Fanconi anemia

MONDO:0019391

Fanconi anemia (FA) is a hereditary DNA repair disorder characterized by progressive pancytopenia with bone marrow failure, variable congenital malformations and predisposition to develop hematological or solid tumors.

Also known as: Fanconi anemia, Fanconi pancytopenia, Fanconi's anemia, Panmyelopathy, Fanconi, pancytopenia, congenital, primary erythroid hypoplasia

54 clinical trials for this condition and its sub-types, 29 tagged with Fanconi anemia itself.

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Where it sits in the disease tree

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Part of

↑ Bone marrow disorder (1763) ↑ Developmental anomaly of metabolic origin (384) ↑ Multiple congenital anomalies/dysmorphic syndrome without intellectual disability (163) ↑ DNA repair disease (105) ↑ Congenital anemia (58) ↑ Inherited aplastic anemia (53)

Sub-types of Fanconi anemia

Fanconi anemia complementation group D1 6 trials
Fanconi anemia complementation group A 4 trials
Fanconi anemia complementation group E 4 trials
Fanconi anemia complementation group N 2 trials
Fanconi anemia, complementation group S 2 trials
Fanconi anemia complementation group B 0 trials
Fanconi anemia complementation group C 0 trials
Fanconi anemia complementation group D2 0 trials
Fanconi anemia complementation group F 0 trials
Fanconi anemia complementation group G 0 trials
Fanconi anemia complementation group I 0 trials
Fanconi anemia complementation group J 0 trials
Fanconi anemia complementation group L 0 trials
Fanconi anemia complementation group O 0 trials
Fanconi anemia complementation group P 0 trials
Fanconi anemia complementation group Q 0 trials
Fanconi anemia complementation group R 0 trials
Fanconi anemia complementation group T 0 trials
Fanconi anemia complementation group U 0 trials
Fanconi anemia complementation group V 0 trials
Fanconi anemia, complementation group W 0 trials
Fanconi anemia, complementation group 10 0 trials

Including sub-types (54) Tagged with Fanconi anemia (29)

Trials to join now! 14 Not yet recruiting 3 Not yet finished but already full! 9 Completed 2 Terminated 1

Gene therapy aims to fix bone marrow failure in fanconi anemia

Disease control Stopped early

This trial tests a gene therapy for Fanconi anemia, a rare genetic disorder that causes bone marrow failure and increases cancer risk. Participants receive their own stem cells that have been genetically corrected with a lentiviral vector to fix the faulty FANCA gene. The study e…

Sponsor: Shenzhen Geno-Immune Medical Institute • Aim: Disease control

Last updated Aug 26, 2026 00:00 UTC

Fanconi anemia

Where it sits in the disease tree

Gene therapy aims to fix bone marrow failure in fanconi anemia