San Diego-based Celladon has made a big splash in the U.K. British scientists are playing a prominent role in a pair of clinical trials of Celladon's Mydicar, a gene therapy for heart failure which uses a benign virus to insert the Serca2a gene into heart cells. The treatment is designed to spur an ailing heart to pump up blood flow in patients, and following the pioneering approval of the world's first gene therapy last fall it's become a prime example of the burgeoning hopes for the field.
"It looks as though we may have stumbled on an important pathway that nature uses to regulate heart contractility," Chuck Murry, director of the Centre for Cardiovascular Biology, tells Reuters' Ben Hirschler, who wrote an in-depth report on the experimental treatment and the rise, fall and return of gene therapy work.
"It is a great example of the slow burn of good laboratory science translating into a potential clinical treatment," noted Peter Weissberg, the medical director of the British Heart Foundation, which is helping to finance one of the two trials.
The Financial Times explains that the UK will host one of the international arms of the Mydicar study, while a separate study looks at the effectiveness of the gene therapy in combination with mechanical heart pumps.
"Once heart failure starts, it progresses into a vicious cycle where the pumping becomes weaker and weaker, as each heart cell simply cannot respond to the increased demand," cardiologist Alexander Lyon tells the FT. "Our goal is to fight back by targeting and reversing some of the critical molecular changes arising in the heart when it fails."
Heart drugs traditionally require years of late-stage research and huge trials, and Mydicar is no exception. Its backers, though, include three Big Pharma venture arms and the company, a 2012 Fierce 15 biotech, has rounded up some impressive financing.
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