文章核心观点 - 泰莎基因疗法公司宣布在2025年国际雷特综合征基金会科学会议上展示支持TSHA - 102项目的最新数据，该项目有望改善雷特综合征患者生活质量 [2][3] 公司信息 - 泰莎基因疗法公司是临床阶段生物技术公司，专注腺相关病毒（AAV）中枢神经系统单基因疾病基因疗法开发，其TSHA - 102项目用于治疗雷特综合征 [2][7] - 公司管理团队有基因疗法开发和商业化经验，利用制造工艺和AAV9衣壳将治疗方法从实验室转化到临床 [7] 产品信息 - TSHA - 102是鞘内注射的自互补AAV9研究性基因转移疗法，旨在通过向中枢神经系统细胞递送功能性MECP2解决雷特综合征的遗传根源问题，采用miRARE技术调节MECP2水平且无过度表达风险 [5] - TSHA - 102获得FDA再生医学先进疗法、快速通道、孤儿药和罕见儿科疾病指定，欧盟委员会孤儿药指定，以及英国药品和保健品监管局创新许可和准入途径指定 [5] 行业信息 - 雷特综合征是由X连锁MECP2基因突变引起的罕见神经发育障碍，主要影响女性，是严重智力残疾常见遗传原因，目前无获批的疾病修饰疗法 [6] - 美国、欧盟和英国约1.5万至2万名患者受致病性/可能致病性MECP2突变导致的雷特综合征影响 [6] 会议信息 - 公司将在2025年6月9 - 11日于波士顿举行的国际雷特综合征基金会科学会议上进行三次口头报告和一次专题研讨会，报告也将在6月9日海报环节展示 [2][4] - 报告内容包括REVEAL青少年/成人和儿科临床试验中TSHA - 102的安全性和有效性数据、护理人员对雷特综合征基因疗法有意义改善的见解、非人类灵长类动物中rAAV9载体在脑和脊髓的生物分布，专题研讨会聚焦发育里程碑的获得和恢复 [1][4]

This article is published by Terry Chrisomalis, who runs the Biotech Analysis Central pharmaceutical service on Seeking Alpha Marketplace. If you like what you read here and would like to subscribe to, I'm currently offering a two-week free trial period for subscribers to take advantage of. My service offers a deep-dive analysis of many pharmaceutical companies. The Biotech Analysis Central SA marketplace is $49 per month, but for those who sign up for the yearly plan will be able to take advantage of a 33. ...

Taysha Gene Therapies Inc. TSHA on Wednesday announced details of its planned pivotal Part B trial design for TSHA-102 following written alignment from the U.S. Food and Drug Administration (FDA).Single-arm, open-label trial with patients serving as their control (intend N=15).Enrollment of females in the developmental plateau population of Rett syndrome (≥ 6 years).The primary endpoint will assess developmental milestone gain or regain.The safety of TSHA-102 will be evaluated in females in the pre-developm ...

Natural history data analysis established patients ≥ six years of age are in developmental plateau, with a ~0% likelihood of gaining/regaining developmental milestones across the core functional domains of Rett syndrome Written alignment from FDA supports single-arm, open label pivotal trial with primary endpoint of developmental milestone gain/regain in the developmental plateau population (≥ 6 years, intend N=15) with each patient serving as their own control 100% of patients in REVEAL Part A (N=10, 6-21 ...

Taysha Gene Therapies, Inc. (TSHA) came out with a quarterly loss of $0.08 per share in line with the Zacks Consensus Estimate. This compares to loss of $0.10 per share a year ago. These figures are adjusted for non-recurring items.A quarter ago, it was expected that this company would post a loss of $0.08 per share when it actually produced a loss of $0.07, delivering a surprise of 12.50%.Over the last four quarters, the company has surpassed consensus EPS estimates two times.Taysha Gene Therapies, which b ...

Taysha Gene Therapies Inc (TSHA) Q1 2025 Earnings Call May 15, 2025 08:30 AM ET Company Participants Hayleigh Collins - Senior Director - Corporate Communications & Investor RelationsSean Nolan - CEO & ChairmanSukumar Nagendran - President & Head - Research & DevelopmentKamran Alam - Chief Financial OfficerWhitney Ijem - Managing DirectorMalcolm Hoffman - Senior BioPharma Equity Research Associate Conference Call Participants Kristen Kluska - AnalystLydia Erdman - Biotech Equity Research AnalystGil Blum - S ...

Taysha Gene Therapies Inc (TSHA) Q1 2025 Earnings Call May 15, 2025 08:30 AM ET Speaker0 Greetings, and welcome to the Tatia Gene Therapies First Quarter twenty twenty five Earnings Call. At this time, all participants are in listen only mode. A question and answer session will follow the formal presentation. As a reminder, this conference is being recorded. I would now like to turn the conference over to your host, Hayley Collins, Director, Head of Corporate Communications. Please go ahead. Speaker1 Thank ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2025 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ___________ to ___________ Commission File Number: 001-39536 Taysha Gene Therapies, Inc. (Exact Name of Registrant as Specified in its Charter) | Delaware | 84- ...

Exhibit 99.1 Taysha Gene Therapies Reports First Quarter 2025 Financial Results and Provides Corporate Update Obtained written alignment from the FDA on key elements of TSHA-102 pivotal Part B trial design and next steps on enabling study initiation The FDA advised the Company to submit pivotal Part B trial protocol and associated statistical analysis plan as an amendment to the IND application, eliminating the need for formal end-of-phase meeting and potentially expediting study initiation and registration ...

High dose and low dose of TSHA-102 continue to be generally well tolerated with no treatment-related SAEs or DLTs in the pediatric, adolescent and adult patients treated (high dose, n=6; low dose, n=4) across both REVEAL trials as of April 10, 2025, data cutoff Program update on pivotal trial design, Rett syndrome natural history data analysis and clinical data from cohort two (high dose) and cohort one (low dose) of both REVEAL trials expected in Q2 2025 in conjunction with IRSF Rett Syndrome Scientific Me ...