Horizon Discovery & Pharmahungary Report Positive Early-Stage Results for Novel Micro-RNA Therapeutic for Ischemic Heart Dise...
March 31 2020 - 10:09AM
Business Wire
Micro-RNA compound found suitable for further
development following favorable pharmacokinetic and pharmacodynamic
study results
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.”
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version on businesswire.com: https://www.businesswire.com/news/home/20200331005537/en/
Zyme Communications (Trade and Regional Media) Lorna
Cuddon Tel: +44 (0)7811 996 942 Email:
lorna.cuddon@zymecommunications.com
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