CRISPR-Cas12a Gene Editing of HBG1 and HBG2 Promoters to Treat Sickle Cell Disease - PubMed
21 hours ago
- #Gene Therapy
- #Sickle Cell Disease
- #CRISPR-Cas12a
- Reni-cel is a CRISPR-Cas12a gene-edited therapy targeting BCL11A binding sites in HBG1/HBG2 promoters to reactivate fetal hemoglobin for sickle cell disease treatment.
- A phase 1-2 study in 28 patients showed increased total hemoglobin from 9.8 g/dL to 13.8 g/dL and fetal hemoglobin from 2.5% to 48.1% at 6 months, sustained thereafter.
- No vaso-occlusive events occurred in 27 of 28 patients post-infusion; adverse events aligned with standard myeloablative conditioning and stem-cell transplantation.
- The study, terminated early due to sponsor priorities, supports further investigation of this gene-editing approach for severe sickle cell disease.