CRISPR_SCD001

CRISPR_SCD001 is an investigational, patient-specific gene-editing therapy designed to treat sickle cell disease (SCD). The therapy uses the CRISPR/Cas9 system to directly correct the disease-causing mutation in a patient's own hematopoietic stem cells. These edited stem cells are then transplanted back into the patient after high-dose chemotherapy conditioning, with the goal of producing healthy red blood cells and eliminating sickle cell symptoms. Unlike some other gene-editing therapies, CRISPR_SCD001 does not use viral vectors for delivery; instead, it employs electroporation to introduce gene-editing components into harvested stem cells. The product was developed by a consortium including UCSF, UCLA, and UC Berkeley through the Innovative Genomics Institute (IGI), with clinical trial leadership at UCSF and UCLA. The primary indication is severe sickle cell disease[1][3][4][5][7].