Investigational gene-edited autologous cell therapy products for sickle cell disease
| Product . | Sponsor . | Technology . | Effect . | Activation . | Status . |
|---|---|---|---|---|---|
| CTX001 | Vertex Pharmaceuticals Incorporated | CRISPR/Cas9 Editing | CRISPR-Cas9/BCL11A erythroid enhancer (antisickling) | 2018 | Submitted BLA spring 2023 |
| BIVV003 (now called SAR445136) | Sangamo Therapeutics | CRISPR/Cas9 editing | ZFN/BCL11A erythroid enhancer (antisickling) | 2019 | Further development discontinued |
| OTQ923/HIX763 | Novartis Pharmaceuticals | CRISPR/Cas9 Editing | CRISPR-Cas9/BCL11A (antisickling) | 2020 | Further development discontinued |
| GPH101 | Graphite Bio, Inc. | CRISPR/Cas9 HDR | CRISPR-Cas9/bS > bA (nonsickling) | 2021 | Further development discontinued |
| EDIT-301 | Editas Medicine, Inc. | CRISPR/Cas912a editing | CRISPR-Cas9/HGB1/2 (antisickling) | 2021 | Active, data not reported |
| CRISPR_SCD001 | UCSF Benioff Children's Hospital Oakland | CRISPR/Cas9 HDR | CRISPR-Cas9/bS > bA (nonsickling) | 2021 | Not yet recruiting |
| BEAM-101 | Beam Therapeutics | Base Editing | Base editing BCL11A erythroid enhancer (antisickling) | 2022 | Not yet recruiting |
| Product . | Sponsor . | Technology . | Effect . | Activation . | Status . |
|---|---|---|---|---|---|
| CTX001 | Vertex Pharmaceuticals Incorporated | CRISPR/Cas9 Editing | CRISPR-Cas9/BCL11A erythroid enhancer (antisickling) | 2018 | Submitted BLA spring 2023 |
| BIVV003 (now called SAR445136) | Sangamo Therapeutics | CRISPR/Cas9 editing | ZFN/BCL11A erythroid enhancer (antisickling) | 2019 | Further development discontinued |
| OTQ923/HIX763 | Novartis Pharmaceuticals | CRISPR/Cas9 Editing | CRISPR-Cas9/BCL11A (antisickling) | 2020 | Further development discontinued |
| GPH101 | Graphite Bio, Inc. | CRISPR/Cas9 HDR | CRISPR-Cas9/bS > bA (nonsickling) | 2021 | Further development discontinued |
| EDIT-301 | Editas Medicine, Inc. | CRISPR/Cas912a editing | CRISPR-Cas9/HGB1/2 (antisickling) | 2021 | Active, data not reported |
| CRISPR_SCD001 | UCSF Benioff Children's Hospital Oakland | CRISPR/Cas9 HDR | CRISPR-Cas9/bS > bA (nonsickling) | 2021 | Not yet recruiting |
| BEAM-101 | Beam Therapeutics | Base Editing | Base editing BCL11A erythroid enhancer (antisickling) | 2022 | Not yet recruiting |
BLA, biologic license application; HDR, homology-directed repair; ZFN, zinc finger nuclease.