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

Inhaled treatment designed to deliver healthy CFTR mRNA to cells in the lungs

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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The Cystic Fibrosis Foundation is investing up to $15 million in ReCode Therapeutics to support the development of the company’s inhaled mRNA-based therapy for cystic fibrosis (CF).

“We are excited to expand our relationship with the CF Foundation with this investment in our CFTR mRNA program for cystic fibrosis,” said Shehnaaz Suliman, MD, ReCode’s CEO, in a company press release.

The foundation is one of a number of investors who took part in in ReCode’s Series B financing, which totaled $210 million.

CF is caused by mutations in the CFTR gene, which provides instructions for making a protein of the same name. The CFTR protein normally sits on the surface of cells like a gate and controls the movement of water and certain salts into and out of it.

The protein is absent or dysfunctional in CF, resulting in abnormally thick and sticky mucus being produced, which causes most disease symptoms.

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Recently, a class of medications called CFTR modulators have become widely available. They can improve the CFTR protein’s function in people with certain CF-causing mutations. But these therapies aren’t effective for about 10% of patients — people who don’t produce any CFTR protein at all.

When a gene like CFTR is “read” to make protein, a temporary copy of the genetic code that provides instructions for making the protein called mRNA (messenger RNA) is produced. This copy is sent to the cell’s protein-making machinery, called ribosomes, which use the mRNA as a template for making protein.

ReCode’s therapy is designed to deliver healthy CFTR mRNA to cells in the lungs so ribosomes in lung cells can use the mRNA to make a functional CFTR protein. The mRNA is packaged into lipid nanoparticles, bubbles of fatty molecules that can easily deliver the mRNA into cells.

This approach is expected to work for all patients regardless of the underlying mutation type, including those who aren’t eligible for modulator therapy.

“Messenger RNA therapy has the potential to help all people with CF, including those who don’t respond to CFTR modulator treatment,” said Steven Rowe, MD, chief scientific officer at the CF Foundation, in a press release.

ReCode presented data last year showing the aerosolized therapy could restore CFTR function in CF lung cell models.

The CF Foundation funding includes an initial $10 million investment, which will help fund preclinical studies to support an application to begin clinical testing, as well as early clinical testing. The Foundation has committed to an additional $5 million if certain milestones are met, according to ReCode.

“In addition to the funding, our team will have further access to the CF Foundation’s world-renowned researchers and lab facilities to accelerate our development of a novel mRNA treatment for CF patients not eligible for current treatments,” Suliman said.

The funding is part of the CF Foundation’s Path to a Cure initiative, aimed at accelerating the development of treatments.

“ReCode is testing whether they can optimize their RNA therapy to reach the correct cells in the lungs using its novel lipid nanoparticles, restoring CFTR activity. While a complex process, this would represent a critical and exciting step in developing a successful therapy,” Rowe said.

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