SynerK, a developer of RNA-targeted therapies with operations in Boston, US, and Beijing and Suzhou, China, has announced the completion of first patient dosing in its Phase I clinical study for an investigational small interfering RNA (siRNA). This marks the first time such a therapy has entered the clinical phase in China.
siRNA Drug Development and GalNAc Technology
The drug, developed using SynerK’s proprietary GalNAc liver targeted delivery platform technology, is designed to safely and efficiently deliver siRNA to hepatocytes. By leveraging the natural process of RNA interference, it directly interferes with the synthesis of liver target proteins at the mRNA level, achieving therapeutic goals. This new siRNA drug demonstrates superior activity and safety, with improved stability in vivo compared to previous siRNA drugs.
Study Design and Objectives
The multi-center, randomized, double-blind, placebo-controlled study is designed to rigorously assess the safety and tolerability of SynerK’s siRNA drug. It also aims to explore the drug’s pharmacokinetics and preliminary efficacy, providing valuable insights into its potential as a treatment option. This trial represents a significant step forward in the development of RNA-targeted therapies, particularly for liver-related diseases.-Fineline Info & Tech
