Award
NIH Reporter #5R01NS120485-05
Design, Synthesis and Efficacy of New Small Molecule Therapeutics to Impede Myotonic Dystrophy
Recipient
State University of New York at Albany, NY
Award Amount
$502,351.00
Ceiling
$502,351.00
Awarded
April 02, 2026
Identifier
5R01NS120485-05
The award funds research on new small molecule therapeutics to treat Myotonic Dystrophy by studying modified polycyclic compounds to understand their mechanism and therapeutic potential in cell and animal models.
Description
This proposal uses in vitro, in cellulo, and in vivo strategies to understand the mechanism of action of a new class of small molecules, modified polycyclic compounds (MPCs), to address the unmet need for therapeutics for Myotonic Dystrophy (DM). A design-model-test cycle will be used to understand the mechanism(s) of action of these new compounds, refine their efficacy, and test their therapeutic potential in patient-derived DM cell lines and animal models. Successful completion will provide a new class of small molecules, a better understanding of their mechanism of action, and in vivo data supporting their future therapeutic potential.