Cystic Fibrosis Foundation Launches Inaugural Golden Ticket Competition to Find the Next Genetic Therapies for CF

April 18, 2022 Off By BusinessWire

Early stage researchers, life science entrepreneurs, and companies are encouraged to apply starting May 2

BETHESDA, Md.–(BUSINESS WIRE)–Today, the Cystic Fibrosis Foundation announced its first-ever “Golden Ticket” Competition to find the next promising treatment for cystic fibrosis in a collaboration with Bakar Labs, the incubator at the University of California, Berkeley’s Bakar BioEnginuity Hub. Researchers developing new technologies in gene editing, gene delivery, and gene therapy/gene insertion that have potential application to cystic fibrosis are invited to apply for the competition starting May 2. Up to three winners will receive one year of free laboratory space and support at Bakar Labs to help advance their transformative work.

“As part of our relentless pursuit of a cure, we are dedicated to investing in early stage science. We know that to make progress toward our bold mission to cure CF, we need to attract bold, like-minded researchers,” said Martin Mense, PhD, senior vice president of drug discovery and director of the Cystic Fibrosis Foundation Therapeutics Lab in Lexington, Mass. “This Golden Ticket Competition provides the incredible resources of both the Foundation and Bakar Labs to help great minds bring ambitious ideas closer to reality.”

The CF Foundation is offering winning applicants an opportunity to tap into its unparalleled research infrastructure along with access to the world’s foremost research and knowledge in cystic fibrosis. This includes scientific experts and advice, lab/research tools and techniques, cell and sample collections, an extensive Patient Registry, clinical trial design support, and a Therapeutics Development Network of more than 90 clinical trial sites in the U.S.

Mense continued, “We refuse to wait for scientific advances to come to us. Instead, we are constantly exploring new approaches to drive research, to bring tomorrow’s breakthrough technologies into cystic fibrosis research today.”

Much of the Cystic Fibrosis Foundation’s success is attributed to its long history of taking risks on early stage science. Starting with funding academic research grants, over time the Foundation began investing in companies to accelerate the pace of research. Over the past two decades, the Foundation has invested in close to 200 companies to help them de-risk upfront investments.

By spearheading this competition, the Foundation is taking a step further — than it ever has before — by extending an invitation to be part of its legacy to a new audience of scientists who are in the very earliest stages of translating their research into a potential therapy. Some of the most exciting research today is being done at academic labs by scientists who want to launch new biotech companies, and the Foundation hopes to champion these burgeoning researchers in those efforts.

Applications for the Golden Ticket Competition will be open from May 2-30, 2022. Finalists will be invited to present to a panel of judges from the Cystic Fibrosis Foundation and Bakar Labs during an in-person pitch day event on July 20, 2022 at Bakar Labs.

For more information on eligibility and to apply, visit the Golden Ticket Competition page.

Building on a Legacy of Progress in Cystic Fibrosis

Cystic fibrosis is a recessive, genetic disease caused by mutations in the gene that produces the cystic fibrosis transmembrane conductance regulator (CFTR) protein. Mutations in the CFTR gene can disrupt the normal production or functioning of the CFTR protein found in the cells of the lungs and other parts of the body. More than 30,000 people in the U.S. and 70,000 worldwide of every racial and ethnic group have cystic fibrosis – a disease that is life-limiting, causes long-lasting lung infections, limits the ability to breathe over time, and leads to many other complications throughout the body.

In the more than 65 years since the Foundation was established, tremendous advances in research and care — including more than 16 approved therapies — have helped extend the lives of people with CF. Some of those treatments — called CFTR modulators — address the underlying cause of disease for many people with CF.

Recently, the median age of survival for people with CF born between 2016 and 2020 was estimated at 50 years – monumental progress from when children rarely made it to kindergarten in the 1950s. Improvements in care and the dedication from the CF community have added hope and decades of life for people with CF. Despite significant progress in treatments and care, there is no cure yet.

While many people with CF have had incredible improvements in their health because of CFTR modulators, the disease can be caused by some mutations that can’t benefit from these treatments. People who have two nonsense mutations (also known as “x” or stop mutations) or other rare mutations that don’t produce any CFTR protein are such examples. That’s why in 2019 the CF Foundation established its $500 million Path to a Cure to accelerate treatments and drug development for the underlying cause of the disease and ultimately deliver a cure for all people with cystic fibrosis, regardless of their mutations. The Golden Ticket Competition is just one approach on the Path to a Cure in pursuit of the mission to help people with CF live longer, healthier lives. Learn more about the Foundation’s approach to research and clinical trials on cff.org.

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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