The Next Generation of Genetic Medicine Delivery
ReCode’s selective organ targeting (SORT) lipid nanoparticle (LNP) platformis 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 VersatilityWhen 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.
Optimized Genetic CargoA diverse range of cargoes can be engineered for higher quality and potency, including mRNA, siRNA, DNA, gene correction components and even mixed cargoes
Precision DeliveryHighly selective and predictable organ and cell targeting for the precise delivery of genetic medicines, beyond the liver
Capability to employ a variety of routes of administration for targeted delivery and biodistribution, including intravenous, inhaled, subcutaneous, intramuscular, and intrathecal
Primary Ciliary Dyskinesia (PCD)ReCode’s lead program for primary ciliary dyskinesia (PCD) is in Phase 1 clinical development
Cystic Fibrosis (CF)The company’s lead mRNA candidate for cystic fibrosis (CF) caused by Class I mutations is advancing into the clinic in early 2024
Future IndicationsReCode is also expanding and diversifying its pipeline of therapeutics using genetic cargoes beyond mRNA to include gene correction modalities in rare genetic diseases including musculoskeletal, central nervous system, liver and infectious disease indications
Nebulized LNP-formulated DNAI1 mRNA Therapy to Restore Mucociliary Clearance for the Treatment of Primary Ciliary Dyskinesia
2023 Cilia, Mucus and Mucociliary Interactions GRC