Novel Therapeutics and Druggable Targets for Non-malignant Hematological Diseases

An American pharmaceutical company with a focus on rare diseases is seeking novel therapeutics for non-malignant hematological diseases. Drug molecules that have a defined target, mechanism of action, and proven efficacy for a number of rare blood diseases are in scope for this campaign.

Approaches of Interest:

• Diseases of interest include, but are not limited to, hereditary hemorrhagic telangiectasia, sickle cell disease, thalassemia, hereditary hemochromatosis, hereditary spherocytosis, diamond Blackfan anemia, Fanconi’s anemia, erythropoietic protoporphyria...