GeneSpire Srl

Six months after launching with a mega-round of financing, Seaport Therapeutics has raised $225 million more to advance a lead drug candidate that could overcome some limitations of currently available treatments for depression. Seaport’s platform technology, called Glyph, designs oral drugs that leverage the lymphatic system to deliver the medicine in a way that makes more of the active pharmaceutical ingredient available in the body to confer its therapeutic effect. The Boston-based company also says its drugs reduce complications such as elevated liver enzyme levels, a sign of drug toxicity that can limit the use of some molecules. Lead Seaport program SPT-300 is a prodrug, a compound that converts inside the body into the active drug allopregnanolone. This neurosteroid modulates the GABA-A receptor to spark effects on the central nervous system. Seaport is advancing the drug into Phase 2b testing as a potential treatment for major depressive disorder with or without anxious distress. Seaport says the new capital will go toward advancing its pipeline through important clinical milestones. The company also plans to further advance the capabilities of its Glyph technology platform. presented by Health IT Accelerating Claim Processing: Strategies to Shorten the Life of a Claim These strategies and practices can significantly shorten the life cycle of claims, leading to quicker resolutions and improved financial outcomes. By Greenway Health Seaport was spun out of PureTech Health, a company that forms startups around platform technologies. In April, Seaport launched, revealing its science, its lead drug candidate, and $100 million in funding. That Series A round was led by Arch Venture Partners and Sofinnova Investments. General Atlantic led Seaport’s new Series B financing. Other participants in the latest round included T. Rowe Price Associates, Foresite Capital, Invus, Goldman Sachs Alternatives, CPP Investments, and other unnamed new investors. Founding investors Arch and Sofinnova also joined the round, as did Third Rock Ventures and PureTech Health. Seaport is part of a bevy of life science companies that have secured new capital. Here’s a recap of recent biotech financing news: —Radiopharmaceuticals developer Alpha-9 Oncology closed a $175 million Series C round to support its pipeline, including A9-3202, which began a Phase 1 test earlier this year in advanced melanoma. Lightspeed Venture Partners and Ascenta Capital led Alpha-9’s new financing. —AvenCell Therapeutics has $112 million in Series B funding to continue Phase 1/2 testing of AVC-101, a CAR T-therapy that can be switched “on” or “off” to address safety and efficacy challenges of currently available cell therapies, which are always “on.” AvenCell was formed in 2021 by Intellia Therapeutics and Cellex Cell Professionals. The company’s latest financing was led by Novo Holdings. Sponsored Post The Future of Hospitals and Pharma Companies Will Depend on Strength of Healthcare Analytics Insights PurpleLab® stands out from others in this sector by providing its data analytics services to several different groups of users across healthcare and pharma companies. By Stephanie Baum —Tolerance Bio launched with $17.2 million to support development of allogeneic stem cell-based cell therapies. These therapies are intended to preserve the thymus, the organ that trains T cells to defend against threats and preserve autoimmunity. The Philadelphia-based biotech’s seed financing, led by Columbus Venture Partners, will be used to advance toward the clinic with potential therapies for immune diseases. —March Biosciences, spun out of the Center for Cell and Gene Therapy, unveiled $28.4 million in Series A financing. Lead program MB-105 is a CAR T-therapy designed to target CD5, a protein expressed on cancerous T cells. A Phase 1 test is underway enrolling patients with refractory T cell lymphoma and leukemia. Mission BioCapital and 4BIO Capital led March Bio’s financing. —Be Biopharma closed $82 million in financing as the cell therapy developer moves into the clinic with BE-101, a potential treatment for hemophilia B. The biotech’s therapies are made by engineering B cells to produce therapeutic proteins. Be Bio last raised money in 2022, a Series B financing that totaled $130 million. —Terray Therapeutics, a biotech that uses generative AI to discover and develop small molecule drugs, unveiled a $120 million Series B financing. Los Angeles-based Terray will use the capital to advance internal programs into clinical trials for indications that were not disclosed. The financing was led by new investor Bedford Ridge Capital and earlier investor NVenture, the investment arm of NVIDIA. Here’s