HEAL-MI: HMGA1-Enabled AAV-based Therapy for Left Ventricular Repair after Myocardial Infarction
Proof-of-Concept in a relevant pig model paving the way to treatment of Myocardial Infarction patients
Hubrecht Institute and Myogene Therapeutics enter a public-private partnership to develop an innovative gene therapy that can regenerate heart muscle cells after a myocardial infarction and thereby preventing heart failure.
Heart Failure is the common outcome in 30% of patients after suffering a myocardial infarction, since the adult heart muscle cells, so called cardiomyocytes, cannot regenerate after injury and are replaced by scar tissue. Current treatments are symptomatic, preserve only the residual cardiac capacity and do not lead to restoration of heart tissue and function. Therefore, Myocardial Infarction-induces Heart Failure remains a severe unmet medical need with a burden on healthcare costs of more than 30 B$ per year.
Unlike the mammalian heart, zebrafish possess a remarkable ability to regenerate their heart tissue following injury. This regenerative capacity has led to the identification of a lead candidate gene, HMGA1, that upon introduction to damaged heart in mice reduces scar formation and induces proliferation of cardiomyocytes leading to improved heart function.
This HEAL-MI project will 1) select the most effective and safe delivery route of the HMGA1 gene to the injured heart and 2) proof the concept of HMGA1-induced heart regeneration in a relevant Myocardial Infarction model in pigs whose hearts are functionally closest to human hearts.
In the end, this HMGA1 gene therapy aims to activate the heart’s own ability to regenerate in a spatially and temporally controlled way, moving beyond symptom management to true heart regeneration and improving patient outcomes post myocardial infarction.
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