18-month Double-blind, Randomized, Placebo-controlled, Multicenter, Phase 3 Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease and in Late-infantile and Juvenile-onset Forms of GM1 Gangliosidosis or GM2 Gangliosidosis

An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in late-infantile and juvenile forms of Niemann-Pick type C disease and in late-infantile and juvenile-onset forms of GM1 gangliosidosis or GM2 gangliosidosis

开始日期2025-06-30

申办/合作机构

Azafaros BV

CTIS2023-510058-17-00

/ Recruiting临床1期

- AZA-001-31-03

开始日期2024-04-17

申办/合作机构

Azafaros BV

CTIS2023-504916-14-00

/ Not yet recruiting临床1期

- AZA-001-31-02

开始日期2023-07-13

申办/合作机构

Azafaros BV

100 项与 Nizubaglustat 相关的临床结果

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100 项与 Nizubaglustat 相关的转化医学

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100 项与 Nizubaglustat 相关的专利（医药）

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项与 Nizubaglustat 相关的文献（医药）

2024-01-01·Molecular genetics and metabolism

First-in-human single-dose study of nizubaglustat, a dual inhibitor of ceramide glucosyltransferase and non-lysosomal glucosylceramidase: Safety, tolerability, pharmacokinetics, and pharmacodynamics of single ascending and multiple doses in healthy adults

Article

作者: Polasek, Thomas M ; Paquet Luzy, Cecile ; Giorgino, Ruben ; Doppler, Emilie

Nizubaglustat is a novel, orally available, brain penetrant, potent, and selective dual inhibitor of ceramide glucosyltranferase and non-lysosomal neutral glucosylceramidase (NLGase), which is currently under development for the treatment of subjects with neurological manifestations in primary and secondary gangliosidoses. The objectives of this first-in-human study were to evaluate the safety and tolerability, pharmacokinetics, and pharmacodynamics (PD) of single oral doses of nizubaglustat after single (1, 3, and 9 mg) and multiple oral doses (9 mg once per day (QD) over 14 days) in healthy adults. Nizubaglustat was rapidly absorbed and systemic exposure was dose-proportional. Steady-state was achieved after three days of QD multiple dosing with minimal accumulation. Renal clearance accounted for around 15% of nizubaglustat elimination. Following multiple dosing, plasma concentrations of glucosylceramide (GlcCer), lactosylceramide (LacCer), and monosialodihexosylganglioside (GM3) decreased to a nadir at Day 10. PD target engagement of GCS inhibition was shown by a median decrease from baseline of plasma concentrations of GlcCer, LacCer, and GM3 ganglioside by 70%, 50%, and 48%, respectively. NLGase inhibition was also manifested by increased concentrations of GlcCer in cerebrospinal fluid from Day 1 to Day 14. Nizubaglustat was safe and well-tolerated at all doses tested. Consistent with the high selectivity, and the absence of intestinal disaccharidases inhibition, no cases of diarrhea were reported. No decreased appetite or weight loss was noted. Only treatment-emergent adverse events with preferred terms belonging to the system organ class skin and subcutaneous disorders of mild intensity were reported as drug-related in the nizubaglustat arm, in line with the pharmacological mechanism targeting glucosylceramide metabolism. Taken together, these data support QD dosing of nizubaglustat and its ongoing development in patients with primary and secondary forms of gangliosidoses.

2017-10-11·Journal of the American Chemical Society1区 · 化学

A Fluorescence Polarization Activity-Based Protein Profiling Assay in the Discovery of Potent, Selective Inhibitors for Human Nonlysosomal Glucosylceramidase

1区 · 化学

Article

作者: Breen, Imogen ; van den Berg, Richard J. B. H. N. ; Boot, Rolf G. ; Kuo, Chi-Lin ; Aerts, Johannes M. F. G. ; Wu, Liang ; van der Stelt, Mario ; Geurink, Paul P. ; Ghisaidoobe, Amar T. ; van den Nieuwendijk, Adrianus M. C. H. ; Liu, Bing ; Overkleeft, Herman S. ; Lahav, Daniel ; Davies, Gideon J. ; Ovaa, Huib ; Ferraz, Maria J. ; Wennekes, Tom ; van der Marel, Gijsbert A.

