Nanite Inc. has announced an investment from the Cystic Fibrosis Foundation of up to $2 million to develop gene delivery technologies for the lung. This builds on the $6 million seed financing recently announced by the company.\n\n\n\nOne of the most significant challenges in developing new therapies for patients with cystic fibrosis is effective drug delivery. Polymer nanoparticles (PNPs) are an innovative delivery modality and can offer several advantages over other means of genetic therapy delivery into cells. \n\n\n\nPNPs may be less likely to trigger an immune response than viruses and lipid nanoparticles, and may be more stable and versatile than lipid nanoparticles, which can be more limited in the amount of drug payload released to the cells.\n\n\n\nNanite platform\n\n\n\nNanite’s proprietary platform, SAYER, couples high-throughput synthesis with artificial intelligence (AI).\n\n\n\nIt is designed to explore a large chemical space to identify the best PNP candidates for genetic material delivery to diverse tissues. \n\n\n\nWith the new funding from the Cystic Fibrosis Foundation, Nanite will work on initially developing polymer nanoparticles that can accurately deliver messenger RNA therapies to the lung while potentially better resisting the thick, sticky mucus that clogs the lungs of patients with CF. Eventually, Nanite hopes to expand these advances to deliver any type of gene therapy.\n\n\n\nThis could then target other organs, such as the pancreas, affected by cystic fibrosis.\n\n\n\nSean Kevlahan, CEO and co-founder of Nanite, said that the SAYER platform allows for the design of programmable polymer nanoparticles to deliver a broad range of genetic cargoes with tissue specificity. \n\n\n\n“We plan to work with additional patient advocacy groups and biotech partners to develop targeted drug delivery vehicles across multiple different indications,” Kevlahan said.\n\n\n\nAbout Nanite\n\n\n\nNanite is a next-generation non-viral gene delivery company developing a new class of programmable polymer nanoparticles for a range of modalities and indications. Nanite is headquartered in Boston, one of the leading biotech hubs in the world.