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ReCode Therapeutics Presents Preclinical Data from mRNA-based Cystic Fibrosis Program at 2021 North American Cystic Fibrosis Conference

– Data show ability to rescue cystic fibrosis transmembrane conductance regulator (CFTR) function using proprietary lipid nanoparticle (LNP) platform to deliver optimized CFTR mRNA as an aerosol to patient-derived human bronchial epithelial cells

Optimized LNP formulation delivered mRNA to therapeutically relevant lung secretory and basal cells

Excerpt from the Press Release:

MENLO PARK, Calif. & DALLAS–(BUSINESS WIRE)–ReCode Therapeutics, a biopharmaceutical company pioneering disease-modifying genetic medicines using its proprietary LNP delivery platform, presented new preclinical data today from the Company’s inhaled mRNA-based therapeutic program for cystic fibrosis (CF) during an oral poster session at the 2021 North American Cystic Fibrosis Conference (NACFC).

“These data validate our mRNA-based approach to treating CF which leverages our powerful LNP platform and offers the potential to restore CFTR function in CF patients. We are excited to advance our approach, particularly with an initial focus on those CF patients with mutations that are not amenable to treatment with approved CFTR modulator therapies,” said David J. Lockhart, Ph.D., CEO & President of ReCode Therapeutics. “Today’s data support the continued development of an inhaled mRNA therapy for the treatment of CF, and we look forward to advancing this program toward an IND submission.”

Data presented today show:

  • ReCode’s LNPs can be nebulized using commercially available mesh nebulizers and deliver mRNA efficiently into fully-differentiated patient-derived human bronchial epithelial (hBE) cells.
  • ReCode’s CFTR mRNA can be delivered into key target cell types (e.g., secretory and basal cells) in patient-derived hBE cells.
  • Treatment of patient-derived hBEs with CFTR mRNA increased CFTR protein function to levels comparable to those achieved with approved CFTR modulators.

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