Table 3.

CRISPR clinical trials for inherited diseases listed in this review.

Disease	Study title	Strategy	Study phase	Study type	Participants (No., Age)	Company	NCT Number	Website
Transfusion- Dependent β-thalassemia	A Safety and Efficacy Study Evaluating CTX001 in Subjects With Transfusion-Dependent β-Thalassemia	CTX001	Phase 1Phase 2	Interventional	45 patients, ≥18 and ≤35 years of age	Vertex Pharmaceuticals Incorporated & CRISPR Therapeutics	NCT03655678	https://clinicaltrials.gov/ct2/show/NCT03655678
Sickle Cell Disease	A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease	CTX001	Phase 1Phase 2	Interventional	45 patients, ≥18 and ≤35 years of age	Vertex Pharmaceuticals Incorporated & CRISPR Therapeutics	NCT03745287	https://clinicaltrials.gov/ct2/show/NCT03745287
β-thalassemia	iHSCs With the Gene Correction of HBB Intervent Subjests With β-thalassemia Mutations	HBB HSC-01	Early Phase 1	Interventional	12 patients, ≥ 2 and ≤ 60 years of age	Allife Medical Science & Technology Co., Ltd.	NCT03728322	https://clinicaltrials.gov/ct2/show/NCT03728322
Leber congenital amaurosis LCA10	Single Ascending Dose Study in Participants With LCA10	AGN-151587	Phase 1Phase 2	Interventional	18 patients, ≥ 3 Years	Allergan & Editas Medicine, Inc.	NCT03872479	https://clinicaltrials.gov/ct2/show/NCT03872479

Data from https://clinicaltrials.gov/