The company's discovery platform uses RNA biology to identify novel cancer targets that drive tumor growth and cancer progression. Their lead therapy, RGX-104, is a small molecule immunotherapy that blocks the ability of tumors to evade the immune system and has obtained FDA Orphan Drug Designation in several solid tumor types. Inspirna's clinical programs include first-in-class oral small molecules in development for patients with RAS mutant CRC (RGX-202; Phase 1b) as well as SCLC and NSCLC (RGX-104; Phase 1b/2). Their goal is to develop effective therapies against these targets that will provide durable and meaningful responses for cancer patients who lack effective therapies.