TSHA 最新10-Q变化

相对上期新增的文字 · 来源:10-Q · 2026-08-11

• We have completed dosing of the 12 patients aged 6 to 21 years in Part A of both REVEAL trials, which includes eight patients in cohort two (high dose, 1x1015 total vg) and four patients in cohort one (low dose, 5.7x1014 total vg).

• Longer-Term Results from Part A of REVEAL Trials as of May 2026 Data Cutoff

• Developmental milestones are defined as a functional gain of one or more of the 28 milestones defined in the natural history study. This is the primary evidence of efficacy that will support our planned BLA submission and the FDA’s review of efficacy in the REVEAL pivotal trial. In REVEAL Part A, de…

• The 12 patients treated in REVEAL Part A ranged from 6 to 21 years of age at dosing, with post-treatment follow-up spanning 12 to 30 months. We enrolled patients across a broad spectrum of disease severity, as assessed by the Clinical Global Impression-Severity, or CGI-S, scale, a 7-point, clinician…

• As of the May 2026 data cutoff, 100% of the 12 patients in REVEAL Part A gained or regained ≥ one developmental milestone across the core functional domains of fine motor, gross motor and communication post-treatment with TSHA-102. These results were seen despite the broad range in age and disease s…

相对上期删除的文字 · 来源:10-Q · 2026-05-06

• We have completed dosing of the 12 patients in Part A of both REVEAL trials, which includes eight patients in cohort two (high dose, 1x1015 total vg) and four patients in cohort one (low dose, 5.7x1014 total vg). We reported positive clinical data in May 2025, demonstrating that the first 10 patient…

• be generally well tolerated with no treatment-related serious adverse events, or SAEs, or dose-limiting toxicities, or DLTs, in all patients treated in the REVEAL Phase 1/2 and REVEAL pivotal trials as of the May 2026 data cutoff.

• An update on longer-term safety and efficacy data from the Part A REVEAL Phase 1/2 trials (n=12) is expected in the second quarter of 2026.

• The TSHA-102 clinical development program is designed to support the potential future approval of TSHA-102 for a broad population of patients aged 2 years and older with Rett syndrome through an efficient and rigorously designed pathway. In close collaboration with the United States Food and Drug Ad…

• The REVEAL pivotal trial, also known as Part B, is a single-arm, open-label study evaluating the efficacy and safety of TSHA-102 in females with Rett syndrome, with each patient serving as their own control. Each participant will receive a single administration of the high dose (1x1015 total vg) of …

相对上期新增的文字 · 来源:10-Q · 2026-08-11

• reasonable assurance that the information required to be disclosed by us in this Quarterly Report on Form 10-Q was (a) reported within the time periods specified by SEC rules and regulations, and (b) communicated to our management, including our Chief Executive Officer and Chief Financial Officer, t…

• There were no changes in our internal control over financial reporting identified in management’s evaluation pursuant to Rules 13a-15(d) or 15d-15(d) of the Exchange Act during the period covered by this Quarterly Report on Form 10-Q that materially affected, or are reasonably likely to materially a…

相对上期删除的文字 · 来源:10-Q · 2026-05-06

• During the three months ended March 31, 2026, we implemented an enterprise resource planning system, NetSuite, which resulted in certain changes to existing business processes and internal control over financial reporting. Other than this transition, there were no other changes in our internal contr…