A phase II study of efficacy, toxicity, and the potential impact of genomic alterations on response to eribulin mesylate in combination with trastuzumab and pertuzumab in women with human epidermal growth factor receptor 2 (HER2)+ metastatic breast cancer

2区 · 医学

Article

作者: Li, Tianyu ; Wagle, Nikhil ; Buendia-Buendia, Jorge E ; Tayob, Nabihah ; Isakoff, Steven J ; Jain, Esha ; Vaz-Luis, Ines ; Lin, Nancy U ; Shannon, Erin ; Tung, Nadine M ; Balch, Sara M ; Helvie, Karla ; Freedman, Rachel A ; Krop, Ian E

PURPOSEThere are limited data on trastuzumab-pertuzumab (HP)-based treatments beyond the first-line, HER2+ metastatic breast cancer (MBC) setting. We conducted a phase II study of eribulin mesylate, which extends survival in MBC, with HP in patients with previously treated HER2+ MBC to evaluate efficacy, toxicity, and genomic alterations driving therapeutic response.METHODSAfter a run-in phase for eribulin dosing, two cohorts were enrolled (Cohort A-no prior pertuzumab; Cohort B-prior pertuzumab). All patients received eribulin 1.4 mg/m² on days 1, 8 with standard-dose HP on day 1 (21-day cycles). The primary endpoint was objective response rate (ORR). Genomic characterization via whole exome sequencing (WES) was completed on tumor DNA and matched germline DNA from 19 patients.RESULTSThe six-patient run-in established a dose of eribulin 1.4 mg/m² with HP. Cohorts A and B enrolled 17 and 7 patients, respectively. Accrual stopped early due to an evolving treatment landscape and slow enrollment. The ORR was 26.3% (95% Confidence Interval [CI] 9.2-51.2%) in Cohort A and 0% in Cohort B (95% CI 0-41.0%). WES revealed more frequent alterations in TP53 (p < 0.05, q > 0.05) in patients without clinical benefit (disease control for < 24 weeks) which was not significant after multiple hypothesis correction.CONCLUSIONEribulin-HP had manageable toxicity and modest clinical activity in patients without prior pertuzumab exposure. This study provides a preliminary landscape of somatic alterations in this patient cohort. Our data add to the literature on how genomic alterations may predict for therapy response/resistance, as we work to individualize choices in a quickly evolving HER2+ MBC treatment landscape.TRIAL REGISTRATIONwww.clinicaltrials.gov , NCT01912963. Registered 24 July 2013.

2021-02-18·Nature1区 · 综合性期刊

Author Correction: A structural variation reference for medical and population genetics

1区 · 综合性期刊

作者: Francioli, Laurent C ; Rotter, Jerome I ; Talkowski, Michael E ; Lander, Eric ; Margolin, Lauren ; Wang, Harold ; Karczewski, Konrad J ; Sharpe, Ted ; Valkanas, Elise ; Gauthier, Laura D ; Post, Wendy S ; Zhao, Xuefang ; Nusbaum, Chad ; Whelan, Christopher W ; Laricchia, Kristen M ; Walker, Mark ; O'Donnell-Luria, Anne ; Khera, Amit V ; Banks, Eric ; Collins, Ryan L ; Gabriel, Stacey ; Philippakis, Anthony ; Daly, Mark J ; Lin, Henry J ; Alföldi, Jessica ; Solomonson, Matthew ; Baumann, Alexander ; Huang, Yongqing ; Chen, Yii-Der Ida ; Ruano-Rubio, Valentin ; Cusick, Caroline ; Lowther, Chelsea ; Brand, Harrison ; Munshi, Ruchi ; Stone, Matthew R ; Stevens, Christine ; Neale, Benjamin M ; Brookings, Ted ; Gupta, Namrata ; MacArthur, Daniel G ; Fu, Jack ; Taylor, Kent D ; Rich, Stephen S ; Tiao, Grace ; Kathiresan, Sekar ; Watts, Nicholas A

A Correction to this paper has been published: https://doi-org.libproxy1.nus.edu.sg/10.1038/s41586-020-03176-6.