Human nonlysosomal glucosylceramidase (GBA2) is one of several enzymes that controls levels of glycolipids and whose activity is linked to several human disease states. There is a major need to design or discover selective GBA2 inhibitors both as chemical tools and as potential therapeutic agents. Here, we describe the development of a fluorescence polarization activity-based protein profiling (FluoPol-ABPP) assay for the rapid identification, from a 350+ library of iminosugars, of GBA2 inhibitors. A focused library is generated based on leads from the FluoPol-ABPP screen and assessed on GBA2 selectivity offset against the other glucosylceramide metabolizing enzymes, glucosylceramide synthase (GCS), lysosomal glucosylceramidase (GBA), and the cytosolic retaining β-glucosidase, GBA3. Our work, yielding potent and selective GBA2 inhibitors, also provides a roadmap for the development of high-throughput assays for identifying retaining glycosidase inhibitors by FluoPol-ABPP on cell extracts containing recombinant, overexpressed glycosidase as the easily accessible enzyme source.

2014-11-13·Journal of medicinal chemistry1区 · 医学

Identification and Development of Biphenyl Substituted Iminosugars as Improved Dual Glucosylceramide Synthase/Neutral Glucosylceramidase Inhibitors

1区 · 医学

Article

作者: Aerts, Johannes M. F. G. ; van den Berg, Richard J. B. H. N. ; van der Stelt, Mario ; Donker-Koopman, Wilma E. ; van den Nieuwendijk, Adrianus M. C. H. ; Strijland, Anneke ; Koomen, Gerrit-Jan ; Overkleeft, Herman S. ; Ghisaidoobe, Amar T. ; Wennekes, Tom ; Butt, Saleem S. ; van Boeckel, Constant A. A. ; van Loevezijn, Arnold ; Leemhuis, Mark ; Scheij, Saskia ; van der Marel, Gijsbert A.

This work details the evaluation of a number of N-alkylated deoxynojirimycin derivatives on their merits as dual glucosylceramide synthase/neutral glucosylceramidase inhibitors. Building on our previous work, we synthesized a series of D-gluco and L-ido-configured iminosugars N-modified with a variety of hydrophobic functional groups. We found that iminosugars featuring N-pentyloxymethylaryl substituents are considerably more potent inhibitors of glucosylceramide synthase than their aliphatic counterparts. In a next optimization round, we explored a series of biphenyl-substituted iminosugars of both configurations (D-gluco and L-ido) with the aim to introduce structural features known to confer metabolic stability to drug-like molecules. From these series, two sets of molecules emerge as lead series for further profiling. Biphenyl-substituted L-ido-configured deoxynojirimycin derivatives are selective for glucosylceramidase and the nonlysosomal glucosylceramidase, and we consider these as leads for the treatment of neuropathological lysosomal storage disorders. Their D-gluco-counterparts are also potent inhibitors of intestinal glycosidases, and because of this characteristic, we regard these as the prime candidates for type 2 diabetes therapeutics.

