GENE THERAPY
Clinical trials for GENE THERAPY explained in plain language.
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New gene therapy offers hope for children with bubble boy disease
Disease control Recruiting nowThis study tests a gene therapy for children under 5 with X-linked severe combined immunodeficiency (SCID-X1), also known as bubble boy disease. Patients receive a low dose of busulfan (a chemotherapy drug) followed by an infusion of their own blood stem cells that have been corr…
Matched conditions: GENE THERAPY
Phase: PHASE1, PHASE2 • Sponsor: David Williams • Aim: Disease control
Last updated Jun 17, 2026 02:58 UTC
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Gene therapy builds CAR-T cells inside the body to fight myeloma
Disease control Recruiting nowThis early-stage study tests a new gene therapy called KLN-1010 for people with multiple myeloma that has come back or not responded to treatment. Instead of removing cells and engineering them in a lab, this therapy delivers a gene directly into the body to create cancer-fightin…
Matched conditions: GENE THERAPY
Phase: PHASE1 • Sponsor: Kelonia Therapeutics, Inc. • Aim: Disease control
Last updated Jun 16, 2026 12:53 UTC