One-Time gene therapy for fatal childhood brain disease: does it last?
Cure
Ongoing
Researchers are following 64 people with cerebral adrenoleukodystrophy (CALD) for 15 years after they received a one-time gene therapy called eli-cel in earlier studies. The therapy uses the patient's own stem cells, modified to carry a working copy of the gene that is faulty in …
Sponsor: Genetix Biotherapeutics Inc. • Aim: Cure
Last updated Sep 16, 2026 00:00 UTC