Yesterday, at a reception on the Bakar Labs campus, the Cystic Fibrosis Foundation and University of California, Berkeley’s Bakar BioEnginuity Hub selected AVECRIS Pte Ltd and Nosis Biological Sciences as winners of the Foundation’s first-ever Golden Ticket Competition. They were each awarded one year of free office and lab space at Bakar Labs and access to the Foundation’s robust network of resources to develop their genetic therapy technologies to apply to cystic fibrosis.
As part of its relentless pursuit of a cure for cystic fibrosis, the Foundation teamed up with Bakar Labs for this competition to bring early-stage researchers who are developing new technologies in gene editing, gene delivery, and gene therapy/gene insertion into CF. Genetic therapies represent one of the most promising approaches for developing a cure for cystic fibrosis.
“Our winners are great examples of the companies the Foundation wants to attract – bold, innovative, and unafraid of the challenges ahead,” said Martin Mense, PhD, senior vice president of drug discovery, director of the Cystic Fibrosis Foundation Therapeutics Lab in Lexington, Mass., and Golden Ticket Competition judge. “These are the types of early-stage technologies we need to nurture today to bring us closer to a cure for CF tomorrow.”
About the Golden Ticket Competition Winners
Winners were selected by a judging panel composed of research and investment staff from the CF Foundation and leaders from Bakar Labs. They used criteria that included the technologies’ potential impact on people with CF and whether the company employed a novel approach to address CF.
Genetic therapies hold enormous promise to treat diseases like CF, but delivering them to affected tissues remains a key challenge. The winners for the Golden Ticket Competition are pursuing different ways of addressing this challenge.
AVECRIS is developing a novel non-viral gene delivery technology, which they call a “dumbbell DNA” vector, that can be inhaled or injected with the goal of it only working in the intended tissue.
With machine learning and high-throughput biological screening, Nosis is developing a platform to improve the on-target delivery and safety of genetic therapies.
Building on a Legacy of Progress in Cystic Fibrosis
In the more than 65 years since the Foundation was established, tremendous advances in research and care — including 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 2017 and 2021 was estimated at 53 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.
Although many people with CF have had incredible improvements in their health because of CFTR modulators, some people have mutations that can’t benefit from these treatments. This includes those who have two nonsense mutations (also known as “x” or stop mutations) or other rare mutations that don’t produce any CFTR protein.
By attracting new companies into CF research and de-risking early-stage science to develop treatments for CF, the Foundation is evolving its successful venture philanthropy model to meet these new challenges. That’s why in 2019 the CF Foundation established its $500 million Path to a Cure initiative 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.