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.
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.
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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.
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The Foundation’s award will support preclinical studies of a potential phage therapy to treat resistant Pseudomonas infections.
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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.
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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.
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The Cystic Fibrosis Foundation awarded up to $4.7 million to EnBiotix Inc. to study the potential use of inhaled colistin as an additional option to treat Pseudomonas infections in people with cystic fibrosis who are not responding to current treatments.
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The Cystic Fibrosis Lung Transplant Consortium Biorepository and Patient Registry, in collaboration with Cleveland Clinic, will provide critical clinical data and samples to support future research investigating complications of lung transplant.
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Today, the Cystic Fibrosis Foundation announced a collaboration with Deep Science Ventures to accelerate treatments that address the underlying cause of CF for every person with the disease. The collaboration will focus on uncovering and designing new technologies to address key scientific challenges on the Path to a Cure.
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New funding awards include up to $2.6M to Eloxx Pharmaceuticals to identify potential therapies for CF nonsense mutations
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