Orna Therapeutics, Inc.

▎药明康德内容团队编辑据创鉴汇不完全统计，上周（5月22日至5月28日）全球大健康领域共披露融资事件34起，覆盖RNA疗法、癌症疫苗、多肽、KRAS抑制剂等领域。其中，ReNAgade Therapeutics获超3亿美元融资，其递送平台有望克服RNA疗法的主要局限性；Carmot Therapeutic致力于针对糖尿病和肥胖等代谢疾病研发新药，获多家投资机构青睐；超亿元助力斯微生物（StemiRNA）研发基于mRNA技术的肿瘤治疗和传染病预防疫苗；Quanta Therapeutics利用高通量光学平台，研发针对RAS的新型小分子抗癌药物，有望解决肿瘤靶向疗法的耐药性挑战 ...... #01ReNAgade Therapeutics完成超3亿美元A轮融资，加速RNA疗法的开发  关键词：RNA最新融资：超3亿美元A轮本轮投资机构：MPM BioImpact、F2 Ventures ReNAgade Therapeutics是一家专注于开发RNA疗法的公司，旨在将RNA药物递送到以前无法接触的组织和细胞中，有望克服RNA疗法的主要限制。该公司建立了一个全面的互补平台，将其专有的递送技术与编码、编辑和基因插入工具结合在一个全RNA系统中，研发新药。ReNAgade已与Orna Therapeutics建立合资企业，将ReNAgade的递送平台与Orna的环状RNA技术结合。随后，Orna在2022年8月与默沙东达成合作，使用Orna与ReNAgade合作下开发的技术。 #02Carmot Therapeutics完成1.5亿美元E轮融资，推进治疗肥胖和糖尿病的多肽产品  关键词：多肽、小分子药物最新融资：1.5亿美元E轮本轮投资机构：Deep Track Capital、5AM Ventures、Franklin Templeton、Frazier Life Sciences、Janus Henderson Investors、Millennium Management、TCGX、The Column Group、Venrock Healthcare Capital Partners、Willett Advisors、RA Capital Management Carmot Therapeutics致力于针对肥胖和糖尿病等代谢性疾病研发新药，利用药物发现平台Chemotype Evolution识别新的肠促胰岛素受体信号转导靶点，研发减轻体重并改善血糖控制的药物。目前该公司有3个临床阶段的管线：CT-388是一款双重GLP-1/GIP受体调节剂，目前正在进行1/2a期临床试验，针对超重和肥胖成人，其中还包括2型糖尿病（T2D）患者；CT-996是一款口服小分子GLP-1受体激动剂；双重GLP-1/GIP受体调节剂CT-868已完成针对超重和肥胖健康的成人的1期临床试验，以及针对患有T2D的肥胖患者的2期试验，以评估其对葡萄糖稳态的影响。此外，他们还有一种长效肽酪氨酸-酪氨酸（PYY）类似物处于后期临床前开发阶段。 #03斯微生物完成数亿元Pre-D轮融资，推进疫苗管线开发并持续布局mRNA序列设计等技术  关键词：癌症疫苗、mRNA最新融资：数亿元Pre-D轮 斯微生物是一家mRNA新药研发企业，拥有自主知识产权的mRNA全产业链技术平台和制剂结构专利LPP技术。他们利用基于云计算技术的抗原分析、预测、序列优化平台以及完整的mRNA疫苗分析及质控技术平台，致力于研究开发基于mRNA技术的肿瘤治疗和传染病预防疫苗。其个性化肿瘤疫苗管线已经在澳洲开展1期临床试验，并已同步在中国多家医院开展IIT研究；编码细胞因子IL-12的非复制mRNA瘤内注射剂SW0715已在中国递交新药临床试验（IND）申请。据悉，本轮资金将加快肿瘤治疗疫苗及传染病疫苗管线的开发，并持续布局人工智能算法及mRNA序列设计优化等技术。 #04Quanta Therapeutics完成5070万美元D轮融资，推进其别构KRAS抑制剂管线  关键词：别构剂最新融资：5070万美元D轮本轮投资机构：Avidity Partners、艾伯维风投（AbbVie Ventures）、BVF Partners、GC&H Investments、Longitude Capital、Sofinnova、Surveyor Capital、Vida Ventures、Wilson Sonsini Goodrich & Rosati Quanta Therapeutics致力于研发靶向疗法治疗RAS驱动的癌症，RAS是癌症中突变最频繁的致癌基因。Quanta通过应用高通量平台，利用SHG光学技术来识别膜结合蛋白复合物的变构调节剂，研发针对RAS的新型小分子抗癌药物。据新闻稿报道，Quanta的别构剂可能扩大可治疗的KRAS突变型癌症范围，同时规避已知的耐药机制。本轮资金将用于研发两种化学性质不同的别构KRAS抑制剂QTX3034和QTX3046，临床前数据显示这两种药物口服生物利用度和脑渗透能力良好，表现出有效的抗肿瘤活性。预计于2024年启动针对KRAS驱动实体瘤适应症的临床试验。此外，Quanta Therapeutics还开展了一项RAF1发现项目，其口服小分子先导候选药物已显示出体内概念验证活性。 #05OnKure Therapeutics完成5400万美元C轮融资，推进肿瘤小分子靶向药物研发  关键词：小分子靶向药最新融资：5400万美元C轮本轮投资机构：Acorn Bioventures、BlackRock、Deep Track Capital、Perceptive Advisors、Samsara BioCapital、Surveyor Capital、鱼鹰资管 OnKure Therapeutics致力于发现和开发小分子靶向药，改善癌症患者的生活质量。该公司使用基于结构的药物设计平台，正在建立抗肿瘤候选药物管线，旨在实现最佳疗效和耐受性。目前，OnKure正在开发bocodepsin（OKI-179），这是一种口服选择性I类HDAC抑制剂，用于治疗血液肿瘤和实体瘤。据悉，本轮融资将支持新型精准药物差异化管线的持续开发，包括推进其先导管线OKI-219进入临床试验阶段，这是一种选择性PI3K α H1047R抑制剂。 读者们请星标⭐创鉴汇，第一时间收到推送免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：本文由药明康德内容团队根据公开资料整理编辑，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转发/复制至其他平台。转发授权请在「创鉴汇」微信公众号留言联系我们。更多数据内容推荐   点击“在看”，分享创鉴汇健康新动态

