It is exactly what we were hoping for. Exactly what we dreamed about long ago. Big Pharma in the form of GSK has made a deal with Prosensa. PPMD and many other organizations have provided seed funding… funding in small bits to grow the work at each step along the way with the understanding that success would attract money and partnerships. The strategy worked!
Today is monumental for Duchenne.
Some months ago, I presented at BIO. Rare Disease was a focus of one of the talks. The gist of the discussion was that there would be significantly less interest and investment for common conditions. Who needs a better statin drug for cholesterol? The panel agreed, the focus will be rare disease for several reasons:
1. high unmet medical need
2. successful partnerships with advocacy (disease specific groups)
3. advocacy efforts are essential to seed projects and break down barriers
4. Genzyme and others have demonstrated investment with return in rare disease.
It is our day. PTC and Genzyme have partnered on Ataluren/PTC124. GSK and Prosensa partnering on exon skipping. And there will be more.
Thankfully, the sun is shining on Duchenne.