This is a single center, non-randomized, open label, single-dose study in subjects with Sickle Cell Disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (BRL-101)
Subject (or their legally authorized representative or guardian) will sign and date an informed consent form (ICF) and, where applicable, an assent form.
Subjects 3 to 35 years of age, inclusive, on the date of informed consent.
Clinically confirmed severe SCD, genotypes include: βS/βS, βS/β + or βS/β0. Severe SCD is defined as having at least 2 VOC events per year during the 2 years prior to screening and requiring appropriate supportive care, including a pain management program, HU therapy (if indicated).
Karnofsky performance status of ≥80% for subjects ≥16 years of age. Lansky performance status of ≥80% for subjects <16 years of age (see Appendix 1 and 2).
Known contraindications, intolerance, or hypersensitivity to hematopoietic stem cell mobilizers, busulfan injection, or dimethyl sulfoxide (DMSO) or study drug-related components.
Eligible for allogeneic hematopoietic stem cell transplantation and have found HLA-identical donors.
Prior allo-HSCT, gene therapy or gene editing therapy.
Clinically significant and active bacterial, viral, fungal, or parasitic infection as determined by the investigator.
Single group
BRL-101 autologous hematopoietic stem and progenitor cells injection
DrugCD34 + autologous hematopoietic stem and progenitor cells edited at the BCL11A gene.
Proportion of stem cell engrafted subjects
Stem cell engraftment was defined as an absolute peripheral blood neutrophil count of ≥ 0.5 × 109/L for 3 consecutive days following BRL-101 intravenous infusion.
Time to neutrophil engraftment
Defined as Day 1 of absolute peripheral blood neutrophil count ≥ 0.5 × 109/L for 3 consecutive days
Frequency, severity, and relationship to BRL-101 of adverse events over 12 months following BRL-101 infusion..
Adverse events assessed according to NCI-CTCAE v5.0 criteria
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Bioray Laboratories
Lead sponsor
Institute of Hematology & Hospital of Blood Diseases, Chinese Academy of Medical Sciences & Peking Union Medical College
Collaborator
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