The Cystic Fibrosis Foundation and University of California, Berkeley’s Bakar Labs announced  Positivo Biotechnology as the winner of this year’s CF Foundation-sponsored Golden Ticket Competition, which awards one year of free office and lab space at Bakar Labs to a company developing genetic therapy technologies that could be applied to cystic fibrosis. To help advance the company’s technologies, the Cystic Fibrosis Foundation will also provide access to its disease-specific resources and expertise. 

This year’s CF Foundation Golden Ticket Competition solicited applications from companies developing novel genetic therapy delivery technologies. Genetic therapies such as RNA therapy, gene therapy, and gene editing, are the most promising approaches to treating — and ultimately curing — all people with CF; but delivery of a genetic therapy to people with cystic fibrosis remains a challenge. Successful delivery mechanisms must be able to contain a large therapeutic cargo, avoid the lungs’ natural immune response, and target the correct cells.  

“Nascent biotech companies are working on some of today’s most exciting genetic therapy technologies, but they face many obstacles in the discovery and preclinical stages of research,” 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. “The Golden Ticket Competition enables the CF Foundation to use our resources and expertise to attract companies with promising technologies to CF research and de-risk these early stages of development.” 

About the Golden Ticket Winner 

Positivo Biotechnology was selected by a judging panel composed of research and investment leadership from the CF Foundation and Bakar Labs. Judges used criteria that included the technologies’ potential impact on people with CF and whether the company employed a novel approach to addressing challenges in genetic therapy delivery.  

Positivo Biotechnology is developing nonviral DNA gene delivery technologies to create proteins to treat various diseases including cystic fibrosis. It aims to address current challenges in developing gene therapies, including unwanted immune system response and toxicity, to safely and effectively deliver and express DNA-encoded therapeutics.

Mense continued, “We are thrilled to begin working with Positivo Biotechnology. The company is focusing on DNA delivery with novel lipid nanoparticles, a nonviral approach that, if successful, may be able to overcome several genetic therapy challenges, such as the need for frequent redosing. Positivo fits our strategy to continue identifying novel approaches to delivering CF genetic therapies, and ultimately moving forward the field of genetic therapies for other pulmonary and rare diseases waiting for their breakthroughs.” 

The winner of the last CF Foundation Golden Ticket Competition, Nosis Bio, is currently working at Bakar Labs developing specialized molecules that aim to more precisely target the relevant cells in the lungs. In addition to the Golden Ticket, the Foundation later funded Nosis with up to $2 million to progress their work. 

The CF Foundation’s Ecosystem of Research Support 

By working with the CF Foundation, companies benefit from access to the Foundation’s robust and diverse resources. 

Those resources include: 

• Comprehensive health information on people with CF in the U.S. from the CF Foundation Patient Registry, the largest CF-dedicated registry in the work which includes the majority of people in the U.S. with CF 

• Support from the CF Foundation Therapeutics Lab, a one-of-a-kind facility that is conducting research to advance CF genetic therapies, including an incubation space for biotech companies to work alongside CF Foundation scientists 

• Access to the CF Foundation-funded Therapeutics Development Network, the largest CF clinical trials network in the world 

• Connections to CF and genetic research expertise at the CF Foundation and institutions around the world 

Visit the CF Foundation’s academic and industry funding pages to learn more about open funding opportunities.