Tackling Cystic Fibrosis from Multiple Angles
CF is a progressive, fatal genetic disease which, if left untreated, can cause persistent lung infections and respiratory failure.
CF is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene and impacts approximately 105,000 people worldwide.
Despite advancements in CFTR modulator treatments, approximately 10% of the CF community have genetic mutations that do not benefit from these life-changing therapeutics.
ReCode is advancing two distinct, innovative approaches to CF treatment: RCT2100, an inhaled mRNA therapy that instructs lung cells to produce functional CFTR protein, and separately, a gene editing collaboration leveraging CRISPR technology with Intellia Therapeutics, designed to precisely correct CF-causing mutations at the genomic level.

John G. Matthews, MBBS, MRCP, Ph.D.
Senior Vice President, Clinical Development“The promise of gene correction therapies is that we’ll be able to go in once, treat the underlying genetic cause of the disease, and give patients the benefit of a repaired lung function indefinitely. It’s an amazing prospect for a new type of therapy.”

Marco Weinberg
Head of ResearchChanging the landscape of treatment options for CF
Using our SORT LNP platform, ReCode is targeting the underlying cause of the disease so that more people with CF may benefit.

ReCode’s CFTR mRNA (RCT2100) is a clinical-stage candidate in development to treat the 10 percent of people with CF who have mutations that do not respond to current CFTR modulators. RCT2100 is an inhaled therapy designed to deliver CFTR mRNA directly to target cells in the lungs, instructing them to produce a functional version of the CFTR protein. By providing CFTR mRNA to individuals who are missing this protein, RCT2100 aims to address the underlying cause of CF, potentially improving CFTR protein function in the lungs rather than just managing symptoms.

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