Key Takeaways Intellia stock has gained 29.8% in a month, outperforming the industry, sector and the market. Intellia advances late-stage gene therapy trials but faces safety concerns and future competition. Intellia aims to become a commercial-ready organization by the end of 2026.Intellia Therapeutics (NTLA) has put up a stellar performance in the past month. Shares of the company have rallied 29.8% compared with the industry’s rise of 0.4%. The stock has also outperformed the sector and the S&P 500 ind ...

GLP-1 agonists, a type of drug used to treat diabetes and weight loss, could be the hottest growth story in healthcare today. Experts at Morgan Stanley estimate that the market could grow to a $150 billion opportunity over the next decade, representing a tenfold increase from its sales last year. Eli Lilly (LLY -0.40%) has captured approximately 35% of the GLP-1 market, alongside arch-rival Novo Nordisk, the current market leader at 65%. However, Eli Lilly could gain on, perhaps even surpass, its rival over ...

Key Takeaways VRTX fell 8.9% over three months due to soft Q1 results and slower uptake of new and CF drugs. Alyftrek, Journavx, and Casgevy sales are expected to pick up in the second half of 2025. VRTX's CF franchise remains strong, with 2025 sales projected to grow 8% amid pipeline expansion.Vertex Pharmaceuticals Incorporated (VRTX) stock has declined 8.9% in the past three months.The deterioration was due to soft first-quarter results, slower-than-expected uptake of its newer drugs as well as CF drug ...

-New Phase 1 clinical data for CTX310™ continues to demonstrate dose-dependent reductions in triglycerides (TG) and low-density lipoprotein (LDL), with peak reduction of up to 82% in TG and up to 86% in LDL, with a well-tolerated safety profile- -Complete Phase 1 data presentation for CTX310 anticipated at a medical meeting in the second half of 2025- -Data update for CTX320™, targeting the LPA gene, now expected in the first half of 2026- -Preclinical in vivo cardiovascular program CTX340™ advancing toward ...

Core Insights - Biotech stocks present significant growth opportunities due to ongoing research and potential breakthroughs in treatments [1] - Wall Street identifies two biotech candidates with projected gains exceeding 80% and 200% in the next 12 months [2] Company Summaries Viking Therapeutics - Viking Therapeutics (VKTX) experienced a surge in stock price following positive phase 2 trial results for its weight loss drug VK2735, but has since returned to pre-announcement levels [4] - The company is advancing VK2735 in both injectable and pill forms, with high demand for weight loss drugs, indicating potential for future growth [5] - Viking is set to begin phase 3 trials for the injectable version in Q2 and expects phase 2 data for the pill version in the second half of the year, with the weight loss drug market projected to reach $100 billion [6] - Wall Street forecasts a 240% increase in Viking's stock price, contingent on trial outcomes [7] CRISPR Therapeutics - CRISPR Therapeutics (CRSP) saw its stock rise leading up to the approval of its first product, Casgevy, but has since declined as investors locked in gains [8] - The company is positioned for potential growth as Casgevy requires a longer rollout process, with expectations for significant increases in new patient initiations this year [10] - Positive phase 1 data for a gene-editing candidate targeting high cholesterol has been reported, with further updates expected on candidates for lipoprotein(a), oncology, and autoimmune diseases [11][12] - Wall Street anticipates an 84% gain for CRISPR Therapeutics, making it a potential buying opportunity for growth investors [12]

撰文丨王聪 编辑丨王多鱼 排版丨水成文 Cas13a 是由 张锋 教授发现的一种 VI 型 CRISPR-Cas 效应蛋白，被认为是一种 RNA 特异性核酸酶，具有 RNA 介导的 RNA 切割活性，。此外，它还具有 依 赖于靶标单链 RNA 的反式切割活性，因此， 张锋 将 Cas13a 用于病毒检测，实现了对 RNA 病毒的 高灵敏度、高特异性、快速检测。 这项研究揭示了 LbuCas13a 对 DNA 的单核苷酸特异性相较于 RNA 有所增强 。这种特异性的增强归因于 CRISPR RNA （crRNA） 对 DNA 的亲和力较低，从 而提高了 crRNA 与 DNA 结合的能量障碍。 在上述发现的基础上，研究团队推出了 基于 LbuCas13a 的卓越通用快速增强特异性检测技术 （简称为 SUREST ） ，作为一种新型分子诊断技术平台， SUREST 能够检测低至 0.3 阿摩尔的 CYP2C19 （rs4986893） DNA 浓度。该基因编码的酶参与多种药物代谢，其活性受基因多态性影响，因此常被用于评估 个体对特定药物的代谢能力差异，以指导精准用药、帮助制定个性化治疗方案。 研究团队还将 SUR ...

