FANCONI ANEMIA COMPLEMENTATION GROUP A
Clinical trials for FANCONI ANEMIA COMPLEMENTATION GROUP A explained in plain language.
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Gene therapy for rare blood disease passes 15-Year safety watch
Disease control OngoingThis study follows 9 people with Fanconi Anemia who already received a gene therapy that adds a working FANCA gene to their blood stem cells. Researchers will check their health and blood counts for 15 years to see if the treatment remains safe and keeps working. No new treatment…
Matched conditions: FANCONI ANEMIA COMPLEMENTATION GROUP A
Sponsor: Rocket Pharmaceuticals Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:07 UTC
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Experimental gene therapy aims to stop bone marrow failure in kids with rare disease
Disease control OngoingThis phase 2 trial tests a gene therapy called RP-L102 for children with Fanconi anemia subtype A, a rare genetic disorder that leads to bone marrow failure. Doctors take the child's own blood stem cells, fix the faulty gene in a lab, and infuse the corrected cells back. The goal…
Matched conditions: FANCONI ANEMIA COMPLEMENTATION GROUP A
Phase: PHASE2 • Sponsor: Rocket Pharmaceuticals Inc. • Aim: Disease control
Last updated Jun 27, 2026 08:06 UTC