Novel micro-RNA therapeutic hits early targets in ischemic heart diseases

Horizon Discovery has announced positive early-stage results for a novel micro-RNA therapeutic for ischemic heart diseases.

The RNA oligonucleotides, custom-manufactured by Horizon in partnership with Pharmahungary Group, 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 companies have confirmed that the micro-RNA was identified as suitable for further development based on favourable pharmacokinetic results as well as pharmacodynamic data at the molecular level.

The pharmacokinetic study is an “important step towards the development of this and ProtectomiRs” explained Péter Ferdinandy, chief executive officer & founder of Pharmahungary Group.

He continued to say, “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.”

Ischemic heart disease, also called coronary heart disease (CHD) or coronary artery disease, is the term given to heart problems caused by narrowed coronary arteries that supply blood to the heart muscle.

Earlier this year, Horizon announced a new collaboration and license agreement with Mammoth Biosciences, in order to access the company’s novel CRISPR platform and facilitate a new generation of genetically engineered CHO cells.

The collaboration means that the companies will put their heads together to optimise certain CRISPR tools to “rapidly develop proprietary CHO cell lines”, in which selected genes are ‘knocked out’ to improve performance parameters.

The San Fransisco-based biotech built the first CRISPR-based detection platform for diagnostics, which can be used and built on by partners across a variety of industries.

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