Company to host webcast on Tuesday, June 18, 2024, at 8:00 AM Eastern Time Oral presentation by Elsa Rossignol, M.D., FRCP, FAAP, Principal Investigator of the REVEAL adolescent and adult trial at CHU Sainte-Justine and Colleen Buhrfiend, M.D., of RUSH University Medical Center at the 2024 IRSF Rett Syndrome Scientific Meeting on Wednesday, June 19, 2024, at 11:00 AM Mountain Time DALLAS, June 12, 2024 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (NASDAQ:TSHA), a clinical- stage biotechnology company foc ...

DALLAS, June 07, 2024 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (NASDAQ:TSHA), 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 announced that, on June 3, 2024, the Compensation Committee of Taysha's Board of Directors granted three new employees, in the aggregate, options to purchase 360,000 shares of the Company's common stock in connection with their employment. The ...

财务数据和关键指标变化 - 研发费用从2023年第一季度的1250万美元增加到2024年第一季度的2070万美元,主要是由于制造工艺批次活动的增加 [75][76][77] - 一般及行政费用从2023年第一季度的880万美元下降到2024年第一季度的710万美元,主要是由于员工人数减少和咨询费用降低 [77][78] - 2024年第一季度净亏损2410万美元,每股0.10美元,而2023年第一季度净亏损1760万美元,每股0.28美元 [78] - 截至2024年3月31日,公司现金及现金等价物为1.24亿美元,预计可支持公司运营和资本需求到2026年 [79] 各条业务线数据和关键指标变化 - 公司目前正在评估TSHA-102基因疗法在青少年、成人和儿童患者中的安全性和初步疗效 [20][33] - 已完成低剂量组的两名成人患者的长期随访,观察到多个临床领域的持续改善,包括运动功能、手功能、自主功能和癫痫发作的减少 [27][28][29][30][31][32] - 已给第二名儿童患者给予低剂量TSHA-102,预计将在2024年中期报告初步数据 [38][39] 各个市场数据和关键指标变化 - 公司正在加快在美国和加拿大扩大青少年和成人试验的临床试验点,同时也在英国启动儿童试验 [72][73] - 公司获得FDA的RMAT(Regenerative Medicine Advanced Therapy)指定,这将使公司能够与FDA高级管理层进行更密切的沟通和合作 [40][41][92][93] 公司战略和发展方向及行业竞争 - 公司认为TSHA-102有潜力成为治疗雷特综合征的变革性疗法,因为它可以适当调节中枢神经系统中MECP2蛋白的表达 [19][46][47][48][49][50][51] - 公司计划继续推进TSHA-102在不同年龄段和疾病阶段患者中的临床评估,以获得更多长期临床数据 [16][42][43] - 公司认为TSHA-102有望成为治疗雷特综合征这一罕见神经发育障碍的首个获批的疾病改善疗法 [17][18] 管理层对经营环境和未来前景的评论 - 管理层对TSHA-102在成人和儿童患者中的持续改善效果感到鼓舞,认为这支持该基因疗法的持久和变革性潜力 [32][65] - 管理层表示将继续密切关注TSHA-102在不同年龄段和疾病阶段患者中的临床数据,以指导后续研究计划 [42][43] - 管理层认为RMAT指定将有助于与FDA加强沟通和合作,加快TSHA-102的开发进程 [40][41][92][93] 问答环节重要的提问和回答 问题1 **Kristen Kluska 提问** 对于年轻患者的疗效评估,公司有何预期?[86][87][88][89] **Sean Nolan 和 Sukumar Nagendran 回答** - 公司希望在儿童患者中看到与成人患者类似的一致性改善模式,包括运动功能、手功能、交流社交等多个领域 [87][88][89][127][128][129][130][131][132][133][134][135][136][137][138][139][140][141] - 尽管儿童患者群体更为异质,但公司预计TSHA-102仍能在不同基因型患者中产生一致的治疗效果 [136][137][138][139][140][141] 问题2 **Salveen Richter 提问** 中期报告将包括哪些内容?RMAT指定对后续开发意味着什么?[94][95][96] **Sean Nolan 和 Sukumar Nagendran 回答** - 中期报告将更新成人低剂量组两名患者的最新临床观察和评估结果,以及儿童患者的初步数据 [95] - RMAT指定将使公司能与FDA高层进行更密切的沟通和合作,有助于加快TSHA-102的开发进程 [96][97][98]

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, 2024 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 84-3199 ...

Taysha Gene Therapies, Inc. (NASDAQ:TSHA) Q4 2023 Earnings Conference Call March 19, 2024 4:30 PM ET Company Participants Hayleigh Collins - Director, Head of Corporate Communications and Investor Relations Sean Nolan - Chief Executive Officer Sukumar Nagendran - President and Head of Research and Development Kamran Alam - Chief Financial Officer Conference Call Participants Whitney Ijem - Canaccord Elizabeth Webster - Goldman Sachs Kristen Kluska - Cantor Fitzgerald Gil Blum - Needham & Company Mehdi Gouda ...

Corporate Presentation March 2024 Legal disclosure FORWARD LOOKING STATEMENTS This presentation contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, contained in this presentation, including statements regarding our strategy, future operations, future financial position, future revenues, projected costs, prospects, plans and objectives of management, are forward-looking statements. The words “anticipate,” “believe,” ...

Exhibit 99.1 Taysha Gene Therapies Reports Full Year 2023 Financial Results and Provides Corporate and Clinical Updates Data from first adult patient in REVEAL Phase 1/2 trial showed TSHA-102 (low dose, 5.7x1014 total vg) was well-tolerated with no treatment-emergent SAEs as of 35-week assessment, with sustained improvement across key efficacy measures at decreased steroid levels and new improvement in RSBQ at month six Data from second adult patient showed TSHA-102 (low dose, 5.7x1014 total vg) was well-to ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-K (Mark One) ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2023 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 84-3199512 ( ...

Taysha Gene Therapies, Inc. (NASDAQ:TSHA) Q3 2023 Results Conference Call November 14, 2023 4:30 PM ET Company Participants Hayleigh Collins - Director and Head of Corporate Communications Sean Nolan - Chief Executive Officer Sukumar Nagendran - President and Head of Research and Development Kamran Alam - Chief Financial Officer Conference Call Participants Whitney Ijem - Canaccord Genuity Kristen Kluska - Cantor Fitzgerald Salveen Richter - Goldman Sachs Gil Blum - Needham & Co Yanan Zhu - Wells Fargo Secu ...

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 September 30, 2023 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- ...