RARE Daily

CF Foundation Invests Up to $2 Million in Nanite to Explore a Novel Delivery Method of Genetic Therapies to the Lung

March 16, 2023

Rare Daily Staff

The Cystic Fibrosis Foundation is investing up to $2 million in Nanite to develop gene delivery technologies for the lung.

This investment builds on a $6 million seed financing announced by the company in mid-February.

One 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. 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 proprietary platform, SAYER, couples high-throughput synthesis with artificial intelligence, 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 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 and target other organs affected by CF, such as the pancreas.

“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,” said Sean Kevlahan, CEO and co-founder, Nanite. “We plan to work with additional patient advocacy groups and biotech partners to develop targeted drug delivery vehicles across multiple different indications.”

Nanite also entered into a partnership with the Charcot-Marie-Tooth Research Foundation in February to design delivery vehicles for genetic medicines to Schwann cells via PNPs. Approximately two-thirds of the world’s more than 3 million CMT cases are caused by mutations in genes that are expressed solely or almost solely within myelinating Schwann cells, making them the primary target of genetic therapies in most cases of CMT. But myelinating Schwann cells lie behind the blood-nerve barrier, making the delivery of genetic medicines to Schwann cells a major challenge that the partnership hopes to overcome.

Photo: Sean Kevlahan, CEO and co-founder, Nanite

Stay Connected

Sign up for updates straight to your inbox.