HONG KONG, GERMANTOWN, Md. and SUZHOU, China, March 12, 2024 /PRNewswire/ -- Sirnaomics Ltd. (the 'Company'; stock code: 2257, together with its subsidiaries, the 'Group' or 'Sirnaomics'), a leading biopharmaceutical company engaging in discovery and development of advanced RNAi therapeutics, will present its preclinical and clinical development of its GalAhead™ technology-based programs, focusing on an innovative dual-targeted-GalNAc design, muRNA platform drug candidates, at the upcoming 2024 OPT (Oligonucleotide & mRNA Therapeutics) Congress, which took place in Boston, USA from March 13-14, 2024.

2024年3月12日，马里兰州香港日耳曼敦和中国苏州，PRNewswire/-Sirnaomics Ltd.（以下简称“公司”；股票代码：2257，连同其子公司“集团”或“Sirnaomics”），一家致力于发现和开发先进RNAi治疗剂的领先生物制药公司，将介绍其基于GalAhead™技术的项目的临床前和临床开发，在即将于2024年3月13日至14日在美国波士顿举行的2024 OPT（寡核苷酸和mRNA治疗）大会上，专注于创新的双靶向GalNAc设计，muRNA平台候选药物。

Based on Sirnaomics' continued progress in targeting single genes with GalAheadTM mxRNA, Company is now pioneering GalAheadTM muRNA, a proprietary RNAi-based platform that allows for simultaneous downregulating of multiple genes. GalAheadTM muRNA, alike mxRNA employs GalNAc ligands, a clinically validated concept for RNAi trigger delivery to liver hepatocytes providing subsequent treatment of liver associated diseases.

基于Sirnaomics在用GalAheadTM mxRNA靶向单个基因方面的持续进展，该公司现在正在开创GalAheadTM muRNA，这是一种专有的基于RNAi的平台，可以同时下调多个基因。GalAheadTM-muRNA与mxRNA一样使用GalNAc配体，这是一种临床验证的RNAi触发递送至肝肝细胞的概念，可随后治疗肝脏相关疾病。

The muRNA concept carries a unique RNAi design, consisting of a duplex that carries two antisense strands, two complementary adaptor strands and site-specific engineered labile spots. The cleavage points allow for the muRNA after cellular delivery to break into two individual RNAi-triggers in the endo-lysosomal pathway.

muRNA概念具有独特的RNAi设计，由携带两条反义链，两条互补衔接子链和位点特异性工程不稳定点的双链体组成。切割点允许细胞递送后的muRNA在内溶酶体途径中断裂成两个单独的RNAi触发器。

The individualized RNAi triggers will then subsequently target their respective genes..

然后，个体化的RNAi触发器将随后靶向它们各自的基因。

From a biological perspective, the muRNA concept allows for modulation of converging overactivated biological pathways while still allowing a window to perform their normal physiological function. In a different scenario, muRNA can simultaneously address two or more non-associated indications where patient populations have a considerable overlap.

从生物学的角度来看，muRNA概念允许调节会聚的过度激活的生物途径，同时仍然允许一个窗口来执行其正常的生理功能。在不同的情况下，muRNA可以同时解决两个或多个非相关适应症，其中患者人群有相当大的重叠。

GalAheadTM muRNA offers a new and inspiring venue in the RNAi space providing a safe and long-lasting effect in treating patients with unmet medical needs..

GalAheadTM muRNA在RNAi空间提供了一个新的鼓舞人心的场所，为治疗未满足医疗需求的患者提供了安全而持久的效果。

2024 OPT (Oligonucleotide & mRNA Therapeutics) Congress Presentation Details

2024 OPT（寡核苷酸和mRNA治疗学）大会介绍详情

Presentation Title: GalAhead™ muRNA: A Proprietary GalNAc-RNAi Therapeutic Platform for simultaneous Downregulation of Multiple Genes

演讲标题：GalAhead™muRNA：一种专有的GalNAc RNAi治疗平台，可同时下调多个基因

Presenter: Jim Weterings, PhD, Vice President for Research, RNA Therapeutics & Delivery of Sirnaomics

主持人：Jim Weterings博士，RNA治疗与Sirnaomics交付研究副总裁

Presentation content:

演示内容：

Introduction to GalAhead™, Sirnaomics' GalNAc-RNAi therapeutic platform

GalAhead™简介，Sirnaomics的GalNAc RNAi治疗平台

Validation of the GalAhead™ muRNA technology in vitro and in vivo

GalAhead™muRNA技术的体内外验证

Progress report on muRNA GalAhead-based program

muRNA GalAhead项目进展报告

Dr. Weterings will join the Plenary Keynote Session, starting at 3:40 pm on the same date. As the Plenary Chairperson, he was invited to share a remark about the functions of RNA therapeutics, as well as future applications..

Weterings博士将于同日下午3:40开始参加全体会议的主题演讲。作为全体会议主席，他被邀请分享关于RNA疗法的功能以及未来应用的评论。

About Sirnaomics (stock code: 2257)

关于Sirnaomics（股票代码：2257）

Sirnaomics is an RNA therapeutics biopharmaceutical company that focuses on the discovery and development of innovative drugs for indications with unmet medical needs and large market opportunities. Sirnaomics is the first clinical-stage RNA therapeutics company to have a strong presence in both Asia and the United States.

Sirnaomics是一家RNA治疗生物制药公司，专注于发现和开发创新药物，用于未满足医疗需求和巨大市场机会的适应症。Sirnaomics是第一家在亚洲和美国都有强大影响力的临床阶段RNA治疗公司。

Based on its proprietary delivery technologies, a polypeptide nanoparticle RNAi platform, and GalNAc RNAi platform, GalAhead™, Sirnaomics has established an enriched drug candidate pipeline. STP122G, which represents the first drug candidate utilizing the Company's GalAhead™ technology, is currently in Phase I development.

基于其专有的递送技术，多肽纳米颗粒RNAi平台和GalNAc RNAi平台GalAhead™，Sirnaomics建立了丰富的候选药物管道。STP122G是第一个使用该公司GalAhead™技术的候选药物，目前正处于I期开发阶段。

The Company has also had multiple successes with oncology applications through its clinical programs for STP705 and STP707. With the establishment of its manufacturing facility in China, Sirnaomics is undergoing a transition from a biotech company to a biopharma corporation.

该公司还通过其针对STP705和STP707的临床计划在肿瘤学应用方面取得了多项成功。随着其在中国制造工厂的建立，Sirnaomics正在从一家生物技术公司过渡到一家生物制药公司。