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Positive early-stage results for novel micro-RNA therapeutic for ischemic heart diseases

Horizon Discovery and Pharmahungary have reported a micro-RNA compound suitable for further development

1 Apr 2020
Tom Casburn
Associate Editor

Industry news

Horizon Discovery 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, have 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.

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

“The pharmacokinetic study was an important step towards the development of this and ProtectomiRs," stated Péter Ferdinandy, CEO & Founder of Pharmahungary Group. "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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Gene Expression and Molecular CloningMolecular cloning is a set of techniques that utilizes vectors to transfer recombinant DNA into host cells and is an essential tool for investigating the expression of genes and proteins in bacterial or mammalian cells. A variety of vectors optimized for gene cloning and expression in a range of host organisms are available, alongside competent cells for genetic replication. Here, you can explore a range of molecular tools, high-quality genomic and cDNA libraries, premade clones, transformation and transfection reagents and mutagenesis or gene expression detection assays and expression arrays. Find the best gene expression and molecular cloning products in our peer-reviewed product directory: compare products, check customer reviews and receive pricing direct from manufacturers.RNA InterferenceRNA interference (RNAi) uses siRNA or miRNA for transcriptional silencing, gene knockdown and regulation of gene expression. RNAi requires chemical synthesis, introduction of DNA vectors into cells, an assay of RNAi effects and RNAi quantification or analysis. Consider target sequence selection, reagent preparation, controls, high specificity and effectiveness and low non-specific gene knockdown.Clinical GeneticsMolecular Genetics covers the analysis of hereditary genetic disease and chromosomal abnormalities. Genetics can be analysed using DNA, RNA, and protein microarrays, PCR, RT PCR and DNA sequencing. Genetic equipment includes genetic workstations, thermal cyclers, cooling blocks and electrophoresis products. Diagnostic kits are used for DNA / RNA extraction and purification.Research and DevelopmentCardiovascular DiseaseCardiovascular disease refers to a group of disorders of the heart and blood vessels, including heart attacks, strokes, and high blood pressure. Early diagnosis and effective treatment are essential for improving patient outcomes. Explore cardiovascular disease research and diagnostic tools in our peer-reviewed product directory; compare products, check reviews, and get pricing directly from manufacturers.TherapeuticsTherapeutics are treatments designed to alleviate or cure diseases. These include pharmaceuticals, biologics, and gene therapies, which work by targeting specific disease mechanisms. Advances in personalized medicine and biologics offer new hope for patients with conditions that were previously difficult to treat. Browse our peer-reviewed product directory to find therapeutic solutions for various diseases; compare products, check user reviews, and get pricing directly from manufacturers.miRNA

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Positive early-stage results for novel micro-RNA therapeutic for ischemic heart diseases