Clinical Study to Evaluate the Safety and Efficacy of Single Dose Intravenous Infusion of CRISPR/Cas9-edited Autologous CD34 + Hematopoietic Stem/Progenitor Cells (BRL-101) in the Treatment of Severe Sickle Cell Disease
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).
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⁃ Subjects must meet all the following inclusion criteria to be eligible for enrolment into the study:
• 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).
• Eligible for autologous stem cell transplant as per investigator's judgment.
• Willing and able to comply with scheduled visits, treatment plan, laboratory tests, contraceptive guidelines, and other study procedures.
• Willing to participate in an additional long-term follow-up study after completion of this study .
• Subjects of childbearing potential must use effective contraception for at least 6 months after BRL-101 infusion during the study.