Cambridge, UK, and Budapest, Hungary, 31 March 2020: 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, under which several miRNA compounds for cytoprotection in ischemic diseases, termed “ProtectomiRs”, have been identified. The aim is to develop a curative treatment for ischemic heart disease, the number one cause of mortality worldwide. This is a major unmet medical need, as currently only symptomatic treatment is available.

Horizon’s Dharmacon™ custom RNA synthesis service is able to support the broadest variety of projects, including generating long RNA oligos, custom RNA inhibitors for the study of microRNA biology, custom phosphoramidites, specialized chemical modifications or modified bases, and larger scale orders for in vivo and preclinical development. 

Terry Pizzie, CEO, Horizon Discovery said: “The expertise in custom synthesis of oligonucleotides at our Boulder facility has been well known for many years. We are extremely excited to be part of this innovative approach to developing new therapeutics.”

Péter Ferdinandy, MD, PhD, MBA, CEO & Founder of Pharmahungary Group, commented: “The pharmacokinetic study was an important step towards the development of this and ProtectomiRs. We look forward to continuing our partnership with Horizon Discovery, to work toward our goal of developing novel drugs for treatment of ischemic heart diseases.”