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 mosaicism associated with stable or improved counts in all PB cell lineages. (If T-lymphocyte chromosomal fragility analysis indicates potential mosaicism, a medically significant decrease in at least one blood lineage over time must be documented to enable eligibility). * Lansky performance status ≤60%. * Any concomitant disease or condition that, in the opinion of the Principal Investigator, renders the subject unfit to participate in the study. * Pre-existing sensory or motor impairment ≥grade 2 according to the NCI CTCAE v5.0 criteria. * Pregnant or breastfeeding women. * Hepatic dysfunction as defined by either: * Bilirubin \>3.0 × upper limit of normal (ULN) or * Alanine aminotransferase (ALT) \> 5.0 × ULN or * Aspartate aminotransferase (AST) \> 5.0 × ULN * Renal dysfunction requiring either hemodialysis or peritoneal dialysis. * Pulmonary dysfunction as defined by either: * Need for supplemental oxygen during the prior 2 weeks in absence of acute infection. * Oxygen saturation by pulse oximetry \<90%. * Evidence of active metastatic or locoregionally advanced malignancy for which survival is anticipated to be less than 3 years. * Subject is receiving androgens (i.e. danazol, oxymethalone).