2020-02-01·Nature Structural & Molecular Biology1区 · 生物学

BRCA1 and S phase DNA repair pathways restrict LINE-1 retrotransposition in human cells

1区 · 生物学

Article

作者: Bader, Joel S ; Mita, Paolo ; Keegan, Sarah ; Boeke, Jef D ; Sun, Xiaoji ; Wudzinska, Aleksandra ; Li, Donghui ; Kahler, David J ; Agmon, Neta ; Yun, Chi ; Fenyö, David

Long interspersed element-1 (LINE-1, or L1) is the only autonomous retrotransposon that is active in human cells. Different host factors have been shown to influence L1 mobility; however, systematic analyses of these factors are limited. Here, we developed a high-throughput microscopy-based retrotransposition assay that identified the double-stranded break (DSB) repair and Fanconi anemia (FA) factors active in the S/G2 phase as potent inhibitors and regulators of L1 activity. In particular, BRCA1, an E3 ubiquitin ligase with a key role in several DNA repair pathways, directly affects L1 retrotransposition frequency and structure and plays a distinct role in controlling L1 ORF2 protein translation through L1 mRNA binding. These results suggest the existence of a 'battleground' at the DNA replication fork between homologous recombination (HR) factors and L1 retrotransposons and reveal a potential role for L1 in the genotypic evolution of tumors characterized by BRCA1 and HR repair deficiencies.

项与 Cellarity Inc. 相关的新闻（医药）

2024-10-01

·FierceBiotech

Florey Biosciences spins out of Harvard's Wyss Institute to cook up probiotic medical foods