None  Welcome to this week's Chutes & Ladders, our roundup of hirings, firings and retirings throughout the industry. Please send the good word—or the bad—from your shop to Max Bayer or Gabrielle Masson, and we will feature it here at the end of each week.  Serial exec Amit Munshi returns to biotech arena with Moderna alumni ReNAgade Therapeutics Amit Munshi, to no one’s surprise, has landed back on his feet and walked into a new C-suite, just over a year after Arena Pharmaceuticals was bought by Pfizer. The serial biopharma executive is taking the helm of ReNAgade Therapeutics, an all-in-one RNA shop that launched this week with $300 million from a series A. It was the largest single fundraising round of the year for a few hours, before ElevateBio took the crown the following day. The former Arena CEO was sold on the company’s RNA delivery focus, working to expand which tissues the polymeric molecules can enter. The company plans to incorporate gene editing tech directly into the platform to widen the diseases RNA can treat.  But arguably more valuable to the company’s early hype are the other C-suite members, including CSO Pete Smith, Ph.D., who was previously the SVP of nonclinical early development work at Moderna before moving to Alnylam. He’ll be joined by Chief Operating Officer Ciaran Lawlor, Ph.D., a principal scientist at Moderna who then worked for Boston Consulting Group.  The company has already launched a joint venture with fellow circular RNA-focused biotech Orna Therapeutics and inked a deal with Merck for up to five targets. ReNAgade’s leadership did not disclose financial details but said they already unlocked a milestone payment for identifying a target in the vaccine space. Fierce Biotech Larkspur launches with all-female leadership team Larkspur Biosciences One of the more persistent diversity trends in biotech is the lack of female executives. Data from a 2022 BIO report found that women make up 34% of biotech executives but 49% of all employees, and 37% of responding companies reported “little change” in the number of female executives in 2021 compared to 2020.  Larkspur Biosciences is helping tip the scales toward equity. The company unveiled this week with an all-female leadership team led by CEO Catherine Sabatos-Peyton, Ph.D., a former executive director at the Novartis Institute for BioMedical Research. She’s joined by Chief Scientific Officer Krista Goodman, Ph.D., and Emily Corse, Ph.D., EVP of biology and translation. Goodman jumps from British Big Pharma GSK where she was global head and senior director of flexible medicinal chemistry and medicine design. Corse was the executive director of cancer immunology at Boehringer Ingelheim.  