Moderna Therapeutics: Medicinal futurism
The industry is very closely following the development of innovative therapy based on matrix RNA
Moderna Therapeutics, one of the most secretive biotech companies, talks about itself with great reluctance. Indeed, why devote competitors to developments in which an abundance of funds has been invested. And yet, interest in her achievements in the innovative therapeutic field based on matrix RNA (mRNA) is warmed up as never before.
Instead of being engaged in the discovery and development of low–molecular and biological drugs - familiar to the industry, or working on a much more mature gene therapy, "Moderna" turns to transcriptional mechanisms. We are talking about delivering mRNA instructions to cells in order to force them to replace, inhibit or, conversely, synthesize proteins that help prevent or cope with the disease.
The "Modern" approach is seen as promising from the standpoint of cost and speed. It is enough to figure out once how to synthesize and deliver mRNAs so that they can be "stamped" on an unlimited scale, only changing the set of encoded instructions. Traditional pharmacy involves launching a full cycle of creating a new drug compound every time from the very beginning, which is why the emergence of breakthrough drugs costs the industry at least $ 1.5 billion and 14 years of labor.
Let little is publicly known about the company, but the major players in the pharmaceutical industry are very well aware of its ideas. For nothing, AstraZeneca, Alexion Pharmaceuticals, Merck & Co. and Vertex Pharmaceuticals have decently invested in a partnership with Moderna.
Moreover, a number of projects are sponsored by the Office of Advanced Biomedical Research and Development (BARDA) under the U.S. Department of Health and Human Services and the Office of Advanced Research Projects (DARPA) under the U.S. Department of Defense.
In January, the company issued a copious statement shedding light on the specifics of its activities regarding mRNA drugs aimed at infectious diseases, immuno-oncology, and cardiovascular diseases. And now, after eight months of silence, I have shared my next achievements and intentions.
Firstly, 16 drug projects have been assembled on the Moderna conveyor, seven of which are undergoing Phase I clinical trials.
Secondly, Moderna has abandoned the business model of research and development based on venture enterprises. Previously, the company's structure included four separate firms: Onkaido Therapeutics, Valera, Elpidera and Caperna, dealing with oncology, infections, rare pathologies and personalized cancer vaccines. Now all of them have been eliminated, moving under the wing of a single brand "Modern".
Thirdly, the construction of a production site for conducting clinical trials of mRNA preparations is underway. The facility with an area of 18.5 thousand square meters will be put into operation by mid-2018.
Fourth, in July, the agreement concluded in January 2014 with Alexion was broken – it decided to restructure its business. There is nothing wrong with this, given that thanks to fruitful cooperation, Moderna has accumulated the necessary expertise in the field of orphan diseases. One of the previous joint projects, mRNA-3704, has been put on the development priority: it is aimed at the therapy of a rare liver disease – methylmalonic acidemia (MMA).
Fifth, the world's first clinical trials of mRNA therapy have been successfully completed. The experimental AZD-8601 encoding vascular endothelial growth factor-A (VEGF-A) and studied jointly with AstraZeneca completed phase I in patients with type 2 diabetes, proving the validity of the approach. It is preparing to enter phase IIa on patients with heart failure undergoing coronary artery bypass grafting.
Local tissue administration of AZD-8601 will help the formation of additional blood vessels, thereby improving blood supply. Such a regenerative mechanism is useful for patients with heart failure or after a heart attack, as well as those suffering from diabetic wounds and ischemic vascular diseases.
Finally, phase I clinical trials of the first Modern immuno-oncological candidate drug, mRNA–2416, which encodes the ligand of the costimulating membrane-bound protein OX40, or CD134, which enhances the functionality of T-lymphocytes in their ability to attach and attack cancer cells, have begun. The experimental mRNA drug is tested by intra-tumor injection.
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18.09.2017