CF Foundation Invests up to $15 Million in ReCode Therapeutics for Development of mRNA Therapy

The Cystic Fibrosis Foundation said it will invest up to $15 million in ReCode Therapeutics for the development of its messenger RNA therapy that could eventually provide a treatment option for all people with cystic fibrosis regardless of their mutations.

Photo: Steven Rowe, chief scientific officer at the Cystic Fibrosis Foundation

The CF Foundation joins other institutional and strategic investors who participated in ReCode’s Series B financing, totaling $210 million.

The investment will help fund ReCode’s preclinical research necessary to advance the program toward clinical trials. The funding will also be used to support early-stage clinical trials. It is part of the Foundation’s $500 million Path to a Cure, a research initiative to accelerate treatments for everyone with CF and ultimately deliver a cure.

CF is caused by a defective or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes—one from each parent—to have CF. While there are many different types of CFTR mutations that can cause the disease, the vast majority of people with CF have at least one F508del mutation. CFTR mutations lead to CF by causing the CFTR protein to be defective or by leading to a shortage or absence of CFTR protein at the cell surface. The defective function or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus, chronic lung infections and progressive lung damage that eventually leads to death for many patients.

ReCode is developing an inhaled mRNA therapy, designed to provide a correct copy of the cystic fibrosis transmembrane conductance regulator (CFTR) mRNA to lung cells to make a functional CFTR protein. To deliver the mRNA therapy into lung cells, ReCode is using a unique, selective organ-targeting lipid nanoparticle—an alternative to other delivery systems such as engineered viruses and an advancement over the conventional lipid nanoparticles (LNPs) used to deliver the mRNA COVID-19 vaccines. These LNPs may allow genetic therapies to enter lung cells more easily and importantly may allow safe redosing of the CFTR mRNA.

“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, chief scientific officer at the Cystic Fibrosis Foundation. “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.”

In lab tests, ReCode demonstrated its mRNA therapy can be delivered in an aerosol form to human lung cells. CFTR protein function improved in treated lung cells with two F508del mutations and cells with one F508del mutation and one G542X mutation. The improvement in function was equal to levels seen with the two-drug CFTR modulator combinations in cells with two F508del mutations.