To avoid the pitfalls of microbiome drug development, Florey is aiming to get FLR-101 into the world as a medical food instead. Antibiotics are one of the most common medicines in the world, with about 236 million prescriptions in the U.S. in 2022 alone, according to the Centers for Disease Control and Prevention. These drugs are great for clearing out bacterial infections but also have an unintended side effect: They wreak havoc on the healthy microbes that call our guts home.“There's a lot of research and clinical data that shows that the disruption of the beneficial microbes in your gut actually is a high risk factor for many diseases,” Andrés Cubillos-Ruiz, Ph.D., a microbiologist and synthetic biologist at the Wyss Institute for Biologically Inspired Engineering at Harvard University, told Fierce Biotech in an interview. “This is a huge problem that is still a highly unmet need.”Florey Biosciences, a company spun out of Cubillos-Ruiz’s work in the MIT and Wyss Institute lab of James Collins, Ph.D., seeks to fill this unmet need with genetically modified microbes and a unique regulatory approach. The company launched Oct. 1 with pre-seed funding from Seerave Ventures, the investment arm of the microbiome-focused Seerave Foundation, according to a release. The team hopes to close out a seed funding round by mid-2025, Cubillos-Ruiz said. In addition to Cubillos-Ruiz, who is CEO, and Collins, Raphaël Gayet, Ph.D., is also a co-founder. Collins is a synthetic biologist known for his entrepreneurial spirit; he’s a co-founder of the Flagship Pioneering-launched Cellarity as well as the publicly traded Synlogic Therapeutics and Senti Biosciences, among others.Florey’s lead program is a genetically modified yeast called FLR-101. This yeast produces an enzyme that breaks down beta-lactam antibiotics, which make up about 50% of all antibiotics used, Cubillos-Ruiz said. Because the yeast is localized to the gut, its anti-antibiotic activity shields the microbiome there while the antibiotic is still able to clear an infection elsewhere in the body. The team first tested this approach using modified bacteria in a 2022 paper, and has now improved upon the method.“We have technologies that prevent these genes from jumping into bacteria,” Cubillos-Ruiz said, so that the yeast’s antibiotic-destroying power can’t be picked up by other microbes.When it came to forming a strategy for Florey, Cubillos-Ruiz learned from predecessors like Seres Therapeutics and Rebiotix (acquired by Ferring Pharma) about the perils of developing drugs for the microbiome. “You were going to incur huge development costs, and then you're going to create a product that doesn't have the market access and the market opportunity,” he said. To avoid those pitfalls, Florey is aiming to get FLR-101 into the world as a medical food instead of as a drug.Medical foods are products that are meant to be taken orally for the management of a disease or condition under the supervision of a physician but that don’t require a prescription. While drugs require studies of safety and efficacy, reporting of side effects and a thorough label review before hitting the market, medical foods do not.There is no formal stamp of approval that the FDA gives to medical foods, but the agency does provide guidelines for the products. Once FLR-101 is recognized as safe to consume, it could be put on the market right away along with the countless other probiotics that claim to support gut health.However, in order to market the modified yeast as being able to manage the microbiome dysfunction that comes from antibiotic use, Florey plans to run human trials to test whether FLR-101 paired with antibiotics leads to less microbiome damage than antibiotics alone. If successful, Florey would be able to add that information to FLR-101’s label and distinguish it from the other probiotics alongside it on the shelf. To bolster FLR-101’s chances of successfully navigating the medical foods regulatory pathway, Florey is adding Peter Hutt, former chief counsel of the FDA, to its board of directors, the company said in the release. At the FDA, Hutt helped craft the medical food regulatory framework, and he also played a pivotal role in the creation of nutrition labels, according to the release.“We have all the pieces of the puzzle to have a biotech product that, in its core, its technology is highly advanced, but we can put it in the market using different ways,” Cubillos-Ruiz said.Putting out products as consumer goods rather than prescription drugs presents a marketing challenge: How do you make sure patients, physicians and pharmacists know about your product? Cubillos-Ruiz sees an opportunity in FLR-101’s partner, antibiotics.“There is a big push from government agencies and within medical societies to be better antibiotic stewards,” he said. Hospitals don’t want readmissions of patients who develop health problems due to antibiotic-weakened gut microbiomes, and antibiotics tend to have slim profit margins. Pairing the drugs with a proven probiotic could be a way to boost revenues, Cubillos-Ruiz suggested.The company is currently preparing in vivo safety studies in preparation for human trials, Florey said in the release. If all goes well, Florey hopes to launch its flagship product in three years, Cubillos-Ruiz said.First-time biotech CEO Cubillos-Ruiz thinks that in 10 years, we’ll all look back and wonder how we could let antibiotics run roughshod over our gut microbes for so long. It will be like “going to a beach day and not using sunscreen to protect yourself from the sun,” he said.

