Cystic Fibrosis News Today
The successful administration of billions of doses of mRNA COVID-19 vaccines and continued progress with novel RNA and gene correction therapeutics have catapulted the world into a new era of possibility for genetic medicines. ReCode’s innovative lipid nanoparticle delivery technology is engineered with unique properties that have the potential to greatly improve the effectiveness of the next generation of genetic medicines.
ReCode’s selective organ targeting (SORT) lipid nanoparticle (LNP) platform
is the foundation for our pipeline of disease-modifying mRNA and gene correction therapeutics for genetically defined diseases for which there are few or no current treatments. The SORT LNPs are used to package and deliver genetic medicine cargoes such as mRNA, siRNA, and gene correction modalities.
Engineered for Precision and Versatility
When delivered into the blood, first-generation LNPs are primarily taken up by the liver, which limits their utility for broad therapeutic applications. ReCode’s SORT LNPs are engineered with a biochemically distinct fifth lipid to help the body “sort” and direct the LNPs to other targeted organs such as the lung and spleen, with the ability to bypass the liver, if desired.
Engineered with a biochemically distinct fifth lipid, ReCode’s SORT LNP platform powers a library of LNPs that are tuned to reach specific organs and cells.
Highly selective and predictable organ and cell targeting for the precise delivery of genetic medicines, beyond the liver
Optimized Genetic Cargo
A diverse range of cargoes can be engineered for higher quality and potency, including mRNA, siRNA, DNA, gene correction components, and even mixed cargoes
Capability to employ a variety of routes of administration for targeted delivery and biodistribution, including IV, inhaled, subcutaneous, intramuscular, and intrathecal
Functional Rescue of CFTR by Aerosolized Delivery of Optimized CFTR mRNA Using ReCode LNPs in Primary Human Bronchial Epithelial Cells Derived From Patients With Cystic Fibrosis
American Thoracic Society (ATS) 2022 International Conference
ReCode is advancing its lead programs for primary ciliary dyskinesia (PCD) and cystic fibrosis (CF) caused by Class I mutations into the clinic. The company is also expanding and diversifying its pipeline of therapeutics using genetic cargoes beyond mRNA including siRNA and gene correction modalities in rare genetic diseases including lung, liver, CNS, and cancer indications.