Experimental drug TPN-101 studied for rare genetic brain disorder
Disease control
Terminated
This early study tested an experimental drug called TPN-101 in people with Aicardi-Goutières syndrome (AGS), a rare genetic disorder that causes severe brain inflammation. The goal was to see if the drug could reduce abnormal immune system activity and check for side effects. Onl…
Phase: PHASE2 • Sponsor: Transposon Therapeutics, Inc. • Aim: Disease control
Last updated May 01, 2026 16:00 UTC