排名前五的靶点	数量

AGT(血管紧张素原)	2
17β-HSD13(17β-羟基类固醇脱氢酶13)	2
APOC3(载脂蛋白C-Ⅲ)	2
PCSK9(前蛋白转化酶枯草杆菌蛋白酶Kexin-9)	1
ANGPTL3(血管生成素样蛋白-3)	1

关联

项与苏州瑞博生物技术股份有限公司相关的药物

Cosdosiran

靶点

caspase 2

作用机制

caspase 2抑制剂 [+2]

在研机构

原研机构

在研适应症

非在研适应症

最高研发阶段临床3期

首次获批国家/地区-

首次获批日期-

RBD-5044

靶点

APOC3

作用机制

APOC3抑制剂

在研机构

原研机构

在研适应症

非在研适应症

最高研发阶段临床2期

首次获批国家/地区-

首次获批日期-

SR-059

靶点

F11

作用机制

F11抑制剂

在研机构

苏州瑞博生物技术股份有限公司 [+1]

原研机构

苏州瑞博生物技术股份有限公司

在研适应症

稳定型心绞痛 [+5]

非在研适应症-

最高研发阶段临床2期

首次获批国家/地区-

首次获批日期-

项与苏州瑞博生物技术股份有限公司相关的临床试验

NCT07042594

/ Not yet recruiting临床1期

A Randomized, Single-blind, Placebo-Controlled Phase I Trial to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single Ascending Doses of Subcutaneously Administered RBD1119 in Healthy Participants

This is a Randomized, Single-blind, Placebo-Controlled Phase I Trial to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single Ascending Doses of Subcutaneously Administered RBD1119 in Healthy Participants.

开始日期2025-08-01

申办/合作机构

苏州瑞博生物技术股份有限公司

NCT06797401

/ Recruiting临床2期

A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Phase 2 Trial Evaluating the Efficacy and Safety of RBD5044 in Patients With Mixed Dyslipidemia

The goal of this clinical trial is to learn if drug RBD5044 works to treat mixed dyslipidemia in adults. It will also learn about the safety of drug RBD5044. The main questions it aims to answer are:
Does drug RBD5044 reduce the triglyceride levels? What medical problems may participants experience when taking drug RBD5044? Researchers will compare drug RBD5044 to a placebo to see if drug RBD5044 works to treat mixed dyslipidemia.
Participants will:
Receive RBD5044 or placebo twice during the trial (Day 1 and Day 84). Visit the clinic 11 times during 12 months (visit every 4-8 weeks) for checkups and tests.

开始日期2025-01-28

申办/合作机构

Ribocure Pharmaceuticals AB

CTIS2023-510369-92-00

/ Recruiting临床2期

A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Phase 2 Trial Evaluating the Efficacy and Safety of RBD5044 in Patients with Mixed Dyslipidemia - RC02T001

开始日期2025-01-28

申办/合作机构

Ribocure Pharmaceuticals AB

100 项与苏州瑞博生物技术股份有限公司相关的临床结果

登录后查看更多信息

0 项与苏州瑞博生物技术股份有限公司相关的专利（医药）

登录后查看更多信息

项与苏州瑞博生物技术股份有限公司相关的文献（医药）

2025-09-01·Molecular Therapy-Nucleic Acids

A potent GalNAc-siRNA drug, RBD1016, leads to sustained HBsAg reduction and seroconversion in mouse models of HBV infection

Article

作者: Gao, Shan ; Zheng, Shuquan ; Gan, Li-Ming ; Sun, Qi ; Guo, Chun ; Yan, Xu ; Liang, Zicai ; Zhang, Min ; Zhang, Hongyan ; Cao, Huiqing

