Cystic Fibrosis Publications
Cystic Fibrosis
Poster/Publication
Journal/Date
Title
Overview
Title
LUNAR® -CFTR mRNA replacement therapy restores CFTR expression and function in human bronchial epithelial cellsOverview
Presentation:LUNAR®-CFTR successfully transfected and expressed a functional CFTR protein in dF508 and G542X/R1162X null HBE cells in a dose dependent manner.
In presence of a Cl- gradient, LUNAR-CFTR treatment restored chloride currents in CF cells to levels approximating those in wild type cells.
In absence of a Cl- gradient, LUNAR-CFTR induced Cl- current at 60% of WT current levels but was comparable to those corrected by ETI, the clinically approved CFTR modulator for CF patients.
Repeated dosing of 5 mg of LUNAR-CFTR administered at half-life time intervals (3 days) achieved Cl- currents comparable to a single 20 mg dose and comparable to ETI induced currents, indicating beneficial effects of dose accumulation.
LUNAR®-CFTR is currently being studied in a Phase 1 clinical trial in healthy volunteers and adults with CF.
Title
LUNAR® -CFTR mRNA replacement therapy restores CFTR expression and function in human bronchial epithelial cellsOverview
Poster:LUNAR®-CFTR successfully transfected and expressed a functional CFTR protein in F508del and G542X/R1162X null HBE cells in a dose dependent manner.
In presence of a Cl- gradient, LUNAR-CFTR treatment restored chloride currents in CF cells to levels approximating those in wild type cells.
In absence of a Cl- gradient, LUNAR-CFTR induced Cl- current at ~60% of wild type current levels but was comparable to those corrected by ETI, the clinically approved CFTR modulator for CF patients.
Repeated dosing of 5 μg of LUNAR-CF administered at half-life time intervals (3 days) achieved Cl- currents comparable to a single 20 μg dose and comparable to ETI induced currents, indicating beneficial effects of dose accumulation.
LUNAR®-CFTR is currently being studied in a Phase 1 clinical study in healthy volunteers and adults with CF.
Title
ARCT-032 (LUNAR®-CFTR) Improves Mucociliary Clearance in CF FerretsOverview
LUNAR® platform demonstrates efficient delivery of mRNA payloads and transfection in various cell types in ferret airways.ARCT-032 (LUNAR®-CFTR) improved CFTR activity in CF ferret model as demonstrated by significantly improved MCC.
These in vivo results complement finings from in vitro studies in human bronchial epithelial cells (HBEs) derived from CF patients where ARCT-032 restored CFTR-mediated chloride conductance.
ARCT-032 is currently being studied in a Phase 1 trial in healthy volunteers and adults with CF.
Title
NACFC Arcturus Therapeutics Plenary 1 Hope for All Addressing the Needs of Those with Untreated CF MutationsOverview
LUNAR®-hCFTR mRNA (ARCT-032) Restores CFTR Expression & Function in vitroLUNAR®-mRNA Transduces Epithelium in Presence of CF Mucus
Title
LUNAR®-CF: An mRNA Replacement Approach for Cystic Fibrosis Lung DiseaseOverview
Arcturus Technology targeting cystic fibrosis lung diseaseTitle
LUNAR®-CF: An mRNA Replacement Approach for Cystic Fibrosis Lung DiseaseOverview
Mutation agnostic treatment for CF lung diseaseTitle
LUNAR®-CF, A Novel mRNA Therapeutic Approach to Correct the Root Cause of Cystic Fibrosis Lung DiseaseOverview
LUNAR®-CF is an aerosolized mRNA replacement therapy to treat the root cause of Cystic Fibrosis (CF) Lung Disease (CFLD) and is agnostic to genotype. We presentrobust preclinical data to support the advancement of LUNAR®-CF as a therapeutic approach for CFLD. A codon-optimized human CFTR mRNA (hCFTR) encapsulated in
LUNAR®, a proprietary lipid nanoparticle, was developed to deliver hCFTR mRNA into airways epithelium. Physicochemical properties of LUNAR® were stable after
aerosolization.
Title
LUNAR®-CF, an aerosolized mRNA replacement Therapy for Cystic Fibrosis Lung DiseaseOverview
LUNAR®-CF is an aerosolized mRNA replacement therapy to treat Cystic Fibrosis (CF) Lung Disease, a therapeutic approach agnostic to a patient’s genotype. A healthy copy of the human CFTR mRNA is encapsulated into lipid nanoparticles (LUNAR®-hCFTR), aerosolized to patient’s airways using a vibrating mesh nebulizer to directly deliver a de novo human CFTR mRNA into epithelial cells. This human CFTR mRNA encodes a fully functional human CFTR protein that will be beneficial to facilitate mucociliary clearance and improve CF lung disease.Title
LUNAR® selective delivery of nebulized mRNA into murine lung epithelial cellsOverview
LUNAR® lipid nanoparticles carrying the mRNA payload reaches the target cell, where it fuses with the plasma membrane forming an intracellular endosome. This endosomic particle undergoes a pH-mediated disruption that causes the breakdown of the biodegradable nanoparticle and the delivery of the mRNA into the cytoplasm.Title
LUNAR-CF a mRNA Replacement Therapy for Cystic FibrosisOverview
Arcturus Therapeutics is a nucleic acid medicines company focused on developing RNA therapeutics to treat rare diseases. Our proprietary LUNAR® lipid-mediated delivery technology enables the efficient delivery of any mRNA into a variety of cell types and tissues, and can be optimized for multiple routes of administration.