2024-09-27

·生物制品圈

12项交易超257亿美元！礼来和诺和诺德斥资扩展肥胖症药物研发管线，不止GLP-1

诺和诺德和礼来无疑处于减肥症市场的顶端。今年第二季度，诺和诺德的Wegovy销售额为17亿美元，礼来的Zepbound销售额为12亿美元，这两家公司都赚取了丰厚的利润。在未来几年内，预计其还将继续主导肥胖症市场，而不会面临太大挑战。为了确保在竞争对手进入市场时保持领先地位，这两家公司正在斥资数十亿美元进行许可和并购交易。根据Pitchbook 和Morning Star在9月9日发布的一份报告，这两家巨头可能会继续占据这一地位，直到竞争对手在2026年左右开始进入市场。但是，来自拓臻生物（Terns Pharmaceuticals）、硕迪生物（Structure Therapeutics）、辉瑞（Pfizer）和安进（Amgen）等公司的下一波药物看起来可能会击败目前的产品。据Pitchbook估计，到2031年，所有这些公司都可以联合起来争夺每年约2000亿美元的市场。对此，礼来和诺和诺德并没有放任其不管，而是采取了一些行动。他们一直在收购公司并达成交易，以分散对肥胖症治疗的赌注。报告指出，这两家制药公司都非常有望在该领域进行进一步收购。两家公司还通过许可协议进行了小规模的尝试，以探索新的靶点或下一代GLP-1方法。诺和诺德高管在3月份的投资者日演讲中表示，肥胖症是公司的主要投资重点。诺和诺德在业务开发方面最为高产，迄今为止，其拥有37个专注于心血管代谢疾病和肥胖症的合作伙伴关系。值得注意的是，该公司特别专注于寻找新的作用机制。例如，诺和诺德正在大力投资多个治疗领域的siRNA技术，其中大部分是糖尿病和肥胖症。该公司还在建立蛋白质、肽和单克隆抗体的内部能力，同时通过合作和收购为其肥胖症产品线增加小分子选择。与此同时，礼来在上半年为业务发展总共分配了14亿美元，包括里程碑付款、已完成的收购和股权投资。另外还投入了44亿美元用于研发；该公司有11种针对肥胖症的新分子正在开发中，其中包括1个强大的后期项目和9个正在进行的试验。 “在肥胖症领域，我们的战略是全面解决这场全球公共卫生危机，寻求针对各种合理的机制、适应症和剂型的机会，”礼来首席执行官David Ricks在8月初的公司第二季度财报电话会议上表示。下面分别盘点了近年来诺和诺德和礼来为保持其在肥胖领域的领先地位而进行的交易。诺和诺德 1、合作/交易方：Metaphore 日期：2024年5月9日价值：6亿美元资产：两种GLP-1受体激动剂诺和诺德与Flagship Pioneering及该公司旗下子公司Metaphore Biotechnologies达成战略合作，共同开发至多两种下一代肥胖治疗药物。Metaphore将与Pioneering Medicines（Flagship 的内部药物开发部门）和 Novo Nordisk联合开展研发项目，Metaphore方负责产品的开发和临床前研究，诺和诺德则负责将这些项目推进至临床阶段。本次合作重点是针对GLP-1受体开发多靶点长效药物，无需频繁给药即可达到减重目的。根据协议条款，诺和诺德将有望支付高达6亿美元的前期、开发和商业里程碑付款，以及许可产品年度净销售额的分级特许权使用费，这些费用将由Metaphore和Pioneering Medicines 分享；诺和诺德还将报销研发费用并参与Metaphore未来的融资。据悉，这是诺和诺德与Flagship签订的第三份合作。早在2024年1月，诺和诺德已与Flagship孵化的Omega Therapeutics和Cellarity公司达成新药研究合作，开发用于肥胖管理的表观遗传药物和针对代谢相关脂肪性肝炎（MASH）的新型疗法，每项合作总额可能高达5.32亿美元。 