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

MENLO PARK, CA & DALLAS, TX, USA I November 03, 2021 IReCode 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.

Details on ReCode’s NACFC Presentation

Poster: Variant-agnostic CFTR Rescue after Aerosolized Delivery of CFTR mRNA Formulated with a SORT-LNP in Primary Human Bronchial Epithelial Cells Derived from Patients with Cystic Fibrosis
Presenter: Michael Torres, Ph.D., Vice President, R&D, ReCode Therapeutics
Poster Number: 583
Date: Wednesday, November 3, 2021
Live Poster Session: 1:10 – 1:50 p.m. ET

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 disease-modifying therapeutics using its powerful LNP delivery technology 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 proprietary LNP platform and nucleic acid technologies and utilizing systemic and direct delivery for mRNA-mediated replacement and gene editing/correction in target cells, including stem cells. For more information, visit and follow us on Twitter @ReCodeTx and LinkedIn.

SOURCE: ReCode Therapeutics