The field of genetic therapies seems to be making a collective shift toward the exploration of delivery systems—and the Cystic Fibrosis Foundation (CFF) is no different. The nonprofit recently announced that they are making an investment of up to $2 million to explore a novel method to deliver genetic therapies to the lung. They will do this by funding the development of an innovative delivery approach—using polymer nanoparticles, comprising small polymeric structures combined with a genetic cargo—developed by the Boston-based company Nanite. Nanite is a nonviral gene delivery company developing a new class of programmable polymer nanoparticles for a range of modalities and indications. This investment builds on the $6M seed financing recently announced by the company.
One of the most significant challenges in developing new therapies for patients with cystic fibrosis (CF) is effective drug delivery. Polymer nanoparticles (PNPs) are a delivery modality that may offer several advantages over other means of genetic therapy delivery into cells. Specifically, PNPs 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.
Nanite’s platform, SAYER, couples high-throughput synthesis with artificial intelligence (AI), and is designed to rapidly explore a large chemical space to identify the best PNP candidates for genetic material delivery to diverse tissues.
With this new funding from the CFF, Nanite will work on 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 and target other organs affected by CF, such as the pancreas.
“We’re thrilled to announce this investment from the Cystic Fibrosis Foundation, which we will use to explore a new way to deliver genetic therapies into the lungs of CF patients,” said Sean Kevlahan, PhD, CEO and co-founder of Nanite. “Our proprietary SAYER platform combines automated polymer synthesis and high-throughput in vivo screening with AI, allowing the design of programmable polymer nanoparticles to deliver a broad range of genetic cargoes with tissue specificity. We plan to work with additional patient advocacy groups and biotech partners to develop targeted drug delivery vehicles across multiple different indications.”