Positivo wins ‘Golden Ticket’ to advance CF gene therapy platform
Award created last year by CF Foundation, University of California, Berkeley
Positivo Biotechnology, a company working to develop new gene therapy technologies for cystic fibrosis (CF), has been selected as the winner of this year’s Golden Ticket Award.
The award, launched last year by the Cystic Fibrosis Foundation and the University of California, Berkeley’s Bakar Labs, aims to help companies that are developing new genetic medicines for CF. As this year’s winner, Positivo will receive one year of free office and lab space at Bakar Labs to work on its technology — and can access the expertise of the CF Foundation.
“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,” Martin Mense, PhD, senior vice president of drug discovery and director of the CF Foundation Therapeutics Lab, said in a press release from the nonprofit.
“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,” added Mense, who also serves as one of the contest’s judges.
Positivo’s CF gene therapy candidate uses different strategy
CF is caused by mutations in the gene CFTR, which result in defective or absent CFTR protein. Without a functional version of this protein, the body produces unusually thick and sticky mucus, which builds up in organs to drive most CF symptoms. Treatments called CFTR modulators can boost the functionality of the defective protein, but they only work in patients with specific disease-causing mutations.
The basic idea behind gene therapy for CF is to deliver a healthy version of the CFTR protein to the body’s cells, allowing the cells to produce functional CFTR protein. In theory, this approach has a notable advantage in that it should work equally well in all patients regardless of their specific mutation.
But getting a new, healthy gene into the body’s cells can be a tricky process. Many experimental gene therapies aim to deliver genetic cargo using viral vectors, which essentially are viruses that have been engineered to safely deliver a therapeutic gene rather than cause an infection.
Viral vectors have shown promise, but they have some notable drawbacks — in particular, the body’s immune system may respond to vectors the same way it responds to an infection. This can increase the risk of side effects, and it generally means that viral gene therapies can only be used once, because the immune system will develop immunity to the vector just as it would to an infection.
Positivo is working to develop a different strategy for CF gene therapy. Instead of a modified virus, the company aims to deliver genetic cargo using lipid nanoparticles, which basically are small bubbles of fat molecules that can carry molecules like DNA into cells.
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.
“We are thrilled to begin working with Positivo Biotechnology,” Mense said, noting that “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,” Mense said.
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