项与 Nizubaglustat 相关的新闻（医药）

2025-07-04

·动脉网

B轮融资1.32亿欧元，这款CNS小分子药如何挺进全球Ⅲ期临床

2025年5月，专注于罕见遗传代谢病的欧洲生物技术公司Azafaros成为行业焦点：公司宣布完成1.32亿欧元B轮融资，由Jeito Capital领投，Forbion Growth联合领投，Seroba、Pictet Group和其他现有投资者参投。此轮融资将主要用于推进其核心候选药物nizubaglustat（AZ-3102）进入全球Ⅲ期临床试验阶段，为LSDs（Lysosomal Storage Disorders，溶酶体贮积症）患者带来治疗曙光。虽为罕见病，但每2315-7000名新生儿中就有1例LSDs患者[1]，这些患儿大多在童年期就会出现不可逆的神经损伤。面对这一医学难题，传统疗法为何屡屡碰壁？Azafaros的创新药物又将如何破局？01科学家引领，行业老将护航，Azafaros点亮LSDs治疗灯塔成立于2018年的Azafaros致力于将基础研究成果转化为临床可及的新疗法，其基础研究成果源于荷兰莱顿大学与阿姆斯特丹大学医学中心在罕见遗传代谢病领域多年的科研积累。Azafaros总部设于荷兰莱顿，聚焦开发具备CNS（Central Nervous System，中枢神经系统）活性的口服小分子药物，靶向三种具有高度致残性与致死性的LSDs：GM1神经节苷脂病（GM1 gangliosidosis）、GM2神经节苷脂病（GM2 gangliosidosis）和NPC病（Niemann-PickTypeC，尼曼-匹克C型）。公司名称“Azafaros”蕴含深意：“Aza”代表其候选药物核心结构——氮杂环（azacycle），而“faro”在希腊语中意为“灯塔”，象征着希望与方向。正如Azafaros愿景所表达的那样：希望成为“严重罕见代谢紊乱患者的灯塔”，在黑暗中点亮罕见病治疗新路径，为患者寻找治疗的新可能。团队方面，Azafaros由一支兼具深厚科研背景与产业化能力的管理队伍组成。现任CEO Carlo Incerti博士拥有超过30年生物医药行业经验，曾在罕见病领域领军企业Genzyme Corporation担任高管，为公司注入全球商业化经验。首席科学家Kyle Landskroner博士是早期药物发现和开发方面经验丰富的专家，为Azafaros带来了丰富的药物开发知识。医疗行业老将护航临床转化，科学家赋能产业，Azafaros在罕见病赛道中开辟出一条临床可行、资本认可、技术独特的路径。图1：Azafaros现任CEO及首席科学家画像简介02可穿越血脑屏障、双重机制的口服小分子管线在LSDs治疗领域，血脑屏障长期以来都是一座难以逾越的“堡垒”。以GM1、GM2神经节苷脂病及NPC为例，虽然外周症状可通过ERT（酶替代疗法，Enzyme Replacement Therapy）部分缓解，但由于外源性酶无法穿透血脑屏障，中枢神经系统病变始终难以控制。此外，ERT通常需频繁静脉输注，费用高昂、依从性差，令多数患者望而却步。SRT（底物还原疗法，Substrate Reduction Therapy）虽提供了口服替代路径，但多数药物靶点单一、疗效有限，难以根本改变疾病进程。在这一背景下，Azafaros开发的nizubaglustat作为一款具备中枢渗透性的小分子药物，开辟了新的治疗思路。它具备“双重机制”：一方面抑制葡萄糖脑苷脂合酶（GCS），另一方面靶向非溶酶体葡萄糖脑苷脂酶（GBA2），双管齐下调控神经节苷脂（GSLs）代谢，减少病理性脂质在脑组织和外周系统中的堆积。更重要的是，nizubaglustat为口服制剂，可在居家条件下每日服用，大幅提升依从性，特别适用于需长期照护的儿童患者。在临床层面，根据Azafaros披露的Ⅱ期RAINBOW研究数据表明，nizubaglustat具有积极的安全性，并且在该研究的参与者中耐受性良好。在监管层面，nizubaglustat已获得FDA和EMA针对GM1、GM2及NPC的孤儿药资格，并获得FDA快速通道认证。作为一款兼具脑渗透性、口服优势与双靶点机制的候选药物，nizubaglustat有望突破当前治疗瓶颈，开启LSDs治疗的新范式。03从认知疾病到验证疗效：nizubaglustat的全周期临床布局围绕其核心候选小分子药物nizubaglustat，Azafaros构建了一条以认知疾病机制为起点、逐步过渡至安全性与疗效验证的“渐进式”临床路径，先后推进PRONTO、Ⅱ期RAINBOW和NAVIGATE三项研究，为神经系统罕见病患儿的治疗奠定坚实的临床基础。表1：Azafaros开展的三项研究具体信息● PRONTO：疾病自然史研究，奠定疗效评估基线作为临床开发的起点，PRONTO研究聚焦晚婴儿期与青少年期发病的GM1和GM2神经节苷脂病患者，系统评估疾病在神经系统中的自然进展过程。研究纳入年龄在2至20岁之间的遗传确诊患者，采用多维度评估方案：医师使用评定量表进行专业评估；照护者通过问卷提供日常观察，同时借助医疗设备监测，实现全方位数据采集。