Novo Nordisk has moved into sickle cell disease through a licensing deal (PDF) with EpiDestiny. The $400 million deal gives Novo worldwide rights to an epigenetic treatment of the disease that is closing in on the start of a phase 2 trial.
People who suffer from the inherited disease sickle cell disease have defects in their hemoglobin, the blood molecule that transports vital oxygen throughout the body. But a small percentage of patients with the disease have beneficial gene mutations that lessen their symptoms. The mutations switch on a gene that produces fetal hemoglobin—a type of the blood molecule that the body normally stops producing in infancy. The lifelong production of fetal hemoglobin helps shield those sickle cell patients from the worst effects of the disease.
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