Trials begin on gene therapy for hypertrophic cardiomyopathy

31st March 2015

A company in America has begun trialling a gene therapy treatment for hypertrophic cardiomyopathy (HCM).

The company, MyoKardia, says it is the first ever therapy designed to target the underlying cause of hypertrophic cardiomyopathy in patients with a particular genetic make- up.

People with HCM have mutations that cause the heart muscle to thicken and stiffen, which can cause the heart to contract too much.

The company says it has begun phase one of a clinical study designed to correct one of the most common molecular mechanisms causing HCM (identified from genetic testing) and reduce heart muscle contractility.

A spokesman said it is an important milestone for patients and the doctors who care for them.

Dr Tassos Gianakakos, chief executive officer of MyoKardia, said that by targeting an underlying molecular defect causing HCM, it was hoped the treatment (MYK-461) could restore normal heart muscle contraction and relaxation, and reduce or prevent disease progression.

MYK-461 is an oral, selective, small molecule allosteric modulator of cardiac myosin. The MYBPC3 gene provides instructions for making the cardiac myosin binding protein C (cardiac MyBP-C).

The phase 1 clinical trial will assess the safety, tolerability and effects of oral doses of MYK-461 in healthy volunteers. In parallel, there will also be a phase 1 study of MYK-461 in patients with HCM.