r2,2019 BARRON'S 17
By Josh Nathan-Kazis
Moderna, Translate Bio, and others are developing
drugsthatusemessengerRNAtocuredisease.
Whythiscouldbethenextthinginbiotech.
ILLUSTRATION BY ISRAEL G. VARGAS
Foroveradecade, Michael Heart-
lein has been working on tricking the human
body into curing itself.
The idea struck him in 2008, when he was
working at the pharmaceutical company Shire.
What if you could slip a molecule into the body
that would tell the cells to make proteins that
would cure that person’s illness?
Lots of diseases are caused by a single miss-
ing protein. If you could get the trick to work on
one of those diseases, it could work for many of
them. Dial in the technique and you could start
cranking out new drugs one after the other, like
new generations of the iPhone.
It’s a thrilling idea, and Heartlein is one of
the scores of scientists swept up over the past
few years in the quest to make a drug out of
messenger ribonucleic acid, or mRNA, the mole-
cule that couriers blueprints from a cell’s DNA
to its protein factories.
“Despite a lot of skepticism initially, because
messenger RNA is such a difficult modality to
think about turning into a drug, we generated a
lot of very interesting data really quickly,”
Heartlein said.
Since then, the dazzling theoretical simplicity
of the concept has attracted more than $5 billion
in capital to the four biotech companies leading
the research on the idea, including Translate
Bio (ticker: TBIO), where Heartlein is chief
technical officer. And some of that investment
has come from the biggest names in Big Pharma:
Merck (MRK), Sanofi (SNY), AstraZeneca
(AZN), and GlaxoSmithKline (GSK).
The mRNA drugs wouldn’t just compete with
gene therapies as treatments for diseases caused
by missing proteins. They could also challenge
long-entrenched parts of the vaccine industry
and offer new options for cancer patients.
It is a multibillion-dollar opportunity.
“It opens up a different scope of applications
and diseases,” said Christian Koch, part of the
investment management team at BB Biotech, a
Swiss investment company that is one of the
largest institutional holders of Moderna
(MRNA), the largest mRNA company.