ) | (67 | ) | |||||||||
| Change in fair value of term loan | (1,534 | ) | (1,703 | ) | (4,525 | ) | (4,035 | ) | ||||||||
| Interest income | 3,169 | 2,107 | 6,354 | 5,240 | ||||||||||||
| Interest expense | (15 | ) | (24 | ) | (51 | ) | (80 | ) | ||||||||
| Other expense | (37 | ) | (81 | ) | (261 | ) | (44 | ) | ||||||||
| Total other income, net | 1,291 | 374 | 1,054 | 1,014 | ||||||||||||
| Net loss | $ | (32,733 | ) | $ | (25,524 | ) | $ | (81,144 | ) | $ | (70,513 | ) | ||||
| Net loss per common share, basic and diluted | $ | (0.09 | ) | $ | (0.10 | ) | $ | (0.26 | ) | $ | (0.29 | ) | ||||
| Weighted average common shares outstanding, basic and diluted | 353,309,524 | 267,824,045 | 307,175,982 | 244,052,057 | ||||||||||||
| Taysha Gene Therapies, Inc.Condensed Consolidated Balance Sheet Data(in thousands, except share and per share data) | ||||||||
| September 30,2025 | December 31,2024 | |||||||
| ASSETS | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 297,344 | $ | 139,036 | ||||
| Restricted cash | 449 | 449 | ||||||
| Prepaid expenses and other current assets | 2,158 | 2,645 | ||||||
| Total current assets | 299,951 | 142,130 | ||||||
| Restricted cash | 2,151 | 2,151 | ||||||
| Property, plant and equipment, net | 6,805 | 7,485 | ||||||
| Operating lease right-of-use assets | 7,463 | 8,381 | ||||||
| Other non-current assets | 184 | 217 | ||||||
| Total assets | $ | 316,554 | $ | 160,364 | ||||
| LIABILITIES AND STOCKHOLDERS' EQUITY | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 5,438 | $ | 3,592 | ||||
| Accrued expenses and other current liabilities | 17,708 | 12,862 | ||||||
| Deferred revenue | 5,485 | 9,773 | ||||||
| Total current liabilities | 28,631 | 26,227 | ||||||
| Term loan, net | 50,852 | 43,942 | ||||||
| Operating lease liability, net of current portion | 16,506 | 17,361 | ||||||
| Other non-current liabilities | 1,576 | 1,309 | ||||||
| Total liabilities | 97,565 | 88,839 | ||||||
| Stockholders' equity | ||||||||
| Common stock, $0.00001 par value per share; 700,000,000 shares authorized and 273,915,373 issued and outstanding as of September 30, 2025, and 400,000,000 shares authorized and 204,943,306 issued and outstanding as of December 31, 2024 | 3 | 2 | ||||||
| Additional paid-in capital | 903,578 | 677,859 | ||||||
| Accumulated other comprehensive loss | (1,143 | ) | (4,031 | ) | ||||
| Accumulated deficit | (683,449 | ) | (602,305 | ) | ||||
| Total stockholders’ equity | 218,989 | 71,525 | ||||||
| Total liabilities and stockholders' equity | $ | 316,554 | $ | 160,364 | ||||