Table 3.
Conditions Targeted | Gene Target | Edit Type | Therapeutic | Sponsor | Clinical Trial ID |
---|---|---|---|---|---|
Sickle cell disease or β-thalassemia |
BCL11A | KO (NHEJ) | exa-cel | CRISPR Therapeutics and Vertex Pharmaceuticals | NCT03655678 [85] |
NCT05477563 [86] | |||||
NCT03745287 [87] | |||||
NCT05356195 [88] | |||||
NCT05329649 [89] | |||||
NCT04208529 [90] | |||||
ET-01 | EdiGene (GuangZhou) Inc. | NCT04925206 [95] | |||
NCT04390971 [96] | |||||
BRL-101 | Bioray Laboratories | NCT05577312 [97] | |||
β-globin | HDR | nula-cel | Graphite Bio, Inc | NCT04819841 [100] | |
CRISPR_SCD001 | UCLA, UC Berkeley | NCT04774536 [101] | |||
iHSCs with corrected β-globin | ALLIFE Medical Science and Technology | NCT03728322 [102] | |||
β-thalassemia | γ-globin promoter | KO (NHEJ) | BRL-101 | Bioray Laboratories | NCT04211480 [103] |
EDIT-301 | Editas Medicine, Inc. | NCT05444894 [104] | |||
Sickle cell disease | NCT04853576 [105] | ||||
Type 1 diabeties | proprietary | VCTX210A | CRISPR Therapeutics and ViaCyte | NCT05210530 [106] | |
Leber congenital amaurosis 10 |
CEP290 | EDIT-101 | Editas Medicine, Inc. | NCT03872479 [107] | |
Hereditary angioedema | KLKB1 (liver) | NTLA-2002 | Intellia Therapeutics | NCT05120830 [108] | |
Duchenne muscular dystrophy |
Dp427c | Exon skipping |
CRD-TMH-001 | Cure Rare Diseases, Inc | NCT05514249 [109] |