ViaNautis’ non-viral delivery mechanism, polyNaut®, aims to improve delivery of genetic therapies to the cells of people with CF.
Since 2011, the Foundation has awarded $2.5 million across 35 different projects to improve cystic fibrosis newborn screening across the country.
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The Cystic Fibrosis Foundation is providing additional funding to Arcturus Therapeutics to develop an inhaled messenger RNA therapy for CF that provides lung cells with the correct instructions to make functional cystic fibrosis transmembrane conductance regulator protein. The funding brings our total commitment to Arcturus to approximately $25 million.
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Nosis Bio, an inaugural winner of the Foundation’s Golden Ticket Competition, receives funding to further explore design of novel ligands, which are specialized molecules that could help more precise targeting of relevant cells in the lung.
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The Cystic Fibrosis Foundation is providing additional funds to TB Alliance to advance the development of a compound that could be used to treat infections caused by nontuberculous mycobacteria.
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Funding will support clinical trials for therapy that could help improve digestion in people with CF
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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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Funding will support preclinical research and early-stage clinical trials
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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.
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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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