First ALS patients receive experimental gene therapy in groundbreaking safety trial
Disease control
Recruiting now
This early-stage study is testing the safety of a new gene therapy called CTx1000 in people with ALS. The therapy aims to target a protein called TDP-43 that builds up in ALS. Researchers will give a single dose to 15 participants and closely monitor for side effects while also c…
Phase: PHASE1 • Sponsor: Celosia Therapeutics Pty Ltd • Aim: Disease control
Last updated Mar 31, 2026 12:11 UTC