RARECast is a Global Genes podcast hosted by award-winning journalist Daniel Levine. It focuses on the intersection of rare disease with business, science, and policy.
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.