Experimental drug TPN-101 tested in rare childhood brain disease
Disease control
Terminated
This study tested a drug called TPN-101 (censavudine) in people with Aicardi-Goutières syndrome, a rare genetic disorder that causes severe brain inflammation. The trial enrolled only 4 participants and aimed to see if the drug could reduce immune system overactivity and check fo…
Phase: PHASE2 • Sponsor: Transposon Therapeutics, Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:05 UTC