Trials of genome-edited T cells with published or interim clinical data available
| Study . | Investigational product . | Modifications . | Indication . | Lymphodepletion . | Number . | Toxicity . | Outcomes . |
|---|---|---|---|---|---|---|---|
| Great Ormond Street Hospital Special’s License | UCART19 | LV-CAR19 TALEN ko of TRAC & CD52 | B-ALL | F,C, AntiCD52 | 2 | Grade2 GVHD | CR 100%29 |
| Servier/Allogene NCT02808442 NCT02746952 | UCART19 | LV-CAR19 TALEN ko of TRAC & CD52 | B-ALL | F,C, ± AntiCD52 | 21 | Grade 3+ CRS 15%; Grade 3+ infections 39% | CR 67%68 |
| Allogene NCT04416984 NCT03939026 | ALLO-501A ALLO-501 | LV-CAR19 TALEN ko of TRAC & CD52 | LBCL | F,C, AntiCD52 | 47 | Grade 3+ CRS: 2%; Grade 3+ infections 24% | CR 50%113 |
| Allogene NCT04093596 | Allo-715 | LV-anti BCMA TALEN ko of TRAC & CD52 | MM | F,C, AntiCD52 | 26 (DL3,4) | Grade 3 infections 13% | ORR 61%114 |
| Cellectis NCT04150497 | UCART22 | LV-CAR22 TALEN ko of TRAC & CD52 | B-ALL | F,C, ± AntiCD52 | 9 | No Grade 3 CRS or infection | No CR115 |
| Precision Bio NCT03666000 | PBCAR0191 | Arcus ko TRAC AAV site specific inserted CAR19 | B-ALL NHL | F, C | 27 | Grade 3+ CRS 6%; Grade 3+ infections 31%-80% | CR/CRi 62%-80%72,73 |
| CRISPR Tx NCT04035434 | CTX110 | CRISPR/Cas9 TRAC & B2m AAV site specific inserted CAR19 | LBCL | F,C | 24 DL2+ | Grade 3+ infections 9%; Grade 3+ ICANS 4% | CR 38%74 |
| Great Ormond Street Hospital NCT04557436 | TT52CAR19 | CRISPR/Cas9 TRAC, CD52 LV CAR19 | B-ALL | F,C, AntiCD52 | 6 | NCT04557436, TT52CAR19' Grade 3+ ICANS 17% | CR/CRi66%70 |
| Beijing Chinese PLA General Hospital NCT03166878 | U-Car | CRISPR/Cas9 TRAC, B2m LV CAR19 | DLBCL | F,C | 2 | No CR71 | |
| Zhejiang University Nanjing Bioheng Biotech | CTA 101 | CRISPR/Cas9 TRAC,CD52 LV CAR19/22 | B-ALL | F,C, AntiCD52 | 6 | Grade 3 CRS 16% Grade 3 infections 50% | CR/CRi83%116 |
| Gracell ChiCTR1900025311 | GC027 | CRISPR TRAC CD7 LV-CAR7 | T-ALL | F,C ±Mel | 6 | Grade 3 CRS 100% Grade 3 infections 50% | CR/CRi83%84 |
| CRISPR Tx NCT04502446 | CTX130 | CRISPR/Cas9 TRAC, B2m, CD70 AAV-site specific | TCL | F,C | 15 | Grade 3+ infections 7%; | CR29%75 |
| Study . | Investigational product . | Modifications . | Indication . | Lymphodepletion . | Number . | Toxicity . | Outcomes . |
|---|---|---|---|---|---|---|---|
| Great Ormond Street Hospital Special’s License | UCART19 | LV-CAR19 TALEN ko of TRAC & CD52 | B-ALL | F,C, AntiCD52 | 2 | Grade2 GVHD | CR 100%29 |
| Servier/Allogene NCT02808442 NCT02746952 | UCART19 | LV-CAR19 TALEN ko of TRAC & CD52 | B-ALL | F,C, ± AntiCD52 | 21 | Grade 3+ CRS 15%; Grade 3+ infections 39% | CR 67%68 |
| Allogene NCT04416984 NCT03939026 | ALLO-501A ALLO-501 | LV-CAR19 TALEN ko of TRAC & CD52 | LBCL | F,C, AntiCD52 | 47 | Grade 3+ CRS: 2%; Grade 3+ infections 24% | CR 50%113 |
| Allogene NCT04093596 | Allo-715 | LV-anti BCMA TALEN ko of TRAC & CD52 | MM | F,C, AntiCD52 | 26 (DL3,4) | Grade 3 infections 13% | ORR 61%114 |
| Cellectis NCT04150497 | UCART22 | LV-CAR22 TALEN ko of TRAC & CD52 | B-ALL | F,C, ± AntiCD52 | 9 | No Grade 3 CRS or infection | No CR115 |
| Precision Bio NCT03666000 | PBCAR0191 | Arcus ko TRAC AAV site specific inserted CAR19 | B-ALL NHL | F, C | 27 | Grade 3+ CRS 6%; Grade 3+ infections 31%-80% | CR/CRi 62%-80%72,73 |
| CRISPR Tx NCT04035434 | CTX110 | CRISPR/Cas9 TRAC & B2m AAV site specific inserted CAR19 | LBCL | F,C | 24 DL2+ | Grade 3+ infections 9%; Grade 3+ ICANS 4% | CR 38%74 |
| Great Ormond Street Hospital NCT04557436 | TT52CAR19 | CRISPR/Cas9 TRAC, CD52 LV CAR19 | B-ALL | F,C, AntiCD52 | 6 | NCT04557436, TT52CAR19' Grade 3+ ICANS 17% | CR/CRi66%70 |
| Beijing Chinese PLA General Hospital NCT03166878 | U-Car | CRISPR/Cas9 TRAC, B2m LV CAR19 | DLBCL | F,C | 2 | No CR71 | |
| Zhejiang University Nanjing Bioheng Biotech | CTA 101 | CRISPR/Cas9 TRAC,CD52 LV CAR19/22 | B-ALL | F,C, AntiCD52 | 6 | Grade 3 CRS 16% Grade 3 infections 50% | CR/CRi83%116 |
| Gracell ChiCTR1900025311 | GC027 | CRISPR TRAC CD7 LV-CAR7 | T-ALL | F,C ±Mel | 6 | Grade 3 CRS 100% Grade 3 infections 50% | CR/CRi83%84 |
| CRISPR Tx NCT04502446 | CTX130 | CRISPR/Cas9 TRAC, B2m, CD70 AAV-site specific | TCL | F,C | 15 | Grade 3+ infections 7%; | CR29%75 |
C, cyclophosphamide; DLBCL, diffuse large B-cell lymphoma; F, fludarabine; ICANS, immune cell associated neurotoxicity syndrome; LV, lentivirus; Mel, melphalan; TCL, T-cell lymphoma; ko, knockout.