Gene Replacement Therapy for Lamin-Related Cardiomyopathies
Phlox Therapeutics and the research team of Prof. Dr. Eva van Rooij at the Hubrecht Institute have joined forces in the RELAMIN project, a public-private partnership aimed at developing a groundbreaking gene therapy for lamin-related dilated cardiomyopathy. This collaboration seeks to address the pressing medical need for a cure for this severe inherited heart disease, leveraging cutting-edge science to deliver transformative outcomes for patients.
Lamin-related cardiomyopathies, caused by mutations in the Lamin gene, are life-threatening conditions that disrupt the heart's electrical and mechanical functions, often leading to severe heart failure or sudden cardiac death at a young age. Globally, 194,000 people are affected, including 420 patients in the Netherlands. Current treatments focus on managing symptoms with medications, defibrillators, and heart transplants, yet these options fail to address the underlying genetic cause, underscoring the urgent need for innovative solutions. This condition significantly reduces quality of life, increases mortality, and poses a societal and economic burden.
The RELAMIN project aims to develop a gene replacement therapy to restore normal cardiac function. Using advanced research tools such as human-derived cell cultures and genetically engineered mice, the project aims to replace the harmful Lamin gene in cardiac cells with a healthy copy. This approach is designed to precisely target the genetic cause of the disease, offering a potentially curative and long-lasting therapeutic solution.
The primary deliverable of the RELAMIN project will be a validated gene therapy candidate ready for clinical development. If successful, this innovative therapy could transform the treatment landscape for Lamin-related cardiomyopathies, providing hope to thousands of patients and positioning the Netherlands as a leader in advanced therapies for rare genetic diseases.
