文章核心观点 基因编辑公司Editas Medicine宣布有五篇摘要被美国基因与细胞治疗学会第28届年会接受展示，将呈现临床前数据支持体内基因编辑药物开发 [1] 公司进展 - 公司体内药物在临床前开发方面取得显著进展，有望在年会分享概念验证数据，其tLNP递送进展凸显“即插即用”方法在多组织执行基因上调策略的潜力 [2] 会议展示信息 口头报告 - 标题为“使用脂质纳米颗粒将HBG1/2启动子编辑载体体内递送至人源化小鼠和非人灵长类动物的造血干细胞”，时间为2025年5月14日下午1:30 - 1:45 CT，会议主题为“碱基和引物编辑器的转化应用”，地点在265 - 268房间，摘要编号为AMA353 [3] 海报展示 - 标题为“用于体内造血干细胞编辑的改良LNP靶向配体的设计与开发”，时间为2025年5月13日下午6:00 - 7:30 CT，会议主题为“周二海报招待会”，地点在12号海报厅，摘要编号为AMA245 [3] - 标题为“用于提高LNP递送的基因编辑载体效力的化学修饰AsCas12a引导RNA的设计”，时间为2025年5月13日下午6:00 - 7:30 CT，会议主题为“周二海报招待会”，地点在12号海报厅，摘要编号为AMA420 [3] - 标题为“使用LNP递送的AsCas12a核酸酶在非人灵长类动物中对多个肝脏靶点进行体内基因编辑和降低疾病相关生物标志物”，时间为2025年5月14日下午5:30 - 7:00 CT，会议主题为“周三海报招待会”，地点在12号海报厅，摘要编号为AMA640 [3][5] - 标题为“体内CRISPR编辑基因调控区域导致小鼠靶蛋白功能上调和疾病相关生物标志物显著降低”，时间为2025年5月14日下午5:30 - 7:00 CT，会议主题为“周三海报招待会”，地点在12号海报厅，摘要编号为AMA351 [5] 展示数据内容 - 展示使用靶向脂质纳米颗粒将HBG1/2启动子编辑载体递送至骨髓造血干细胞和/或祖细胞的体内临床前数据 [4] - 展示使用体内CRISPR编辑上调目标蛋白表达并降低相关小鼠疾病模型中疾病相关生物标志物的未披露肝脏靶点的临床前概念验证 [4] - 展示首次体内小鼠和非人灵长类动物研究的概念验证结果，证明静脉注射Genevant的LNP递送的AsCas12a信使RNA和化学修饰引导RNA后肝脏中靶基因编辑水平高以及相应的生物标志物反应 [4] - 展示更多体内基因编辑能力的临床前数据，包括引导修饰和靶向部分优化以提高效力和改善体内基因编辑结果 [4] 公司简介 - 作为领先的基因编辑公司，专注于将CRISPR/Cas12a和CRISPR/Cas9基因组编辑系统的潜力转化为针对严重疾病的体内药物管线，目标是发现、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，是博德研究所Cas12a专利以及博德研究所和哈佛大学Cas9专利在人类药物方面的独家被许可方 [6]

NEW YORK, April 28, 2025 (GLOBE NEWSWIRE) -- Blade Air Mobility, Inc. (Nasdaq: BLDE, “Blade” or the “Company”), will release financial results for the first quarter ended March 31, 2025 on Monday, May 12, 2025 before the market opens. The company will hold a conference call the same day at 8:00 am Eastern Time to discuss the results. The call will be hosted by Rob Wiesenthal, Blade’s Chief Executive Officer, and Will Heyburn, Blade’s Chief Financial Officer, and will include a question-and-answer session fo ...

NEW HOPE, Pa., April 28, 2025 (GLOBE NEWSWIRE) -- Orchestra BioMed Holdings, Inc. (Nasdaq: OBIO, “Orchestra BioMed” or the “Company”), a biomedical company accelerating high-impact technologies to patients through risk-reward sharing partnerships, today announced that company management will participate in multiple upcoming institutional investor conferences. Details on the Company’s participation appear below: Chardan Capital’s Trending Issues in Drug Development Conference – April 29, 2025 (Virtual) David ...

GIG HARBOR, Wash., April 28, 2025 (GLOBE NEWSWIRE) -- Heritage Distilling Holding Company, Inc. (“HDC” or “Heritage” or the “Company”) (Nasdaq: CASK), a leading craft distiller of innovative premium brands, today reported it regained compliance with the Nasdaq regarding the filing of periodic reports after filing its 2024 Annual Report of Form 10-K with the Securities and Exchange Commission on April 28, 2025. On April 22, 2025, Nasdaq notified the Company that it no longer met the periodic filing requireme ...

SINGAPORE, April 28, 2025 (GLOBE NEWSWIRE) -- SAIHEAT Limited (“SAIHEAT” or the “Company”) (NASDAQ: SAIH), today reported audited financial results for the fiscal year ended December 31, 2024. Financial Highlights for the Year Ended December 31, 2024 Total revenues were US$5.54 million, representing a decrease of 18% compared to US$6.78 million in 2023 primarily due to revenue decreases across business segments of sales of products, hosting service and mining pool, which was partially offset by 125% revenue ...

Hong Kong, April 28, 2025 (GLOBE NEWSWIRE) -- Phoenix Asia Holdings Limited (the “Company” or “PHOE”), a premier substructure contractor for public and private sector projects in Hong Kong, today announced the closing of its initial public offering (the “Offering”) of 1,600,000 ordinary shares (the “Ordinary Shares”) at a public offering price of $4.00 per share for total gross proceeds of $6,400,000, before deducting underwriting discounts and other offering expenses. The Offering closed on April 28, 2025, ...

SAN RAFAEL, Calif., April 28, 2025 (GLOBE NEWSWIRE) -- Ekso Bionics Holdings, Inc. (Nasdaq: EKSO) (the “Company”), an industry leader in exoskeleton technology for medical and industrial use, today announced that it will release financial and business results for the first quarter of 2025 after the close of trading on Monday, May 5, 2025. Management will host a conference call beginning at 1:30 p.m. PT / 4:30 p.m. ET to review the financial results and discuss recent business developments. To participate in ...

- Oral presentation on tau silencing gene therapy VY1706, which has previously shown up to 73% knockdown of tau mRNA in NHPs in the CNS following a single IV dose of 1.3e13 vg/kg - - Featured data also include anti-amyloid gene therapy for Alzheimer’s disease, as well as multiple presentations on Voyager’s continued enhancements to its highly BBB penetrant novel capsids - LEXINGTON, Mass., April 28, 2025 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to lev ...

SOUTH SAN FRANCISCO, Calif. and SAN DIEGO, April 28, 2025 (GLOBE NEWSWIRE) -- ORIC Pharmaceuticals, Inc. (Nasdaq: ORIC), a clinical stage oncology company focused on developing treatments that address mechanisms of therapeutic resistance, today announced the presentation of a poster at the 2025 American Association for Cancer Research (AACR) Annual Meeting, highlighting preclinical data on ORIC-944, a potent, highly selective, orally bioavailable allosteric inhibitor of PRC2, which demonstrated synergistic ...

NEW YORK, April 28, 2025 (GLOBE NEWSWIRE) -- Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today unveils research data on TALEN®-mediated non-viral transgene insertion for advancing cellular and gene therapies, and advancements in genetic editing using TALE base editors (TALEB), at the Society of Gene and Cell Therapy (ASGCT) annual meeting, that will be held ...