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 Scientists say mRNA has the potential to target diseases that cannot
be reached by conventional drugs.
U.S. emergency authorization of the vaccines - one from Moderna Inc
and another from partners Pfizer Inc and BioNTech SE – and their
gene-based manufacturing processes has shown that the Food and Drug
Administration is open to broader use of the previously unproven
technology, according to interviews with eight top experts in the
field of mRNA.
Billions of dollars in capital have flowed into mRNA-focused
companies based on these raised expectations – hundreds of millions
in the last few months alone. And with FDA acceptance, the field is
attracting top scientific talent, experts said.
"There is more attention and focus on mRNA now that it has proven to
be a technology that works," said Glenn Hunzinger, U.S.
pharmaceutical and life sciences leader at consulting firm
PricewaterhouseCoopers.
Regulatory authorization of the vaccines "accelerates the process"
for other mRNA therapies, providing real-world evidence that it is
possible to secure fragile mRNA components during manufacturing and
distribution, said Josh Kellar, co-lead of Boston Consulting Group's
COVID-19 team.
The technology is often likened to the operating system on a
computer, allowing drugmakers to alter their target by inserting new
genetic code into a manufactured form of mRNA, a natural chemical
messenger that instructs the body to produce specific proteins.
Its advantage in vaccines is versatility and speed compared to
standard technology requiring long lead times to produce and purify
proteins and create a vaccine.
TREATMENTS REACH HUMAN TESTING
In 2020, given the urgent need created by the coronavirus pandemic,
more than $5.2 billion was invested in companies engaged in
developing mRNA vaccines and therapeutics - up from $596 million in
2019, according to India-based research firm Roots Analysis.
In just the last three months, mRNA company CureVac NV raised $518
million, Arcturus Therapeutics Holding raised $150 million and
Gilead Sciences Inc agreed to a therapeutic HIV vaccine deal worth
up to $785 million to mRNA company Gritstone Oncology.
Over 150 mRNA vaccines and therapeutics are in development globally,
Roots Analysis said. Most are still in early animal testing, but
more than 30 have reached human testing.
mRNA can be very difficult to work with, making future successes
with treatments uncertain.
Instructions from mRNA are fleeting and non-specific about where in
the body it goes, which works well when directing cells to make a
harmless fragment of the coronavirus to trigger an immune response.
It is more difficult to send those orders to a specific tissue, like
the lungs or heart muscle, which requires different delivery methods
or encapsulation to protect the fragile mRNA molecule.
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 Most of last year's funding
went to COVID-19 projects but enabled companies
to also push forward with other disease
categories.
Moderna, for instance, is working on treatments
for heart disease, cancer, and rare diseases.
Its most advanced non-COVID program is a vaccine
for cytomegalovirus, the leading cause of birth
defects in the United States.
Translate Bio Inc may be the first to market with an mRNA treatment
if its inhaled drug for cystic fibrosis is shown to succeed at
delivering instructions to make a protein known as CFTR to the
lungs, according to Chief Executive Ron Renaud.
The company expects to have interim results from a mid-stage trial
in the second quarter of this year. If safety and efficacy results
are encouraging, it would conduct a larger trial and then
potentially apply for U.S. approval.
People with cystic fibrosis, a life-shortening lung disease, have
mutations in the CFTR gene that cause the protein to malfunction or
not be made at all, leading to a buildup of thick mucus, which
causes lung infections and other serious health complications.
"Most drugs treat the downstream effects of disease ... mRNA is in
the business of replacing what's missing," said Joe Payne, CEO of
Arcturus, which is working to develop mRNA vaccines for COVID-19 and
influenza, as well as medicines for liver disease and cystic
fibrosis.
Dr. Drew Weissman, professor of infectious diseases at the
University of Pennsylvania’s Perelman School of Medicine, is one of
the two scientists credited with a groundbreaking 2005 discovery of
how to alter the molecular structure of mRNA to keep it stable
enough to get past the body's defenses.
In the past nine months, Weissman said 20 companies working in the
field of mRNA have asked him to join their boards of directors, and
the number of labs asking to collaborate with Penn on mRNA work has
nearly tripled.
As fields like cell therapy start to mature, scientists angling for
cutting-edge jobs are reaching out to mRNA companies, said Jacob
Becraft, CEO at Strand Therapeutics, which is working on technology
to control the activity of mRNA therapies.
"A lot of those people are inbounding to my inbox," he said.
(Reporting By Deena Beasley; Editing by Caroline Humer and Bill
Berkrot)
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