. Author manuscript; available in PMC: 2024 Oct 8.

Published in final edited form as: Lancet Haematol. 2023 Jul 11;10(8):e633–e686. doi: 10.1016/S2352-3026(23)00096-0

Table 4:

Overview of ongoing or recently completed gene addition therapy studies

	Modality	Lead group	Status	Clinical trial number	Results

Globin gene addition	Addition of modified β-globin gene (b^T87Q; LentiGlobin=lovotibeglogene autotemcel; bb1111)	bluebird bio	Phase 3; recruiting	NCT02140554 and NCT04293185	35 Group C patients infused by July, 2021, with a median duration of follow-up 20·9 months (range 8·5–28·5); 28 (97%) of 29 evaluable patients were free of severe VOCs
Globin gene addition	Addition of γ-globin gene (ARU-1801)	Aruvant	Phase 1/2; not recruiting	NCT02186418	As of November, 2021, four patients have been infused; three (75%) patients with >12 months follow-up had reduced or absent VOCs
Globin gene addition	Addition of modified β-globin gene, (bAS3; DREPAGLOBE)	Assistance Publique—Hôpitaux de Paris	Phase 1/2; not recruiting	NCT03964792	No clinical trial results posted yet
Globin gene addition	Addition of modified β-globin gene (bAS3-FB)	University of California, Los Angeles	Phase 1; recruiting	NCT02247843	No clinical trial results posted yet
HbF induction	shRNA knockdown of BCL11A gene (BCH-BB694)	Boston Children’s Hospital	Phase 2; recruiting	NCT03282656 and NCT05353647	Ten patients infused by November, 2022, with a median duration of follow-up of 30·5 months (range 2–50); nine (90%) of ten patients had reduced or absent VOCs

VOCs=vaso-occlusive crises. shRNA=short hairpin RNA.