A Clinical Trial to Evaluate the Safety of RP-L102 in Pediatric Subjects With Fanconi Anemia Subtype A

Inclusion Criteria: * Fanconi anemia, as diagnosed by chromosomal fragility assay of cultured T-lymphocytes in the presence of DEB or a similar DNA-crosslinking agent. * Subjects of Fanconi Anemia complementation group A. * Minimum age: 1 year and a minimum of 8 kg. * Maximum age: 12 years. * At least one of the following hematologic parameters below lower limits of normal: * Hemoglobin * Absolute neutrophils * Platelets * At least 30 CD34+ cells/μL are determined in one BM aspiration within 3 months prior to initiation of CD34+ cell collection. * If the number of C34+ cells/ μL in BM is in the range of 10-29, PB parameters should meet two of the three following criteria: * Hemoglobin: ≥11g/dL * Neutrophils: ≥900 cells/μL * Platelets: ≥60,000 cells/μL * Provide informed consent in accordance with current legislation. * Women of childbearing age must have a negative urine pregnancy test at the baseline visit and accept the use of an effective contraception method during participation in the trial. Exclusion Criteria: * Subjects with an available and medically eligible human leukocyte antigen (HLA)-identical sibling donor. * Evidence of myelodysplastic syndrome or leukemia, or cytogenetic abnormalities predictive of these conditions in BM aspirate analysis. This assessment should be made by valid studies conducted within the 3 months before the subject commences the stem cell mobilization/collection procedures of the clinical trial. * Subjects with somatic mosa…