GENE THERAPY
Clinical trials for GENE THERAPY explained in plain language.
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Bubble boy disease gene therapy trial offers hope for a normal life
⭐️ CURE ⭐️ 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 chemotherapy followed by an infusion of their own blood stem cells that have been corrected with a healt…
Matched conditions: GENE THERAPY
Phase: PHASE1, PHASE2 • Sponsor: David Williams • Aim: ⭐️ CURE ⭐️
Last updated Apr 29, 2026 15:03 UTC
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Gene therapy makes its own CAR-T cells to battle bone marrow cancer
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 taking cells out of the body to make them fight cancer, this therapy delivers a gene directly into the body to create canc…
Matched conditions: GENE THERAPY
Phase: PHASE1 • Sponsor: Kelonia Therapeutics, Inc. • Aim: Disease control
Last updated Apr 30, 2026 15:51 UTC