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 cure
⭐️ 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 May 18, 2026 11:59 UTC
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Inside-Out attack: gene therapy builds cancer killers in the body
Disease control Recruiting nowThis early-stage trial tests a new gene therapy called KLN-1010 for people with multiple myeloma that has come back or not responded to treatment. The therapy works by giving a single injection that instructs the body's own immune cells to become cancer-fighting cells (CAR-T cell…
Matched conditions: GENE THERAPY
Phase: PHASE1 • Sponsor: Kelonia Therapeutics, Inc. • Aim: Disease control
Last updated May 26, 2026 10:51 UTC