Table 1. Selected examples of CRISPR-based clinical trials for inherited blood disorders.

Disease indication	Therapeutic strategy	Sponsor	Phase	Clinical trial
Transfusion-dependent β thalassemia	Nuclease-driven editing at the HBG1/2 promoters using CRISPR-Cas12b	Institute of Hematology and Blood Diseases, China	NA	NCT06041620
Sickle cell disease	Nuclease-driven correction (HDR) at HBB using CRISPR-Cas9	Kamau Therapeutics, USA	I, II	NCT04819841
Sickle cell disease	Nuclease-driven editing at BCL11A using CRISPR-Cas9	Bioray Laboratories, China	NA	NCT06287099
Hemophilia B	Nuclease-driven (HDR) coagulation factor IX gene insertion using CRISPR-Cas9	Regeneron Pharmaceuticals, USA	I, II	NCT06379789
Transfusion-dependent β thalassemia and sickle cell disease	Nuclease-driven editing at BCL11A using CRISPR-Cas9	Vertex Pharmaceuticals, USA	III	NCT05477563
Transfusion-dependent β thalassemia and sickle cell disease	Nuclease-driven combined enhancer editing at BCL11A using CRISPR-Cas9	Boston Children’s Hospital, USA	I	NCT06647979
X-linked chronic granulomatous disease (X-CGD)	Gene correction at CYBB using base editing	National Institute of Allergy and Infectious Diseases (NIAID), USA	I, II	NCT06325709
Sickle cell disease	Modification of the HBG1/2 promoters using base editing	CorrectSequence Therapeutics, China	I	NCT06565026
Sickle cell disease	Modification of the HBG1/2 promoters using base editing	Beam Therapeutics, USA	I, II	NCT05456880
Chronic granulomatous disease (CGD)	Gene correction at NCF1 using prime editing	Prime Medicine, USA	I, II	NCT06559176

The NIH National Library of Medicine clinical trials database (ClinicalTrials.gov) was searched on January 5^th, 2025, for active clinical trials using the keywords "CRISPR", "Hematopoietic stem cells", "Base editing", and "Prime editing". NA, not applicable.