Genethon
Clinical trials sponsored by Genethon, explained in plain language.
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Experimental gene therapy aims to fix immune system in rare childhood disease
Disease control TerminatedThis early-phase trial tested a gene therapy for X-linked chronic granulomatous disease (X-CGD), a rare genetic disorder that leaves boys unable to fight off bacteria and fungi. The treatment involved taking the patient's own blood stem cells, adding a corrected gene, and infusin…
Phase: PHASE1, PHASE2 • Sponsor: Genethon • Aim: Disease control
Last updated Jun 27, 2026 12:06 UTC
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Experimental gene therapy targets rare immune disorder
Disease control TerminatedThis trial tested a gene therapy for X-linked chronic granulomatous disease (X-CGD), a rare genetic disorder that leaves boys unable to fight off infections. The treatment involved taking the patient's own blood stem cells, adding a working copy of the faulty gene, and returning …
Phase: PHASE1, PHASE2 • Sponsor: Genethon • Aim: Disease control
Last updated Jun 27, 2026 12:06 UTC