BETHESDA, Md. — The Cystic Fibrosis Foundation will invest up to $15 million in ReCode Therapeutics for the development of its messenger RNA (mRNA) therapy that could eventually provide a treatment option for all people with cystic fibrosis regardless of their mutations. The CF Foundation joins other institutional and strategic investors who participated in ReCode’s Series B financing, totaling $210 million.
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, MD, chief scientific officer at the 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.”
The investment will help fund ReCode’s preclinical research necessary to advance the program toward clinical trials. Funding will also be used to support early-stage clinical trials.
In lab tests, ReCode demonstrated their mRNA therapy can be delivered in an aerosol form to human lung cells. This preclinical data was presented at the North American Cystic Fibrosis Conference in November. 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.
The funding is part of the Foundation’s $500 million Path to a Cure, an ambitious research initiative to accelerate treatments for everyone with CF and ultimately deliver a cure.
About the Cystic Fibrosis Foundation
The Cystic Fibrosis Foundation is the world's leader in the search for a cure for cystic fibrosis. The Foundation funds more CF research than any other organization, and nearly every CF drug available today was made possible because of Foundation support. Based in Bethesda, Md., the Foundation also supports and accredits a national care center network that has been recognized by the National Institutes of Health as a model of care for a chronic disease. The CF Foundation is a donor-supported nonprofit organization. For more information, visit cff.org.