more about Terray and its technology platform, tNova, which it unveiled in 2022. —Genetic medicines have been difficult to deliver to the kidney. Judo Bio is developing oligonucleotide drugs that target a particular receptor to reach the organ, and the Cambridge-based startup unveiled $100 million in seed and Series A financing to advance its research toward the clinic. The Series A round was co-led by Atlas Venture, TCG, and Droia Venture. —Purespring Therapeutics, a developer of gene therapies for kidney disease, raised £80 million (about $105 million). The London-based company’s therapies target the podocyte, a special cell implicated in 60% of renal diseases. Proceeds will be used to advance lead program PS-002 into a Phase 1/2 clinical trial for immunoglobulin A nephropathy, a rare disease currently treated with chronically administered therapies. Sofinnova Partners led Purespring’s Series B financing. —Artificial intelligence biotech company Basecamp Research revealed a $60 million Series B financing to scale its collection of data and strengthen its AI capabilities. The London-based startup also announced a multi-year collaboration with David Liu of the Broad Institute of MIT and Harvard. Basecamp said this partnership will research new approaches to programmable genetic medicines with potential applications in a range of diseases. —City Therapeutics launched with $135 million to develop next-generation RNA interference (RNAi) drugs. The company says its engineering platform can design therapies in smaller sizes that have novel cellular mechanisms and improved potency, efficiency, and specificity. The Cambridge-based biotech, co-founded by scientists involved in the first generation of RNAi therapies, expects to enter the clinic by the end of 2025. Arch Venture Partners led City’s Series A round. —Resolution Therapeutics raised £63.5 million to advance to Phase 1/2 testing with RTX001, an engineered autologous macrophage cell therapy that could offer anti-fibrotic and anti-inflammatory effects in patients with end-stage liver disease. The company, which maintains operations in London and Edinburgh, Scotland, said this trial will be conducted in the United Kingdom and Spain. Syncona led the Series B round, which will also support research in other inflammatory fibrotic conditions such as graft-versus-host disease and lung fibrosis. —Triveni Bio raised $152 million to support a pipeline whose most advanced program, TRIV-573, is a bispecific antibody that blocks two targets associated with atopic dermatitis. The Watertown, Massachusetts-based biotech aims to file an investigational new drug application for this program in the first quarter of 2025. Triveni launched last year supported by a $92 million Series A financing. Goldman Sachs Alternatives led the company’s Series B. —Kailera Therapeutics launched with $400 million to support its GLP-1 and GIP receptor agonist drugs for metabolic indications, including obesity. Kailera’s four programs were licensed from China-based Jiangsu Hengrui Pharmaceuticals. The Series A financing was co-led by Atlas Venture, Bain Capital Life Sciences, and RTW Investments. —Radiopharmaceutical developer Aktis Oncology unveiled $175 million in financing to support development of a pipeline led by AKY-1189, an alpha radiotherapy that targets Nectin-4, a tumor-associated antigen found in a range of solid tumors. The Series B round was led by RA Capital Management and co-led by RTW Investments and Janus Henderson Investors. Boston-based Aktis emerged from stealth in 2021 backed by $72 million in Series A financing. —Genespire raised €46.6 million (about $52 million) to advance to Phase 1/2 testing with GENE202, an in vivo gene therapy in development for methylmalonic acidemia, a rare inherited disorder that impairs the metabolism of certain amino acids and fats. The Milan, Italy-based startup’s Series B round was co-led by Sofinnova Partners, XGEN Venture, and CDP Venture Capital. —Vaccines developer Vicebio unveiled $100 million in financing as it begins a Phase 1 test of VXB-241, a bivalent vaccine designed to address both respiratory syncytial virus (RSV) )and human metapneumovirus (hMPV). Data are expected in mid-2025. The next Vicebio program is VXB-251, a trivalent vaccine for RSV, hMPV, and parainfluenza virus 3. The London-based company’s Series B round was led by TCGX. —GC Therapeutics launched with $65 million in financing. The biotech’s next-generation cell therapies come from a platform technology called TFome, which programs induced pluripotent stem cells. GC’s initial therapeutic areas of focus include gastrointestinal, neurological, and immunological diseases. Cormorant Asset Management led Cambridge, Massachusetts-based