In 1989, a team of scientists supported by the Foundation discovered the cystic fibrosis
People with CF and their loved ones were hopeful that this discovery would quickly lead to new drugs to fight this rare and fatal disease. But the gene discovery alone wasn’t enough to spur innovation. The challenge of taking scientific knowledge from the laboratory and applying it to develop effective treatments for CF was a larger obstacle than anyone had anticipated.
By the late 1990s, two important CF drugs that helped battle some of the devastating symptoms of CF had been approved with the Foundation’s support. However, there were no breakthrough treatments in the pipeline to address the underlying cause of the disease.
By 1998, the median predicted age of survival for a person with CF was 32 years, and people with CF and their loved ones were desperate for faster progress. With a growing sense of impatience, Robert J. Beall, PhD, then president and CEO of the Foundation, landed on an idea called venture philanthropy to break through the pharmaceutical industry’s reluctance to get involved in cystic fibrosis research.
Venture philanthropy, where a nonprofit organization provides funding to a for-profit company to de-risk an investment, was an entirely new approach to funding research. This was a monumental shift because most disease nonprofits had traditionally focused their fundraising dollars on academic and medical research. It was considered an unconventional strategy and a major gamble with no guarantees.
With the support of our Board of Trustees, Dr. Beall began working the phones to find a willing partner. Only two drug companies returned his calls. In 2000, the Foundation made its first large investment: $40 million with Aurora Biosciences (now Vertex Pharmaceuticals) to discover compounds that might correct the core
It became clear in 2012 that our venture strategy was a success. The U.S. Food and Drug Administration (