Genetic medicines aim straight for the heart
, a firm founded by Christine Seidman and her husband Jonathan Seidman and later acquired by Bristol Myers Squibb, it works by decreasing the elevated contractility that arises from excessive cross-bridge formation between actin and myosin, the proteins responsible for generating the force that gives rise to heart contractions. Cytokinetics is in phase 3 in obstructive HCM with another oral myosin inhibitor, aficamten.
Tenaya Therapeutics is several years behind Rocket Pharma but is about to move its lead gene therapy program, TN-201, into a phase 1b trial in patients with HCM due to mutations in thegene, which encodes myosin-binding protein C3. The protein is involved in the organization and function of sarcomeres, the basic contractile units of cardiac muscle.
Olson is a co-founder of Tenaya, but rights to his more recent gene editing work in cardiovascular disease have yet to be assigned. Olson’s group developed both adenine base editing and prime editing approaches to correct pathogenic
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