CAMBRIDGE, U.K.--(BUSINESS WIRE)--Healx, an AI-enabled, clinical-stage biotech company specializing in rare diseases, announced it has raised $47 million in a Series C round. The Series C round was co-led by Silicon Valley-based R42 Group and Atomico, one of Europe’s largest venture capital firms, with participation from new and existing investors including Balderton, Jonathan Milner, Global Brain, btov, Ayana Capital, o2h and VU Venture Partners.

英国剑桥——（商业新闻短讯）——Healx是一家人工智能支持的临床阶段生物技术公司，专门研究罕见疾病，它宣布在C轮融资中筹集了4700万美元。C轮由总部位于硅谷的R42集团和欧洲最大的风险投资公司之一Atomico共同领导，包括Balderton、Jonathan Milner、Global Brain、btov、Ayana capital、o2h和VU venture Partners在内的新投资者和现有投资者参与。

Proceeds of the financing will be used to advance the company’s pipeline of medicines in rare oncology, renal and neurodevelopmental disorders, including advancing its lead program HLX-1502 through a Phase 2 clinical trial for the treatment of neurofibromatosis Type 1 (NF1). In conjunction with the financing, Stanford Medicine Adjunct Professor Ronjon Nag, Ph.D., founder of R42 Group and 2024 Silicon Valley Hall of Fame AI inductee, joins the board of Healx..

融资收益将用于推进该公司在罕见肿瘤学，肾脏和神经发育障碍方面的药物管道，包括通过治疗1型神经纤维瘤病（NF1）的2期临床试验推进其领先项目HLX-1502。在融资的同时，斯坦福医学副教授Ronjon Nag博士，R42集团创始人和2024年硅谷名人堂AI诱导者加入了Healx董事会。。

“It is truly a pivotal time at Healx,” said Tim Guilliams, Ph.D., co-founder and CEO of Healx. “By December 2024, we will advance HLX-1502 into a Phase 2 clinical trial in patients with NF1 both for plexiform neurofibroma, a genetic condition with limited treatment options and for cutaneous neurofibroma, for which there are no FDA approved options.

“这确实是希克斯的关键时刻，”希克斯联合创始人兼首席执行官蒂姆·吉利亚姆博士说。“到2024年12月，我们将HLX-1502推进NF1患者的2期临床试验，用于丛状神经纤维瘤（一种治疗选择有限的遗传病）和皮肤神经纤维瘤，目前尚无FDA批准的选择。

We are also generating important preclinical data for multiple additional compounds identified using our novel generative AI drug discovery engine. Those compounds target rare diseases with significant unmet needs, including additional nerve-related tumor disorders, autosomal dominant polycystic kidney disease and neurodevelopmental disorders such as Angelman syndrome..

我们还为使用我们的新型生成性AI药物发现引擎鉴定的多种其他化合物生成了重要的临床前数据。这些化合物针对的是需求尚未得到满足的罕见疾病，包括其他与神经相关的肿瘤疾病，常染色体显性多囊肾病和神经发育障碍，例如Angelman综合征。。

The Healx drug discovery pipeline is powered by Healnet, an AI-driven discovery platform designed to identify clinically de-risked therapeutic opportunities for rare diseases. Healnet incorporates recent advances in generative AI to find connections between biological and chemical entities that could be turned into new treatments..

Healx药物发现管道由Healnet提供支持，Healnet是一个人工智能驱动的发现平台，旨在识别罕见疾病的临床去风险治疗机会。Healnet结合了生成人工智能的最新进展，以发现生物和化学实体之间的联系，这些联系可以转化为新的治疗方法。。

Healx was co-founded in Cambridge (UK) by its chairman and Viagra co-inventor David Brown, Ph.D., and Tim Guilliams, Ph.D., with a mission to apply emerging technologies to speed the discovery of rare disease treatments.

Healx由其董事长兼伟哥联合发明人DavidBrown博士和TimGuilliams博士在英国剑桥共同创立，其使命是应用新兴技术加速罕见疾病治疗的发现。

U.S. FDA IND Clearance

U、 美国FDA IND许可

Healx also announced that it has received clearance from the U.S. Food and Drug Administration (FDA) to proceed with its Phase 2 clinical trial of HLX-1502. This trial will focus on treating adults with NF1 and inoperable plexiform neurofibroma.

