Table 2.
ARI-0001 Product Specification List and Acceptance Criteria
| Parameter | Method | Acceptance Criteria | ARI0001/01 | ARI0001/02 | ARI0001/03 |
|---|---|---|---|---|---|
| Appearance | visual inspection | cloudy liquid solution | cloudy liquid solution | cloudy liquid solution | cloudy liquid solution |
| Number of CAR19+ cells | Neubauer cell counting and flow cytometry | 35 × 106 cells for a 70-kg patient (>0.5 × 106 cells/kg) | 1,495 × 106 | 1,746 × 106 | 1,340 × 106 |
| CAR19+ cells (%) | flow cytometry | ≥20% | 26.0 | 41.9 | 39.7 |
| CD3+ cells (%) | flow cytometry | ≥70% | 97.9 | 98.6 | 98.6 |
| Cell viability (%) | Neubauer cell counting with trypan blue exclusion | ≥70% | 97.9 | 98.1 | 98.0 |
| Sterility | microbial growth | sterile | sterile | sterile | sterile |
| Mycoplasma | PCR | absent | absent | absent | absent |
| Endotoxin | chromogenic assay | ≤0.5 EU/mL | ≤0.5 EU/mL | ≤0.5 EU/mL | ≤0.5 EU/mL |
| Adventitious viruses | PCR | absent | absent | absent | absent |
| Number of transgene copies/cell | real-time PCR | ≤10 copies/cell | 1.45 | 2.32 | 2.51 |
| RCL (replication-competent lentivirus) | real-time PCR | absent | absent | absent | absent |
| Cytotoxic potency | flow cytometry | (a) NALM6 cell surviving fraction using CART19 ratio E:T 1:1 <70% or (b) difference in NALM6 cell surviving fraction between CART19 versus untransduced T using ratio E:T 4:1 >50% | (a) 14.9; (b) 90.9 | (a) 40.4; (b) 64.6 | (a) 43.0; (b) 67.0 |