Verge Genomics takes AI-sourced drug for ALS into clinic

Verge Genomics has joined a select group of biotechs who have taken a drug discovered and developed using artificial intelligence into human testing. The small-molecule PIKfyve inhibitor – called VRG50635 – has been administered to the first subject in the phase 1 trial involving healthy volunteers, according to the San Francisco-based biotech, which was founded in 2015 by Alice Zhang and Jason Chen. VRG50635 was discovered using Verge's AI-powered discovery platform ConVERGE which maps out the biological underpinnings of diseases using data on DNA, RNA, and protein profiles to identify new targets and drugs that can interact with them. The company focuses on diseases of the central nervous system, starting with neurodegenerative diseases amyotrophic lateral sclerosis (ALS) – the indication for VRG50635 – and Parkinson's disease. PIKfyve is an enzyme thought to be involved in an underlying disease process in ALS linked to the function of lysosomes, organelles involved in processing waste materials in cells.