First human trial for gene therapy targeting rare heart disease
Disease control
Not yet recruiting
This early-stage trial is testing a new, single-dose gene therapy called AFTX-201 for adults with a specific inherited form of heart failure. The main goal is to see if the treatment is safe and well-tolerated. Researchers will also look for early signs that it might improve hear…
Phase: PHASE1, PHASE2 • Sponsor: Affinia Therapeutics • Aim: Disease control
Last updated Mar 27, 2026 12:38 UTC