Boston-based startup Nanite has netted $6 million in seed funding to launch its artificial intelligence platform for gene delivery. Founded in 2021, the company applies machine learning to designing polymer nanoparticles for nonviral delivery of genetic therapies.
A drug’s delivery mechanism can be as important as the drug itself. For gene therapies, the most well-established delivery vehicle is adeno-associated viruses (AAVs). This method harnesses the ability of viruses to infect cells and impose their genes onto their new hosts. However, AAVs have a limited payload size, and they can trigger an immune response.
Lipid nanoparticles, which are used in COVID-19 mRNA vaccines, are an alternative delivery vehicle. But lipid nanoparticles tend to get stuck in the liver, so they aren’t suited for tissue-specific delivery.
Polymer nanoparticles are the latest nonviral gene delivery alternatives. Their main advantage is their chemical diversity, says Sean Kevlahan, Nanite’s CEO and cofounder. This versatility allows scientists to tailor the polymers for different cargo and tissue targets in the body. Tens to hundreds of nanometers in diameter, Nanite’s polymeric nanoparticles can transport genetic sequences as short as 10 base pairs, as well as large accessory proteins that would enable gene editing such as CRISPR, the company says.
The polymer capsule involves many design considerations, such as the monomer chemistry, polymer architecture, and surface charge distribution on the nanoparticle. Nanite employs AI to predict the design for the polymer vehicle, depending on the cargo and the destination tissue. The company’s technology is based on the work of University of Minnesota chemist Theresa Reineke, who is also a company cofounder.
AI “gives us the ability to explore the polymer universe without having to actually empirically determine each star in that universe,” Kevlahan says. “We believe that polymers are going to take over the whole [gene delivery] space.”
Kevlahan says that the company is in talks with potential partners to offer its in-silico design and automated synthesis services.