文章核心观点 公司单剂量专有的靶向脂质纳米颗粒（tLNP）在非人类灵长类动物（NHPs）的造血干细胞（HSCs）中实现了治疗相关水平的HBG1/2启动子编辑，支持开发治疗镰状细胞病和β - 地中海贫血的新型体内方法 [1][2] 研究成果 - 单剂量高效HSC递送在五个月后实现了58%的平均编辑，证明使用临床验证策略达到治疗相关编辑水平 [1] - 公司专有的tLNP制剂将HBG1/2启动子编辑货物递送至NHPs的HSCs，单次静脉注射tLNP在五个月后使HSCs中HBG1/2启动子区域的平均靶向编辑水平达到58%，远超治疗所需的≥25%的预测编辑阈值 [3] - 公司tLNP在NHPs中的生物分布数据显示与标准LNP相比肝脏脱靶显著 [3] 研究意义 - 体内HSC项目的数据证实公司有能力在NHPs中实现高效递送、治疗相关编辑水平和良好的生物分布，验证了公司专有的HSC - tLNP用于编辑HBG1/2启动子治疗镰状细胞病和β - 地中海贫血的进一步开发 [4] 研究方法 - 体内HSC项目靶向HBG1/2启动子以模拟胎儿血红蛋白遗传性持续存在（HPFH）的自然发生机制，并利用专有的AsCas12a进行高效编辑并减少脱靶编辑 [5] 研究展示信息 - 公司将在2025年6月14日下午6:30 - 7:30 CEST（美国东部时间中午12:30 - 1:30）于意大利米兰举行的欧洲血液学协会（EHA）2025年大会的海报会议上详细介绍数据 [2] - 海报展示标题为“非人类灵长类动物中的靶向脂质纳米颗粒递送实现β - 血红蛋白病的体内HBG1/2启动子编辑”，地点在米兰会议中心Allianz MiCo，属于海报会议2 [7] 公司概况 - 作为一家开创性的基因编辑公司，专注于将CRISPR/Cas12a和CRISPR/Cas9基因组编辑系统的潜力转化为针对全球严重疾病患者的体内药物管线，旨在发现、开发、制造和商业化变革性、持久性、精准的体内基因编辑药物 [7] - 是博德研究所Cas12a专利资产以及博德研究所和哈佛大学Cas9专利资产用于人类药物的独家被许可方 [7]

It has been about a month since the last earnings report for Editas Medicine (EDIT) . Shares have added about 40% in that time frame, outperforming the S&P 500.Will the recent positive trend continue leading up to its next earnings release, or is Editas due for a pullback? Before we dive into how investors and analysts have reacted as of late, let's take a quick look at the most recent earnings report in order to get a better handle on the important catalysts.How Have Estimates Been Moving Since Then?It tur ...

Over the past few years, investors have moved away from somewhat speculative and unprofitable companies to put their money into safer, steadier investments. Editas Medicine (EDIT -4.01%), a gene-editing-focused clinical-stage biotech, is firmly in the speculative camp, which is why its shares are down by 97% since early 2021.The stock is trading for about $1.50 right now. However, its average price target of $3.38 (according to Yahoo! Finance) implies a potential upside of about 125%. Should investors scoop ...

Explore the exciting world of Editas Medicine (EDIT 1.09%) with our expert analysts in this Motley Fool Scoreboard episode. Check out the video below to gain valuable insights into market trends and potential investment opportunities!*Stock prices used were the prices of April 9, 2025. The video was published on May 15, 2025. ...

Editas Medicine, Inc. (NASDAQ:EDIT) BofA Securities 2025 Healthcare Conference May 14, 2025 8:15 PM ET Company Participants Amy Parison - CFO Christi Bernett - SVP of IR Conference Call Participants Alec Stranahan - BofA Securities Alec Stranahan Welcome to the last fireside for day 1 of the 2025 Bank of America Healthcare Conference. Thanks for joining this session with Editas. My name is Alec Stranahan. I'm senior biotech analyst at BofA, covering Editas. And I'm pleased to be joined by Amy Parison, Chie ...

