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.
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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 providing additional funds to TB Alliance to advance the development of a compound that could be used to treat infections caused by nontuberculous mycobacteria.
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.
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.
ViaNautis’ non-viral delivery mechanism, polyNaut®, aims to improve delivery of genetic therapies to the cells of people with CF.
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.
Funding will support preclinical research and early-stage clinical trials
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.
As someone who does not qualify for Trikafta®, I took particular interest in the first plenary of the North American Cystic Fibrosis Conference, which discussed potential treatments for people with nonsense and rare mutations. I learned that researchers are exploring a variety of options, and some early research has shown promising results.