Ultragenyx (RARE) shares ended the last trading session 5.1% higher at $41.44. The jump came on an impressive volume with a higher-than-average number of shares changing hands in the session. This compares to the stock's 5.1% gain over the past four weeks.The sudden rise in the stock price can be attributed to the positive investor mindset regarding Ultragenyx’s key late-stage pipeline progress. A regulatory application, seeking the FDA approval of UX111, an AAV gene therapy candidate being developed for th ...

- SGT-501 is a novel gene therapy for rare, life-threatening, genetic arrhythmogenic disease with no approved therapies - - SGT-501 has received Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA - - Solid expects to initiate Phase 1b clinical trial of SGT-501 in the fourth quarter of 2025 - - Expands Solid’s clinical pipeline to include first cardiac indication with urgent unmet medical need - CHARLESTOWN, Mass., July 08, 2025 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SL ...

访谈 ｜ 杨轩 海若镜 文 ｜ 杨轩 Byran Johnson，可能是全球最快成名的抗衰达人。他身上有很多标签：硅谷抗衰富豪、永生科技狂 人、"姨化吸血鬼"、"试药小白鼠"、"天山童姥"等等。 在众多渴望长寿的科技新贵中，Bryan不一定是氪金投资最多的，但他应该是对自己最狠的。为了抗衰 逆龄，他遵从着"苦行僧"般的作息：每晚8点半睡觉、早4点半起床；每天摄入卡路里低于2000大卡，过 午不食；每周运动7天。除了每天吃下几十粒保健品，他还尝试了一系列监管尚未批准的前沿科技：换 血、注射干细胞、基因疗法等。 从2021年启动计划至今，47岁的他每年氪金200万美元，宣称已将生理年龄追回了5.1岁，自己的衰老速 度已经降低至0.48，相当于一年只老去6个月。他还公布了自己多项生物标志物数据，比如睡前静息心 率44次/分，夜间勃起时长超过3小时，卧推110公斤...... 如今，"Don't die不死"和"逆转年龄"，已经成为他的第一目标和使命。 Bryan Johnson：我21岁的时候从厄瓜多尔回来，在那边和极度贫困的人们一起生活了两年。回到美国 后，我感到很幸运。因为我们有充足的食物、有房子、有医疗、有厄 ...

Core Viewpoint - Rocket Pharmaceuticals, Inc. is facing a class action lawsuit due to allegations of providing misleading information regarding its clinical trials, particularly concerning the safety of its gene therapy product RP-A501 for Danon disease [3][4]. Group 1: Lawsuit Details - The class action lawsuit is titled Ho v. Rocket Pharmaceuticals, Inc. and covers purchasers of Rocket Pharmaceuticals securities from February 27, 2025, to May 26, 2025 [1]. - The lawsuit alleges that Rocket Pharmaceuticals and a top executive violated the Securities Exchange Act of 1934 by concealing material adverse facts about the safety and clinical trial protocol of RP-A501 [3]. - It is claimed that the company failed to disclose serious adverse events, including the death of a participant, which occurred after a protocol amendment that introduced a new immunomodulatory agent [3][4]. Group 2: Impact on Stock - Following the announcement of a clinical hold by the U.S. Food and Drug Administration on May 27, 2025, due to a serious adverse event, Rocket Pharmaceuticals' stock price experienced a decline [4]. Group 3: Legal Process - Investors who purchased Rocket Pharmaceuticals securities during the class period can seek appointment as lead plaintiff in the lawsuit, which allows them to act on behalf of other class members [5]. - The lead plaintiff can choose a law firm to represent the class, and participation as lead plaintiff does not affect the ability to share in any potential recovery [5]. Group 4: About Robbins Geller - Robbins Geller Rudman & Dowd LLP is a leading law firm specializing in securities fraud and shareholder litigation, having secured over $2.5 billion for investors in 2024 alone [6]. - The firm has been ranked 1 in the ISS Securities Class Action Services rankings for four out of the last five years, indicating its strong track record in obtaining monetary relief for investors [6].

证券代码：301080 证券简称：百普赛斯 北京百普赛斯生物科技股份有限公司 投资者关系活动记录表 编号：2025-003 值得特别关注的是，公司在拥有 GMP 级别质量管理体系平台基础上， 结合细胞治疗药物生产规范，以严格的质量管理和药品级放行检测标准，已 成功开发超 50 款高质量的 GMP 级别产品，包括细胞因子、细胞激活用抗体 和磁珠、全能核酸酶以及 Cas 酶等，适用于 CGT 药物 CMC、商业化生产和 临床研究。公司还可提供用于临床前研究的 Premium (Pre-GMP) 级别原料， 助力客户实现从临床前开发到临床阶段的无缝过渡。另外，公司针对细胞治 疗应用进行针对性的蛋白结构设计，能够提供从基因合成、载体构建到蛋白 质表达、纯化的 GMP 级别蛋白的一站式开发服务。 问：公司在 ADC 领域的布局？ 抗体偶联药物（ADC）作为新一代抗癌免疫治疗药物的代表，其独特之 处在于将高靶向性的抗体与强大的化疗药物相结合。这种结合方式不仅能在 肿瘤细胞内部实现精准投药，有效避免化疗药物对正常细胞的伤害，还显著 减少治疗过程中的不良反应。鉴于 ADC 在肿瘤等疾病治疗中展现出的巨大 潜力，现已成为抗体药 ...

