Therapeutics: small molecule activators

We are working with the Center for the Development of Therapeutics from the Broad Institute of MIT and Harvard to identify small molecule drugs that could increase DHPS enzyme activity in patients by enhancing activity of the hypomorph DHPS N173S variant. This program is at an early stage, in assay development.

We are also collaborating with Dr. Przemysław Grudnik from the Jagiellonian University in Kraków to test in our in vitro models the small molecule DHPS activators that his group is developing by combining cryo-EM analysis of DHPS enzyme and a fragment-to-drug screen (see previous publication). This program is also at an early stage, in hit identification.