2、合作/交易方：Catalent 日期：2024年2月5日价值：165亿美元资产：3个灌装-成品基地 2月5日，诺和诺德宣布公司已同意从诺和诺德的控股股东Novo Holdings收购3个灌装-成品基地，作为Novo Holdings同意通过165亿美元合并收购Catalent（康泰伦特）的交易的一部分。康泰伦特是一家总部位于美国新泽西州萨默塞特的全球合同开发和生产组织。根据协议条款，诺和诺德将以110亿美元的预付款收购这三个生产基地。此次收购将在康泰伦特和Novo Holdings子公司合并后尽快完成，预计将在2024年底满足各种惯例成交条件后完成，包括康泰伦特股东和监管机构的批准。在收购完成之前，康泰伦特将继续独立运营，独立于Novo Holdings和Novo Nordisk。交易完成后，诺和诺德将履行诺和诺德正在收购的三个康泰伦特工厂的所有客户义务。此次收购灌装厂符合诺和诺德的战略，即让更多的糖尿病和肥胖症患者接受目前和未来的治疗。收购实现了生产规模和速度的扩张，同时为诺和诺德现有的供应网络提供了未来的选择性和灵活性。预计此次收购将从2026年起逐步提高诺和诺德的灌装能力。 3、合作/交易方：EraCal Therapeutics 日期：2024年1月24日价值：2.55亿美元资产：口服小分子项目诺和诺德通过与瑞士生物技术公司EraCal Therapeutics签署的2.55亿美元的许可协议，增加了一种新的肥胖作用机制。该交易授予诺和诺德对EraCal 口服小分子计划的独家权利，该计划旨在控制食欲和体重以治疗肥胖症。两家公司自2022年以来一直在合作，以寻找食物摄入调节和其他代谢表型的靶点。EraCals基于斑马鱼的技术平台源于苏黎世大学和哈佛大学的研究成果。它能够在一种完整的活生物体中大规模地分析像食物摄入这样的复杂表型。这种技术不但能够根据体内生物利用度和毒性对化合物进行筛选，还能够检测潜在候选药物在中枢神经系统中的不良反应，这些特征让它能够识别在体内更具特异性的化合物。 4、合作/交易方：Omega Therapeutics 日期：2024年1月5日价值：5.32亿美元资产：肥胖管理潜在新疗法诺和诺德、Omega Therapeutics和Cellarity宣布，诺和诺德已分别与Flagship Pioneering旗下的这两家生物平台（bioplatform）公司达成研究合作，每项合作总额可能高达5.32亿美元。其中，诺和诺德与Omega的合作将利用Omega专有平台技术开发一种表观基因组控制器，旨在增强代谢活性，作为肥胖管理潜在新疗法；诺和诺德与Cellarity的合作则旨在解开代谢功能障碍相关脂肪性肝炎（MASH，原名为非酒精性脂肪性肝炎[NASH]）的新型生物驱动因子，并将利用Cellarity的平台开发针对该疾病的小分子疗法。 5、合作/交易方：Embark Biotech 日期：2023年8月30日价值：5.12亿美元资产：Embark Biotech，来自Embark Laboratories的资产诺和诺德收购Embark Biotech，包括其先导代谢项目，以开发治疗肥胖和相关合并症的新型药物。交易金额可能超过4.7亿欧元（合5.12亿美元）。根据收购协议，诺和诺德将获得先导项目开发和商业化的全部权利，而Embark股东将获得1500万欧元预付款现金，并有资格获得高达5.12 亿美元的潜在开发、监管和商业里程碑付款。作为协议的一部分，诺和诺德还与Embark Laboratories进行了为期三年的研发合作。 6、合作/交易方：Inversago Pharma 日期：2023年8月10日价值：10.75亿美元资产：Inversago Pharma 诺和诺德收购Inversago pharma，囊获其用于治疗肥胖、糖尿病及其他代谢性疾病的临床阶段管线。根据协议，诺和诺德将支付6亿美元的预付款，如果里程碑达到，交易总额可高达10.75亿美元。据悉，Inversago致力于开发大麻素1型受体（CB1r）的反向激动剂，用于糖尿病肾病、肥胖、NASH、1型糖尿病等代谢性疾病。 