Cystic Fibrosis Foundation Strikes First-of-Its-Kind Deal With Pioneering Medicines, a Flagship Pioneering Initiative

Today, the Cystic Fibrosis Foundation announced a first-of-its-kind collaboration with Pioneering Medicines, an initiative of Flagship Pioneering, to spur the development of genetic-based therapies for cystic fibrosis. As part of its Path to a Cure research agenda, the Foundation expects to invest up to $110 million in the collaboration, with an initial commitment of $20 million and the option to invest additional funds as promising therapies arise. Through this agreement, Pioneering Medicines will combine technologies from several Flagship-founded companies to develop a separate company specifically focused on potential treatments for cystic fibrosis.

“By combining the CF Foundation’s scientific expertise with Pioneering Medicines’ and Flagship Pioneering’s emerging technologies and track record of innovation, we believe this agreement has the potential to push the field forward and accelerate progress toward future treatments for everyone with CF, regardless of their mutation,” said William Skach, MD, chief scientific officer of the Cystic Fibrosis Foundation.

Pioneering Medicines partners with multiple Flagship-founded companies to develop new technologies and medicines. Through this agreement, Pioneering Medicines and the Cystic Fibrosis Foundation will leverage several emerging technologies from companies within the Flagship ecosystem, including Gene Writing^TM from Tessera Therapeutics, targeted delivery modalities, and RNA technologies. The companies will work together to leverage each of their unique technologies in combination to advance research toward a cure for cystic fibrosis.

At the onset, the new company will focus on developing technology to determine whether it can create a functional CFTR protein in lung cells, and a gene writing approach that may enable the simultaneous correction of numerous types of mutations in the CFTR gene. These two strategies will be combined with a delivery approach focused on targeting the proper cells in the lung and potentially other tissues. Additionally, as part of the agreement, the CF Foundation will participate in a joint steering committee with key leaders from Pioneering Medicines and the Flagship companies to facilitate research and ongoing drug development decisions.

“We will not rest until every person with CF has a cure. This collaboration marks an evolution in our approach to venture philanthropy and expands our footprint to bring the best scientific minds and technologies into CF,” said Michael Boyle, MD, president and chief executive officer of the Cystic Fibrosis Foundation.

Our Approach to Innovation

The CF Foundation’s established venture philanthropy model provides early-stage funding to companies to develop breakthrough treatments for adults and children with cystic fibrosis. This approach has super-charged the field of CF by helping to de-risk and accelerate the development of new technologies in CF, resulting in more than 16 approved therapies – including medicines that treat the underlying cause of CF for many people with the disease.

This progress has added decades of life and hope for people with CF, but critical work remains to ensure that every person has an effective therapy to address their underlying cause of disease. In 2019, the Foundation announced its $500 million Path to a Cure, an ambitious research agenda to accelerate treatments for individuals who are still waiting for a breakthrough and cure CF for all people living with the disease.

The Foundation continues to evolve its venture philanthropy approach to take advantage of emerging science with the potential to benefit individuals with CF who are most in need of new therapeutic options. Today, the Foundation is investing more aggressively than ever before to support development of the next generation of transformative therapies to help people with CF live their longest, healthiest lives possible.

About the Cystic Fibrosis Foundation

The Cystic Fibrosis Foundation is the world's leader in the search for a cure for cystic fibrosis. The Foundation funds more CF research than any other organization, and nearly every CF drug available today was made possible because of Foundation support. Based in Bethesda, Md., the Foundation also supports and accredits a national care center network that has been recognized by the National Institutes of Health as a model of care for a chronic disease. The CF Foundation is a donor-supported nonprofit organization. For more information, visit cff.org.

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