AB1 is an oral, once-daily, potentially disease-modifying treatment with the differentiated potential to address all associated morbidities for all patients living with sickle cell and thalassemia.
AB1, is currently in clinical development for the treatment of sickle cell disease as a best-in-class HbF inducer with the potential to act as a ‘functional cure.
AB1 gene control therapy: novel oral small molecule inducer of HbF
Blocked HbF activation leading to sickling of red blood cells (left)
In the diseased state, methylated nucleotides in the HbF promoter region allow repressor complex binding which blocks activation of HbF transcription.
HbF gene activation increases healthy fetal red blood cells (right)
By contrast, AB1 depletion of DNMT1 reduces nucleotide methylation and repressor complex binding allowing HbF gene activation and increasing production of healthy circulating fetal red blood cells.