Greenstone Biosciences has received a Catalyze R61 award from the National Heart, Lung, and Blood Institute to support the discovery of new drug candidates targeting myocardial fibrosis and dilated cardiomyopathy in Duchenne muscular dystrophy. The National Heart, Lung, and Blood Institute is part of the National Institutes of Health.
Duchenne muscular dystrophy is a rare genetic disorder affecting approximately one in every 3,500 baby boys worldwide.
Cardiomyopathy and other heart complications are major causes of mortality among patients with DMD. Although corticosteroids can delay heart failure for some patients, more than one-quarter either cannot tolerate the drugs or do not respond adequately.
Greenstone said there is currently no approved therapy that directly addresses the cardiac fibrosis associated with DMD.
The company will use its induced pluripotent stem cell biobank and cardiomyocyte disease models alongside proteomics, computational drug screening and generative AI to identify and validate potential therapies.
The Catalyze program is structured in two stages. The R61 phase will support target identification and early drug candidate discovery.
Successful programs could advance into an R33 phase supporting compound synthesis, characterization and in vivo testing before potential IND-enabling studies.
Greenstone’s platform includes an iPSC biobank containing samples from more than 2,500 donors, including rare and orphan disease cell lines.
The company uses patient-derived cardiomyocytes, organoids and other iPSC-derived cell types to model human diseases and combines those models with AI-driven target identification and ADMET prediction.
KEY QUOTE:
“DMD cardiomyopathy has gone unaddressed for too long, in part because the tools to model it faithfully in the lab have not existed. This award lets us apply patient-derived cardiomyocyte models and modern computational methods to a disease where the biology has been difficult to reach any other way.”
Dr. Joseph C. Wu, Co-Founder of Greenstone Biosciences
