BIRSA 101

**BIRSA 101** is India's first indigenously developed CRISPR-Cas9-based gene therapy for sickle cell disease (SCD), a genetic blood disorder primarily affecting tribal populations in central and eastern India. It employs precise gene editing using an engineered enFnCas9 CRISPR platform to correct mutations in the HBB gene within patients' hematopoietic stem cells **ex vivo**, enabling production of normal hemoglobin and preventing red blood cell sickling for a potential one-time cure. Developed by CSIR-Institute of Genomics and Integrative Biology (CSIR-IGIB) and transferred to Serum Institute of India for scalable, affordable manufacturing, it aims to drastically reduce costs compared to global therapies like Casgevy (₹15–20 crore), targeting accessibility for underserved communities under the National Sickle Cell Anaemia Elimination Mission.[1][2][7]