The world has stepped into the golden age of life sciences. The constantly outpouring monoclonal antibody drugs have get the venture capitalists satisfactory returns, and those adventurers now start to focus on the field rarely touched in drug inventions: research of mRNA therapy, to treat genetic diseases, cancers, and infectious diseases based on mRNA. Moderna Therapeutics is a global leader in this technology.
Moderna is now using lipid nanoparticles (the top circle in the figure) to shuttle mRNA (the orange-yellow part in the figure) into cells (bottom right in the figure), to translate it into therapeutic proteins (bottom left in the figure).
Let’s return to April 2018. Dozens of venture capitalists attended an investment meeting launched by Moderna Therapeutics in a Marriott Hotel conference hall in Massachusetts. They gathered to listen to Moderna Therapeutics’ introduction of a great project that could be fostered into a "unicorn" in the future. The technology platform of the company was studying injection of carefully designed mRNA into human body to prompt the body to make its own "medicine" to cure diseases. The concept has attracted billions of USD in funding at the meeting.
Stephen Hoge, CEO of Moderna, explained to investors in an interview, "Why are we so passionate about mRNA?" He explained, "In fact, all life that we know flows through mRNA. ... In our language, mRNA is the software of life."
(Note: mRNA is a key substance with DNA genetic information; it decides the amino acid sequence of body’s peptide chain, and regulates various components that eventually produce human body.)
"Our ultimate goal is to put mRNA that can cure diseases into human body, to make it form a drug factory inside you. It will make diseases achieve self-cure inside systemic circulation. We will no longer rely on external small molecule or macromolecular monoclonal antibody drugs."
The idea Dr. Hoge was selling seems straightforward, but it is in fact not. When modified mRNA is injected into the body, it triggers virus-detecting immune sensors. That event causes cells to shut down therapeutic protein transcription, thus "sealing up the factory", and foiling the therapy. This is a challenge that has long troubled mRNA delivery experts.
"Moderna now employs about 600 people, the majority of them scientists, and spends enormous sums—over USD 450 million in the past five years—learning how to make and improve its mRNA therapies. This year, the firm will invest another USD 100 million, and add USD 500 million in the future."
Hoge said at the meeting, "It’s an astonishing sum for a company that is still years away from a marketed product. But what I see is the unprecedented enthusiasm of you siting in front of me. I will get ready for the clinical trial of mRNA therapy using the USD 100 billion received this year."
"Because we don’t want to develop a drug or therapy just waiting to be acquired by big pharmaceutical enterprises. We want to bring a disruptive unicorn to the drug industry, like Amgen, Biogen, and Genentech, did when they began developing protein therapies called biologics in the eighties. Biologics are now the segment with the fastest-growing profits of the drug industry. We believe mRNA could replace them all. This is a 20-year job. We believe we are just starting."
Hoge shouted on the site, "You could ultimately use mRNA to express any protein and perhaps treat almost any disease. It is almost limitless what it can do."
Moderna announced the exciting results that evening; they invited academic experts who had doubts, showed to them some basic academic studies as to how to convert mRNA into product, and how to use mRNA in the rare genetic diseases, cancers, and infectious disease vaccines, which excited the investors on the site. They saw the possibility of therapeutic mRNA development. Moderna completed USD 1.7 billion financing on the site that evening.
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