First-Ever gene therapy trial launches for devastating childhood nerve disease

This is the first study in people to test a single-dose gene therapy called ELP-02 for 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 into the nervous system to slow or stop disease progression. The study will enroll 8 children and young adults (ages 3-20) to first check safety and see if the treatment helps stabilize muscle function and quality of life.