Study confirms the ability of ReCode’s Selective Organ Targeting (SORT) lipid nanoparticle (LNP) platform to deliver optimized, functional CFTR mRNA as an aerosol directly to specific cell targets
Results show consistent rescue of CFTR function across multiple cystic fibrosis patient genotypes, including genotypes that do not respond to approved CFTR modulator treatments
MENLO PARK, Calif. & DALLAS, May 18, 2022--(BUSINESS WIRE)--ReCode Therapeutics, a biopharmaceutical company powering the next wave of genetic medicines through superior delivery, today presented encouraging new preclinical data from the company’s inhaled mRNA-based therapeutic program for cystic fibrosis (CF) at the American Thoracic Society (ATS) 2022 International Conference, taking place May 13-18, 2022 in San Francisco. The company’s mRNA-based program for cystic fibrosis uses ReCode’s first-in-class Selective Organ Targeting (SORT) lipid nanoparticle (LNP) delivery platform which enables the targeting of organs and tissues beyond the liver via a variety of administration routes.
"Today’s presentation confirms that our SORT LNP platform can deliver optimized, functional CFTR mRNA as an aerosol to the intended secretory and basal cells in the airway epithelium," said David Lockhart, Ph.D., Chief Scientific Officer and President, ReCode Therapeutics. "In human bronchial epithelial cells derived from CF patients, we have shown that delivery of our CFTR mRNA results in consistent rescue of CFTR function with multiple genotypes, including those types that account for approximately ten percent of the CF population who do not respond to currently approved CFTR modulators. We are eager to continue development of this promising therapy."
Data from a preclinical proof-of-concept study demonstrate that ReCode’s SORT LNPs successfully delivered nebulized mRNA into primary CF human bronchial epithelial (hBE) cells, a well-validated in vitro model. Furthermore, the studies demonstrate that delivery of CFTR mRNA results in consistent rescue of CFTR function in cells derived from patients with multiple different disease-causing genotypes.
Full details from the preclinical CF data can be accessed via the Presentations section on the company’s website.
About Cystic Fibrosis
Cystic fibrosis (CF) is a progressive, genetic disease which causes persistent lung infections and respiratory failure. CF is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene and impacts approximately 100,000 people worldwide. The absence or dysfunction of the CFTR protein triggers excessive mucus build up in the lungs which leads to recurrent infections, inflammation, respiratory failure, and other complications. Despite advancements in CFTR modulator treatments, no available therapies exist to restore function for Class I mutations in the CFTR gene since no significant CFTR protein is produced.
About the SORT LNP Platform
ReCode’s novel Selective Organ Targeting (SORT) lipid nanoparticle (LNP) technology is a modular genetic medicines delivery platform with broad potential applications across a range of organs and tissues, target cells, and therapeutic modalities. ReCode’s proprietary SORT LNP platform enables the flexibility to target specific organs and tissues while de-targeting the liver.
Beyond its highly selective targeting capability, ReCode’s SORT LNP platform is further distinguished by its versatility in both mode of administration and the diversity of genetic cargoes that can be delivered, which include mRNA and gene correction components. Together, these qualities form the foundation for opportunities to unlock the full potential of genetic medicines.
About ReCode Therapeutics
ReCode Therapeutics is a biopharmaceutical company powering the next wave of genetic medicines through superior delivery. ReCode’s Selective Organ Targeting (SORT) lipid nanoparticle (LNP) platform is a next-generation LNP delivery technology to target organs and tissues beyond the liver. The SORT LNP platform is the foundation for ReCode’s pipeline of disease-modifying mRNA- and gene correction-based therapeutics for genetically defined diseases for which there are few or no current treatments. ReCode’s lead programs are focused on primary ciliary dyskinesia, and cystic fibrosis caused by Class I mutations. ReCode is leveraging its SORT LNP platform and nucleic acid technologies for mRNA-mediated replacement and gene 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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