- Collaboration with a leader in gene editing systems is
Ionis' latest move to expand and diversify its technology
- CRISPR-Cas gene editing is a natural extension of Ionis'
innovative approach to delivering potentially transformative
- Ionis to hold webcast Nov. 14
at 8 a.m. Eastern Time
CARLSBAD, Calif. and EMERYVILLE, Calif., Nov. 14,
2022 /PRNewswire/ -- Ionis Pharmaceuticals, Inc.
(Nasdaq: IONS) and Metagenomi today announced that the companies
have entered a collaboration that will leverage Ionis' extensive
expertise in RNA-targeted therapeutics and Metagenomi's versatile
next-generation gene editing systems to pursue a mix of validated
and novel genetic targets that have potential to expand therapeutic
options for patients. The companies will jointly conduct research
aimed initially at delivering investigational medicines for up to
four genetic targets. Ionis has the right to add four more targets
upon achievement of pre-determined development milestones.
"Ionis was founded over 30 years ago to discover and develop
novel, highly personalized medicines using our powerful
RNA-targeting technology platform. This partnership with Metagenomi
supports Ionis' strategic objective to advance our technology and
expand our capabilities to deliver precision genetic medicines.
Together, we can broaden the application of gene editing by
leveraging Ionis' vast experience in nucleic acid therapeutics to
optimize and extend the reach of gene editing for liver targets and
to new tissues," said Ionis Chief Executive Officer Brett P. Monia, Ph.D. "This alliance brings our
two companies closer to delivering next-generation therapies with
the potential to revolutionize the treatment of diseases."
"Gene editing has the potential to transform chronic therapies
into potentially curative treatments for patients who currently
have limited options," said Brian C.
Thomas, Ph.D., chief executive officer and founder of
Metagenomi. "This collaboration is a strategic step to combine
Metagenomi's leading gene editing toolbox of diverse, specific and
highly efficient nucleases, with Ionis' pioneering expertise of
RNA-targeted therapeutics, to catalyze the next wave of in vivo
gene editing applications. We continue to follow our strategy of
partnering with the most experienced teams in the genetic medicines
field that will allow us to transform the lives of patients."
Under the terms of the agreement, Ionis will pay $80 million upfront to Metagenomi plus the
potential for future milestone payments and royalties. Wells Fargo
Securities acted as financial advisor to Ionis on the
Ionis will conduct a webcast on Nov.
14 at 8 a.m. Eastern Time to
discuss this announcement and related activities. Interested
parties may access the webcast here. A webcast replay will be
available for a limited time at the same address.
CRISPR Gene Editing
CRISPR (clustered regularly interspaced short palindromic
repeats) gene editing is a technology that modifies the sequence of
DNA in cells utilizing a specific RNA-guided nuclease (Cas enzyme).
Gene editing enzymes act as molecular word processors to correct
the genetic code at the precise spot where it is
About Ionis Pharmaceuticals, Inc.
For more than 30 years, Ionis has been the leader in
RNA-targeted therapy, pioneering new markets and changing standards
of care with its novel antisense technology. Ionis currently has
three marketed medicines and a premier late-stage pipeline
highlighted by industry-leading cardiovascular and neurological
franchises. Our scientific innovation began and continues with the
knowledge that sick people depend on us, which fuels our vision of
becoming a leading, fully integrated biotechnology company.
To learn more about Ionis, visit www.ionispharma.com and follow
us on Twitter @ionispharma.
Metagenomi is a gene editing company committed to developing
potentially curative therapeutics by leveraging a proprietary
toolbox of next-generation gene editing systems to accurately edit
DNA where current technologies cannot. Our metagenomics-powered
discovery platform and analytical expertise reveal novel cellular
machinery sourced from otherwise unknown organisms. We adapt and
forge these naturally evolved systems into powerful gene editing
systems that are ultra-small, extremely efficient, highly specific
and have a decreased risk of immune response. These systems fuel
our pipeline of novel medicines and can be leveraged by partners.
Our goal is to revolutionize gene editing for the benefit of
patients around the world. For more information, please visit
Ionis' Forward-looking Statements
This press release includes forward-looking statements regarding
our collaboration and license agreement with Metagenomi, Inc., the
therapeutic and commercial potential of genetic targets using gene
editing technologies, Ionis' technologies and Ionis' other products
in development. Any statement describing Ionis' goals,
expectations, financial or other projections, intentions or beliefs
is a forward-looking statement and should be considered an at-risk
statement. Such statements are subject to certain risks and
uncertainties, including those related to the impact COVID-19 could
have on our business, and including but not limited to those
related to our commercial products and the medicines in our
pipeline, and particularly those inherent in the process of
discovering, developing and commercializing medicines that are safe
and effective for use as human therapeutics, and in the endeavor of
building a business around such medicines. Ionis' forward-looking
statements also involve assumptions that, if they never materialize
or prove correct, could cause its results to differ materially from
those expressed or implied by such forward-looking statements.
Although Ionis' forward-looking statements reflect the good
faith judgment of its management, these statements are based only
on facts and factors currently known by Ionis. As a result, you are
cautioned not to rely on these forward-looking statements. These
and other risks concerning Ionis' programs are described in
additional detail in Ionis' annual report on Form 10-K for the year
ended Dec. 31, 2021, and our most
recent Form 10-Q quarterly filing, which are on file with the
Securities and Exchange Commission. Copies of these and other
documents are available at www.ionispharma.com
In this press release, unless the context requires otherwise,
"Ionis," "Company," "we," "our," and "us" refers to Ionis
Pharmaceuticals and its subsidiaries.
Ionis Pharmaceuticals® is a trademark of Ionis
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SOURCE Ionis Pharmaceuticals, Inc.