uniQure (QURE) Q1 2025 Earnings Call May 09, 2025 08:30 AM ET Company Participants Chiara Russo - Senior Director - IRMatt Kapusta - CEOWalid Abi-Saab - Chief Medical Officer & Member of Management BoardChristian Klemt - CFODebjit Chattopadhyay - Senior Managing DirectorPaul Matteis - Managing Director, Head of Therapeutics ResearchPatrick Trucchio - Managing DirectorJenny Gonzalez-Armenta - Equity Research AssociateUy Ear - Vice PresidentSuzanne van Voorthuizen - Head of Life Sciences Equity Research Confe ...

华福证券 2025 年 05 月 08 日 策 略 研 究 医疗与消费周报——信玖凝获批：我国基因治疗 药物行业发展推进中 投资要点： 近期观点 1、医药指数本周（4.28-4.30）关注的 6 个子行业三分之二录得正 收益，表现良好。 策 略 定 期 报 告 2、2025 年 4 月 11 日，中国首个血友病 B 基因治疗药物信玖凝（波 哌达可基注射液）获批上市，从研发到上市仅用不到七年。基因疗法 是通过导入外源基因置换或纠正致病基因，实现"一次治疗，长期有 效"。医麦克《基因治疗药物行业发展白皮书》显示，2025 年中国基 因疗法市场规模预计达 25.9 亿美元，2025-2027 年有望达 500 亿元。 中国罕见病约 2000 种，基因疗法潜力巨大。中国丰富的基因组学数据 为研究提供支持，但基因疗法仍面临技术安全性、伦理、法规及成本 等挑战，新的基因编辑工具和递送系统为其发展提供动力。 团队成员 分析师： 赵月(S0210524050016) zy30563@hfzq.com.cn 相关报告 风险提示 技术研发不及预期；宏观需求变化；地缘政治影响 证 券 研 究 报 告 诚信专业 发现价值 1 请务必 ...

分组1 - Voyager Therapeutics reported a quarterly loss of $0.53 per share, which was worse than the Zacks Consensus Estimate of a loss of $0.35, and compared to a loss of $0.20 per share a year ago, indicating an earnings surprise of -51.43% [1] - The company posted revenues of $6.47 million for the quarter ended March 2025, missing the Zacks Consensus Estimate by 66.26%, and down from $19.52 million in the same quarter last year [2] - Voyager Therapeutics shares have declined approximately 38.5% since the beginning of the year, contrasting with the S&P 500's decline of -3.9% [3] 分组2 - The current consensus EPS estimate for the upcoming quarter is -$0.31 on revenues of $19.19 million, and for the current fiscal year, it is -$1.41 on revenues of $90.91 million [7] - The Medical - Biomedical and Genetics industry, to which Voyager Therapeutics belongs, is currently ranked in the top 31% of over 250 Zacks industries, suggesting a favorable outlook compared to lower-ranked industries [8]

Sarepta Therapeutics (SRPT) Q1 2025 Earnings Call May 06, 2025 04:30 PM ET Speaker0 Good afternoon, and welcome to the Sarepta Therapeutics First Quarter twenty twenty five Financial Results Conference Call. As a reminder, today's program is being recorded. At this time, I'll turn the call over to Mary Jenkins, Associate Director, Investor Relations. Please go ahead. Speaker1 Thank you, Marvin, and thank you all for joining today's call. Earlier this afternoon, we released our financial results for the firs ...

世界正迅速进入一个激动人心的医学新时代。曾经设计用于治疗数百万人的大型药物时代，正快速过渡到 个性化治疗的新纪元。依靠普适法则确保治疗方案适用于大多数受疗者，虽然惠及了无数人，但也使得许 多"非典型"患者无法获得有效的治疗解决方案——同时，支付方为无效治疗支付了大量资金。 在2021年，细胞和基因疗法（Cell and Gene Therapies, CGT）仅占全球药物市场总收入的0.8%。但随着超 过500种CGT进入研发状态，它们正准备在与传统技术的竞争中占据越来越大的市场份额。我们估计，全 球CGT市场将在2021年至2026年间以每年超过50%的年化增速增长，届时将有近200种疗法产生约570亿美 元的销售额，占全球药物市场的4%以上（见图1）。确实，CGT正逐步发展成为标准治疗的下一个浪潮， 瞄准一些现代社会中最常见的疾病，包括镰状细胞贫血、2型糖尿病和阿尔茨海默症。 美国FDA和其他监管机构已经批准了CGT（细胞与基因治疗）用于治疗冠状动脉疾病、遗传性视网膜病、 大B细胞淋巴瘤和脊髓性肌萎缩症等多种病理现象。 然而，最近的上市情况喜忧参半，公司在摸索前进时面临着未知的挑战，包括与往往百万美元的定 ...

Precigen, Inc. (NASDAQ:PGEN) Q4 2024 Earnings Conference Call March 19, 2025 4:30 PM ET Company Participants Steven Harasym - IR Helen Sabzevari - President and CEO Rutul Shah - COO Phil Tennant - CCO Harry Thomasian - CFO Conference Call Participants Jason Butler - Citizens Carolina Ibanez Ventoso - Stifel Swayampakula Ramakanth - H.C. Wainwright Jennifer Kim - Cantor Brian Cheng - JPMorgan Operator Good evening, and welcome to the Precigen's Full Year 2024 Financial Results and Business Update Call. At th ...

Sangamo Therapeutics, Inc. (NASDAQ:SGMO) Q4 2024 Earnings Conference Call March 17, 2025 4:30 PM ET Company Participants Louise Wilkie - Vice President, Investor Relations and Corporate Communications Sandy Macrae - Chief Executive Officer Nathalie Dubois-Stringfellow - Chief Development Officer Prathyusha Duraibabu - Chief Financial Officer Conference Call Participants Luis Santos - H.C. Wainwright Maury Raycroft - Jefferies Nicole Germino - Truist Gena Wang - Barclays Operator Good afternoon, and welcome ...

MeiraGTx Holdings (MGTX) Update / Briefing February 21, 2025 08:00 AM ET Speaker0 Good morning, and welcome to the GTX Investor Event. At this time, all attendees are in a listen only mode. A question and answer session will follow the presentations. As a reminder, this call is being recorded and a replay will be made available on the Mirror GTX website following the conclusion of the event. I'd now like to turn the call over to Doctor. Sandy Forbes, President and Chief Executive Officer of Miura GTX. Pleas ...

CANbridge Pharmaceuticals (01228) H2 2022 Earnings Call March 31, 2023 12:00 PM ET Speaker0 Good morning, everyone. Welcome to Cambridge Pharmaceuticals twenty twenty two twelve Month Annual Results Conference Call. Today, it's a great honor to have Doctor. James Shre, Founder, Chairman and CEO of Cambridge Pharmaceuticals Doctor. Gerald Cox, Chief Medical Officer and Chief Development Strategist Glenn Hassan, Chief Financial Officer and Sophie Hsieh, Director of Investor Relations. Management team will giv ...