研究目标是识别可量化的临床终点、理解疾病异质性，并为后续注册试验提供疾病自然史对照依据。该研究于2022年启动，预计将于2025年完成主要随访。● RAINBOW：Ⅱ期短期研究，初步验证剂量、安全性与药效紧随其后，Azafaros于2023年开展了RAINBOW研究，该研究为期12周，纳入年龄≥12岁的晚发型患者，采用随机、双盲、安慰剂对照设计，分别接受高低剂量药物治疗。所有受试者随后进入为期52周的开放标签扩展期。研究显示，nizubaglustat可良好穿越血脑屏障，初步展现降低脑脊液糖脂水平的潜力，未见严重不良反应，为剂量选择与长期治疗提供了依据。图2：RAINBOW研究设计思路● NAVIGATE：即将开展的Ⅲ期注册研究，验证长期疗效作为整个开发路径的关键一环，Azafaros将于近期在全球约15个国家设立35个中心，并同步开展研究工作。NAVIGATE是一项针对晚婴儿期与青少年型GM1、GM2和NPC的Ⅲ期双盲随机对照研究，聚焦nizubaglustat在18个月持续治疗后对共济失调及其他神经表现的改善效果，同时将系统评估其药代动力学、药效学、安全性与耐受性。三项研究虽启动时间不同、作用各异，却形成了从疾病理解到长期疗效评估的临床接力，体现了Azafaros在中枢神经系统罕见病领域由浅入深、风险可控的开发策略。04从科研孵化到Ⅲ期冲刺：Azafaros的成长全纪实Azafaros的成长路径，是一条典型的“早研-临床-资本”融合转化范式。2018年，Azafaros获得一项关键小分子化合物的全球独家开发权——这正是后来成为明星候选药物的nizubaglustat。凭借该分子在LSDs中的广泛潜力，Azafaros吸引了早期风投关注，并迅速完成种子轮融资，正式启动药物开发征程。2020年，Azafaros获得Forbion领投的2500万欧元A轮融资，加速推进nizubaglustat的临床前开发与初步人体研究。2021年，该药物进入I期临床试验，聚焦剂量耐受性与安全性评估。2023年，Azafaros正式启动Ⅱ期RAINBOW研究。2024年，该研究数据显示药物具备良好的中枢神经系统穿透性。2025年5月，Azafaros宣布完成1.32亿欧元B轮融资，该轮融资将直接用于推动Ⅲ期全球注册试验，标志着Azafaros正式迈入后期临床阶段。从一枚学术化合物出发，Azafaros完成了从早期探索、临床验证到资本放大的“三级跳”。这一进程不仅验证了其技术路径的临床可行性，也将其创新药物进一步推向真正服务患者的落地阶段。表2：从实验室走向全球临床：Azafaros发展里程碑（信息来源：Azafaros官网）05从技术创新到生态协同，可复制的Biotech进阶路径一方面，Azafaros敏锐抓住大分子药物难以穿透血脑屏障的技术瓶颈，选择口服小分子策略切入CNS罕见病，为患者提供更可及的治疗方案。对中国药企而言，这提示我们在布局新药时，不应盲目“高大全”，而要立足患者真实需求，在靶点可及性与给药便利性之间寻找突破。另一方面，借助资本与临床的“双轮驱动”，搭建起生态共建的协同效益。Azafaros与莱顿大学不仅是成果对接，更通过共同开发、共享资源，建立起持续迭代的创新机制。同时，在早期即引入国际风险投资与临床专家，使其能高效推进从临床试验到监管沟通的全链条流程。药物创新的核心不在于“多做什么”，而在于“做对什么”。对中国药企而言，真正的“借鉴”不是复制模式，而是从其选择中看到逻辑，从其成功中提炼路径。Azafaros的故事，从“昂贵、注射依赖、通路稀缺”向“可口服、跨血脑屏障、普适性更强”演进。在未来，真正能撬动罕见病治疗格局的，或许并非体量庞大、路径保守的巨型平台，而是那些精准切入未被满足需求、又具备跨学科整合能力的“Azafaros式”公司——它们小而锐、深而专，如灯塔般矗立在临床未解的黑暗之地。参考文献：[1] Paquet Luzy, Cecile et al. “First-in-human single-dose study of nizubaglustat, a dual inhibitor of ceramide glucosyltransferase and non-lysosomal glucosylceramidase: Safety, tolerability, pharmacokinetics, and pharmacodynamics of single ascending and multiple doses in healthy adults.” Molecular genetics and metabolism vol. 141,1 (2024): 108113. doi:10.1016/j.ymgme.2023.108113如果您想对接文章中提到的项目，或您的项目想被动脉网报道，或者发布融资新闻，请与我们联系；也可加入动脉网行业社群，结交更多志同道合的好友。近期推荐声明：动脉网所刊载内容之知识产权为动脉网及相关权利人专属所有或持有。未经许可，禁止进行转载、摘编、复制及建立镜像等任何使用。文中如果涉及企业信息和数据，均由受访者向分析师提供并确认。动脉网，未来医疗服务平台

