CF Foundation Venture Philanthropy Model

Through its venture philanthropy model, the Cystic Fibrosis Foundation provides early stage funding to biotechnology and pharmaceutical companies to develop breakthrough drugs for adults and children with cystic fibrosis.

A Strategy to Change CF History

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

People with CF, and their friends and families, were hopeful that this discovery would quickly lead to new drugs to fight this rare and fatal disease. But progress stalled. The challenge of taking scientific knowledge from the laboratory and transforming it into 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 development to address the underlying cause of the disease.

Did You Know?

The CF Foundation supported development of vital drugs to treat CF including lumacaftor/ivacaftor (Orkambi®), ivacaftor (Kalydeco®), aztreonam (Cayston®) and inhaled tobramycin (TOBI®).

At the time, pharmaceutical companies had little to no interest in rare diseases and were focused on developing the next big blockbuster drug -- one that would help tens of millions of people and result in large profits.

In 1998, the median predicted survival age of a person with CF was 32 years. People with CF and their families were desperate for faster progress. With a growing sense of impatience, Robert J. Beall, Ph.D., then president and CEO of the Cystic Fibrosis Foundation, landed on an idea that would break through the pharmaceutical industry's reluctance to get involved in cystic fibrosis research.

The CF Foundation pioneered venture philanthropy and led the movement of voluntary health organizations funding drug development with for-profit companies. This was a monumental shift as traditionally most disease nonprofits had focused their fundraising dollars on academic and medical research.

This unconventional strategy was considered a major gamble with no guarantees. 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 genetic defect in people with CF. It was unknown that such a compound existed. The Foundation chose to work with Aurora because the company specialized in high through-put screening, a unique technology that used robots to test the therapeutic properties of thousands of chemical compounds a day in cells in laboratory dishes.

Building the CF Drug Pipeline

Aurora was the first company to tackle CF research supported by funding from the Foundation, but many were to follow.

Today the Foundation is funding research programs with leading biopharmaceutical companies worldwide, including Pfizer, Genzyme, Editas, and Corbus. In total, the Foundation has invested $425 million as part of its venture philanthropy model to accelerate drug development for cystic fibrosis.

Breakthrough Therapies for People With CF

January 31, 2012, marked a historic day for the CF community. The U.S. Food and Drug Administration (FDA) approved the first drug to address the underlying cause of cystic fibrosis, ivacaftor (Kalydeco®). The Foundation's targeted investment with Aurora Biosciences had paid off for thousands of people with CF.

Many people taking the drug have experienced a profound difference in their day-to-day lives. After Lindsay Shipp started taking ivacaftor as part of a clinical trial, she was told at a regular check-up that her lungs were functioning at 96 percent -- almost at the same level as someone without the disease. “I realized I would have a full life at that moment,” she told the Los Angeles Times.

Ivacaftor treats a minority of the CF population, but a wider swath of the community received a treatment option when the FDA approved the first combination therapy in July 2015. Lumacaftor/ivacaftor (Orkambi®) addresses the underlying cause of CF in half of the CF population. More clinical trials are testing potential therapies that would help nearly all people with CF.

“The unique and mutually beneficial partnership that led to the approval of Kalydeco serves as a great model for what companies and patient groups can achieve if they collaborate on drug development.”

- Margaret A. Hamburg, M.D., former FDA Commissioner

Super-Charging the Search for a Cure

In 2014, the Foundation sold royalty rights for CF treatments developed by Vertex for $3.3 billion, bringing resources to the fight against CF never thought possible. Under terms of the agreement with Royalty Pharma, it is possible the Foundation may receive additional funds in future years.

The funds will be used to accelerate the development of lifesaving new therapies, to provide high-quality care and programs that support people with CF and their families, and to pursue daring new opportunities to one day find a lifelong cure.

Praise for the Venture Philanthropy Model

The Foundation's model has been nationally recognized and praised by leaders in medicine, business, and health care advocacy.

One of the highest honors came from President Barack Obama. During his 2015 State of the Union address, he cited the story of cystic fibrosis as an example of how nonprofits, the pharmaceutical industry, researchers, patients, and their families can work together to produce more targeted and effective treatments for diseases.

The approach has been widely emulated by many other rare disease nonprofits, and a National Institutes of Health initiative has adopted CF Foundation strategies to advance drug development for rare and neglected diseases.

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