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 German and U.S. firms are leading the way in synthetic messenger
RNA, or mRNA technology, a new approach to tackling a range of
hard-to-treat diseases.
In theory, the promise of mRNA is enormous, ranging from cancer to
infectious diseases to heart and kidney disorders, since it could be
used to tackle the 80 percent of proteins that are difficult to
affect with existing medicines.
Despite a recent sell-off in biotech stocks, sparked by U.S.
Democratic Presidential candidate Hillary Clinton's threat to crack
down on drug pricing, enthusiasm for mRNA, is rising.
Four-year-old Moderna Therapeutics, based in Massachusetts, which
raised a record-breaking $450 million in a private funding round in
January, valuing it at $3 billion, has so far hogged the limelight
but German companies are flexing their muscles.
Privately-held CureVac in the university town of Tuebingen, which
already has backing from Microsoft co-founder Bill Gates thanks to
its vaccine work, last week raised $110 million from new investors,
valuing it at $1.6 billion.
 And Mainz-based BioNTech clinched a deal possibly worth up to $1.5
billion with Sanofi to use mRNA to fight cancer. BioNTech is owned
by the Struengmann family who sold generic drugmaker Hexal to
Novartis in 2005.
Driving the point home, the third International mRNA Health
Conference is being held in Berlin this week. Moderna, CureVac and
BioNTech are all sponsoring the event.
Today's biotech medicines use complex proteins or antibodies to
treat disease, while traditional tablets such as aspirin or Viagra
are simple chemicals. Harnessing synthetic mRNA is a different model
altogether.
In effect, mRNA serves as software that can be injected into the
body to instruct ribosomes, the "3D-printers" found inside cells, to
churn out desired proteins.
"This is a radically different approach from conventional
approaches, where therapeutic proteins are produced outside the
human body and active ingredients need to be isolated, purified and
cooled and then be inserted back into the human body at great
complexity and cost," said CureVac co-founder and CEO Ingmar Hoerr.
LONG-TERM BET
Single-stranded RNA, which helps to relay information encoded in
double-helix DNA molecules, for a long time attracted only academic
interest because it was seen as too unstable to handle.
But advances in chemistry have increased both stability and potency,
paving the way to the creation of potential drugs, although big
challenges remain to prove their clinical value.
"It's still a long-term bet," said Andy Smith, chief investment
officer at fund manager Mann Bioinvest. "I don't think it is ready
for prime time just yet."
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Still, ever more big pharmaceutical companies are lining up to do
deals with mRNA firms.
Some of the most advanced work to date has been in vaccines, where
CureVac is a leader with clinical trials in prostate cancer and
rabies. Overall, the company has tested its products on more than
300 clinical trial participants and its relatively maturity means it
could be the first mRNA player to go public.
The company says it is getting ready for a possible initial public
offering, but declines to say when.
Moderna has yet to start clinical trials, although it has ambitious
plans for parallel studies with multiple drug candidates, alongside
partners including AstraZeneca, Merck & Co and Alexion.
Marcus Schindler, vice president for cardiovascular and metabolic
drug discovery at AstraZeneca, is particularly excited about the
promise of mRNA in producing a radical new treatment for heart
failure, by regenerating cardiac tissue.
AstraZeneca plans to start testing mRNA as an agent to improve
coronary blood flow in human trials in 2016. This follows promising
data in animals that suggests synthetic mRNA can be delivered
accurately and produce the right amount of protein.
"The field is moving very rapidly," Schindler said. "I predict it
will have a significant impact." As a chemically synthesized
product, Schindler believes mRNA should be cheaper to make than
current costly antibody drugs.
But potential obstacles remain in the form of optimizing drug
delivery and possible side effects, while the history of biotech
advances suggests some unforeseen setbacks are likely as the
technology moves into large clinical trials.
Other companies working in the mRNA area include Argos Therapeutics,
eTheRNA, Ethris and Factor Bioscience.
(editing by David Stamp)
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