Horizon Discovery Group plc (LSE: HZD) (“Horizon”, “the Company” or “the Group”), a global leader in the application of gene editing and gene modulation for cell line engineering, and Pharmahungary Group, a preclinical and clinical R&D company, today announced positive early-stage results for a novel micro-RNA therapeutic for ischemic heart diseases. RNA oligonucleotides, custom-manufactured by Horizon at its Boulder, Colorado site (formerly Dharmacon, Inc.), were used in successful studies that have paved the way for in vivo proof-of-concept efficacy studies in further animal models of acute myocardial infarction and post-infarction heart failure.
The micro-RNA (an miRNA-125b*-mimic compound) was identified as suitable for further development based on favorable pharmacokinetic results as well as pharmacodynamic data at the molecular level. This study also represents a significant development in understanding, as very little is currently known about the pharmacokinetic properties of miRNA compounds.
The potential therapeutic has been developed under Pharmahungary’s ProtectomiR™ program, ...