First-Ever gene therapy trial launches for devastating childhood nerve disease
Disease control
Not yet recruiting
This is the first human study to test a single-dose gene therapy called ELP-02 for children and young adults (ages 3-20) with a rare, inherited nerve disease called Charcot-Marie-Tooth type 4J (CMT4J). The therapy aims to deliver a working copy of a faulty gene directly to the ne…
Phase: PHASE1, PHASE2 • Sponsor: Elpida Therapeutics SPC • Aim: Disease control
Last updated Apr 01, 2026 14:41 UTC