Menlo Park, Calif. and Dallas, Texas – June 11, 2021 – ReCode Therapeutics (the “Company”), a biopharmaceutical company pioneering disease-modifying medicines for the treatment of life-limiting respiratory diseases, today presented encouraging preclinical data from the Company’s RNA-based molecular therapy program for cystic fibrosis (CF) delivered using the Company’s LNP platform, including three-component Zwitterionic Amino Lipids (ZALs) and five-component selective organ targeting lipid nanoparticles (SORT-LNPTM), during an oral session at the 44th European Cystic Fibrosis Conference.
“These data showcase the potential of our differentiated RNA-based molecular therapies to treat cystic fibrosis. By leveraging our LNP platform to deliver genomic medicines, we have demonstrated the ability to restore cystic fibrosis transmembrane conductance regulator (CFTR) function through both tRNA and mRNA approaches,” said David Lockhart, Ph.D., CEO & President, ReCode Therapeutics. “Our RNA-based approaches and broad LNP platform enable us to tackle CF from multiple angles in order to specifically target the underlying causes of the disease and maximally help patients. These findings are an important step as we advance our novel CF program closer to Investigational New Drug (IND)-enabling studies and an IND submission to the FDA in 2022.”
The data presented show that RCT223, the Company’s tRNA agent for CF nonsense mutations, restores CFTR function in the gold-standard human bronchial epithelial (hBE) cell model, both with single and repeat dosing. The three-component ZAL LNP formulation was also shown to be generally well-tolerated in patient-derived hBE cell cultures. RCT223 was shown to restore CFTR function for at least 72 hours in patient-derived hBE cells after a single administration. With twice-weekly repeated doses, CFTR functional levels continued to increase.
The data presented also show that RTX0001, the Company’s mRNA replacement agent for CF, restores CFTR function in the gold-standard CF patient-derived hBE cell model. The mRNA payload was successfully nebulized and delivered onto hBEs and into the lungs of mice as an aerosol, using a commercially available mesh nebulizer. In CF hBE cells, the delivered mRNA significantly restored CFTR function after a single, low dose. Additionally, CFTR activity was maintained for at least 72 hours after twice-weekly administration. Overall, the nebulized formulations were well tolerated in hBE cells and in mice.
ReCode’s Oral Presentation at the 44th European Cystic Fibrosis Conference
Abstract title: Rescue of CFTR function in primary bronchial epithelial cells from patients with cystic fibrosis using lipid nanoparticle delivery of RNA-based therapies
Abstract number: WS09.3
Session title: Workshop 9 – Future therapeutic approaches and insights into our current practice
Presenter: Michael Torres, Ph.D., Vice President, R&D, ReCode Therapeutics
The presentation is now available to registered conference attendees on the ECFS website. Learn more about the full program here.
About Cystic Fibrosis
Cystic fibrosis (CF) is a progressive, genetic respiratory disease that causes persistent lung infections and limits the ability to breathe over time. In people with CF, mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene cause the CFTR protein to become dysfunctional, triggering the mucus in various organs to become thick and sticky. In the lungs, the mucus clogs the airways and traps germs, leading to infections, inflammation, respiratory failure and other complications.
About ReCode Therapeutics
ReCode Therapeutics is an integrated genetic medicines company developing targeted, disease-modifying therapeutics using its powerful, proprietary SORT-LNPTM platform to target organs and tissues beyond the liver. The Company’s pipeline includes lead programs for patients with life-limiting genetic respiratory diseases, including cystic fibrosis and primary ciliary dyskinesia. The Company is leveraging its SORT-LNPTM and nucleic acid technologies, utilizing systemic and direct delivery for mRNA-mediated replacement and gene editing/correction in target cells, including stem cells. For more information, visit www.recodetx.com and follow us on Twitter @ReCodeTx and LinkedIn.
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