Award
NIH Reporter #3R01NS120485-05S1
Design, Synthesis and Efficacy of New Small Molecule Therapeutics to Impede Myotonic Dystrophy
Recipient
State University of New York at Albany
Award Amount
$13,263.00
Ceiling
$13,263.00
Awarded
September 02, 2026
Identifier
3R01NS120485-05S1
The award funds research to develop and evaluate new small molecule therapeutics for Myotonic Dystrophy using a design-model-test cycle to refine compounds and assess efficacy in cell lines and animal models, aiming to support future clinical studies.
Description
This project 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, systematically refine their efficacy and test their therapeutic potential in patient-derived DM cells lines and animal models. The successful completion of this project will provide a new class of small molecules, a better understanding of their mechanism of action and in vivo data from multiple animal models supporting their future therapeutic potential – all critical steps towards future clinical studies for this complex and devastating disease.