Drugs Discovery Assets:


SMA Foundation

The SMA Foundation has invested more than $40M on developing a portfolio of drug discovery assets essential for moving treatments from the laboratory through the clinic. These assets include validated in vivo and in vitro research tools, biomarker and natural history of SMA, and a trial-ready clinical network. The SMA Foundation has spearheaded the implementation of licensing policies that help make existing tools available to the SMA research community. In cases where critical tools do not exist, the SMA Foundation has worked to generate the necessary assets. Our mandate is to ensure that all assets we have developed or supported are made readily and rapidly available to researchers at minimal cost or obligation, thereby significantly lowering the barriers to entering the field for researchers or companies interested in SMA drug development.