Core Viewpoint - The FDA is investigating the risk of acute liver failure associated with Sarepta Therapeutics' Elevidys treatment, which has resulted in serious outcomes including hospitalization and death [1][2]. Group 1: FDA Investigation - The FDA has received two reports of fatal acute liver failure in non-ambulatory pediatric male patients with Duchenne Muscular Dystrophy (DMD) following treatment with Elevidys [2]. - The agency is evaluating the need for further regulatory action based on these reports [1]. Group 2: Patient Outcomes - In the reported cases, patients exhibited elevated transaminases and were hospitalized within two months after receiving Elevidys [3]. - Both fatalities appear to be directly related to the treatment with Elevidys [2]. Group 3: Analyst Reactions - HC Wainwright has reiterated a Sell rating for Sarepta, maintaining a price forecast of $10, citing the likelihood of the FDA requiring updated warnings regarding liver failure or death [4]. - The worst-case scenario for Sarepta would involve the FDA mandating the withdrawal of Elevidys from the U.S. market [4][5]. Group 4: Company Valuation - If Elevidys were to be withdrawn, analysts believe there would be little intrinsic value remaining in Sarepta [5]. - The skepticism from FDA officials regarding the initial approval of Elevidys suggests potential challenges for the company's continued marketing efforts [6]. Group 5: Stock Performance - Following the news, Sarepta's stock (SRPT) has seen a decline of approximately 9.96%, trading at $17.09 [6].

Intra-patient, double-blind, multicenter, placebo-controlled study with crossover design Approximately 16 subjects will be enrolled in the IOLITE study. The sample size was calculated based on the average symptomatic days per month and standard deviation data from subjects enrolled in the natural history study who would be otherwise eligible for IOLITE and provides 90% power to detect an effect size of at least 25%, allowing for a dropout rate up to 20%. Enrolled patients will initially receive either a sin ...

专家建议，预防糖尿病足，糖尿病患者每日需用38℃以下温水泡脚，擦干后检查趾间有无破损，穿 透气棉 袜与宽松鞋，并每年至少做一次足部血管神经专项检查。 国际糖尿病联盟最新数据显示，我国20—79岁糖尿病患者人数到2030年预计将突破1.64亿人。随着 糖尿病患病率的持续攀升，糖尿病足这一严重并发症的患病人数也呈逐年递增趋势。临床统计表明，我 国50岁以上糖尿病患者中，糖尿病足1年新发率达8.1%，总截肢率高达19.3%。 令人振奋的是，广州医科大学与中国科学院联合团队近期通过基因工程改造干细胞技术，实现实验 室环境下14天内糖尿病足伤口愈合率超96%，为众多患者带来希望。与此同时，抗生素骨水泥、脊髓电 刺激器植入术（SCS）、血管重建与修复等治疗技术也取得突破性进展，为糖尿病足的临床干预开辟了 新路径。 血糖失控渐进性破坏足部组织 "糖尿病是一种表现为血糖异常升高的代谢性疾病。糖尿病对双足的影响，本质是高血糖引发的'血 管—神经—免疫'三重打击。"天津医科大学第二医院手足显微外科副主任医师苗平指出，糖尿病足的发 生、发展背后是血糖失控对足部组织的渐进性破坏。 多项突破为解决临床难题带来希望 糖尿病足作为糖尿病最 ...

政策动向 国家药监局征求意见：纳入创新药临床试验审评审批30日通道的申请应当为中药、化学药品、生物制品 1类创新药 6月16日，国家药监局综合司公开征求《关于优化创新药临床试验审评审批有关事项的公告（征求意见 稿）》意见，其中提出，纳入创新药临床试验审评审批30日通道的申请，应当为中药、化学药品、生物 制品1类创新药，能够按要求提交申报资料，并需满足以下条件之一：（一）国家支持的重点创新药。 获国家全链条支持创新药发展政策体系支持的具有明显临床价值的重点创新药品种。（二）入选国家药 品监督管理局药品审评中心儿童药星光计划、罕见病关爱计划的品种。（三）全球同步研发品种。全球 同步研发的Ⅰ期、Ⅱ期临床试验和III期国际多中心临床试验，以及中国主要研究者牵头开展的国际多中 心临床试验。 药械审批 "多动症"新药上市申请获受理并纳入优先审评 6月16日，上海爱科百发生物医药技术股份有限公司（下称" 爱科百发"）宣布其新药爱智达（通用名： 丝右哌甲酯/右哌甲酯复方胶囊；代号：AK0901）的新药上市申请（NDA）已经获得中国国家药品监督 管理局（NMPA）药品审评中心（CDE）正式受理，并将其已纳入"优先审评审批"程序 ...

MALVERN, Pa., June 16, 2025 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) amendment to initiate a Phase 2/3 pivotal confirmatory trial of OCU410ST, a modifier gene therapy candidate being developed for all Stargardt disease (ABCA4-associated retinopathies). The FDA previously granted Rare Pediatr ...