Healx还宣布，它已获得美国食品和药物管理局（FDA）的批准，将继续进行HLX-1502的2期临床试验。该试验将重点治疗患有NF1和无法手术的丛状神经纤维瘤的成年人。

“This IND approval marks another significant milestone in our efforts to harness powerful AI to develop a new treatment for NF1-associated plexiform neurofibroma,” said Tim Guilliams, Ph.D., CEO of Healx. “We are committed to advancing this promising investigational treatment through clinical development and bringing it one step closer to patients in need.'.

Healx首席执行官Tim Guilliams博士说：“这次IND批准标志着我们利用强大的人工智能开发NF1相关丛状神经纤维瘤新疗法的另一个重要里程碑。”。“我们致力于通过临床开发推进这种有前途的研究性治疗，并使其更接近有需要的患者。”。

HLX-1502 is a tablet taken orally that works differently than other treatments and offers a new and differentiated investigational treatment option for patients with NF1.

HLX-1502是一种口服片剂，其作用与其他治疗方法不同，为NF1患者提供了一种新的差异化研究治疗选择。

NF1 is a rare genetic disorder associated with predisposition to develop multiple benign and malignant tumors. It affects approximately 1 in 2,500 individuals. Two notable types of tumors associated with NF1 are plexiform neurofibromas and cutaneous neurofibromas. Plexiform neurofibromas are complex tumors growing aggressively along nerves, which often leads to significant morbidity and risk of malignant transformation.

NF1是一种罕见的遗传疾病，与易患多种良性和恶性肿瘤有关。它影响大约2500个人中的1个人。与NF1相关的两种显着类型的肿瘤是丛状神经纤维瘤和皮肤神经纤维瘤。丛状神经纤维瘤是沿着神经侵袭性生长的复杂肿瘤，通常会导致严重的发病率和恶性转化风险。

These tumors can affect various parts of the body, causing functional impairments, disfigurement and pain and requiring multidisciplinary management. Currently, there is only one treatment option available for some children with plexiform neurofibromas which is, however, associated with tolerability and safety concerns including gastrointestinal, heart, eye and skin toxicity..

这些肿瘤可以影响身体的各个部位，导致功能障碍，毁容和疼痛，需要多学科管理。目前，对于一些患有丛状神经纤维瘤的儿童，只有一种治疗选择，然而，这与耐受性和安全性有关，包括胃肠道，心脏，眼睛和皮肤毒性。。

Cutaneous neurofibromas are benign tumors that often cause significant symptoms, leading to impairment in quality of life, and also result in considerable cosmetic concerns. There are no approved treatments for NF1-associated cutaneous neurofibromas, leaving a major unmet need for the estimated 3 million people with NF1 worldwide..

皮肤神经纤维瘤是良性肿瘤，通常会引起明显的症状，导致生活质量受损，并导致相当大的美容问题。与NF1相关的皮肤神经纤维瘤尚无批准的治疗方法，因此全球约有300万NF1患者的主要需求尚未得到满足。。

HLX-1502 has received Orphan Drug and Rare Pediatric Disease designations from the FDA for treating NF1. These FDA designations provide several benefits to encourage the development of treatments for rare diseases and further highlight HLX-1502’s potential to significantly improve the lives of NF1 patients..

HLX-1502已获得FDA的孤儿药和罕见儿科疾病指定，用于治疗NF1。这些FDA名称提供了一些好处，以鼓励开发罕见疾病的治疗方法，并进一步突出了HLX-1502显着改善NF1患者生活的潜力。。

“I could not be more excited to start this trial soon. Our treatment – with its novel mechanism of action and potential for a compelling and differentiated safety profile – represents a significant advancement in the NF1 field and a much needed hope for the NF1 community. It has the potential to greatly improve NF1 patients’ lives, which is by far what matters the most and what drives us,” said Simone Manso, Healx head of neurofibromatosis therapy development..

Healx神经纤维瘤病治疗开发负责人西蒙·曼索（Simone Manso）表示：“很快开始这项试验，我感到无比兴奋。我们的治疗方法——其新颖的作用机制和潜在的引人注目和差异化的安全性——代表了NF1领域的重大进步，也是NF1社区急需的希望。它有可能大大改善NF1患者的生活，这是迄今为止最重要的，也是推动我们的因素。”。。

Healx has an investment agreement with its long-term research partner, Children’s Tumor Foundation (CTF), whereby Healx will receive milestone-driven payments from CTF to support the advance of Healx’s NF programs including its lead candidate, HLX-1502. CTF has been a longtime partner of Healx and actively supports its mission to deliver much-needed therapies to this patient group..

