Angelini Pharma, Inc.

▎药明康德内容团队编辑 据创鉴汇不完全统计，上周（9月23日至9月29日）全球大健康领域共披露融资事件27起，总额近60亿元。按照金额划分，亿元及以上融资12起。按照已披露的融资轮次划分，早期融资（B轮以前）13起，中后期融资（B轮及以后）10起。生物医药领域共8家公司获融资，涉及药物类型包括疫苗、抗体、基因疗法、小分子药物等。 #01  BioAge Labs完成IPO 关键词：小分子药物 最新融资：1.98亿美元IPO 9月26日，BioAge Labs在纳斯达克上市，募资1.98亿美元，该融资所得将用于推动其减肥管线的临床试验。BioAge Labs是一家专注于肥胖症治疗的临床阶段生物技术公司。BioAge Labs的主要候选药物Azelaprag是一种口服小分子药物，属于Apelin受体激动剂。该产品正在与礼来GLP-1/GI受体激动剂Tizepatide联合开展减肥2期临床试验，它还将于明年上半年与诺和诺德减肥产品Wegovy联合进入2期试验。 #02 Vicebio完成1亿美元B轮融资 关键词：疫苗 最新融资：1亿美元B轮 本轮投资机构：Avoro Ventures、Goldman Sachs Alternatives、Medicxi、TCG Crossover、UniQuest等 9月23日，Vicebio宣布完成1亿美元的B轮融资，获得的资金将用于推动其双价疫苗VXB-241的1期临床试验，该疫苗靶向呼吸道合胞病毒（RSV）和人偏肺病毒（hMPV）。这笔资金还将支持Vicebio多价疫苗管线的加速开发，包括VXB-251，这是一种靶向RSV、hMPV和副流感病毒3型（PIV3）的三价疫苗。Vicebio致力于开发针对呼吸道病毒的下一代疫苗，采用其专有的分子钳技术（molecular clamp）。该技术由昆士兰大学发现，能够独特地稳定病毒糖蛋白处于高度免疫原性的融合前构象，这对于引发强有力的保护性免疫反应至关重要。 #03 Genespire完成4660万欧元B轮融资 关键词：基因疗法 最新融资：4660万欧元B轮 本轮投资机构：CDP Venture Capital、Sofinnova Partners、XGen Ventures 9月25日，Genespire宣布完成4660万欧元B轮融资，获得资金将支持该公司的核心候选产品GENE202的开发，直至进入治疗甲基丙二酸血症（MMA）的1/2期临床试验。此外还将加强公司产品管线，促进针对多种其他遗传疾病候选疗法的发现和临床前研究。GENE202是一种现货型基因疗法，利用了该公司的免疫屏蔽慢病毒载体（ISLV）平台，设计为可静脉注射。ISLV的作用机制使Genespire的治疗方法特别适用于当前面临最紧迫未满足医疗需求的遗传病儿童患者，同时也能惠及成人患者。 #04 天辰生物完成近亿元B2轮融资 关键词：抗体 最新融资：近亿元B2轮 本轮投资机构：启明创投 9月24日，天辰生物宣布成功完成近亿元B2轮融资。通过本轮融资，天辰生物将推进核心项目在中国的临床研究，加速产品上市进程，并助力国际布局。天辰生物成立于2020年，是一家致力于过敏和补体领域大分子创新药开发的中国生物技术公司。今年6月，天辰生物进展较快的新一代抗IgE抗体药物LP-003注射液，启动治疗季节性过敏性鼻炎患者的3期临床试验。此外，LP-005是潜在“first-in-class”C5与C3补体双功能抗体，已启动阵发性睡眠性血红蛋白尿症（PNH）的2期临床试验。根据天辰生物官网，该公司临床前在研管线还包括AAV介导的补体抑制剂LP-008项目，拟开发治疗年龄相关性黄斑变性（AMD）等。 #05 Aptadir Therapeutics完成160万美元pre-种子轮融资 关键词：寡核苷酸 最新融资：160万美元pre-种子轮 本轮投资机构：Angelini Ventures、Evotec、EXTEND 9月24日，Aptadir Therapeutics宣布正式成立，致力于开发基于RNA的创新疗法，治疗难治性癌症和遗传疾病。Aptadir从EXTEND技术转移生物技术中心获取了160万美元的pre-种子资金，用以优化其主要资产Aptadir Ce-49。该药物正在开发用于治疗骨髓增生异常综合征，目标是在2025年底进入临床开发阶段。该公司的技术平台基于一种能够与DNA甲基转移酶（DNMTs）相互作用的RNA分子。这些RNA分子能够精准地阻断特定基因的异常DNA甲基化，重新激活先前由于高度甲基化而不表达的基因。某些基因高度甲基化是癌症和遗传性疾病的一个关键特征。通过重新激活特定基因，有望针对一些疾病的根本原因进行治疗，比如骨髓增生异常综合征（MDS）和脆性X综合征。 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

Aptadir Therapeutics is based in Milan Italian biotech Aptadir Therapeutics has launched with the promise that its pipeline of preclinical RNA inhibitors could crack intractable cancers.The Milan-based company was founded by RNA leaders Annalisa Di Ruscio, M.D., Ph.D., of Harvard Medical School's Beth Israel Deaconess Medical Center and Vittorio De Franciscis, Ph.D., of the Italian Research National Council alongside leukemia expert Daniel Tenen, M.D., of the Cancer Science Institute of Singapore and oligonucleotide-focused Marcin Kortylewski, Ph.D., of the City of Hope National Medical Center.At the center of this joint venture is a new class of RNA inhibitors called DNMTs interacting RNAs (DiRs), which are able to block aberrant DNA methylation at a single gene level. The theory is that this reactivates previously hypermethylated genes, considered to be a key feature in cancers as well as genetic disorders.Reactivating specific genes offers the hope of reversing cancers and genetic conditions for which there are either no or limited curative options, such as the blood cancer myelodysplastic syndrome (MDS) in adults and the neurodevelopmental disorder fragile X syndrome in children.Aptadir is hoping to get the most advanced of its DiRs, a MDS-focused candidate dubbed Ce-49, into clinical trials by the end of 2025. To help reach this milestone, the biotech has received $1.6 million in pre-seed funding from the Italian National Technology Transfer Hub’s EXTEND initiative. The hub was set up Italian VC manager CDP Venture Capital SGR. Aptadir is the first biotech to come out the EXTEND initiative, which is partly funded by Rome-based VC firm Angelini Ventures as well as German biotech Evotec.EXTEND’s goal is to “develop high quality science coming from top Italian universities and to help build new start-ups that can develop that science for the benefit of future patients,” CDP Venture Capital’s Claudia Pingue explained in the release.Giovanni Amabile, entrepreneur in residence of EXTEND, has been appointed CEO of Aptadir, having previously helmed autoimmune biotech Enthera.