7、合作/交易方：Flagship Pioneering 日期：2022年5月价值：未披露资产：多家公司合作相关研究项目 2022年5月，Flagship Pioneering和诺和诺德宣布合作创建一系列新颖的研究项目来进行药物开发转化。两家公司将探索应用Flagship创新生物平台，以应对心脏代谢和罕见疾病领域的科学挑战，并启动相关研究项目。诺和诺德将为启动的研究项目提供资金，并拥有每个项目的独家许可选择权，目标则是在合作的前三年内启动三到五个研究项目。这种新颖的合作方式，为公司之间提供了广泛的创新机会，可以利用多家生物技术公司的技术平台，创建跨多个疾病领域的药物组合。 8、合作/交易方：Dicerna Pharmaceuticals 日期：2021年11月18日价值：33亿美元资产：Dicerna Pharmaceuticals 诺和诺德于2019年与Dicerna Pharmaceuticals合作寻找新的RNAi疗法，合作研究了30个肝细胞靶点，这些靶点可能在MASH、2型糖尿病、肥胖症和罕见病中产生新的疗法。诺和诺德对此感到满意，于2021年11月以约33亿美元的价格完全收购了该公司。除了肥胖之外，诺和诺德还看到了Dicerna的技术有可能找到治疗糖尿病、心血管疾病、MASH以及内分泌和出血性疾病的药物。 9、合作/交易方：Kallyope 日期：2018年6月21日、2024年9月价值：未披露资产：肥胖配体这笔交易进展缓慢。诺和诺德和Kallyope最初于2018年6月达成了一项合作研究和期权协议，共同开发治疗肥胖症和糖尿病的新型多肽类药物。当时没有公布财务细节，但该交易涉及预付费用和研究支持。诺和诺德获得了最多六种产品的权利，并提供了许可费（如果有）。Kallyope正在探索肠脑轴，正在进行四个新陈代谢项目。本月早些时候，有了一些关于该合作的进展情况。据悉，诺和诺德行使了许可Kallyope确定的用于肥胖的配体的选择权。同样，财务细节也处于保密状态。诺和诺德表示，其相信自己在Kallyope计划中发现了一种新的肥胖机制。现在，诺和诺德将接管临床前和临床开发、制造和商业化。Kallyope已收到预付款，并且可能会在开发和销售里程碑以及特许权使用费方面收到额外付款。礼来 1、合作/交易方：Haya Therapeutics 日期：2024年9月5日价值：高达10亿美元资产：RNA靶向疗法今年9月，礼来与Haya Therapeutics宣布达成一项总额高达10亿美元的多年协议。两家公司将应用HAYA的RNA引导基因调控平台来支持肥胖和相关代谢疾病的临床前药物研发工作。两者将确定多个能够调控基因表达的RNA药物靶点，以治疗这些慢性疾病。 Therapeutics是一家精准医疗公司，致力于开发可编程疗法，靶向不编码蛋白的调节性RNA，以重新编程包括心血管疾病、代谢疾病和癌症在内多种疾病的病理细胞状态。该公司正在利用其创新平台获得有关疾病细胞状态生物学和相关长链非编码RNA（lncRNA）的新见解。HAYA的主打项目是HTX-001，正在开发用于治疗心力衰竭。HAYA还在开发一系列靶向lncRNA的精准疗法，用于针对其他组织疾病的细胞特异性治疗。 HAYA专有的基因调控发现平台能够识别组织、疾病和细胞特异性的lncRNA靶标，并开发相对应的RNA靶向疗法，从而重新编程细胞并潜在使之恢复成健康状态。与现有疗法相比，这类疗法可能更有效且毒性更小。此次合作将利用HAYA的基因调控平台来识别、检视和验证多个创新lncRNA靶标，旨在开发出治疗肥胖症和相关代谢疾病的新疗法。 2、合作/交易方：BioAge 日期：2023年10月价值：未披露资产：azelaprag与Zepbound的组合疗法礼来与BioAge同意携手开展一项临床试验，将后者的apelin受体APJ激动剂azelaprag与礼来的Zepbound进行组合。