AI spurs gene therapy, drug development

From the discovery of novel drug candidates to the delivery of advanced genetic therapies, enterprises are beginning to show how artificial intelligence can generate breakthroughs that may reshape the global pharmaceutical landscape. As AI shifts from concept to clinical testing in life sciences, China is emerging as one of the strongest drivers of this transformation.

That momentum was reflected in MIT Technology Review's 2025"50 Smartest Companies" list, also known as the TR50, which highlights firms that combine technological breakthroughs with market foresight. Life sciences ranked among the top four sectors, with a notable rise of companies integrating AI and biotech in transformative ways.

One example is Insilico Medicine, a global AI-driven company that has built its key research and development team in Shanghai. Its return to the TR50 reflects an evolution from a single milestone to a scalable model. In 2022, it gained international attention when an AI-designed drug for idiopathic pulmonary fibrosis entered clinical trials. The breakthrough, featured in Nature Biotechnology, showed that AI could compress both the time and cost of early-stage research.

"In 2022, our recognition was for a singular, groundbreaking achievement," said Alex Zhavoronkov, founder and CEO of Insilico Medicine. "By 2025, the recognition was no longer for a single project, but for the proven replicability of our AI platform and the sustainability of our business model."

The progress is clear. Insilico has nominated more than 20 pre-clinical candidates, secured 10 Investigational New Drug Application approvals, and advanced seven programs into clinical development. Its lead therapy became the first AI-discovered drug worldwide to demonstrate efficacy in a phase 2a trial, an industry milestone, even as further studies are needed to confirm long-term safety and effectiveness.

Zhavoronkov described the company's long-term vision as "Pharmaceutical Superintelligence", a multimodal AI system capable of designing medicines end-to-end. "Imagine an autonomous AI system that operates as the ultimate drug hunter. When faced with any disease, it will independently engineer the optimal therapeutic solution," he said.

If drug discovery is one side of the equation, delivery is the other. In this space, Beijing-based biotech unicorn METiS TechBio has captured attention with its debut on the TR50. The company's Nano-Forge platform uses AI to design lipid nanoparticles (LNPs) that deliver RNA and gene-editing therapies directly to specific organs.

The results have been significant. METiS reports liver-targeting LNPs that exceeded clinical benchmarks and advanced into human trials, lung-targeting systems that carried mRNA into alveolar cells in primates, and first-in-class LNPs for heart and skeletal muscle tissue, which could open new treatment paths for genetic heart conditions and muscular dystrophy.

"Delivery is the rocket of modern biotech. We're building the rockets that will carry tomorrow's therapeutic satellites," said Chris Lai, co-founder and CEO of METiS TechBio. Lai said rising investor confidence is fueling closer collaboration between Chinese and foreign firms.

The rise of such players reflects the maturing of China's innovation ecosystem. According to Stanford University's AI Index 2024, the country accounted for 61.1 percent of global AI patent filings in 2022, far ahead of the United States at 20.9 percent. Analysts point to a decade of sustained investment in AI talent, biomedical research and supportive policies as the foundation for this acceleration.

"The convergence of AI and life sciences was inevitable," Zhavoronkov said. "China's accumulated strengths in both fields are now producing paradigm-shifting momentum." He said China is moving beyond "model innovation" toward "foundational innovation", a more difficult but enduring form of progress.

Wang Hongwei, professor at the School of Life Sciences and vice-president of Tsinghua University, said: "With the rapid development of AI, we have seen an increasing number of multinational pharmaceutical corporations working with emerging biopharmaceutical companies in China, in early drug development, as well as diagnosis and treatment. This kind of collaboration has already been formed as an innovative ecosystem in China."