Hutchinson-Gilford progeria syndrome (HGPS), an extremely rare genetic disorder, arises when a silent point mutation in the gene encoding the nuclear envelope protein lamin A, LMNA, leads to abnormal splicing of LMNA mRNA.
The University of Minnesota has patented flavones having senolytic activity that are described as useful for the treatment of aging and Hutchinson-Gilford progeria syndrome.
Researchers from the University of Maryland in collaboration with the National Institutes of Health (NIH) and Duke University have identified angiopoietin-2 (Ang2) as a targetable protein to reverse cardiovascular dysfunction in Hutchinson-Gilford progeria syndrome (HGPS).
Although Eiger Biopharmaceuticals Inc. sees more would-be opportunities with ultra-rare disease-targeting Zokinvy (lonafarnib), the company’s vice president of clinical and development operations, Colin Hislop, said that “at the moment, we’re very clearly focused on the population identified in the label, because it fits most closely with the mechanism of action.”
