Current and upcoming studies of gene therapy in SCD
| Study name . | LentiGlobin . | DREPAGLOBE . | CLIMB . | PRECIZN-1 . | Genetic silencing of BCL11A . | MOMENTUM . | CEDAR . |
|---|---|---|---|---|---|---|---|
| Type of gene therapy | Gene addition | Gene addition | Gene editing | Gene editing | Gene silencing | Gene addition | Gene correction |
| Editing tool | NA | NA | CRISPR-Cas9 RNP | Zinc finger | ShRNA | NA | HiFi CRISPR-Cas9 RNP |
| Type of stem cell manipulation | Transduction | Transduction | Electroporation | Transfection with zinc finger nuclease mRNA | Transduction | Transduction | Electroporation |
| Vector (y/n) | BB305 LVV | DROBE 1 LVV | None | None | BCH-BB694 LVV that encodes a microRNA-adapted shRNA | γG16D LVV | Nonintegrating AAV6 donor DNA repair template |
| Genetic target (y/n) | NA | NA | Erythroid lineage- specific enhancer of the BCL11A gene | 11A (BCL11A) locus (erythroid enhancer) | BCL11A mRNA | N/a | Sickle mutation (adenosine— > thymine [A— > T] |
| Drug product | LentiGlobin BB305 | DREPAGLOBE | CTX001 | BIVV003 | BCH-BB694 | ARU-180126 | GPH101 |
| Protein product | HbAT87Q | βAS3, an antisickling β-globin protein (AS3) containing 3 amino acid substitutions in the wild-type HBB | HbF | HbF | HbF | HbFG16D | HbA |
| Study name . | LentiGlobin . | DREPAGLOBE . | CLIMB . | PRECIZN-1 . | Genetic silencing of BCL11A . | MOMENTUM . | CEDAR . |
|---|---|---|---|---|---|---|---|
| Type of gene therapy | Gene addition | Gene addition | Gene editing | Gene editing | Gene silencing | Gene addition | Gene correction |
| Editing tool | NA | NA | CRISPR-Cas9 RNP | Zinc finger | ShRNA | NA | HiFi CRISPR-Cas9 RNP |
| Type of stem cell manipulation | Transduction | Transduction | Electroporation | Transfection with zinc finger nuclease mRNA | Transduction | Transduction | Electroporation |
| Vector (y/n) | BB305 LVV | DROBE 1 LVV | None | None | BCH-BB694 LVV that encodes a microRNA-adapted shRNA | γG16D LVV | Nonintegrating AAV6 donor DNA repair template |
| Genetic target (y/n) | NA | NA | Erythroid lineage- specific enhancer of the BCL11A gene | 11A (BCL11A) locus (erythroid enhancer) | BCL11A mRNA | N/a | Sickle mutation (adenosine— > thymine [A— > T] |
| Drug product | LentiGlobin BB305 | DREPAGLOBE | CTX001 | BIVV003 | BCH-BB694 | ARU-180126 | GPH101 |
| Protein product | HbAT87Q | βAS3, an antisickling β-globin protein (AS3) containing 3 amino acid substitutions in the wild-type HBB | HbF | HbF | HbF | HbFG16D | HbA |