Novartis' sickle cell disease drug candidate shows promise in animal studies

Novartis has reported encouraging results for its experimental SCD treatment, a “glue degrader” small molecule. In animal studies, the drug effectively increased fetal hemoglobin (HbF) levels, crucial for managing SCD symptoms. The drug, dWIZ-2, targets the WIZ transcription factor, a previously unrecognized repressor of HbF expression. In non-human primates, it demonstrated gene up-regulation and HbF production over 28 days, with no signs of side effects. Pamela Ting, PhD, associate director of hematology at Novartis, emphasized the potential of this approach to provide a more accessible treatment option for SCD patients worldwide.

At OxyDial, we are intrigued by Novartis’ progress in developing a small-molecule drug that can potentially offer a widely accessible treatment option compared to current gene therapies.