ReCode Therapeutics Shares Preclinical Data on Cystic Fibrosis at 2024 NACFC in Boston

ReCode Therapeutics, a clinical-stage company specializing in genetic medicines, recently shared promising preclinical data at the North American Cystic Fibrosis Conference (NACFC) in Boston, Massachusetts. Their mRNA-based treatment for cystic fibrosis (CF), known as RCT2100, showed significant efficacy in restoring CFTR function in human bronchial epithelial (HBE) cells derived from CF patients. Remarkably, the therapy achieved up to 135% of the effectiveness observed with the current standard triple combination therapy (elexacaftor/tezacaftor/ivacaftor) in cells with specific genetic mutations.

The studies included in vivo experiments using a G551D CF ferret model. The results indicated a notable improvement in mucociliary clearance (MCC) following treatment with RCT2100. MCC is crucial for lung function and patient health, and this improvement underscores RCT2100’s potential to meet the needs of approximately 10% of CF patients who do not respond to existing CFTR modulator therapies.

Shehnaaz Suliman, M.D., MBA, M.Phil., CEO of ReCode Therapeutics, emphasized the significance of these findings, noting that RCT2100 could provide a new therapeutic option for CF patients who are resistant to current treatments. The data demonstrate RCT2100’s effectiveness in restoring CFTR function in vitro and enhancing mucociliary clearance in a CF ferret model, which are critical steps toward improving patient outcomes.

Dr. Marco Weinberg, Head of Research at ReCode Therapeutics, highlighted the importance of the results. According to Weinberg, the ability of RCT2100 to restore CFTR function in HBE cells at levels comparable to triple combination therapies is a significant advancement. In the G551D CF ferret model, RCT2100 treatment resulted in increased MCC after two weeks, mirroring the response seen with current modulator therapies but achieved with fewer doses. These findings collectively highlight the potential for RCT2100 to offer a much-needed treatment alternative for CF patients unresponsive to current therapies.

Cystic fibrosis is a hereditary condition that profoundly affects the respiratory system, causing persistent lung infections and respiratory failure due to mutations in the CFTR gene. Despite significant advancements in CFTR modulator treatments, about 10% of the CF population has genetic mutations that render these treatments ineffective. This underscores the need for new therapeutic approaches like RCT2100.

ReCode Therapeutics is at the forefront of developing innovative genetic medicines, leveraging its proprietary Selective Organ Targeting (SORT) lipid nanoparticle (LNP) platform. This technology allows precise and targeted delivery of genetic medicines to specific organs, tissues, and cells, thereby enhancing treatment efficacy and potency. The company’s lead programs include RCT1100 for primary ciliary dyskinesia and RCT2100 for cystic fibrosis patients with non-responsive genetic mutations. Both therapies are inhaled and designed using the SORT LNP platform.

ReCode Therapeutics is also broadening its pipeline to address a range of rare and common genetic diseases, targeting areas such as the musculoskeletal and central nervous systems, liver, and infectious diseases. The company's efforts in genetic medicine have garnered recognition, earning it a spot as one of the Best Places to Work by the San Francisco Business Times and Silicon Valley Business Journal.