临床3期

2025-06-09

·azafaros.com

Azafaros to Present at BIO International Convention 2025 Following Successful €132M Series B Financing

Leiden, the Netherlands – 09 June 2025 – Azafaros, a clinical-stage biotechnology company developing novel therapies for rare lysosomal storage disorders (LSDs), today announced that it will present at the 2025 BIO International Convention taking place in Boston, USA, from 16-19 June. The company’s presentation is scheduled for Monday, June 16 at 16:45 ET in Room 153B. The presentation will highlight Azafaros’ progress in developing nizubaglustat, its lead investigational compound for the treatment of rare lysosomal storage disorders with neurological involvement, including GM1 and GM2 gangliosidoses and Niemann-Pick disease type C (NPC). This announcement follows Azafaros’ recently completed and oversubscribed €132 million Series B financing round, which will support advancing nizubaglustat into pivotal clinical development. The company is on track to initiate two Phase 3 trials in GM1/GM2 gangliosidoses and NPC in 2025. Earlier this year, Azafaros reported positive topline results from its ongoing Phase 2 study evaluating nizubaglustat in patients with GM2 gangliosidosis and NPC. The current Ph2 extension study demonstrated a favorable safety profile after more than 12 months of treatment. In addition, preliminary improvements or stabilization in key clinical endpoints were observed in the majority of patients, underscoring promising efficacy trends for nizubaglustat. “We are excited to share our latest progress at BIO 2025, including the continued clinical development of nizubaglustat which holds strong potential as a meaningful treatment option for patients with devastating neurodegenerative lysosomal disorders,” said Stefano Portolano, CEO at Azafaros. “With the support of our recent financing round and encouraging data from our ongoing trials, we can continue our rapid momentum to bring this product into pivotal clinical studies as we work to develop a potential new solution for this area where there is a significant unmet need.” About nizubaglustat Nizubaglustat is a small molecule, orally available and brain penetrant azasugar with a unique dual mode of action, developed as a potential treatment for rare lysosomal storage disorders with neurological involvement, including GM1 and GM2 gangliosidoses and Niemann-Pick disease type C (NPC). Nizubaglustat has received Rare Paediatric Disease Designations (RPDD) for the treatment of GM1 and GM2 gangliosidoses and NPC, Orphan Drug Designations (ODD) for GM1 and GM2 gangliosidosis (Sandhoff and Tay-Sachs Diseases) and NPC, as well as Fast Track Designation and IND clearance for GM1/GM2 gangliosidoses and NPC from the US Food and Drug Administration (FDA). Additionally, nizubaglustat has been awarded Orphan Medicinal Product Designation (OMPD) for the treatment of GM1 and GM2 gangliosidoses by the European Medicines Agency (EMA) and Innovation Passport for the treatment of GM1 and GM2 gangliosidoses from the UK Medicines and Healthcare Products Regulatory Agency (MHRA). About GM1 and GM2 gangliosidoses GM1 gangliosidosis and GM2 gangliosidosis (Tay-Sachs and Sandhoff diseases) are lysosomal storage disorders caused by the accumulation of GM1 or GM2 gangliosides respectively, in the central nervous system (CNS). This results in progressive and severe neurological impairment and premature death. These diseases mostly affect infants and children, and no disease-modifying treatments are currently available. About Niemann-Pick disease Type C (NPC) Niemann-Pick disease Type C is a progressive, life-limiting neurological lysosomal storage disorder caused by mutations in the NPC1 or NPC2 gene and aberrant endosomal-lysosomal trafficking, leading to the accumulation of various lipids, including gangliosides in the CNS. The onset of disease can happen throughout the lifespan of an affected individual, from prenatal life through adulthood. About Azafaros Azafaros is a clinical-stage company founded in 2018 with a deep understanding of rare genetic disease mechanisms using compound discoveries made by scientists at Leiden University and Amsterdam UMC and is led by a team of highly experienced industry experts. Azafaros aims to build a pipeline of disease-modifying therapeutics to offer new treatment options to patients and their families. By applying its knowledge, network and courage, the Azafaros team challenges traditional development pathways to rapidly bring new drugs to the rare disease patients who need them. Azafaros is supported by leading healthcare investors including Jeito Capital, Forbion Growth, Seroba, Pictet Group and a syndicate of leading Dutch and Swiss existing investors including Forbion Ventures, BioGeneration Ventures (BGV), BioMedPartners, Asahi Kasei Pharma Ventures, and Schroders Capital. For further information: Azafaros B.V. Email: info@azafaros.com www.azafaros.com

