Stem Cell Gene Therapy for Cystinosis

This study is a Phase 1/2 clinical trial that will assess the safety and efficacy of enriched gene-corrected hematopoietic stem cells isolated from patients affected with cystinosis. (Investigational Product: CTNS-RD-04, including product manufactured with and without the transduction enhancer LentiBOOST \[CTNS-RD-04-LB\])

Inclusion Criteria: The following criteria must be met by all subjects considered for study participation. 1. Cohorts 1 and 2: Male or female subject is ≥ 18 years of age. 2. Cohort 3: Male or female subject is ≥ 14 years of age. 3. Subject is diagnosed with cystinosis, i.e., early onset of Fanconi syndrome, and history of elevated white blood cell cystine level and/or history of or presence of cystine crystals in the eye. 4. Subject has a Karnofsky Performance Status or age-dependent Lansky Performance of ≥ 60. 5. If subject has had a kidney transplant, he or she must be at least one-year post kidney transplant status. 6. Subject has adequate hematologic function: 1. Absolute neutrophil count (ANC) ≥ 1.5 x 1000/mm\^3 2. Platelet count ≥ 100 x 1000/mm\^3 3. Hemoglobin ≥ 9.0 gm/dL 7. Subject has an adequate hepatic function: 1. Bilirubin ≤ 2.0 mg/ dL 2. ALT ≤ 3 x institution's upper limit of normal (ULN) U/L 8. Subject has an adequate renal function: a. Serum creatinine \