Potent small interfering RNA (siRNA) drugs that could achieve hepatitis B surface antigen (HBsAg) seroconversion and functional cure are currently lacking. The purpose of this study is to evaluate the therapeutic potential of RBD1016, a novel GalNAc-conjugated siRNA drug targeting transcripts of hepatitis B virus (HBV), in animal models. RBD1016 was designed to target X gene of HBV (HBx) and showed strong activities on HBV DNA and HBsAg for multiple HBV genotypes and nucleoside-resistant viral strains in in vitro studies. Through single and repeated administration of RBD1016 in two different HBV animal models, a robust reduction of both serum HBsAg and HBV DNA (maximum reduction 3.7 log) was demonstrated. The durable anti-HBV effects of RBD1016 could still be observed on day 169. Importantly, repeated or single administration of RBD1016 caused de novo production of hepatitis B surface antibody (HBsAb), i.e., seroconversion in animals, which is an important hallmark of immune response induction. Furthermore, the combination of RBD1016 and entecavir (ETV) demonstrated an enhanced effect on serum HBV DNA inhibition. A strong and durable anti-HBV effect of RBD1016, both alone and in combination with nucleoside analogues (NAs), indicates significant therapeutic potential for chronic hepatitis B (CHB) patients.

2025-06-02·ORAL DISEASES

Chinese Expert Consensus on Management of Betel-Chewing-Related Oral Squamous Cell Carcinoma.

Review

作者: Liu, Binjie ; Tang, Zhangui ; Peng, Xin ; Xu, Pu ; Ren, Guoxin ; Meng, Jian ; Xing, Lu ; Bu, Rongfa ; Li, Longjiang ; Guo, Wei ; Sun, Moyi ; Gao, Shan ; Hu, Yanjia ; Shang, Wei ; Bin, Xin ; Li, Bo

OBJECTIVE:

Betel-chewing-related oral squamous cell carcinoma (BCR-OSCC) has become a global health issue with increasing incidence year by year around the world. Active prevention of the occurrence of BCR-OSCC, monitoring the population exposed to Betel Nuts, and early diagnosis and treatment are very important to maintain and improve the quality of life of patients. However, there is currently no consensus or guideline that provides targeted guidance on the management of BCR-OSCC.

SUBJECTS AND METHODS:

A consensus panel consisting of 15 leading Chinese experts from multidisciplinary fields was convened, and a roundtable meeting was held to discuss the topics of BCR-OSCC.

RESULTS:

Based on existing research reports and the experts' clinical experiences, a consensus on staging, diagnosis, and treatment for BCR-OSCC was formed through extensive discussion.

CONCLUSION:

This manuscript presents consensus recommendations and a summary of evidence supporting each recommendation. This consensus may improve clinical practices about BCR-OSCC in China and propel more clinical trials to provide high-level evidence for BCR-OSCC management.

2025-06-01·CLINICAL PHARMACOKINETICS

Prediction of Interspecies Translation for Targeting Delivery Coefficients of GalNAc-siRNA Silencing Apolipoprotein C-III Using a Mechanistic Minimal Physiologically Based Pharmacokinetic/Pharmacodynamic Model

Article

作者: Gao, Shan ; Yang, Zhenzhen ; Tian, Zhiteng ; Chu, Yantao ; Liu, Yanhong ; Luo, Hui ; Song, Ling ; Liu, Dongyang

BACKGROUND AND OBJECTIVE:

The emerging N-acetylgalactosamine-small interfering RNA (GalNAc-siRNA) conjugates lead the way for liver-targeting delivery to exert gene-silencing therapeutic effects. To facilitate the drug development of GalNAc-siRNA, further detailed understanding of the key modality-specific mechanisms underlying the temporal discordance between pharmacokinetics and pharmacodynamics and how these processes can be extrapolated from animals to humans is needed.

METHODS:

A mechanistic minimal physiologically based pharmacokinetic/pharmacodynamic (mPBPK-PD) model for an investigational new apolipoprotein C-III (APOC3)-silencing GalNAc-siRNA (RBD5044) was developed using available pharmacokinetic/pharmacodynamic (PK/PD) data. The aim was to explore hepatic-targeting delivery processes, the PK/PD relationship, and interspecies translation.

RESULTS:

First, multiple PK/PD datasets from mice were satisfactorily fitted using the mPBPK-PD model. Second, we translated the mice model to the monkey model, validated it, and then extrapolated from mice and monkeys to humans to simulate the PK/PD characteristics. We then mechanistically summarized and proposed the essential in vivo delivery processes of GalNAc-siRNA after subcutaneous administration (termed "ADUEB": Absorption [into system circulation], Disposition [distribution to liver target and elimination], Uptake [into hepatocytes], Escape [from endosome and lysosome compartments], and Binding [with argonaute2 to form RNA-induced silencing complex]). The targeting delivery coefficients of these processes achieved with the model using RBD5044 and the published data of another GalNAc-siRNA (fitusiran) quantitatively reflected the delivery efficiency and rate-limiting factors in targeted hepatocytes.

