Gene therapy (Lentiviral vector) · Gene replacement · developed by Rocket Pharmaceuticals
Mozafancogene autotemcel is an autologous gene therapy consisting of CD34+ hematopoietic stem and progenitor cells transduced with a lentiviral vector carrying the FANCA gene. It restores FANCA expression to correct the DNA repair defect in Fanconi anemia subtype A, addressing chromosomal breakage and bone marrow failure.
Summary written with AI from the sources listed at the end of this page.
| Trial | Phase | Condition | Status | Sponsor |
|---|---|---|---|---|
| NCT04248439 | Phase 2 | Fanconi Anemia Complementation Group A | Active not recruiting | RKTO |
Trials of the companies BioRadar tracks, from ClinicalTrials.gov, most advanced phase first.
Fanconi Anemia Complementation Group A
Updated Oct 3, 2026. Something wrong or missing? Report it on the RKTO page.
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