Known as global pioneers of the cardiovascular genetics field, Christine “Kricket” Seidman, MD, and Jonathan Seidman, PhD, have made tremendous progress in research and care for people with genetic heart diseases. The Seidman Lab—based at the Brigham and Harvard Medical School (HMS)—has extensively studied cardiomyopathy, an often-inherited group of heart muscle diseases that put people at high risk for irregular heartbeat, stroke, and heart failure.

The scientists’ discoveries have led to better therapies to manage symptoms as well as genetic testing that can detect specific faulty genes and guide medical intervention. But challenges remain: Treatments are moderately effective, can cause burdensome side effects, and do not cure the underlying problem.

Now, an exciting project of the Seidmans is poised to turn the tide in developing therapies targeting the root issue. Recognizing this ambitious translational research will require flexible funding, an anonymous donor committed $15 million to support efforts including establishing patient registries, identifying biomarkers predictive for disease progression, studying molecular processes in diseased hearts, and developing novel effective therapeutics.

“We’re so thankful for this gift, which comes at a critical point and puts us firmly on the path toward new genetic therapies that precisely correct the faulty genes in the heart itself,” says Kricket Seidman, director of the Cardiovascular Genetics Center and the Smith Professor of Medicine.

With the funding, the team aims to develop therapies that can halt, treat, and cure cardiomyopathy caused by pathogenic variants in a critical protein involved in multifaceted essential functions of the heart.

“This work will create a platform to develop treatments for several genetic heart muscle conditions, which could help millions of people,” says Jonathan Seidman, the Bugher Foundation Professor of Genetics at HMS.

The Seidmans’ work is a flagship project of the Mass General Brigham (MGB) Gene and Cell Therapy Institute, which brings together the health system’s resources to boost groundbreaking research.

“There’s an incredible opportunity to leverage expertise across MGB to better understand and treat disease,” says Paul Anderson, MD, PhD, chief academic officer for MGB and the Austen Professor of Medicine. “This gift will accelerate the Seidmans’ work to treat, and ultimately cure, hereditary heart conditions.”