Rarecast

Ilan Ganot is not your typical biotech entrepreneur. When his son Eytani was diagnosed with Duchenne muscular dystrophy, he quit his job as a hedge fund manager with JPMorgan in London, relocated his family to Boston, and launched a company focused exclusively on finding treatments for Duchenne. We spoke to Ganot, CEO and founder of Solid Ventures, about his company, the thinking behind his business model, and whether his approach is transferable to other rare diseases.

Prosensa, a biotechnology company developing a breakthrough therapy for the rare, fatal, genetic disease Duchenne muscular dystrophy suffered a big setback when disappointing, late-stage trial results of its lead therapeutic led its partner, GlaxoSmithKline, to return rights to the experimental drug in January 2014. Prosensa’s stock plummeted and the future of the company was uncertain. CureDuchenne, which had provided early funding for the company, stepped in once again. This time it made a $7 million commitment to restore the clinical programs and move the company forward. We spoke to Debra Miller, president and CEO of CureDuchenne about her experience with Prosensa, the recent news that BioMarin would acquire the company for up to $840 million, and the role venture philanthropists like her can play in accelerating the development of life saving drugs.

News last month of the Cystic Fibrosis Foundation’s $3.3 billion sale of royalty rights to Kalydeco and other Vertex Pharmaceuticals drugs it helped fund served as a bold statement to the growing prominence and power of venture philanthropy. We spoke to Margaret Anderson, executive director of FasterCures, about the transaction, the role new funding and collaboration models are playing in accelerating drug development, and what traditional investors and disease groups are learning from each other.