It’s a deeply experienced trio that comes equipped with experience that mirrors the biotech’s ambitions. The company is working on a slate of precision immunotherapies to target “bottlenecks” wielded by tumors against the immune system. The company is first aiming its technology at a subtype of patients with colorectal cancer. Release  CytoDyn president takes medical leave  CytoDyn CytoDyn president Cyrus Arman, Ph.D., is taking a medical leave of absence from the company, with Chief Financial Officer Antonio Migliarese replacing him in the meantime. It’s unclear how long Arman may be away from the company, and CytoDyn did not specify what’s keeping him out. Migliarese will have at his side new interim Chief Medical Officer Melissa Palmer and clinical and strategic adviser Salah Kivlighn, Ph.D., both of whom recently joined the company. It’s a difficult time for CytoDyn as it struggles to find any kind of clinical momentum, and Arman’s absence comes just eight months after he took the helm. His first order of business was redirecting the company’s lead asset, leronlimab, away from COVID-19. Then, a month later, the company pulled an FDA approval application for leronlimab as an HIV treatment, citing “systemic issues” with the data from a CRO.  The biotech filed a claim for damages against the CRO and said at the time that resolving the issues with the data would require “significant additional investment.” CytoDyn is now aiming leronlimab at a new, notoriously difficult-to-treat disease—nonalcoholic steatohepatits. Oh, and it’s still exploring potential partnerships. Source  > Jeremy Chadwick, Ph.D., is leaving Takeda to become chief operating officer at Kymera. He held a variety of roles at the Japanese pharma, including SVP and head of the global development office. Release > Talaris Therapeutics has appointed its only C-suite member left, Chief Financial Officer Mary Kay Fenton, as interim CEO and president. Fenton will also continue to serve as CFO and succeeds former CEO Scott Requadt, who left as part of a 95% workforce reduction. Release > BioAge labs has promoted BJ Sullivan to the inaugural chief strategy officer position. He previously served as the company’s chief of staff and VP of strategy and business operations. Release > Obsidian Therapeutics is crystalizing its top brass, naming Madan Jagasia, M.D., as the new CEO and Parameswaran Hari, M.D., as chief development officer. Both new hires come from Iovance Biotherapeutics, with Jagasia serving as the VP of medical affairs while Hari was the VP of clinical science. Release > Ming Yang, Ph.D., has been tapped to lead life biosciences development efforts as SVP of R&D. He previously held the same role at Graybug Vision after working at Genentech. Release > Former medtech