This study is a first-in-human, single-arm, open-label Phase I/II study of nula-cel in approximately 15 participants, diagnosed with severe Sickle Cell Disease. The primary objective is to evaluate safety of the treatment in this patient population, as well as preliminary efficacy and pharmacodynamic data.
≥12 to ≤ 40 years
recurrent severe VOC (≥ 4 episodes in the preceding 2 years)
ACS (≥ 2 episodes in the prior 2 years with at least one episode in the past year)
Lansky/Karnofsky performance status of ≥ 80
Available 10/10 HLA-matched sibling donor
Prior HSCT or gene therapy
Prior or current malignancy or myeloproliferative or a significant coagulation or immunodeficiency disorder
Clinically significant and active bacterial, viral, fungal or parasitic infection
Single group
nula-cel Drug Product
Geneticnula-cel is administered via IV infusion following a myeloablative conditioning regimen
Proportion of patients who reach neutrophil engraftment
Incidence rate of treatment-related mortality
Incidence rate of treatment-related mortality
Overall survival
Time to neutrophil engraftment
Time to platelet engraftment
Evaluation of gene correction levels in peripheral myeloid cells
Evaluation of adult Hgb as a percentage of total Hgb
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