Reni-cel Builds on Promising Early Clinical Gains
Emerging data on renizgamglogene autogedtemcel (reni-cel) continue to position gene editing as a potential “functional cure” for sickle cell disease, with early-phase findings demonstrating sustained increases in fetal hemoglobin and marked reductions in vaso-occlusive events.1
As previously reported, the investigational therapy uses a CRISPR gene editing–Cas12a approach to modify autologous hematopoietic stem cells, enabling increased fetal hemoglobin production and prevention of red blood cell sickling.1,2,3
However, experts caution that current approaches represent an early iteration of gene-based therapies.
Catch up with Part 1 and Part 2 of our conversation with Rabi Hanna, MD, the lead author and chair of the Pediatric Hematology – Oncology & Blood and Bone Marrow Transplant Division at Cleveland Clinic Children’s, for insights into pain crisis reductions and its mechanism as a “functional cure”.