208 - How Drugs Are Designed, Targets, Phenotypes, and Structure Based Design

What this episode covers

Jesse Gordon-Blake, PhD, CSO of Cortexa Therapeutics, explains the process of discovering small-molecule drugs for ALS, contrasting structure-based and phenotype-based design. He details the computational methods used to design molecules that can cross the blood-brain barrier, citing Lipinski's Rule of Five and the CNS MPO score. Gordon-Blake discusses the critical early-stage work at Cortexa, including validating the drug target by linking it to TDP-43 dysfunction in iPSC models, and outlines the company's hybrid funding strategy using both nonprofit donations and venture capital.