The additional funding will support a Phase 2 clinical trial for an inhaled antisense oligonucleotide drug for people with cystic fibrosis who have the splicing mutation 3849+10Kb C-to-T.
Prime Medicine’s prime editing technology has the potential to bring genetic therapies to all people with cystic fibrosis, ultimately paving the way to a cure.
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.
Funding will support preclinical research and early-stage clinical trials
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.
Investment will support discovery research of a novel Gene CodingTM approach that could benefit all people with CF regardless of their mutation
Study reaffirms the Foundation’s commitment to advance solutions to the growing challenge of antibiotic resistance