The TRAIN-HEART consortium, funded by the European Commission (2019-2023), is made up to train a league of 15 promising fellows that harness novel insights in the pathogenesis of ischemic heart failure, study the therapeutic potential of existing RNA therapeutics and improve its efficacy by the design of novel drug delivery systems.
TRAIN-HEART brings together leading academic teams and (biotech) companies covering various disciplines ranging from fundamental research to clinical pharmacology and gene therapy to drug delivery applications, have teamed up in the European Union.
The TRAIN-HEART network aims to gain viable insight in the pathogenesis of ischemic heart failure which will serve as a basis for drug discovery and drug delivery efforts that aim to therapeutically target specific molecules and mechanisms within cardiomyocytes for the treatment of ischemic heart failure.
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News
Covid-19 infections can lead to serious inflammations of the lung and the formation of scar tissue (fibrosis). This can have a long-term impact on lung function and is one of the causes of “long covid”. A team working with Stefan Engelhardt has developed a new RNA-based drug that can prevent these inflammatory lung conditions. When administered via the respiratory passages, it quickly targets immune cells in the alveoli (tiny air sacs in the lungs) and inhibits a microRNA molecule found in these cells.
In Covid patients, misguided immune cells called macrophages play a substantial role in severe inflammatory infections and lung damage. However, when the new drug blocked the microRNA molecule in macrophages in mice, there was a significant reduction in inflammation and lung damage and a considerable improvement in lung function. Stefan Engelhardt is confident that serious infections and thus the kind of lung damage associated with long covid can be prevented in human patients receiving the drug through an inhaler.
RNA drug would be the first in its class
Starting with RCS-21, a sugar-coupled oligonucleotide inhibitor, Engelhardt and his partners are developing a drug to treat Covid-19 infections. The RNA drug would be the first in its class for targeted delivery to macrophages. “Because it influences immune cells, and not the virus itself, RCS-21 should also be effective in fighting omicron infections or even more aggressive variants arising in the future,” says Deepak Ramanujam, co-inventor and a group leader at TUM’s Institute of Pharmacology and Toxicology. A global patent process is underway. The Federal Ministry of Education and Research (BMBF) will provide 7 million euros in funding under the “Directive to support research and development for urgently needed treatments for SARS-CoV-2”.
The Federal Institute for Drugs and Medical Devices (BfArM) issued a positive assessment of RCS-21 in an advice meeting. This is an important prerequisite for the further development of a drug. “We expect to complete the pre-clinical toxicological assessment in the third quarter of 2022. We then plan to enter phase one of the clinical assessment in about a year,” says Stefan Engelhardt. In the first of three clinical trial phases before final approval, a drug is initially administered to a small group of individuals.
Further information:
www.bmbf.de/bmbf/shareddocs/pressemitteilungen/de/2021/12/231221-Therapeutika.html
Wed, 19 January
This project has received funding from the European Union’s Horizon 2020 research and innovation programme under the Marie Skłodowska-Curie grant agreement no. 813716.
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