Deep Genomics

Deep Genomics has developed a proprietary AI-driven drug discovery platform focused on decoding complex RNA biology to identify novel therapeutic targets and design programmable RNA-based medicines. The company claims world-firsts in using AI to discover drug candidates—its lead program in Wilson disease was the first such example. As of mid–2025 it has at least ten preclinical programs targeting rare metabolic and neurological conditions with plans for rapid expansion; no human efficacy data reported yet but expects first-in-human studies imminently. The company collaborates with major pharma partners like BioMarin Pharmaceutical on rare-disease programs.