CONCLUSION:

This study successfully constructed the mPBPK-PD model and conducted interspecies extrapolation for a GalNAc-siRNA targeting APOC3. Promising quantitative insights into a hepatic-targeted GalNAc-siRNA delivery system are provided to characterize the unique temporal disconnection of PK/PD properties and evaluate the key in vivo delivery processes. It will promote model-informed strategies and quantitative mechanistic understanding to support efficient drug development, evaluation, and clinical application of this modality in the future.

694

项与苏州瑞博生物技术股份有限公司相关的新闻（医药）

2025-08-14

·同写意

易慕峰实体瘤CAR-T发表最新研究成果 | 会员动态

免疫疗法细胞疗法临床1期

2025-08-14

·同写意

瀚枢生物：提供一站式神经系统药物研发解决方案 | 写意会员

临床申请

2025-08-13

·CPHI制药在线

First-in-class突破！siRNA疗法半年一针，为心血管健康护航

关注并星标CPHI制药在线 *展会预登记火热进行中，👆扫描二维码免费领票！心血管疾病是全球健康的“头号威胁”之一。高胆固醇（例如低密度脂蛋白胆固醇LDL-C）是诱发动脉粥样硬化的重要因素。传统治疗中，他汀类药物需每日服用，患者因依从性差难以达标。 2025年7月31日，诺华宣布其siRNA（小干扰RNA）疗法inclisiran（商品名：Leqvio®）获FDA批准为一线单药，每年仅需注射两次即可降低成人高胆固醇血症患者的LDL-C水平。一、Leqvio的突破性 Leqvio是一款“first-in-class”、靶向PCSK9 mRNA的siRNA疗法。2021年首次获批时，ORION系列试验（ORION-9、-10、-11）显示，Leqvio较安慰剂持续降低LDL-C达52%，参与试验的3400余名动脉粥样硬化性心血管疾病（ASCVD）或杂合子家族性高胆固醇（HeFH）患者的数据夯实了其疗效。2023年适应症扩展后，其可作为饮食和他汀类药物治疗的辅助用药。今年7月的这次适应症扩展，是FDA主动要求更新Leqvio药品标签，更新后的药品标签去除了Leqvio必须与他汀类药物联合使用或在其基础上使用的限制。同样，本次适应症拓展也源于扎实的临床数据。 2024年公布的V-MONO三期研究数据更具说服力：对未接受降脂治疗的低或中风险ASCVD患者，Leqvio单药疗效优于安慰剂和依折麦布（ezetimibe），LDL-C降低幅度具有临床和统计学意义。这为其成为单药疗法提供了关键依据。此外，“半年一针”的给药方式是Leqvio的核心优势。不同于每日服药，Leqvio由医护人员注射，初始两针间隔3个月，此后每6个月一次。这一给药频率直击传统治疗痛点，美国高达80%的ASCVD患者难以达到指南推荐的LDL-C<70 mg/dL的目标，而长效给药大幅提升了用药依从性。本次批准中，其他更新内容还包括将标签中“原发性高脂血症”一词统一修改为更具体的“高胆固醇血症”，更精准聚焦LDL-C降低，强化了其作为精准降脂疗法的定位，为临床应用提供了清晰指引。二、siRNA技术是长效特性的科学根基 Leqvio的成功，源于siRNA技术的独特优势，它通过靶向PCSK9基因的mRNA，从源头抑制该蛋白合成。PCSK9蛋白会对低密度脂蛋白（LDL）受体的回收与再利用产生抑制作用。所以，若能降低PCSK9蛋白的水平，就可使更多LDL受体重新回到肝细胞表面。这些受体能结合更多的LDL，并将其从血液中清除出去。技术迭代是长效的根基。早期siRNA因递送系统缺陷难以成药，而Leqvio采用了三触角结构N-乙酰半乳糖（GalNAc）偶联技术，GalNAc配体可以精准的与肝细胞膜表面表达的ASGPR结合。图注：Leqvio的结构（来源：参考资料[4]）由于Leqvio抑制PCSK9蛋白的产生是持续性的，而且肝脏清除LDL-C的能力增强，因此这种降LDL-C的效果可以持续较长时间。这使其无需频繁给药，就能持续降脂，解决了慢性病治疗的“依从性魔咒”。图注：Leqvio在体内的作用机制（来源：参考资料[4]）众多药企也布局了siRNA技术。