ReCode Therapeutics has announced that it will secure a strategic investment from the Cystic Fibrosis Foundation (CF Foundation) to develop its inhaled mRNA-based therapeutic for cystic fibrosis (CF).
The inhaled mRNA-based therapeutic is intended for people with CF, including those with genotypes that are not responsive to approved CFTR modulators.
ReCode Therapeutics CEO Shehnaaz Suliman said: “We are excited to expand our relationship with the CF Foundation with this investment in our CFTR mRNA programme for cystic fibrosis.
“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 provide up to $15m including an initial equity investment of $10m to support preclinical studies required for an investigational new drug (IND) application and early clinical development of the company’s CFTR mRNA therapy for CF.
The investment includes a commitment by the foundation to invest another $5m following completion of development milestones.
ReCode’s CFTR mRNA therapy intends to compensate for mutated CFTR, the basic genetic defect in CF.
The CFTR mRNA is provided leveraging the company’s first-in-class selective organ targeting (SORT) lipid nanoparticle (LNP) platform, which is intended to deliver mRNA that would order the body to make the normal, healthy version of the protein.
Preclinical data from ReCode that was presented at the North American Cystic Fibrosis Conference in 2022 showed that CFTR mRNA encapsulated in SORT LNPs can be nebulized and delivered as an aerosol.
Furthermore, the delivered mRNA can rescue CFTR function in human bronchial epithelial cells drawn from CF patients to levels comparable to presently approved modulators.