Moderna Therapeutics, whose unusual and still unproven drug-producing technology has attracted more than a billion dollars in funding and partnerships, is joining the scramble to produce a vaccine to protect against Zika virus infection.
The virus, which has gone from a biological afterthought to a global health crisis in a few years, can cause microcephaly, or stunted head growth, and neurological devastation in unborn babies whose mothers are infected. It spreads through the bite of the Aedes aegypti mosquito and through unprotected sex. It has wreaked the most havoc in Brazil, with more than 1,800 cases of Zika-related defects confirmed so far. (The virus is also tied to the potentially deadly Guillain-Barré syndrome, an autoimmune condition.)
This year, Zika has spread throughout the Americas and to the United States. Florida health officials have zeroed in on two Miami-area neighborhoods as areas of active transmission.
The race to provide a vaccine is on. The National Institutes of Health and the private biotech Inovio Pharmaceuticals (NASDAQ: INO) have begun testing vaccines in people. Cambridge, MA-based Moderna announced today that with an $8 million U.S. biodefense grant in hand, it will move its own vaccine into human testing in coming months if the Food and Drug Administration gives the green light. The grant, from the Biomedical Advanced Research and Development Authority, could expand to $125 million if the vaccine progresses through clinical testing and requires large-scale production, Moderna said.
The NIH and Inovio vaccines, which use the DNA from Zika, began testing this summer and could produce their first human data in early 2017.
Moderna has four satellite companies: two aimed at cancer, one at rare diseases, and one at infectious diseases. All their medicines work in similar fashion. Synthetic messenger RNA (mRNA), small strings of biological code, are injected into a patient’s cells. Cells naturally use mRNA to turn the blueprint of their DNA into the vast array of proteins that make up life. Moderna’s synthesized mRNA are supposed to work the same way, giving cells instructions to make proteins that the body uses to fight disease. For vaccines, however, the Moderna injection would prompt cells to make virus-like proteins that would, in turn, stimulate an immune defense against a future infection.
Moderna says it has tested its mRNA vaccines, aimed at other undisclosed infectious diseases, in about 250 healthy volunteers so far. It will publish data from those tests next year. The Zika vaccine has generated a “robust immune response” in animal testing, Moderna spokeswoman Liz Melone said via email. Its synthesized mRNA triggers cells to make virus-like fragments “that are essentially identical to those produced when the cell is infected,” Melone said. That similarity could be an advantage over vaccines produced outside the body, she said, but that remains conjecture until the different vaccines go through a battery of clinical tests in the coming years.
Moderna also confirmed today that it has raised $474 million, its second funding round of more than $400 million in the past two years. News of the massive financing emerged last week through a regulatory filing.
The company reported it has $1.4 billion in cash, some of which it will invest in manufacturing as several of its programs, both partnered and unpartnered, move into clinical trials. Moderna also said it would expand its mRNA drug technology into new therapeutic areas.
Image of A. aegypti courtesy of the U.S. Department of Agriculture.