Familial severe combined immunodeficiency
MONDO:003152024 clinical trials for this condition and its sub-types.
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Bubble boy disease gene therapy trial launches
Disease control Not yet recruitingThis trial tests a gene therapy given by IV for X-linked severe combined immunodeficiency (SCID-X1), a rare immune disorder. Ten participants with no matched donor will receive a lentiviral vector to correct the genetic defect. The goal is to restore immune function and improve s…
Phase 1/2 • Sponsor: Shenzhen Geno-Immune Medical Institute • Aim: Disease control
Last updated Aug 29, 2026 00:00 UTC
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One-Time gene fix could rescue 'Bubble Boy' disease
Disease control Not yet recruitingThis study tests a gene therapy given by IV for ADA-SCID, a rare and life-threatening immune disorder. The treatment uses a modified virus to deliver a working gene, aiming to restore the immune system. Ten participants will be monitored for safety and immune recovery over one ye…
Sponsor: Shenzhen Geno-Immune Medical Institute • Aim: Disease control
Last updated Aug 28, 2026 00:00 UTC