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NCT02140554

A Phase 1/2 Study Evaluating Gene Therapy by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo With the LentiGlobin BB305 Lentiviral Vector in Subjects With Severe Sickle Cell Disease

Study Summary

This is a non-randomized, open label, multi-site, single dose, Phase 1/2 study in approximately 50 adults and adolescents with severe SCD. The study will evaluate hematopoietic stem cell and progenitor stem cell (collectively referred to as hematopoietic stem and progenitor cells or HSPCs) transplantation using lovo-cel.

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Interventions

lovo-celGENETIC
lovo-cel is administered by IV infusion following myeloablative conditioning with busulfan.

Study Locations

FacilityCityStateCountry
N/ABirminghamAlabamaUnited States
N/AOaklandCaliforniaUnited States
N/AAtlantaGeorgiaUnited States
N/AChicagoIllinoisUnited States
N/ABethesdaMarylandUnited States
N/AHackensackNew JerseyUnited States
N/ANew Hyde ParkNew YorkUnited States
N/ANew YorkNew YorkUnited States
N/AChapel HillNorth CarolinaUnited States
N/APhiladelphiaPennsylvaniaUnited States
N/ACharlestonSouth CarolinaUnited States

Official Trial Information

View on ClinicalTrials.gov

Data sourced from ClinicalTrials.gov. Last updated: April 14, 2026