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:
- Introduction to GalAhead™, Sirnaomics' GalNAc-RNAi therapeutic platform
- Validation of the GalAhead™ muRNA technology in vitro and in vivo
- Progress report on muRNA GalAhead-based program
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..
About Sirnaomics (stock code: 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. 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. 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. Learn more at: www.sirnaomics.com.
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