Delivery of genetic therapies to affected tissues is a key challenge to developing new treatments for people with cystic fibrosis. In addition to investing in viral delivery and lipid nanoparticles, the Cystic Fibrosis Foundation is looking at the potential of a relatively new approach to delivery using an exceptionally small synthetic molecule called a polymer nanoparticle.
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The Cystic Fibrosis Foundation’s investment will go toward conducting preclinical research on a novel gene delivery vehicle. If successful, this gene delivery method could overcome some of the biggest challenges to delivering a gene therapy into the lung cells of people with cystic fibrosis.
The Cystic Fibrosis Foundation is providing up to $5 million to develop a method to deliver a healthy copy of the CFTR gene into the lung cells of people with CF that is unlikely to trigger an immune system response.
Carbon Biosciences is the first company to publicly launch from the Foundation’s collaboration with Longwood Fund and the first to work alongside CF Foundation researchers at the Foundation’s therapeutics lab
New modulators in development by the CF-specific company could provide unique treatment options for the underlying cause of cystic fibrosis for many people with the disease
The Cystic Fibrosis Foundation is awarding up to $15.9 million in additional funding to Eloxx Pharmaceuticals Inc. to expand clinical studies of ELX-02, a potential therapy for people with CF who have nonsense mutations.
The Foundation’s award will support preclinical studies of a potential phage therapy to treat resistant Pseudomonas infections.
This milestone was reached nearly two years ahead of the initiative’s five-year commitment. However, the Foundation aims to continue to support infection research at the same pace as it has in the past three years.
The Cystic Fibrosis Foundation awarded up to $3.5 million to Arrevus Inc. to test a potential treatment for pulmonary exacerbations in people with cystic fibrosis in a late phase clinical trial.
Investment will support discovery research of a novel Gene CodingTM approach that could benefit all people with CF regardless of their mutation