Huayuan Biotech Builds Barriers for lncRNA Drug R&D, and New Delivery System Solves Industry Pain Points

Huayuan Biotech has achieved original breakthroughs across the entire chain from target screening to delivery systems in the field of lncRNA drug R & D, and built a globally competitive R & D pipeline. In terms of target screening, the company innovatively adopts the strategy of "Genome - Wide Association Study (GWAS) + large - scale clinical validation". Relying on the million - level human second - generation sequencing database of the international CHARGE Consortium and collaborating with more than ten thousand clinical samples from Harvard Medical School and the medical research center of the University of Chicago, it precisely identifies liver - specific lncRNA targets, showing significant therapeutic effects on type 2 diabetes (T2D) and metabolic syndrome. Based on the characteristics of lncRNA, it avoids complications and effectively overcomes the industry pain points of short drug efficacy duration and numerous complications associated with traditional methods.

In the delivery system, Huayuan Biotech has collaborated with the Shenzhen Institutes of Advanced Technology, Chinese Academy of Sciences to break through the limitations of traditional four - component LNP design and develop a new liposome delivery system. Through component innovation, this system significantly improves liver targeting efficiency and significantly reduces the toxicity risk caused by non - liver organ enrichment, establishing a new industry standard for the efficient and precise delivery of small nucleic acid drugs.

In drug design, the siRNA drugs developed by the company for original lncRNA targets adopt a unique chemical modification strategy to enhance sequence stability, significantly extending the in - vivo action time of the drugs and successfully breaking through the technical bottleneck of easy degradation of RNAi in the body. Currently, Huayuan Biotech has laid out multiple core patents in key areas such as target identification, delivery systems, and drug optimization, building a solid intellectual property barrier.

In drug efficacy verification, Huayuan has carried out multi - level drug verification on primary cells and animals. The cross - species efficacy of the drugs has been systematically verified. In the experiments, Huayuan's innovative small nucleic acid drugs have demonstrated excellent systematic metabolic regulation ability: key indicators such as blood glucose, body weight, and blood lipids in the cynomolgus monkey model have been continuously improved, and the progression of type 2 diabetes - related complications has been significantly delayed.

In drug safety verification, in addition to the full - chain experiments on primary cells and animals, the whole - genome and transcriptome analysis based on cynomolgus monkeys further shows that Huayuan's innovative small nucleic acid drugs can precisely silence the target lncRNA and regulate the downstream gene network. The drug's specificity and safety have been confirmed from multiple dimensions, including molecular mechanism levels and animal experiment results, as well as dry and wet experiment results.

Compared with existing treatment methods for metabolic diseases, Huayuan Biotech's technology has three disruptive advantages:

1. Disruptive treatment mechanism: Utilizing the function of lncRNA as the "command tower" of the gene network to achieve coordinated regulation of multiple organs such as the liver and pancreas, avoiding side effects such as muscle loss and treating both the symptoms and the root cause;

2. Disruptive delivery system: The new liver - targeting delivery system has been verified to have good safety in the cynomolgus monkey model, with no abnormalities in liver and kidney functions and coagulation indicators, while significantly improving the liver drug enrichment efficiency;

3. Disruptive treatment strategy: The drugs have shown significant therapeutic effects in indications related to metabolic diseases such as non - alcoholic steatohepatitis, liver fibrosis, and hypertension. Huayuan's innovative small nucleic acid drugs are expected to become drugs for multiple indications, with the application prospect of "one drug with multiple effects".

Currently, the company has reached an in - depth strategic cooperation with WuXi AppTec to jointly accelerate the clinical translation process of the "small nucleic acid multi - effect drugs for T2D and metabolic syndrome based on lncRNA". At the same time, derivative pipelines for indications such as non - alcoholic fatty liver and liver fibrosis expanded based on this delivery platform have also been launched, continuously expanding the boundaries of technology application and market imagination space.