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. 2023 Apr 7;12(8):1103. doi: 10.3390/cells12081103

Table 3.

List of CRISPR-Cas9-based gene editing therapies for other non-infectious diseases.

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]