该临床试验的期望是这种组合能够提升礼来这款重磅炸弹级产品的减肥潜力。该临床试验供应协议一直都在进行，但礼来是通过其独立的临床开发组织Chorus来进行这项研究的。该部门与生物技术公司合作，通过临床概念验证来开发资产。礼来还进一步加大了参与力度，于2月参与了BioAge的D轮融资。BioAge筹集了1.7亿美元，投资方包括RA Capital、Cormorant、SV Health、礼来和安进。根据9月18日美国证券交易委员会（SEC）提交的文件显示，这家生物技术公司提交了上市申请，并寻求高达1.533亿美元的融资。 3、合作/交易方：Versanis Bio 日期：2023年7月14日价值：19.25亿美元资产：Versanis Bio 2023年7月，礼来公司宣布以19.25亿美元收购Versanis Bio，这是一家专注于开发治疗心脏代谢疾病新药的私人临床阶段生物制药公司。交易的核心是bimagrumab，这是一种单克隆抗体，旨在在不影响肌肉质量的情况下减少脂肪。诺华开发了治疗肌肉疾病的疗法，但研究未能显示患者身体机能的改善。然而，研究人员确实注意到，服用这种药物的人体重减轻了。快进到席卷制药行业的减肥热潮，礼来通过近20亿美元的支出从诺华的抛售中脱颖而出。Bimagrumab正在针对成人的IIb期BELIEVE研究中进行测试，以实现和保持减脂和健康的身体成分。据ClinicalTrials.gov称，预计该试验将于2025年年中完工。参考资料： [1]Lilly and Novo Spend Billions to Expand Obesity Pipelines Beyond GLP-1s.September 25, 2024.Biospace. [2]165亿美元！诺和诺德收购Catalent.医药魔方Pro.2024年02月05日. [3]速递 | 2.35亿欧元！诺和诺德囊获口服小分子减肥药.药明康德.2024年01月24日. [4]速递 | 开发肥胖、NASH创新疗法，诺和诺德/ Flagship达超10亿美元合作！.药明康德.2024年01月05日. [5]再拿一城！诺和诺德5.12 亿美元收购减肥新秀Embark Biotech.药智头条.2023年09月01日. [6]【深度解析】诺和诺德超10亿美元收购Inversago，和礼来比眼光如何？.萝卜药闻.2023年08月14日. [7]超43亿！诺和诺德出手下一代减肥疗法.动脉新医药.2024年05月11日. [8]最新布局！礼来达成10亿美元合作开发RNA靶向疗法.药明康德.2024年09月05日. 为促进抗体行业的交流与创新，2024年10月16-17日第七届金秋十月抗体产业发展大会如约而至。会议旨在为研究人员提供一个互动交流的平台，有助于推动抗体产业的进一步发展。会议内容时间：2024年10月16-17日地点：上海张江（酒店定向通知）规模：600-800人主办单位：生物制品圈、抗体圈演讲支持：Entegris、瑞孚迪、AS ONE CORPORATION 会议费用：免费FREE!（仅收取100元报名定金，含参会学习、茶歇、会议手册，定金概不退还），先到先得，报完即止！报名方式：扫描下方二维码或点击文章最底部“阅读原文”→ 填写表格 → 报名成功（报名志愿者，承担一定工作，请慎重考虑，免交定金）！组委会获得报名信息后，根据报名信息进行初筛，并进一步与报名者沟通确认，实现精准邀请。最终有机会进入大会微信群（严格审核通过）。日程安排更多嘉宾正在邀请中识别微信二维码，添加生物制品圈小编，符合条件者即可加入生物制品微信群！请注明：姓名+研究方向！版权声明本公众号所有转载文章系出于传递更多信息之目的，且明确注明来源和作者，不希望被转载的媒体或个人可与我们联系(cbplib@163.com)，我们将立即进行删除处理。所有文章仅代表作者观点，不代表本站立场。

