A new lipid nanoparticle delivery platform is able to deliver functional CFTR mRNA as an aerosol to the intended cells in the airway epithelium, according to new preclinical data presented at the American Thoracic Society 2022 International Conference that took place in San Francisco, California between May 13, 2022, and May 18, 2022.

The delivery of the mRNA to human bronchial epithelial cells derived from patients with cystic fibrosis (CF) led to the consistent rescue of the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. This was the case even for genotypes that do not respond to CFTR modulators that are currently approved to treat the disease.

“We are eager to continue development of this promising therapy,” David Lockhart, PhD, chief scientific officer and president of ReCode Therapeutics, said in a press release.

The delivery platform is a first-in-class selective organ targeting (SORT) lipid nanoparticle platform that enables the targeting of specific organs and tissues. It is estimated that CF affects around 100,000 people worldwide. The disease is caused by a mutation in the CFTR gene, which codes for the CFTR protein, a protein essential to maintaining the balance of salt and water on cell surfaces.

Read more about different CFTR modulators and other therapies for CF

There are several CFTR modulator treatments that are available. However, these cannot treat people with so-called class 1 mutations, which account for 10% of the CF population, where no CFTR protein is produced by the body.

The SORT lipid nanoparticle platform could be used to deliver different types of genetic material such as mRNA and gene correction components to target tissues and organs and could be used to treat patients with CF who cannot benefit from currently available treatments.

Reference

ReCode Therapeutics presents new preclinical data from mRNA-based program for cystic fibrosis at the ATS 2022 International Conference. News release. ReCode Therapeutics; May 18, 2022.