Experimental drug TPN-101 targets rare inflammatory brain disease
Disease control
Terminated
This study tested an experimental drug called TPN-101 in people with Aicardi-Goutières syndrome, a rare genetic disorder that causes severe brain inflammation. The goal was to see if the drug could lower abnormal immune activity and check for side effects. Only 4 participants wer…
Phase: PHASE2 • Sponsor: Transposon Therapeutics, Inc. • Aim: Disease control
Last updated Jun 11, 2026 12:11 UTC