biotech’s Series A financing. —Neuroscience startup Nura Bio extended its Series A financing by $68 million to proceed to Phase 2 testing with an oral small molecule designed to block SARM1, an enzyme associated with axonal damage. It faces potential competition from Eli Lilly, which is in early clinical development with a drug that goes after the same target. Nura Bio’s founding investor The Column Group led the startup’s new financing. —F2G raised $100 million for olorofim, which is in late-stage development to treat aspergillosis and other invasive fungal infections. Olorofim is from a novel class of antifungal agents that work by inhibiting the pyrimidine synthesis pathway. The cash will enable the Manchester, U.K.-based company to complete late-stage clinical development, submit regulatory applications, and prepare for U.S. commercialization of the drug. The new financing was led by new investor AMR Action Fund and co-led by ICG. —T cell engagers first reached patients as cancer treatments. Candid Therapeutics aims to bring such drugs to autoimmune disorders, and its efforts are backed by more than $370 million in financing. Candid’s drug candidates came from acquiring and merging two biotechs, Vignette Bio and TRC 2004. The Candid pipeline includes two T cell engagers for cancer now being repositioned for development in autoimmune diseases. These therapies are designed to selectively deplete B cells that drive disease. —Startup creator Flagship Pioneering unveiled its newest company, Mirai Bio. Mirai’s platform technology is applied to the development of genetic medicines for the company’s partners working across a range of therapeutic areas and therapeutic modalities. It says its platform technology unlocks delivery of a drug to any tissue and cell type, optimize design of the drug cargo, and facilitates manufacturing. Like other startups that come from Flagship, Mirai is backed by the customary $50 million in financing. —Superluminal Medicines, a company developing small molecule drugs that target elusive G protein-coupled receptors (GPCRs), closed $120 million in financing. The Boston-based startup says its drugs target membrane receptors that mediate cell signaling and response processes fundamental to human physiology. These small molecule come from a generative biology and chemistry platform technology. The new capital will be used to advance toward the clinic in undisclosed indications. RA Capital Management led the Series A financing. —Riding strong investor interest in metabolic disorder drugs, OrsoBio closed $67 million to advance a pipeline that includes programs for type 2 diabetes, obesity, and the fatty liver disease MASH. The Menlo Park, California-based startup’s drugs address pathways associated with energy metabolism. The Series B round, led by Ascenta Capital and Woodline Partners, comes less than a year after the company unveiled $60 million in Series A financing. Photo by Flickr user Massachusetts Office of Travel & Tourism via a Creative Commons license

在当前生物制药的市场当中，早期或临床前项目获取资金是一件尤为困难的事情，因为投资人更倾向于有商业前景的项目。而一种新技术能够获得投资人的青睐，也说明其具有可观的潜力。 近来，一家意大利的初创公司Genespire宣布完成了4660万欧元（约5200万美元）的B轮融资，用于推进其免疫屏蔽慢病毒载体技术基因治疗的临床研究。 该轮融资由Sofinnova Partners、XGEN Venture和CDP Venture Capital共同领投，并与Indaco SGR共同组成了一个投资者群体。 根据报道，这是迄今为止意大利最大的私人生物技术公司融资之一。 Genespire公司是一家专注于开发儿科基因治疗法的生物技术公司，拥有专有的免疫屏蔽慢病毒载体（ISLV）技术。 免疫屏蔽慢病毒载体（ISLV）技术能够屏蔽免疫系统对慢病毒的识别，克服传统慢病毒基因治疗的缺陷，能够实现更低的剂量、具有更好的耐久性。 这种慢病毒载体能够设计为直接输注到患者的外周血中，从而进入肝脏细胞并将治疗基因插入细胞的DNA内。另外，这种整合允许通过细胞分裂并维持治疗基因，确保其在肝脏中的稳定表达，因此也可以适用于年轻患者。 ISLV平台可应用于多种遗传疾病，有望扩大体内基因治疗的范围，为成人和儿童患者提供一次性的、现成的基因治疗。 根据报道，Genespire公司完成的B轮融资将主要用于推进其首个基因治疗项目的临床开发。 Genespire公司的首个项目GENE202是一款针对甲基丙二酸血症（MMA）的基因治疗，基于该公司专有的免疫屏蔽慢病毒载体（ISLV）技术开发。 甲基丙二酸血症 甲基丙二酸血症（MMA）是一种罕见的遗传病，主要由甲基丙二酸代谢相关基因突变引起，导致甲基丙二酸和相关有毒代谢物在体内的积累。最常见线粒体甲基丙二酰辅酶A突变酶（MMUT）基因突变，导致MMUT酶活性降低或完全丧失，破坏甲基丙二酸的正常代谢。MMA的新生儿患病率约为1/90,000。严重者往往在婴儿期死亡，幸存者经常出现不稳定的健康状况，导致多器官并发症，如复发性代谢危象、神经功能损害、慢性肾病和早期死亡。目前尚无针对这种疾病的批准治疗方法，主要治疗方法包括限制蛋白质摄入和使用降氨剂，但长期效果仍不尽如人意。 今年5月，Genespire公司在ASGCT 2024年会上展示了GENE202的临床前数据。在小鼠MMA模型中，通过静脉输注了编码人类MUT转基因的ISLV。结果显示，小鼠表现出快速、大量、持久的循环甲基丙二酸减少，小鼠体重正常化和完全生存。 同时，ISLV通过静脉给药导致全身效应，肝脏、大脑和肾脏中的甲基丙二酸水平均显著降低，表明通过肝脏介导的解毒实现肝外益处。 除了MMA项目之外，Genespire公司还进一步开发了针对其他遗传性代谢疾病和出血性疾病的ISLV项目。 值得注意的是，Moderna有一款临床MMA项目mRNA-3705。 mRNA-3705的全球1/2期临床试验的中期结果表明，队列2和队列3中患者的甲基丙二酸呈剂量依赖性降低。早期结果显示，与治疗前相比，患者的年化代谢失代偿事件（MDE）、频率和该疾病相关住院率可能降低。 参考资料： 1.https://www.biopharmadive.com/news/genespire-mma-gene-therapy-pediatric-series-b/728054/ 2.https://www.genespire.com/wp-content/uploads/2024/09/240925-Genespire-Series-B-press-release-FINAL.pdf 3.https://www.genespire.com/en/our-science/overview/ 4.https://www.genespire.com/en/programs/ 内容来源于网络，如有侵权，请联系删除。

▎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