CF Foundation invests up to $2M in Nanite’s gene therapy approach

Investment aims to find safer and more efficient ways to deliver gene therapies

Joana Vindeirinho, PhD avatar

by Joana Vindeirinho, PhD |

Share this article:

Share article via email
An illustration of a piggy bank.

The Cystic Fibrosis Foundation is investing up to $2 million in Nanite to develop new non-viral methods of delivering gene therapies into the lungs of people with cystic fibrosis (CF).

“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,” Sean Kevlahan, PhD, CEO and cofounder of Nanite, said in a press release.

These new methods center around the use of tiny synthetic particles called polymer nanoparticles (PNPs), an alternative vehicle for therapy delivery with the potential to be safer and more efficient than other commonly used vehicles.

CF Foundation’s investment joins the previous $6-million seed investment gathered to advance Nanite’s PNP design and synthesis platform.

Recommended Reading
A pile of money points toward a laboratory test tube.

ReCode’s mRNA Therapy Wins $15M CF Foundation Investment

PNPs for gene therapy delivery may be less likely to trigger immune reaction

CF is caused by mutations in the CFTR gene, leading to the malfunction or complete absence of the CFTR protein, a channel in the cell membrane that helps to regulate the flow of water and salts. A malfunctioning or absent CFTR protein causes the accumulation of thick mucus in several organs in the body, such as the lungs and the pancreas.

Because of the disease’s genetic cause, gene therapies have been developed to deliver a version of the CFTR gene, so that a working version of the CFTR protein can be produced. Harmless, modified viruses have been the traditional and most commonly used method of gene therapy delivery, but other methods are being developed, such as lipid nanoparticles.

A main challenge of gene therapy in all diseases is the balance between an efficient delivery of enough therapy to the target tissue and an acceptable safety profile for patients. In CF, this is particularly challenging because of the mucus accumulation in the target organs.

According to Nanite, an attractive feature of PNPs for gene therapy delivery is that they might be less likely to trigger a patient’s immune system than viruses or lipid nanoparticles, improving a therapy’s safety, and they are more stable and versatile than lipid nanoparticles, increasing a therapy’s efficiency.

However, one of the drawbacks of using PNPs is how complex their development is, in both design and interaction with the genetic therapies they will carry.

To overcome these hurdles, Nanite has developed a platform, SAYER, that combines automated synthesis with artificial intelligence-led screening of new PNPs. This allows for a fast exploration of many PNPs to identify the best candidates for specific tissues and therapies.

Recommended Reading
A hand holds up a coin amid images of dollar signs and packets of money.

CF Foundation Invests Another $4.85M to Help Develop AR-501

“Advances in automated synthesis and high-throughput screening now make it possible to build and test PNPs rapidly and have enabled us to sample much larger and more diverse regions of the polymer universe than ever before,” Kevlahan said in the announcement of seed investments. “Combining those data, in a closed loop, with new machine learning techniques allows us to decode the functional landscape of PNPs and rationally design or “program” delivery vehicles in a way that would have seemed impossible only a few years ago.”

Nanite plans to develop PNPs first with the potential to deliver messenger RNA therapies efficiently and to resist the thick mucus present in the lungs of CF patients. Of note, messenger RNA is a temporary copy of the genetic code that provides instructions for making proteins.

The long-term goal of the company is the development of PNPs able to deliver any type of gene therapy and target other organs affected by CF, such as the pancreas.

A Conversation With Rare Disease Advocates

Your CF Community

Visit the Cystic Fibrosis News Today forums to connect with others in the CF community.