Biotech Values

[poorgradstudent]

Re: heart failure / gene therapy success

2. Heart failure explodes on the biopharma scene with gene therapy success

Who is working on it? What projects are in the works? What are they targeting?

I know that BMY and Myokardia are in this area, and Merck is also looking into it.

The primary interest is in sarcomeric proteins like myosin, myosin binding protein-C, actin, etc... They're focusing on the mutations in these proteins found in some hypertrophic and dilated cardiomyopathies. The thinking is that the mutations allow them to streamline the clinical trial process, running smaller and more focused trials with a relatively homogenous patient population to increase chances of success.

However, none of these programs are anywhere near showing clinical gene therapy success next year. The companies are throwing around various sorts of strategies to target these mutations, most of which I'm rather unexcited about. In addition, some of their targets are junk (see: myosin binding protein-C) and I've indicated as such to some people involved.

Time will tell which direction they go, but nothing is close right now.