孤儿药临床2期临床结果快速通道

2025-05-19

·创鉴汇

20家公司获融资！1.32亿欧元支持口服小分子新锐 | 一周融资

据创鉴汇不完全统计，上周（5月12日至5月18日）全球大健康领域共披露融资事件20起，总额超9.5亿美元。从融资轮次来看，35%为早期融资（B轮以前）。Pathos以3.65亿美元D轮融资登顶，Azafaros、Stylus Medicine、Alpheus Medical及Cohere Health的单笔融资超5000万美元。这20家公司中，“AI+生物技术”覆盖小分子抑制剂设计（Pathos）、细胞疗法开发（Somite AI）及计算生物学平台（Pluto Bio）；CGT赛道迭代加速，体内CAR-T（Stylus Medicine）与溶瘤腺病毒疗法（TILT Biotherapeutics）竞逐实体瘤治疗；其他创新药如核素偶联药物（LinqMed）、纤维蛋白靶向抗体（Therini Bio）等拓宽疾病干预边界。#01Pathos AI完成3.65亿美元D轮融资关键词：AI药物最新融资：3.65亿美元D轮5月15日，专注于肿瘤药物研发的生物技术公司Pathos AI宣布完成3.65亿美元D轮融资。本轮资金将用于推进其临床阶段管线及专有多模态数据基础模型的开发。Pathos AI致力于通过整合临床、分子与影像等多维度数据，构建计算驱动的药物开发平台加速肿瘤治疗领域的研发进程。公司核心平台基于多模态数据基础模型，旨在优化临床资产筛选、试验设计及生物标志物发现等关键环节。该模型通过融合大规模临床数据集与分子机制研究，辅助提升药物开发效率。目前，Pathos AI的研发管线聚焦于肿瘤治疗领域，多个临床阶段项目正在推进中。#02Azafaros完成1.32亿欧元B轮融资关键词：口服小分子药物最新融资：1.32亿欧元B轮本轮投资机构：BioGeneration Ventures、Forbion、Jeito Capital、Pictet group、Schroders Capital、Seroba5月13日，Azafaros宣布完成1.32亿欧元B轮融资，将推进其核心管线药物nizubaglustat针对尼曼匹克病C型（NPC）及GM1/GM2神经节苷脂贮积症的两项3期临床研究，并扩展管线至其他适应症。Azafaros成立于2018年，专注于开发罕见溶酶体贮积症的疾病修饰疗法。溶酶体贮积症是一组严重的罕见遗传病，通常会导致进行性神经退行性变。Azafaros的核心候选药物nizubaglustat为口服小分子药物，可穿透血脑屏障，通过双重作用机制调节溶酶体功能，靶向干预脂质代谢异常导致的神经损伤。#03Stylus Medicine正式亮相，获8500万美元A轮融资关键词：体内基因疗法最新融资：8500万美元A轮本轮投资机构：Chugai Venture Fund 、礼来、Khosla Ventures、RA Capital Management、强生创新5月14日，基因疗法公司Stylus Medicine宣布完成8500万美元A融资，将推进其体内基因药物平台开发及管线拓展，重点聚焦体内CAR-T疗法研发。Stylus Medicine专注于利用非病毒递送技术开发精准体内基因疗法。公司核心技术平台将序列特异性基因组整合与细胞靶向脂质纳米粒子（LNP）递送相结合，基于Stylus设计的治疗级重组酶，能够高效、高特异性和高保真地编码多功能多kb有效载荷。该技术旨在实现治疗基因的持久表达与规模化应用。目前，公司研发技术在在肿瘤学、自身免疫、遗传疾病等领域具有潜在的治疗应用。#04Therini Bio获3900万美元A轮融资关键词：单克隆抗体最新融资：3900万美元A轮本轮投资机构：Angelini Ventures、Apollo Health Ventures、礼来、Dementia Discovery Fund、Dolby Family Ventures、赛诺菲风险投资公司等5月14日， Therini Bio宣布完成3900万美元A轮融资，A轮总额达7500万美元。资金将用于推进核心管线药物THN391治疗阿尔茨海默病（AD）及糖尿病黄斑水肿（DME）的1b期临床试验，并支持纤维蛋白/VEGF双特异性抗体的开发。Therini Bio专注于开发靶向纤维蛋白的免疫疗法，聚焦于纤维蛋白介导的神经炎症机制，通过特异性结合纤维蛋白的炎症表位，阻断慢性神经炎症导致的神经元损伤。公司核心候选药物THN391为高亲和力人源化单抗，可选择性抑制纤维蛋白引发的神经炎症而不影响凝血功能。#05TILT Biotherapeutics获2560万美元B轮融资关键词：溶瘤腺病毒免疫疗法最新融资：2560万美元B轮本轮投资机构：Finnish Industry Investment、Lifeline Ventures、Stephen Industries Inc Oy、欧洲创新委员会基金5月13日， TILT Biotherapeutics宣布完成2560万美元（2260万欧元）B轮融资，将推进其核心产品TILT-123在铂类耐药上皮性卵巢癌的2期临床试验，以及联合TILs治疗黑色素瘤的的1b期临床试验。TILT Biotherapeutics专注于溶瘤腺病毒免疫疗法，该腺病毒配备了包括细胞因子在内的分子，可以激活 T 细胞并破坏癌细胞。其专利TILT技术可以通过静脉内、局部或瘤内给药。它改变了肿瘤微环境，通过使“冷”肿瘤变“热”，消除癌症逃避免疫反应的能力，从而增强免疫检查点抑制剂及T细胞疗法的抗肿瘤效应。公司核心管线TILT-123为搭载肿瘤坏死因子（TNF）和白细胞介素-2（IL-2）的溶瘤腺病毒。读者们请星标⭐创鉴汇，第一时间收到推送免责声明：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：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

