Greenstone Biosciences Secures NIH R61 Grant to Develop iPSC and AI-Driven Therapies for DMD Cardiac Fibrosis
核心洞察
Greenstone Biosciences (搜索) received a Catalyze R61 award from the NIH's NHLBI to discover novel drug candidates targeting myocardial fibrosis (搜索) and dilated cardiomyopathy (搜索) in Duchenne muscular dystrophy (搜索).
DMD affects approximately one in every 3,500 baby boys worldwide, with cardiomyopathy representing the leading cause of death and no approved therapy directly addressing heart scarring.
The program leverages patient-derived iPSC cardiomyocyte models, unbiased proteomics, computational drug screening, and generative AI to identify and validate new drug candidates.
Greenstone Biosciences (搜索), a Palo Alto-based biotechnology company focused on New Approach Methodologies (NAMs) for drug discovery, has been awarded a Catalyze R61 grant from the National Heart, Lung, and Blood Institute (NHLBI), part of the National Institutes of Health (NIH). The funding will support a research program aimed at discovering novel drug candidates for myocardial fibrosis (搜索) and dilated cardiomyopathy (搜索) in patients with Duchenne muscular dystrophy (搜索) (DMD).
DMD is a rare genetic disorder affecting approximately one in every 3,500 baby boys worldwide. Cardiac complications, particularly cardiomyopathy, are prevalent among individuals with DMD and represent the primary cause of mortality. While corticosteroids (搜索) can delay heart failure and improve survival, more than a quarter of patients either cannot tolerate these treatments or fail to respond. Critically, no approved therapy exists that directly targets the heart scarring characteristic of the disease — a gap this program is designed to fill.
A Novel iPSC and AI-Driven Discovery Platform
The research program will harness Greenstone's induced pluripotent stem cell (iPSC) biobank and cardiomyocyte disease models, integrating unbiased proteomics, computational drug screening, and generative AI to identify and validate new therapeutic candidates for DMD cardiomyopathy. The Catalyze program follows a two-phase structure: the current R61 phase supports target identification and early candidate discovery, while successful progression to the R33 phase would fund synthesis, characterization, and in vivo testing of promising compounds, ultimately advancing toward IND-enabling studies.
"DMD cardiomyopathy has gone unaddressed for too long, in part because the tools to model it faithfully in the lab have not existed," said Dr. Joseph C. Wu, co-founder of Greenstone Biosciences (搜索) and Director of the Stanford Cardiovascular Institute. "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."
Regulatory Tailwinds for NAMs
The award arrives amid a rapidly shifting regulatory landscape favoring human-relevant models in drug development. The FDA's 2025 Roadmap to Reduce Animal Testing and its April 2026 progress report have made the ISTAND program permanent, established a NAMs Acceptability Database, and approved the agency's first AI-based drug development tool for clinical trials. Concurrently, Congress has advanced the FDA Modernization Act 3.0, which would align agency regulations with the nonclinical testing authority granted in 2022. The bill passed the House on July 20, 2026, and now awaits a final Senate vote before proceeding to the President. These developments represent some of the strongest legislative and regulatory support yet for human-relevant models in drug development, providing a favorable environment for Greenstone's iPSC-based discovery approach.