Healx与其长期研究合作伙伴儿童肿瘤基金会（CTF）签订了投资协议，根据该协议，Healx将从CTF获得里程碑式的付款，以支持Healx NF计划的推进，包括其主要候选人HLX-1502。CTF一直是Healx的长期合作伙伴，并积极支持其为该患者群体提供急需的治疗的使命。。

About Healnet

关于Healnet

Behind advances in our therapeutic pipeline lies Healnet, a unique AI-driven drug discovery engine that is tailored to discover treatments for rare diseases. The platform uses disease multi-omics signature reversal and AI to identify novel therapeutic hits. Using Healx lab-generated phenotypic and transcriptomics profiling data, novel disease biology, mechanisms and targets are identified, which enables the design of second-generation compounds using virtual screening, predictive modeling and phenomics..

在我们的治疗流程取得进展的背后是Healnet，这是一种独特的人工智能驱动的药物发现引擎，专门用于发现罕见疾病的治疗方法。该平台使用疾病多组学特征逆转和AI来识别新的治疗命中。使用Healx实验室生成的表型和转录组学分析数据，确定了新的疾病生物学，机制和靶标，从而可以使用虚拟筛选，预测建模和表型组学设计第二代化合物。。

Healnet is now incorporating modern advances in generative AI, increasing the quantity and quality of its data sources, creating intelligent AI workflows and enabling natural language reasoning over its biological and chemical predictions. To better design optimized novel compounds for our programs, Healx will develop chemically-aware large language models (LLMs).

Healnet现在正在整合生成人工智能的现代进步，提高其数据源的数量和质量，创建智能人工智能工作流程，并实现对其生物和化学预测的自然语言推理。为了更好地为我们的程序设计优化的新型化合物，Healx将开发化学感知的大型语言模型（LLM）。

Moving beyond general text-based large language models like ChatGPT from OpenAI, Healx is exploring how models trained on raw biological data that speak the language of the cell can identify and exploit targets and result in greater understanding of rare disease biology. These technological capabilities empower Healx’s scientists to operate with enhanced expertise, efficiency and accuracy in order to uncover previously undiscoverable treatments..

除了来自OpenAI的基于文本的大型语言模型（如ChatGPT）之外，Healx正在探索如何在讲细胞语言的原始生物数据上训练模型，以识别和利用目标，从而更好地理解罕见疾病生物学。这些技术能力使Healx的科学家能够以更高的专业知识、效率和准确性进行操作，以发现以前无法发现的治疗方法。。

About Healx

关于Healx

Healx is a generative AI enabled, clinical-stage, rare disease biotech pioneering the next generation of drug discovery in order to bring novel, effective treatments to rare disease patients around the world. There are 10,000 known rare diseases that affect 400 million people across the globe, but only 5% of those conditions have an approved treatment.

Healx是一种生成性人工智能的临床阶段罕见疾病生物技术，开创了下一代药物发现的先河，旨在为世界各地的罕见疾病患者带来新颖有效的治疗方法。已知有10000种罕见疾病影响全球4亿人，但只有5%的疾病得到了批准的治疗。

By combining generative AI technology with deep drug discovery and development expertise, Healx can accelerate the pace, increase the scale and improve the chance of success of rare disease treatment development in order to deliver global patient impact. Healx exists because every rare disease patient deserves a treatment..

通过将生成性人工智能技术与深入的药物发现和开发专业知识相结合，Healx可以加快罕见病治疗开发的步伐，扩大规模，提高成功的机会，从而对全球患者产生影响。Healx的存在是因为每个罕见病患者都应该接受治疗。。

Healx was founded in Cambridge, UK, by Tim Guilliams, Ph.D., a biochemical engineer and tech entrepreneur, and David Brown, Ph.D., co-inventor of Viagra and former global head of drug discovery at Roche. Healx has raised approximately $110 million to date. For more information, visit www.healx.ai or follow us on LinkedIn or X..

Healx由生物化学工程师兼科技企业家蒂姆·吉利亚姆博士和伟哥联合发明者、罗氏公司前全球药物研发负责人大卫·布朗博士在英国剑桥创立。迄今为止，Healx已经筹集了大约1.1亿美元。。。