Apheresis parameters of published trials for SCD gene therapy
| Trial . | Patients (reported) . | Premobilization transfusion . | Apheresis cycles . | Apheresis starting material cell target (CD34+ cells per kilogram) . | Gene therapy product cell dose target (CD34+ cells per kilogram) . | Comments . |
|---|---|---|---|---|---|---|
| Lovotibeglogene autotemcel (bluebird)12 | 15 | 60 days | 8 patients, 1 cycle 6 patients, 2 cycles 1 patient, 3 cycles 1.7 apheresis/patient (2 d/cycle) | 15 × 106 | 3 × 106 | Cell products shipped fresh |
| Exagamglogene autotemcel (Vertex)13 | 44 | 8 weeks | Median 2 per cycles 32% of patients ≥3 cycles of 2 days per cycle | 15 × 106 | 3.5 × 106 | Cell products shipped fresh |
| OTQ923 Novartis50 | 3 | 60 days | Three patients with 3, 2, and 2 cycles, respectively, of 2-3 days per patient | 15 × 106 | 2 × 106 | Cryopreserved, 2/3 patients met cell dose target |
| BCH-BB694 (BCH/NHLBI)14 | 6 | 12 weeks | 1 cycle of up to 2 apheresis days | 15 × 106 | 4 × 106 | Cell products shipped fresh |
| Trial . | Patients (reported) . | Premobilization transfusion . | Apheresis cycles . | Apheresis starting material cell target (CD34+ cells per kilogram) . | Gene therapy product cell dose target (CD34+ cells per kilogram) . | Comments . |
|---|---|---|---|---|---|---|
| Lovotibeglogene autotemcel (bluebird)12 | 15 | 60 days | 8 patients, 1 cycle 6 patients, 2 cycles 1 patient, 3 cycles 1.7 apheresis/patient (2 d/cycle) | 15 × 106 | 3 × 106 | Cell products shipped fresh |
| Exagamglogene autotemcel (Vertex)13 | 44 | 8 weeks | Median 2 per cycles 32% of patients ≥3 cycles of 2 days per cycle | 15 × 106 | 3.5 × 106 | Cell products shipped fresh |
| OTQ923 Novartis50 | 3 | 60 days | Three patients with 3, 2, and 2 cycles, respectively, of 2-3 days per patient | 15 × 106 | 2 × 106 | Cryopreserved, 2/3 patients met cell dose target |
| BCH-BB694 (BCH/NHLBI)14 | 6 | 12 weeks | 1 cycle of up to 2 apheresis days | 15 × 106 | 4 × 106 | Cell products shipped fresh |
NHLBI, National Heart, Lung, and Blood Institute.