“Aptadir’s business is based on real innovation—a landmark discovery of a new class of molecules which have the potential to be best-in-class therapeutics for intractable conditions,” Amabile said in a Sept. 24 release.“From data already generated, DiRs are highly selective, stable and non-toxic, and have the potential to be used across multiple indications,” Amabile added. “This is a really exciting new field and we are looking forward to pushing our first candidate forward into the clinic.”

MILAN--( BUSINESS WIRE )--Aptadir Therapeutics (Aptadir), a biotech company developing RNA inhibitor-based therapeutics for treating intractable cancers and genetic conditions, announces its launch and the appointment of Giovanni Amabile as Executive Chairman and acting CEO. Aptadir was founded by Dr Annalisa Di Ruscio and Dr Vittorio De Franciscis, both leaders in the field of RNA biology and DNA methylation, alongside Prof. Daniel Tenen, an expert in MDS and leukaemia, and Prof. Marcin Kortylewski, an expert in oligonucleotide immunotherapeutics. They are based at world class institutions: the Beth Israel Deaconess Medical Center of the Harvard Medical School, the Italian Research National Council, the Cancer Science Institute of Singapore and City of Hope National Medical Center. The basis for the Company was the landmark discovery of a new class of RNA inhibitors called DNMTs Interacting RNAs (DiRs) 1,2 . DiRs are capable of blocking aberrant DNA methylation at a single gene level reactivating previously hypermethylated genes – a key feature in cancer and genetic disorders. By reactivating specific genes, there is the opportunity to target the underlying cause of and potentially reverse specific intractable cancers and genetic conditions in adults, such as Myelodysplastic Syndrome (MDS) and, in children, such as Fragile X Syndrome. Aptadir’s proprietary platform can generate multiple DiRs for specific genes suppressed in cancer and genetic conditions, re-establishing the pathway functionality. The first programme, Aptadir Ce-49 is being developed in MDS and the aim is to enter the clinic by the end of 2025. Leading the management team is Giovanni Amabile who has been appointed Executive Chairman and acting CEO; he is also Entrepreneur in Residence of EXTEND. Previously, he was CEO at Enthera and before then, CSO at ADIENNE Pharma. He has a successful track record in raising seed and Series A funds, and for leading IND approvals, complex clinical trials and regulatory approvals in the US. Giovanni Amabile, Executive Chairman and CEO , said: “ Today represents the launch of Aptadir Therapeutics and is an exciting day for the founders and team. Aptadir’s business is based on real innovation - a landmark discovery of a new class of molecules which have the potential to be best-in-class therapeutics for intractable conditions. From data already generated, DiRs are highly selective, stable and non-toxic, and have the potential to be used across multiple indications. This is a really exciting new field and we are looking forward to pushing our first candidate forward into the clinic.” Aptadir has received $1.6m in pre-seed funding from the EXTEND initiative to optimize