The reni-cel experimental gene therapy for severe sickle cell disease continues to show promising results. Now tested in a full cohort of 40 patients, some with more than two years of follow-up, the one-time infusion of gene-edited stem cells has helped 95% of enrolled patients avoid vaso-occlusive events (VOEs).
“This is remarkable for these patients, most of whom had numerous severe VOEs requiring an average of nearly five hospital admissions each year,” says Rabi Hanna, MD, Chair of Hematology and Medical Oncology at Cleveland Clinic Children’s. “In addition, these patients have reported improvements in their physical and social functioning. Not only is the treatment reducing their pain and functionally curing them of sickle cell disease, but it is also helping them live like people without disease.”
Dr. Hanna presented these latest results of the phase 1/2 RUBY trial at the 2025 American Society of Hematology meeting.