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. Seaport, Boston, USA.

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

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. 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.

2024 OPT (Oligonucleotide & mRNA Therapeutics) Congress Presentation Details

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

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

• Presentation content:

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..