drug-repurposing
(3)
Auransa
Auransa is a clinical-stage, AI-native biopharmaceutical company in Palo Alto, California, that uses a proprietary machine-learning platform - the SMarTR Engine - to mine large, heterogeneous human disease datasets and discover novel cancer therapies without starting from a predefined drug target. Founded in 2014 by Pek Lum and Viwat Visuthikraisee, the company pairs each predicted compound with the patient subtypes most likely to respond, aiming to improve the odds of clinical success. Its lead program, AU-409, an RNA transcription modulator for advanced liver cancer, is in Phase 1 trials, with additional programs including heart-safe chemotherapy.
Melior Discovery
Melior Discovery is an Exton, Pennsylvania preclinical contract research organization that specializes in in vivo pharmacology and drug repositioning. Its proprietary theraTRACE platform runs a candidate compound through a large, multiplexed panel of validated animal disease models simultaneously, systematically surfacing new therapeutic uses for existing or shelved drugs. Founded in 2005 by Andrew Reaume and Michael Saporito, the company both partners with major pharmaceutical firms and builds its own drug pipeline through spinouts, and was named CRO of the Year by Life Sciences Review in 2024.
Unravel Biosciences
Unravel Biosciences is a clinical-stage therapeutics company spun out of Harvard's Wyss Institute that starts with a patient's RNA rather than a drug target. Its BioNAV probabilistic network model of human health screens 40,000-plus compounds in silico - reporting roughly 200x the hit rate of conventional screens - to find existing molecules that can push a disease back toward health. The company validates predictions in engineered whole-organism models (including CRISPR-edited tadpoles) and then in patients, aiming to bring effective, lower-cost therapies to rare and overlooked diseases. Its lead asset, RVL-001 (a proprietary formulation of vorinostat), holds FDA Orphan Drug Designation for Rett syndrome and has entered clinical studies.