Arcturus Therapeutics Holdings Inc. · NASDAQ:ARCT
Arcturus Therapeutics — 45th European Cystic Fibrosis Conference (ECFC) 2022, Rotterdam, Workshop 16
10 slides from Arcturus Therapeutics's conference presentation, Jun 10, 2022
Slide 1: LUNAR-CF: An mRNA Replacement Approach for Cystic Fibrosis Lung Disease
LUNAR®-CF: An mRNA Replacement Approach for Cystic Fibrosis Lung Disease David E Geller, MD 45th ECFC, Rotterdam Workshop 16 10 June 2022
Slide 2: LUNAR-CF Therapy
LUNAR®- CF Therapy mRNA Replacement CAP- 5’UTR ORF 3’UTR Cargo: hCFTR mRNA Delivery vehicle: LUNAR® Delivery format: Aerosol Mutation agnostic treatment for CF lung disease 2
Slide 3: Effects of Aerosolization and Stability in CF Mucus
Effects of Aerosolization and Stability in CF Mucus Aerosolized LUNAR®, A Lung Delivery Platform Encapsulation Efficiency is Aerosolized LUNAR-mRNA LUNAR® Protects mRNA in CF Maintained maintains activity Sputum Pre-Nebulization Post-Nebulization Different mesh nebulizers were tested for Translation
Slide 4: Targeting LUNAR-mRNA Delivery to Airways Epithelium
Targeting LUNAR®-mRNA Delivery to Airways Epithelium LUNAR® -mRNA Transduction is Maintained Across Species 4
Slide 5: Penetration of LUNAR-mRNA through CF mucus
Penetration of LUNAR®-mRNA through CF mucus LUNAR® -Cre in a G551D CF ferret Trachea Bronchus High-res Low-res 5
Slide 6: Epithelial Cell Profiling with LUNAR-mRNA (TdT)
Epithelial Cell Profiling with LUNAR®-mRNA (TdT) Apical transduction in non-CF HBE cell population Transduction Ionocytes Ciliated efficiency ~31% Goblet cells Basal cells 6
Slide 7: Functional Restoration of Cl- Current with LUNAR-hCFTR in vitro
Functional Restoration of Cl- Current with LUNAR®-hCFTR in vitro Ferret BE cells (G551D) HBE cells (F508del+/+) High levels of mature CFTR protein Restored chloride activity 7
Slide 8: Functional Restoration of Cl- Current with LUNAR-hCFTR in vivo
Functional Restoration of Cl- Current with LUNAR®-hCFTR in vivo NPD assessment in G542X Mouse Model after topical nasal instillation of LUNAR-hCFTR LUNAR® buffer control LUNAR®-hCFTR mRNA 8
Slide 9: LUNAR-hCFTR: Summary
Arcturus Technology LUNAR®-hCFTR: Summary targeting CF Lung Disease Aerosolized LUNAR® LNPs are stable and protect mRNA from degradation in CF sputum Target engagement of epithelial cells in vitro and in vivo across species and cell types Codon-optimized hCFTR mRNA generates high levels of mature CF
Slide 10: Thanks …
Thanks … • John Engelhardt • Carlos Perez-Garcia • Xiaoming Liu • Suezanne Parker • Yihua Pei • Steven Rowe • Paloma Redondo • Javier Campos Gomez • Stefen Boehm • Craig Hodges • Rajesh Mukthavaram • Wendy Taylor • Martin Mense • Pad Chivukula • Kevin Coote • Kiyoshi Tachikawa • Yi Cheng • Patty Lim