CG001 (DanausGT Biotechnology)

CG001 is an ex vivo CRISPR-based gene editing cell therapy being developed by DanausGT Biotechnology for the treatment of Pyruvate Kinase Deficiency (PKD). PKD is a rare autosomal recessive blood disorder caused by mutations in the PKLR gene, which encodes the pyruvate kinase enzyme essential for red blood cell metabolism. CG001 utilizes DanausGT's proprietary CRISPR 3.0 platform, which combines CRISPR/Cas9 with Adeno-Associated Virus (AAV) vectors to achieve precise gene replacement or correction in patient-derived hematopoietic stem cells (HSCs). By restoring functional PKLR expression, the therapy aims to provide a one-time curative treatment for chronic hemolytic anemia. The program has received Orphan Drug Designation from both the FDA and the EMA and is currently in pre-clinical development.