Key Takeaways Casgevy, CRSP's first marketed therapy, treats SCD and TDT but saw just $10 million in 2024 sales. The therapy's complex, multi-step process has slowed adoption despite its curative potential. Casgevy faces pressure from cheaper approved drugs and emerging gene-editing competitors.CRISPR Therapeutics’ (CRSP) first marketed product is the one-shot gene therapy Casgevy, approved in late 2023 and early 2024 across the United States and Europe for two blood disorder indications — sickle cell dis ...

撰文丨王聪 编辑丨王多鱼 排版丨水成文 我们每个人的体内都会产生一种名为 白细胞介素-15 （IL-15） 的细胞因子，它能够有效保护我们免受癌症侵害，其作用在于促进能迅速发现并杀死首次出现的 癌细胞的免疫细胞的生成，例如 自然杀伤细胞 （NK 细胞） 。 但问题在于，癌细胞进化出了许多策略来抑制像 NK 细胞这样的免疫细胞，导致免疫逃逸。因此，研究人员尝试使用能够激活免疫细胞上 IL-15 受体的 IL-15 受 体激动剂，从而诱导抗肿瘤免疫反应，但这种方法被证明毒性太大，限制了进一步临床开发。 2025 年 6 月 12 日， oNKo-innate 公司和 澳大利亚莫纳什大学的研究人员合作，在 Cancer Cell 期刊发表了题为： Enhancing anti-tumor immunity of natural killer cells through targeting IL-15R signaling 的研究论文。 该研究表明，在 NK 细胞中关闭一个基因—— UBE2F ，能够显著提高 NK 细胞对低剂量 IL-15 的敏感性，从而增强其抗癌功能，并在临床前模型中减缓 结直肠 癌 的生长。 在 ...

Core Insights - Intellia Therapeutics announced promising three-year follow-up data from the Phase 1 portion of its ongoing Phase 1/2 study for lonvoguran ziclumeran (lonvo-z) in patients with hereditary angioedema (HAE) [1][2] - All 10 patients in the Phase 1 study were attack-free and treatment-free for a median of nearly two years, demonstrating the potential of lonvo-z as a one-time therapy [2][6] - The global Phase 3 HAELO trial has completed screening ahead of schedule, with over half of the patients screened in the United States [4][6] Clinical Results - A single dose of lonvo-z led to a mean reduction in monthly HAE attack rate of 98% compared to pre-treatment baseline [2][6] - Patients showed deep, dose-dependent, and durable reductions in plasma kallikrein protein levels throughout the study [2][6] - The treatment was well tolerated, with the most frequent adverse events being infusion-related reactions, which were mostly Grade 1 and resolved without complications [3][6] Development Plans - The ongoing Phase 3 HAELO trial is randomized, double-blind, and placebo-controlled, assessing the safety and efficacy of lonvo-z at the 50 mg dosage [4][5] - Intellia plans to submit a biologics license application (BLA) in 2026, aiming for a U.S. launch in 2027 [4][8] - New data from the Phase 2 portion of the ongoing Phase 1/2 study is expected to be presented in the second half of 2025 [4][10] About Lonvo-z - Lonvo-z is based on CRISPR/Cas9 technology and aims to prevent HAE attacks by inactivating the kallikrein B1 (KLKB1) gene [8] - The therapy has received multiple regulatory designations, including Orphan Drug and RMAT Designation from the FDA [8][9] - Intellia is focused on leveraging gene editing technology to develop novel therapies that address unmet medical needs [9]

2009 年，荷兰 皇家艺术与科学院 Hubrecht 研究所的 Hans Clevers 等人使用来自小鼠肠道的成体干细胞 培育出首个 肠道类器官 ， 开创了类器官研究时代 。此后，类器官领域研究成果不断，许多新型类器官和 更复杂的类器官不断涌现，为新药研发、精准治疗、再生医学等领域带来了更强大的工具 。 2025 年 6 月 12 日 ， Hans Clevers 教授团队 （ 林琳 为第一作者兼共同通讯作者） 在 Cell 子刊 Cell Stem Cell 上发表了题为 ： Human airway submucosal gland organoids to study respiratory inflammation and infection 的研究论文 【1】 。 该研究开发了人类 气道黏膜下腺体 （SMG） 类器官，用于研究呼吸系统炎症和感染。 撰文丨王聪 编辑丨王多鱼 排版丨水成文 鉴于 SMG 在黏液生成和宿主防御中的关键作用，研究团队从 SAE 和 SMG 区域的初级支气管组织中建立 了人类类器官，以探究它们独特的生理特性。 林 琳 ，2011 年本科毕业于中山大学，2016 年博士毕业 ...