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).