BIOIO-2001

BIOIO-2001 is a small molecule investigational drug developed by BIOIO for the treatment of diabetic neuropathy and potentially other aging-related diseases. It targets serine palmitoyltransferase (SPT), the rate-limiting enzyme in sphingolipid biosynthesis, and is based on the mechanism of action of cationic amphiphilic drugs (CADs) that inhibit sphingolipid biosynthesis. By modulating lipid metabolism, BIOIO-2001 aims to address multiple hallmarks of aging and has been positioned as a gerotherapeutic—an agent designed to target core mechanisms underlying age-related diseases. The compound was discovered using BIOIO’s proprietary MOAT (Mechanism Of Action Technologies) platform, which combines genome-wide screening with AI modeling to elucidate drug mechanisms[1][4]. Funding for its development has included support from VitaDAO using Molecule's IP-NFT framework[3].