A Trial to Evaluate Safety and Efficacy of RP-L401-0120 in Subjects With Infantile Malignant Osteopetrosis

Exclusion Criteria

• •1. Availability of medically-feasible HLA-matched sibling donor for allogeneic HSCT.
• •2. Any medical or other contraindication for either apheresis or autologous transplant as determined by the Investigator.
• •3. Participation in another clinical trial with an investigational drug within 14 days before the informed consent signature. Participation in observational studies is allowed.
• •4. Active hematologic or solid organ malignancy, not including non-melanoma skin cancer or another carcinoma in situ.
• •5. Uncontrolled seizure disorder.
• •6. Renal dysfunction as defined by a glomerular filtration rate \<30 mL/min/1.73m2 or dialysis dependence.
• •7. Serious infections with persistent bloodstream pathogens at time of trial entry
• •8. Pulmonary dysfunction as defined by either:
• •* Need for supplemental oxygen during the prior 2 weeks (in absence of acute infection) or
• •* Oxygen saturation (by pulse oximetry) \<90% resulting from pulmonary conditions (intermittent hypoxia secondary to IMO-related choanal atresia will not be considered exclusionary)