礼来的Lepodisiran在二期ALPACA试验中实现Lp(a)水平平均降低93.9%，一些患者的降低持续了近1.5年[5]。安进的Olpasiran能将Lp(a)降低95%以上，并且在最后一剂后近一年对Lp(a)的降低显示出持久的效果[]。这些临床数据印证了siRNA在降脂领域的巨大潜力，不仅仅是PCSK9，更多靶点正被攻克。中国药企也在加速追赶。齐鲁制药、石药集团等企业的长效siRNA制剂进入临床，靶点覆盖PCSK9、ANGPTL3等。舶望制药与诺华达成超41亿美元合作，其siRNA药物管线进展迅速。瑞博生物的RBD7022靶向ASGPR/PCSK9，腾盛博药的elebsiran聚焦乙肝，维亚臻生物的VSA006靶向17β-HSD13瞄准代谢相关脂肪性肝炎（MASH），展现出本土创新潜力。结语从每日服药到半年一针，从辅助治疗到一线单药，Leqvio的获批是siRNA技术成熟的代表。它证明了RNA干扰不仅能攻克罕见病，更能在心血管等慢性病领域掀起变革。随着更多临床试验的推进，以及更多企业的技术突破，一次给药管半年甚至一年的降脂时代已近在眼前。 siRNA的高度特异性、广靶点潜力和长效特性，将为更多疾病提供新疗法。未来，siRNA疗法有望为全球患者带来更轻松、更有效的健康守护。参考资料： 1.Novartis twice-yearly* Leqvio® (inclisiran) receives FDA approval for new indication enabling first-line use, Jul 31, 2025, www.novartis.com 2.FDA Approves Inclisiran (Leqvio) Label Update as First-Line Monotherapy in Hypercholesterolemia, July 31, 2025, www.hcplive.com 3.Novartis twice-yearly* Leqvio® demonstrated clinically meaningful, statistically significant LDL-C lowering as a monotherapy in patients at low or moderate ASCVD risk, Aug 28, 2024, www.novartis.com 4.Migliorati, Julia M et al. “siRNA drug Leqvio (inclisiran) to lower cholesterol.” Trends in pharmacological sciences vol. 43,5 (2022): 455-456. doi:10.1016/j.tips.2022.02.003 5.Lilly's lepodisiran reduced levels of genetically inherited heart disease risk factor, lipoprotein(a), by nearly 94% from baseline at the highest tested dose in adults with elevated levels, March 30, 2025, investor.lilly.com 6.AMGEN PRESENTS LATE-BREAKING PHASE 2 OLPASIRAN DATA AT ESC 2023, www.amgen.com END 智药研习社近期直播预告来源：CPHI制药在线声明：本文仅代表作者观点，并不代表制药在线立场。本网站内容仅出于传递更多信息之目的。如需转载，请务必注明文章来源和作者。投稿邮箱：Kelly.Xiao@imsinoexpo.com ▼更多制药资讯，请关注CPHI制药在线▼ 点击阅读原文，进入智药研习社~

siRNA临床3期引进/卖出

100 项与苏州瑞博生物技术股份有限公司相关的药物交易

登录后查看更多信息

100 项与苏州瑞博生物技术股份有限公司相关的转化医学

登录后查看更多信息

组织架构

使用我们的机构树数据加速您的研究。

或

查看完整样例数据

管线布局

2025年08月22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

药物发现

临床前

临床1期

临床2期

临床3期

其他

登录后查看更多信息

当前项目

药物(靶点)	适应症	全球最高研发状态

Cosdosiran ( caspase 2 )	缺血性视神经病变更多	临床3期
RBD-1016 ( HBV-X )	乙型肝炎更多	临床2期
SR-059 ( F11 )	血栓栓塞更多	临床2期
RBD-5044 ( APOC3 )	高脂血症更多	临床2期
RBD-7022 ( PCSK9 )	原发性高胆固醇血症更多	临床2期