并购siRNA财报

2024-09-19

·Pharmaceutical Technology

Novo Nordisk and Nanovation sign $600m deal in rare and cardiometabolic diseases

As part of the deal, Novo Nordisk and NanoVation will collaborate on up to seven programmes. Credit: PeopleImages.com – Yuri A via Shutterstock. Novo Nordisk has signed a $600m deal with NanoVation to develop genetic medicines for rare and cardiometabolic diseases. Free Report How is the Biopharmaceutical industry evolving? 2021 was a year of continued innovation and change in the Biopharmaceutical industry. As the COVID-19 pandemic continues to take its toll on businesses worldwide, it’s time to look for new ways to create value, prepare for the future, and remain competitive in the ever-changing landscape. GlobalData’s expansive report examines the business environment and trends that shape the Biopharmaceutical industry. We highlight the most impactful emerging technologies, as well as the industry, regulatory, and macroeconomic factors that influence growth prospects. Access the report to: Benchmark the impact of major themes on the Biopharmaceutical industry. Gain a deeper "on the ground" perspective through exclusive opinions and analysis from industry respondents. Evaluate the effects of COVID-19 on the sector. Download the full report to understand what to expect and how to align your strategies for success. Thank you. You will receive an email shortly. Please check your inbox to download the Report. Go deeper with GlobalData Reports NPVM: Innovent Biologics Inc's Mazdutide Reports NPVM: Novo Nordisk AS's Ideal Pump Insulin Data Insights The gold standard of business intelligence. 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Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address. Novo has secured a licence to use NanoVation’s long-circulating lipid nanoparticle (lcLNP) technology for RNA delivery targeting cells outside the liver. Both companies will collaborate on two programmes to develop base-editing therapies for rare genetic diseases, with plans for up to five future targets for cardiometabolic and rare diseases. Under the agreement, Novo is paying an undisclosed upfront fee. The multi-year deal could be worth up to $600m, with NanoVation set to receive research funding eligible for potential milestone payments, as well as tiered royalties on future product sales. Novo has secured several deals in the past few years to maintain its dominant position in the cardiometabolic disease market. The Danish pharma company entered research collaborations with two US cell and gene therapy biotech startups in January, namely Omega Therapeutics and Cellarity. In February 2024, Novo partnered with US-based biotech Neomorph in a collaboration and licensing agreement to discover and develop multiple molecular glue degraders for cardiometabolic disorders and rare diseases. See Also: AstraZeneca’s Fasenra to challenge GSK’s Nucala in rare vasculitis after FDA nod FDA approves Accord BioPharma’s HERCESSI for cancer treatment Novo has also shown its commitment to developing therapies for disorders with unmet needs such as metabolic dysfunction-associated steatohepatitis (MASH). The company’s collaboration with Cellarity involves the development of therapies for MASH, for which there are no treatments approved by the US Food and Drug Administration (FDA). Novo’s Q1 2024 financial results highlighted that the company’s aspiration to expand the use of its blockbuster obesity products to cardiovascular indications has been successful. In March, its obesity drug Wegovy (semaglutide) was approved by the FDA for reducing the risk of cardiovascular diseases in obese or overweight adults. Semaglutide is also marketed as Ozempic in type 2 diabetes. Wegovy generated $4.5bn in sales for Novo in 2023, as per the company’s financials. According to GlobalData analyst consensus forecasts, the drug is set to make $19.4bn in 2030. GlobalData is the parent company of Pharmaceutical Technology. In the announcement accompanying the deal, NanoVation’s co-founder Pieter Cullis said: “Genetic medicine is at a pivotal moment and this partnership marks a major milestone for NanoVation as an innovator in nucleic acid delivery. By combining NanoVation’s expertise in extrahepatic delivery with Novo Nordisk’s expertise in cardiometabolic and rare diseases we have the potential to create truly transformative therapies.” Free Report How is the Biopharmaceutical industry evolving? 2021 was a year of continued innovation and change in the Biopharmaceutical industry. As the COVID-19 pandemic continues to take its toll on businesses worldwide, it’s time to look for new ways to create value, prepare for the future, and remain competitive in the ever-changing landscape. GlobalData’s expansive report examines the business environment and trends that shape the Biopharmaceutical industry. We highlight the most impactful emerging technologies, as well as the industry, regulatory, and macroeconomic factors that influence growth prospects. Access the report to: Benchmark the impact of major themes on the Biopharmaceutical industry. 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引进/卖出上市批准

100 项与 Cellarity Inc. 相关的药物交易

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100 项与 Cellarity Inc. 相关的转化医学

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管线布局

2024年11月20日管线快照

管线布局中药物为当前组织机构及其子机构作为药物机构进行统计，早期临床1期并入临床1期，临床1/2期并入临床2期，临床2/3期并入临床3期

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药物(靶点)	适应症	全球最高研发状态

CLT-1081 ( Hemoglobins )	镰状细胞血症更多	临床前
Metabolic dysfunction-associated steatohepatitis(Cellarity)	代谢功能障碍相关的脂肪肝病更多	临床前