Scientists find new way to boost fetal hemoglobin for blood disorder treatment
Researchers at Dana-Farber have uncovered a biological switch involving BACH2 and NRF2 that could lead to better therapies for sickle cell disease and beta thalassemia.
Target Discovery 1 article
Target Discovery begins with the biological question of which molecule, pathway or cellular mechanism should be perturbed to change disease. Human genetics, functional genomics, disease models and mechanistic experiments are used to establish causality, tractability and safety before chemistry or therapeutic engineering takes over. The section therefore precedes Molecular Design and Lead Optimization; it is about choosing and validating what to act on, not yet about designing the drug itself.