US-based Morderna Therapeutics, headquartered in Cambridge, Massachusetts, is one of the biggest biotechnology startups in the U.S. Since its 2010 founding they have attracted $1.9 billion from investors, partnered with drug giants. Traditional protein-based biotechnology medicines are grown in vats and harvested from living organisms, a tedious and expensive process. The company wants to make it possible for a patient’s body to make its own medicine using strands of genetic instructions called Messenger RNA, or mRNA. The Company says that mRNA can hijack a cell’s protein-making mechanisms to create a drug within the body. If it works, mRNA might well have many applications in infectious diseases, cardiovascular disorders and rare disease.
However, the technology is unproven, as it is difficult to deliver mRNA to the right cells without the immune system destroying it. Critics of the company said it has a one-size-fits-all strategy.”At Moderna, it’s like the answer is mRNA,” says Andrew Allen, CEO of rival Gritstone Oncology, which is planning to begin a trial of a competing cancer vaccine next year.
Today, “personalized medicine” is biotech’s biggest buzzword. The expense of sequencing a whole genome has fallen to about $2,000, from millions in the early 2000s, unlocking new insights on how diseases work. Cancer drugs now routinely come with diagnostic tests and are given only to patients with the right genetic features.
Moderna’s software produced a set of 20 protein targets specific to the type of cancer patient has. Then the researchers assembled DNA building blocks to generate the recipe for the patient’s treatment. Those DNA templates were delivered to Catalent Biologics in Madison, Wisconsin, where these were transcribed into mRNA and packed as a string of instructions into the glass vial.
Scientists believe that some cancers spread because the immune system doesn’t recognize malignant cells as foreign. Moderna’s vaccine would teach the body how to recognize protein targets that appear only on the cancer cells.
The company expects initial results by the end of 2018. It’s too early for the company to say what price it will seek for its labour-intensive product, but the more personalization is involved, the more cost and logistical challenges—so the result has to justify the extra effort and expense to do it,” says Ronald Levy, a professor of medicine at Stanford University.