细胞疗法基因疗法免疫疗法临床3期引进/卖出

100 项与 Nizubaglustat 相关的药物交易

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研发状态

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适应症	最高研发状态	国家/地区	公司	日期
GM1神经节苷脂贮积病	临床3期	美国	Azafaros BV	2025-06-30
GM1神经节苷脂贮积病	临床3期	阿根廷	Azafaros BV	2025-06-30
GM1神经节苷脂贮积病	临床3期	澳大利亚	Azafaros BV	2025-06-30
GM1神经节苷脂贮积病	临床3期	巴西	Azafaros BV	2025-06-30
GM1神经节苷脂贮积病	临床3期	加拿大	Azafaros BV	2025-06-30
GM1神经节苷脂贮积病	临床3期	法国	Azafaros BV	2025-06-30
GM1神经节苷脂贮积病	临床3期	印度	Azafaros BV	2025-06-30
GM1神经节苷脂贮积病	临床3期	意大利	Azafaros BV	2025-06-30
GM1神经节苷脂贮积病	临床3期	葡萄牙	Azafaros BV	2025-06-30
GM1神经节苷脂贮积病	临床3期	西班牙	Azafaros BV	2025-06-30

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适应症

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT05758922 (RAINBOW, BusinessWire) 人工标引	临床2期	C型尼曼匹克病 \| GM2神经节苷脂贮积症	13	Nizubaglustat	繭壓繭願襯餘鹹觸餘膚(範觸積鬱觸餘壓醖憲醖) = had a positive safety profile and was well-tolerated in the 13 participants in the study. 遞蓋夢鬱鏇鬱積積獵鹹 (構憲願夢繭淵壓選齋廠 ) 更多	积极	2024-09-10
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SSIEM2022	临床1期	山德霍夫病	-	AZ-3102	醖選衊壓壓範鏇糧壓餘(觸網鬱鏇製積艱齋願鹹) = 蓋觸窪鹹簾糧觸願構觸襯鏇艱鹽鏇繭淵製繭鹹 (醖廠鏇鏇鏇製構選餘衊 )	-	2022-08-30
				Vehicle	醖選衊壓壓範鏇糧壓餘(觸網鬱鏇製積艱齋願鹹) = 醖積築範顧顧窪構簾衊襯鏇艱鹽鏇繭淵製繭鹹 (醖廠鏇鏇鏇製構選餘衊 )