The biopharmaceutical field has recently witnessed several disruptive breakthroughs, with innovative achievements, particularly those represented by technologies independently developed in China, attracting global attention. On December 11, the Global Health Drug Discovery Center (GHDDI) officially launched "AI Kongming," China's first end-to-end intelligent drug design AI platform, in Beijing. This platform enables end-to-end intelligent driving from target discovery to candidate drug screening, injecting strong momentum into the global development of new drugs for health diseases. Meanwhile, key advancements in areas such as precise mRNA delivery and gene editing for diabetes treatment are jointly propelling the biopharmaceutical industry into a new phase of efficient innovation and precision medicine.
At the "AI Kongming: Driving the Future of New Drugs" event, GHDDI launched its independently developed platform, marking a significant breakthrough for my country in AI-enabled drug development. Unlike single-stage AI tools on the market, "AI Kongming" constructs a comprehensive system covering the entire process from target structure analysis to AI molecule generation, drug design, activity assessment, and drugability optimization. By integrating generative AI molecular design models and high-precision virtual screening algorithms, it forms an intelligent closed loop of "design-screening-evaluation-optimization-decision," significantly reducing the "trial and error" cycle of traditional drug development.
According to reports, the platform has been validated in dozens of real-world drug development pipelines, including tuberculosis, malaria, and rare diseases, with candidate molecule hit rates and optimization efficiency several to tens of times higher than traditional processes. Of particular note is that "AI Kongming" adheres to the principle of universal accessibility, focusing on globally neglected health diseases such as malaria and tuberculosis, empowering global research through an open and shared model. The platform also features a self-controlled and secure foundation, supporting localized and private cloud deployments to ensure the secure and compliant transfer of core research data. "The ultimate goal of technological breakthroughs is to address clinical needs," emphasized Professor Ding Sheng, Director of GHDDI. As a non-profit organization, the initial motivation for developing "AI Kongming" was to respond to the unmet needs of millions of patients worldwide. In the future, they will promote the platform's application iteration in multiple disease scenarios through a collaborative network, building a new ecosystem for science and technology-driven public welfare.
mRNA Delivery Technology Achieves Precise Targeting, Opening New Spaces for Multi-Organ Therapy
In the field of gene therapy, the latest achievements of the team from the Institute of Zoology, Chinese Academy of Sciences, are equally remarkable. Traditional lipid nanoparticles (LNPs) have the limitation of "liver tropism," making it difficult to meet the treatment needs of extrahepatic diseases. The research team innovatively integrated peptides and ionizable lipid materials to develop peptide-based ionizable lipid (PIL) materials and the PILOT organ-targeting mRNA delivery platform, achieving precise multi-organ delivery without additional ligand modification.
The platform establishes clear "design rules": lysine/arginine modification enables lung-targeted delivery, cysteine/histidine modification corresponds to liver targeting, glutamic acid/aspartic acid modification can precisely target the spleen, lysine-tyrosine dipeptide modification can improve thymus targeting efficiency, and the addition of alendronate enables bone targeting. Based on this, the team achieved a 13.3% editing efficiency in the liver and a 7.4% editing efficiency in the lungs through liver-targeted and lung-targeted PILOT LNP delivery lead editing technology. This marks the first time that efficient lead editing has been achieved in the lungs through systemic administration, laying a technological foundation for the treatment of genetic lung diseases.
JinYi Connect points out that China's independent innovation achievements in fields such as AI-driven drug development and mRNA technology are gradually breaking international technological monopolies and enhancing the inclusiveness of the global biopharmaceutical industry. As these technologies are validated in more disease areas, they are expected to provide more efficient and accessible solutions to global health challenges, propelling the biopharmaceutical industry into a high-quality development stage centered on new productive forces.
