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.
Bioray Laboratories
Lead sponsor
Institute of Hematology & Hospital of Blood Diseases, Chinese Academy of Medical Sciences & Peking Union Medical College
Collaborator