In a medical first, scientists have unveiled an artificial intelligence (AI)-designed drug that could transform the outlook for people living with idiopathic pulmonary fibrosis (IPF)—a deadly age-related lung disease.

The new therapy, rentosertib, is the first drug discovered and developed entirely using generative AI to reach human trials. Developed by Insilico Medicine in collaboration with Peking Union Medical College, the drug targets a novel biological pathway involved in fibrosis, known as TNIK.

Published in Nature Medicine, results from a Phase 2a clinical trial show encouraging signs. Seventy-one patients with IPF received either rentosertib or a placebo over 12 weeks. The treatment was well-tolerated, with side effects no different from placebo. Even more promising, lung function—measured through forced vital capacity (FVC)—tended to improve in patients taking rentosertib, particularly in those not on standard antifibrotic medications.

IPF causes scarring of lung tissue, leaving patients increasingly breathless and with few options beyond slowing the disease’s progression. Currently, there is no cure, and survival after diagnosis is typically three to five years.

What makes this milestone remarkable is not only the drug’s potential but its speed. Thanks to AI, rentosertib advanced from discovery to human trials in less than 30 months—an unprecedented pace in drug development. “Rentosertib brings new hope to IPF patients and highlights AI’s transformative power in medicine,” said Dr. Zuojun Xu, lead investigator of the study.

While larger trials are still needed, rentosertib represents a promising step forward—offering hope to patients and a glimpse of how AI can accelerate solutions for age-related diseases.

 

Reference: https://pmc.ncbi.nlm.nih.gov/articles/PMC12353801/