. Author manuscript; available in PMC: 2023 Aug 1.

Published in final edited form as: Transl Res. 2022 Mar 27;246:1–14. doi: 10.1016/j.trsl.2022.03.010

Table 2:

Clinical Trials for Gene therapy and editing in sickle cell disease

Strategy	NCT Number	Phase/Enrollment/Status	Results	Citations
Lentiviral transfer of γ-globin^G16D – ARU-1801	NCT02186418	Phase 1/2 Enrollment- 10 Status: open	Increased F-cells, no VOE	Grimley M, et al. 2020. Blood
Lentiviral transfer of modified HBB encoding antisickling variant β^A87Thr:Gln[β^A-T87Q] LentiGlobin BB305	NCT02151526	Phase 1/2 Enrollment- 7 Status: closed	Stable CBC, Decreased reticulocyte, decreased RBC sickling under hypoxia and improved RBC deformability	Ribel JA et al. 2017. NEJM
	NCT02140554	Phase 1/2 Enrollment- 50 Status: not recruiting^*
	NCT04293185	Phase 3 Enrollment- 35 Status: recruiting^*
AS3-FB vector transduced peripheral blood CD34+ cells	NCT02247843	Phase 1/2 Enrollment- 6 Status: not recruiting	None reported Pre-clinical: decreased sickling, engraftment of mice	Romero Z. et al. 2013. JCI
Lentiviral targeting of γ-globin repressor BCL11A with BCH-BB694 BCL11A shmiR vector	NCT03282656	Phase 1 Enrollment- 15 Status: SUSPENDED^*	Increased HbF, increased % F-cells, increased HbF per F cells	Esrick, EB. Et al. 2021. NEJM
Reactivation of fetal hemoglobin by using Crisper-Cas (CTX001) editing to suppress BCL11A β-globin switch	NCT03745287	Phase 1/2 Enrollment- 45 Status: recruiting	Increased HbF, reduced transfusion needs and no VOE	Frangoul, H. et al. 2021. NEJM

Studies were paused in March 2021 due to the development of myelodysplastic syndrome and acute myeloid leukemia in patients in NCT02140554 and NCT04293185 prompting suspension of NCT03282656 out of caution. NCT02140554 and NCT04293185 currently active.