Our Venture Philanthropy Model

In 1989, a team of scientists supported by the CF Foundation discovered the cystic fibrosis gene and opened the door to our understanding of the disease at its most basic level.

People with CF and their loved ones hoped 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 proved difficult.

By the late 1990s, two important CF drugs that helped battle some of the devastating symptoms of CF had been approved. 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, Foundation President and CEO Robert J. Beall, PhD, landed on an idea known as venture philanthropy to break through the pharmaceutical industry’s reluctance to get involved in cystic fibrosis research. 

Venture philanthropy, in which a nonprofit organization uses investment strategies to support a for-profit company with the aim of advancing the organization’s mission, was an entirely new approach to funding research and drug development. Most disease nonprofits had traditionally focused their fundraising dollars on academic and medical research. It was considered an unconventional strategy and a big bet with no guarantees. 

In 2000, the Foundation made its first large investment: $40 million with Aurora Biosciences (now Vertex Pharmaceuticals) to discover compounds that might correct the malfunctioning protein in people with CF. Aurora specialized in high throughput screening, a then-novel technology that uses robots to test the therapeutic properties of thousands of chemical compounds per day in cells in laboratory dishes. 

It became clear that our venture strategy was a success in 2012, when the U.S. Food and Drug Administration (FDA) approved the first drug to address the underlying cause of cystic fibrosis: ivacaftor (Kalydeco).  

In the years that followed, our venture philanthropy model supported the development of additional therapies, including Orkambi (lumacaftor/ivacaftor) and Symdeko (tezacaftor/ivacaftor), that benefited more people with CF, including those with the most common mutations.

In 2014, the Foundation sold royalty rights for CF treatments developed by Vertex for $3.3 billion to Royalty Pharma, bringing resources to the fight against CF never thought possible. And in 2020, we secured even more resources when we successfully completed a sale of our remaining stake in royalties related to Vertex’s therapies for an upfront payment of $575 million and a potential future payment of $75 million.

The funds have been used to accelerate the development of lifesaving new therapies, to provide high-quality, specialized care and programs that support people with CF and their families, and to pursue new opportunities that could lead to a lifelong cure.

In 2019, the FDA approved Trikafta (elexacaftor/tezacaftor/ivacaftor), a modulator therapy treating the underlying cause of disease for more people than ever before, marking a major milestone in the development of treatments for CF. Then in 2024, the FDA approved Alyftrek (vanzacaftor/tezacaftor/deutivacaftor), a once-a-day modulator therapy for people with CF aged six and up with at least one responsive mutation, including 31 rare mutations that were previously not responsive to modulators.

While CFTR modulator therapies have continued to change the lives of many people with CF, not everyone can benefit. Our goal is to provide a cure for everyone with the disease. As of 2024, the Foundation has funded more than $500 million in research to advance transformative treatments for the underlying cause of CF.

While we have achieved life-changing gains for people with CF, we have more to do. Today, CF Foundation Mission Ventures harnesses the Foundation’s robust scientific infrastructure and employs venture investment best practices to source, evaluate, fund, and manage projects that advance our mission. 

Through Mission Ventures, the Foundation selectively invests in companies pursuing genetic therapies that offer the best chance of treating the disease’s underlying cause as well as projects aimed at relieving symptom burden for everyone with CF. All financial returns are reinvested into advancing the Foundation’s mission, including treating and ultimately curing CF.