Abstract: The present disclosure provides DCN-1 modulating compounds, pharmaceutically acceptable salts thereof, pharmaceutical compositions, and their use for treating sickle cell disorders, diseases, and conditions. Such compounds are of Formula I: or a pharmaceutically acceptable salt thereof.

Abstract: The present disclosure provides DCN-1 modulating compounds, pharmaceutically acceptable salts thereof, pharmaceutical compositions, and their use for treating sickle cell disorders, diseases, and conditions. Such compounds are of Formula Ia: or a pharmaceutically acceptable salt thereof.

Abstract: Systems and methods of associating a test compound with a reference compound are provided. A fingerprint of a chemical structure of the test compound is obtained. Abundance values for a set of cellular constituents are also obtained, from one or more reference assays, across a plurality of cells that have been exposed to a control solution free of the test compound. The fingerprint and the abundance values are inputted into a model that outputs a predicted similarity between (i) a predicted perturbational effect of the test compound across the set of cellular constituents and (ii) a measured cell-based perturbational effect of the reference compound across the set of cellular constituents. When the predicted similarity achieves a threshold similarity, the test compound is associated with the reference compound.