Preclinical studies achieved therapeutically relevant gene editing levels of the HBG1/2 promoter & favorable biodistribution profile in non-human primates using a clinically validated editing strategy Data reinforces continued development as a potentially transformative, in vivo approach to treating sickle cell disease and beta thalassemia CAMBRIDGE, Mass., May 14, 2025 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a pioneering gene editing company, today announced that new data from a study in ...

纪要涉及的公司 Editas 纪要提到的核心观点和论据 1. **公司战略** - 自CEO Gilmore约三年前加入后，公司目标成为体内基因编辑领域的领军企业，目前已取得令人兴奋的数据并持续推进 [5] - 战略是上调功能性蛋白质，目标是成为体内基因疗法的一流企业，以此指导未来项目和产品线规划 [15][16] 2. **体内基因编辑优势** - 体内基因编辑可扩大市场和潜力，减轻患者和基础设施负担，是治疗镰状细胞病的理想方式 [6][7] - 其效果与体外疗法相当甚至更好，能覆盖更多患者群体 [9] 3. **研发进展** - 借鉴体外项目Runicel的经验用于体内项目，临床验证了hbt12启动子靶点，专注于专有TLNP递送技术，在临床前显示出巨大潜力 [10][11] - 本周在ASGCT展示肝脏项目海报，实现基因靶点的最大编辑，使疾病生物标志物降低超80%；体内HSC项目持续展示后续数据和编辑水平数据，明天有相关口头报告 [32][33] 4. **业务拓展与合作** - 开放所有融资途径，包括合作或授权专利以获取非稀释性资金，因当前市场融资困难 [17] - 专注于产生令人兴奋的科学成果和展示基因编辑潜力，以创造对患者和股东有价值的项目来规划产品线 [18][19] 5. **资金与成本管理** - 公司现金可支撑到2027年第二季度，会谨慎部署资金，确保与战略一致，同时利用在公司的经验提高资金使用效率 [20][21][23] - 对前Exvio项目的收尾成本进行有效管理，有助于维持现金储备 [26] 6. **知识产权与许可** - CAFC裁决将案件发回PTAB重新审查，但公司知识产权仍然有效，现有许可协议不受影响，仍可继续授权 [28][29] 7. **未来催化剂** - 2025年年中宣布两个项目的候选药物，2026年为其中一个候选药物提交IND申请，2026年底至2027年初展示人体机制验证 [36] 其他重要但是可能被忽略的内容 - 肝脏项目的具体适应症尚未披露，将在合适时间公布 [33] - 首席财务官Amy Parson虽新担任该角色，但已在公司工作近三年，能利用经验帮助公司更有效地思考和行动 [23]

For those looking to find strong Medical stocks, it is prudent to search for companies in the group that are outperforming their peers. Editas Medicine (EDIT) is a stock that can certainly grab the attention of many investors, but do its recent returns compare favorably to the sector as a whole? By taking a look at the stock's year-to-date performance in comparison to its Medical peers, we might be able to answer that question.Editas Medicine is one of 1001 individual stocks in the Medical sector. Collectiv ...

Editas Medicine (EDIT) reported an adjusted loss of 43 cents per share in the first quarter of 2025, narrower than the Zacks Consensus Estimate of a loss of 51 cents. The adjusted figure excluded the effect of restructuring and impairment charges in the reported quarter. The company had incurred a loss of 76 cents per share in the year-ago quarter.Collaboration and other research and development (R&D) revenues, which comprise the company’s top line, were $4.7 million in the reported quarter, up significantl ...

Editas Medicine (EDIT) came out with a quarterly loss of $0.43 per share versus the Zacks Consensus Estimate of a loss of $0.51. This compares to loss of $0.76 per share a year ago. These figures are adjusted for non-recurring items.This quarterly report represents an earnings surprise of 15.69%. A quarter ago, it was expected that this genome editing company would post a loss of $0.39 per share when it actually produced a loss of $0.55, delivering a surprise of -41.03%.Over the last four quarters, the comp ...