executive and consultant Ken Mariash is joining Sinaptica Therapeutics as CEO. He was recently founder and managing partner of medtech consulting firm Rubicon Strategy Partners, a company he launched after running marketing efforts at EBT Medical. Release > Arcutis’ chief commercial officer Ken Lock is stepping down to “attend to personal matters,” the company announced this week. He’ll be temporarily replaced by Ayisha Jeter, VP of market access. Release > TScan Therapeutics acting CEO Gavin MacBeath, Ph.D., will be handed the keys to the car after being permanently named chief executive. He took over temporarily in May and will no longer serve as chief scientific officer. Release > In addition to announcing the start of a new phase 2 trial testing a treatment for celiac disease, Topas Therapeutics has hired two new executives to the C-suite. The German biotech has brought on Mireia Gómez-Angelats, Ph.D., has chief business officer and Christian Schröter, Ph.D., as chief operating officer. Release > Cancer detection company Harbinger Health has hired May Orfali, M.D., to be chief medical officer. She joins a leadership team led by former FDA Commissioner Stephen Hahn, M.D., who’s CEO of the company. Release > Rani Therapeutics has appointed Kate McKinley as chief business officer. She was previously founder and CEO of Spark Outcomes. Release

今年以来，多家专注于 RNA 治疗的公司获得大量资金支持，除 ReNAgade  外，Orbital Therapeutics 在今年 4 月的 A 轮融资中筹集了 2.7 亿美元，以帮助推进其 RN 当地时间 5 月 23 日，RNA 治疗领域公司 ReNAgade Therapeutics（以下简称 ReNAgade） 宣布获得由 MPM BioImpact 和 F2 Ventures 领投的 3 亿美元 A 轮融资，从而在 RNA 技术领域崭露头角。公司方面还表示，计划在未来 12 个月内推出其产品线。 ReNAgade 由 MPM BioImpact 创立于 2020 年，总部位于美国剑桥，目前拥有大约 100 名员工。公司的目标是，凭借其递送系统将 RNA 药物递送到体内以前无法进入的组织和细胞，来解决 RNA 疗法的主要局限性——RNA 药物目前受到组织靶向递送和缺乏综合治疗平台的限制。 ReNAgade 结合新型 RNA 递送平台与综合 RNA 平台，建立了一个全面的互补平台，将其专有的递送技术（包括新型脂质纳米颗粒（LNP））与广泛的编码、编辑和基因插入工具结合在一个全 RNA 系统中。可进行编码、编辑和基因插入，以生产新药物。 （来源：ReNAgade官网） 据了解，ReNAgade 有两位高管来自 Moderna。 首席科学官 Peter Smith 博士在 2017 年加入生物制药公司 Alnylam Pharmaceuticals 之前，曾在 Moderna 担任首席早期开发官和高级副总裁兼非临床研发主管；首席运营官 Ciaran Lawlor 博士在波士顿咨询公司工作之前是 Moderna 的首席科学家。 （来源：ReNAgade官网） 董事长兼首席执行官 Amit Munshi 拥有 30 多年领先生物技术公司经验，此前曾担任 Arena Pharmaceuticals 的首席执行官，后来 Arena 被辉瑞公司以 67 亿美元的价格收购。 Amit D. Munshi 透露，“为了开发具有治疗身体任何部位、任何疾病潜力的 RNA 药物，ReNAgade 组建了一支世界级的多学科团队，该团队共取得过 25 项 NDA（包括 4 项 RNA 药物）和 200 项 IND，并且一直是 RNA 药物领域的先驱。” （来源：ReNAgade 官网） 在公司的拓展方面，ReNAgade 已经与 Orna Therapeutics 成立了一家合资企业，将 ReNAgade 的 RNA 递送技术与 Orna 的环状 RNA 技术相结合。 根据资料，Orna 在 2022 年 8 月完成了 2.21 亿美元的 B 轮融资，并与美国默克公司（NYSE: MRK）签署了一项协议，在多个治疗项目中进行合作，其中包括在 Orna/ReNAgade JV 下开发的技术。与默沙东的交易可能会让 Orna 获得总计 35 亿美元的里程碑付款。 今年以来，多家专注于 RNA 治疗的公司获得大量资金支持，除 ReNAgade  外，Orbital Therapeutics 在今年 4 月的 A 轮融资中筹集了 2.7 亿美元，以帮助推进其 RNA 疗法产品组合。此外，COVID-19 以外的潜在 RNA 疫苗也受到诸多关注，例如 BioNTech 的胰腺癌 mRNA 疫苗，其已获得积极的早期数据。