FIX-Gene Therapy

We have an early stage Factor IX gene therapy construct - CB 2679d-GT - for Hemophilia B. This gene therapy product candidate has demonstrated a 2-3-fold higher specific activity and a 4-5-fold reduction in bleeding time in a mouse preclinical Hemophilia B model compared with the Factor IX Padua variant currently being developed by others. Recently, we have shown that combination of a novel AAV gene therapy delivery vector expressing our CB 2679d-GT construct provides significantly enhanced expression and Factor IX activity in mouse hemophilia B model.