BETHESDA, Md. -- The Cystic Fibrosis Foundation announced today that it has invested in SalioGen Therapeutics to support the company’s preclinical research into a novel genetic therapy for cystic fibrosis. SalioGen’s Gene CodingTM approach is designed to turn on, turn off, or modify the function of any gene in the genome.
“Genetic therapies are the key to curing CF and, while it is still very early days in this scientific quest, we’re investing in many different approaches that offer promise,” said Martin Mense, PhD, CF Foundation senior vice president of drug discovery and director of the CF Foundation Therapeutics Lab. “SalioGen’s Gene Coding approach seeks to insert a large piece of healthy CFTR DNA at a precise location within the CFTR gene, which could enable the expression of a functional CFTR protein in essentially all individuals with CF, regardless of their individual mutation.”
SalioGen’s Gene Coding approach aims to modify an individual’s DNA in a highly controlled manner to maintain the normal, regulated expression of genes such as cystic fibrosis transmembrane conductance regulator (CFTR). The company’s focus is to develop investigational treatments which add a healthy copy of genes for other diseases, and the CF Foundation’s investment will enable them to apply their novel approach to CF. SalioGen hopes that by inserting a large piece of the healthy CFTR DNA and enabling the expression of functional CFTR protein in the lungs of people with CF, there will be a reduction in the amount of mucus buildup in the lungs.
About Gene Coding
SalioGen’s unique Gene Coding approach aims to restore natural function of the gene by adding new DNA sequences at a precise location within the faulty native gene. The Gene Coding machinery utilizes a proprietary mammalian enzyme that is designed to allow the integration of DNA sequences without causing double-strand DNA breaks and potential unwanted DNA damage. Its machinery is being delivered using a lipid nanoparticle, which could allow potential re-dosing of the therapy with low risk of dangerous immune responses.
Understanding Genetic Therapies and Their Potential to Cure CF
The Foundation’s investment in SalioGen is part of our $500 million Path to a Cure initiative, an ambitious research agenda to deliver treatments for the underlying cause of the disease and a cure for every person with CF. This initiative funds research centered around three core strategies: repairing broken CFTR protein; restoring CFTR protein when none exists; and replacing or fixing the underlying genetic mutation with a genetic therapy, which is SalioGen’s area of focus.
The Foundation is currently funding many different genetic therapy programs. While this research is still in very early stages, scientists are pursuing several different approaches, including both gene therapy and gene editing.
Gene therapy involves inserting a new version of the CFTR DNA into the cells of people with cystic fibrosis. Although the old, mutated copy of the CFTR gene is still present in the genome, the new, correct copy gives the cells the ability to make normal CFTR protein. With SalioGen’s approach, the goal is to edit and correct the existing CFTR gene by inserting new CFTR DNA at a precise location within the faulty native gene in order to restore normal expression of CFTR protein.
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.
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.