its lead asset, Aptadir Ce-49. The Company is the first to receive funding from EXTEND, which is the Italian National Technology Transfer Hub wholly dedicated to the biopharmaceutical sector for the development of new therapeutic approaches. The Hub was initiated by CDP Venture Capital SGR, and joint funded with co-investors, Angelini Ventures and Evotec SE. Its purpose is to develop translational drug discovery partnerships with highly renowned universities and research centers in Italy to accelerate their promising therapeutics for commercialization. Claudia Pingue, senior partner and head of the Technology Transfer Fund of CDP Venture Capital SGR , added: “ Aptadir is the first biotech company born from EXTEND, National Technology Transfer hub established on the initiative of CDP Venture Capital and funded with co-investment partners, Angelini Ventures and Evotec. EXTEND aims to develop high quality science coming from top Italian universities and to help build new start-ups that can develop that science for the benefit of future patients. Aptadir represents one of the best examples of where science innovation can create a new potential therapeutic for underserved conditions in adults and children, such as MDS and Fragile X Syndrome. We’ll be watching its progress with great interest.” Di Ruscio A et al 2013, Nature Esposito CL et al 2023, Nature Communication Notes to Editors About Aptadir Therapeutics Aptadir Therapeutics is a preclinical biotech company developing a new class of RNA therapeutics. The Company’s therapeutic focus is underserved intractable cancers and genetic conditions, including underserved intractable conditions in the oncology and rare diseases fields such as Myelodysplastic Syndrome and Fragile X Syndrome. The technology was developed and validated by a group of key opinion leaders in the field of RNA therapeutics based at three world class institutions: the Harvard Medical School, Cancer Science institute of Singapore, Italian CNR Institute and City of Hope National Medical Center. The platform is based on a landmark discovery of a new class of RNA inhibitors called DNMTs Interacting RNAs (DiRs) 1,2 . DiRs are capable of blocking aberrant DNA methylation at a single gene level reactivating previously hypermethylated genes – a key feature in cancer and genetic disorders. The first programme is developing a treatment for Myelodysplastic Neoplasms (MDS), a group of blood cancers that occur when immature blood cells in the bone marrow don't mature into healthy blood cells. Sufferers of MDS go on to develop acute myeloid leukaemia (AML). To find out more, please see: www.aptadir.com About EXTEND EXTEND is the Italian National Technology Transfer Hub (the Hub) wholly dedicated to the biopharmaceutical sector for the development of new therapeutic approaches and it was formed as an initiative of CDP Venture Capital to transform the results of this scientific research into new companies. Alongside CDP Venture Capital, the Hub is funded by co-investors, Evotec, which also acts as an entrepreneurial partner by supporting research groups along the path of scientific study up to the establishment of startups ready to raise capital on the market to start the clinical phase, and Angelini Ventures, the new Angelini Industries Group company operating in the VC sector with a focus on digital health and biotech. The scientific promoters of the initiative are IRSCC San Raffaele Hospital in Milan, the Universities of Milan, Florence, Modena and Reggio Emilia, Padua, Trento, La Sapienza in Rome, the regional research district of Puglia through H-Bio, and Human Technopole. To learn more, please visit: www.extend-tt.vc