MENLO PARK, Calif.--(BUSINESS WIRE)--ReCode Therapeutics, a genetic medicines company using superior delivery to power the next wave of mRNA and gene correction therapeutics, announced today that it will receive a strategic investment from the Cystic Fibrosis Foundation (CF Foundation) to develop ReCode’s inhaled mRNA-based therapeutic for people with cystic fibrosis (CF), including those with genotypes that do not respond to approved CFTR modulators.
“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, M.D., MBA, M.Phil., chief executive officer of ReCode Therapeutics. “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.”
The CF Foundation will contribute up to $15 million including an initial equity investment of $10 million to support preclinical studies needed for an Investigational New Drug (IND) application and early clinical development of ReCode’s CFTR mRNA therapy for CF. The investment includes a commitment by the CF Foundation to invest another $5 million upon completion of development milestones.
ReCode’s CFTR mRNA therapy aims to compensate for mutated CFTR, the fundamental genetic defect in CF. The CFTR mRNA is delivered using the company’s first-in-class selective organ targeting (SORT) lipid nanoparticle (LNP) platform, which is designed to deliver mRNA that would instruct the body to make the normal, healthy version of the protein.
Preclinical data from ReCode presented at the North American Cystic Fibrosis Conference in 2022 demonstrated that CFTR mRNA encapsulated in SORT LNPs can be nebulized and delivered as an aerosol. In addition, the delivered mRNA can rescue CFTR function in human bronchial epithelial cells derived from CF patients to levels comparable to currently approved modulators.
About Cystic Fibrosis
Cystic fibrosis (CF) is a progressive, genetic disease which causes persistent lung infections and respiratory failure. CF is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene and impacts approximately 100,000 people worldwide. The absence or dysfunction of the CFTR protein triggers excessive mucus build up in the lungs which leads to recurrent infections, inflammation, respiratory failure, and other complications. Despite advancements in CFTR modulator treatments, no available therapies exist to restore function for Class I mutations in the CFTR gene since no significant CFTR protein is produced.
About ReCode Therapeutics
ReCode Therapeutics is a genetic medicines company using superior delivery to power the next wave of messenger RNA (mRNA) and gene correction therapeutics. ReCode’s selective organ targeting (SORT) lipid nanoparticle (LNP) platform is a next-generation, genetic medicines technology that enables precise delivery to target organs and cells beyond the liver. The SORT LNP platform is the foundation for ReCode’s pipeline of disease-modifying mRNA and gene correction therapeutics. ReCode’s lead programs are focused on primary ciliary dyskinesia and cystic fibrosis. ReCode is leveraging its SORT LNP platform and nucleic acid technologies to expand its pipeline with therapeutics that use mRNA-mediated replacement and gene correction in target organs with precision targeting of disease-relevant cells. In 2022, ReCode was named among Fierce Biotech’s “Fierce 15” as one of the most promising early-stage biotechnology companies in the industry, and was described by Nature as one of the “Seven Technologies to Watch in 2022” for its SORT LNP platform. For more information, visit www.recodetx.com and follow us on Twitter @ReCodeTx and on LinkedIn.