ReCode Therapeutics, a clinical-stage genetic medicines company using tissue-specific delivery to power the next wave of mRNA and gene correction therapeutics, today presented preclinical data from its mRNA-based cystic fibrosis (CF) program at the North American Cystic Fibrosis Conference (NACFC), taking place September 26-28, 2024, in Boston, Massachusetts.

These preclinical data demonstrate that RCT2100 significantly restored CFTR function in human bronchial epithelial (HBE) cells derived from people with CF, achieving up to 135% of the efficacy seen with an elexacaftor/tezacaftor/ivacaftor equivalent in cells with an F508del mutation on one allele and a minimal function mutation on the other allele. Additionally, in vivo studies using a G551D CF ferret model revealed notable improvement in mucociliary clearance (MCC) post-treatment, highlighting RCT2100's potential to address the medical needs of approximately 10% of people with CF not eligible for current CFTR modulator therapy.

"These preclinical data underscore the potential of RCT2100 as a novel therapeutic approach for people with cystic fibrosis who do not respond to existing CFTR modulators,” said Shehnaaz Suliman, M.D., MBA, M.Phil., chief executive officer of ReCode Therapeutics. “In the studies, RCT2100, an inhaled mRNA-based therapy, successfully restored CFTR function in patient-derived cells in vitro and improved mucociliary clearance, which is critical for improving patient outcomes, in a ferret model of CF lung disease. These findings support our goal of developing innovative therapies that address the needs of the entire cystic fibrosis community, especially those still awaiting effective treatment options.”

“The results seen in this preclinical study are significant in that they demonstrate the ability of RCT2100 to improve CFTR function in HBE cells at a level previously only seen with triple combination CF drug modulator treatments,” said Marco Weinberg, Ph.D., Head of Research at ReCode Therapeutics. “In a separate study, the use of RCT2100 demonstrated that when used in the G551D CF ferret model, treatment resulted in a notable improvement in mucociliary clearance. This is significant in that treatment with RCT2100 resulted in an increase in MCC after two weeks, similar to that seen with existing modulator therapy after only two doses administered once per week. Combined, these data highlight the potential of RCT2100 as a treatment option for a group of CF patients that do not respond to current therapy.”

ReCode is pleased to welcome NACFC participants to booth #816 in the Exhibit Hall.

About Cystic Fibrosis

Cystic fibrosis (CF) is a progressive genetic disease that causes persistent lung infections and respiratory failure. CF is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. An estimated 105,000 people have been diagnosed with CF across 94 countries, according to the Cystic Fibrosis Foundation. The absence or dysfunction of the CFTR protein results in a defect in airway hydration, which leads to excessive mucus buildup in the lungs. It also creates a mucociliary clearance defect, recurrent infections, inflammation, respiratory failure, and other complications. Despite advancements in CFTR modulator treatments, approximately 10% of the CF community have genetic mutations that do not benefit from these life-changing therapeutics.

About ReCode Therapeutics

ReCode Therapeutics is a clinical-stage genetic medicines company using precision delivery to power the next wave of mRNA and gene correction therapeutics. ReCode’s proprietary Selective Organ Targeting (SORT) lipid nanoparticle (LNP) platform enables highly precise and targeted delivery of genetic medicines directly to the organs, tissues, and cells implicated in disease, enabling improved efficacy and potency. ReCode’s lead programs include RCT1100 for the treatment of primary ciliary dyskinesia caused by pathogenic mutations in the DNAI1 gene, and RCT2100 for the treatment of the 10% of people with cystic fibrosis who have genetic mutations in the CFTR gene that do not respond to currently approved CFTR modulators. RCT1100 and RCT2100 are inhaled disease-modifying mRNA-based therapies formulated using the SORT LNP delivery platform. ReCode is expanding its pipeline to develop potential therapies for other rare and common genetic diseases, including musculoskeletal, central nervous system, liver, and infectious disease indications.

ReCode has been recognized by the San Francisco Business Times and Silicon Valley Business Journal as a Best Place to Work. For more information, visit www.recodetx.com and follow us on LinkedIn and Instagram.

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