Targeting the cardiac sarcomere: How basic science led to treatment of a deadly disease

Place: INF669, 69120 Heidelberg Seminar Room 2nd floor, Date: Juli 12th, 2023 Time: 4:30 PM

• Speaker: Leslie Leinwand, PhD
• Distinguished Professor - Molecular, Cellular & Developmental Biology
• Chief Scientific Officer - BioFrontiers Institute
• University of Colorado Boulder

Hypertrophic cardiomyopathy (HCM) is the most common inherited heart disease and it is caused by hundreds of mutations in sarcomeric protein genes, the most common of which are in myosin heavy chain and myosin binding protein C. These are usually inherited in an autosomal dominant fashion and are the leading cause of sudden death in young people. The work that will be presented in this seminar describes the work that we and others have done to understand how mutations in myosin cause HCM. We have found that alterations in the motor activity of myosins contribute to the hypercontractility of HCM hearts. I, along with Jim Spudich, Jon Seidman and Kricket Seidman co-founded MyoKardia, Inc. and developed a small molecule inhibitor of myosin’s motor activity. This was approved by the FDA in 2022 for treatment of symptomatic obstructive HCM after very successful clinical trials. Prior to 2022, there were no primary therapies for HCM.

Host: Johannes Backs
Director of the Institute of Experimental Cardiology 
Department of Internal Medicine VIII
University of Heidelberg