The Endosomal Escape Vehicle Platform Safely & Effectively Delivers Oligonucleotide Therapeutics to Skeletal and Cardiac Muscle Tissue for the Potential Treatment of Duchenne Muscular Dystrophy
Time: 3:30 pm
day: Conference Day Two
Details:
- Developing the Endosomal Escape Vehicle (EEV™) family of cyclic cell-penetrating peptides that were developed to overcome limitations of intracellular therapeutics
- Delivering antisense oligonucleotides using EEV-PMO constructs to skeletal and cardiac muscle in preclinical models of Duchenne muscular dystrophy (DMD)
- Discussing how healthy human volunteers were administered a single dose of ENTR601-44, a DMD exon 44 skipping EEV-PMO construct, and showed dose-dependent exon 44 skipping with no adverse events related to study drug
- Demonstrating the therapeutic potential of the EEV platform and using findings to support further clinical development of EEV-PMO constructs in people living with DMD
