Metagenomi, Inc. (Nasdaq: MGX), a precision genetic medicines
company committed to developing curative therapeutics for patients
using its proprietary, comprehensive metagenomics-derived gene
editing toolbox, today announced it has regained full global rights
to research, develop, manufacture, and commercialize its
wholly-owned gene editing technologies, including base editors and
RNA-mediated integration systems (RIGS), which were previously
subject to exclusive rights granted to Moderna, Inc. Metagenomi and
Moderna have mutually agreed to terminate their collaboration on
primary hyperoxaluria type 1 (PH1), and rights to develop the PH1
program, as well as all other rights granted under the
collaboration, will be returned as part of the termination.
“We are pleased to regain full control of the development of
base editing technology and RIGS systems so that we may advance
them in areas of significant need, such as Alpha-1 antitrypsin
deficiency and Wilson’s disease,” said Brian C. Thomas, Ph.D.,
Chief Executive Officer & Founder, Metagenomi, Inc.
Moderna's involvement in the collaboration, which includes the
PH1 program, ended in conjunction with a strategic prioritization
by the company. Moderna remains a Metagenomi shareholder.
This announcement represents a renewed opportunity to advance
curative genetic medicine through the translation of Metagenomi’s
broad toolbox of wholly-owned gene editing technologies, as well as
a broadened ability to engage with partners in target-specific
application of these technologies.
“Over the past several years, we have built internal
capabilities and expertise required to advance programs to the next
phase of development,” said Dr. Thomas. “In addition to exponential
growth in terms of talent and footprint, we have leveraged
significant private and public funding to greatly expand our gene
editing toolbox, and established in-house manufacturing. With this
announcement, we continue to have the opportunity to accelerate our
mission to become the premier gene editing company.”
Conference Call Information
The Company will discuss this announcement on a conference call
today, Wednesday, May 1, at 5:00pm ET. To join and register for the
call please visit the link here.
About RNA-mediated integration systems
(RIGS)
Our RIGS are being developed in order to encode any type of
genomic modification in an RNA template, and thus create any type
of genome modification necessary to address a disease. Little RIGS
describes systems used for prime editing (e.g., for small genomic
replacements such as transversions, transitions, insertions, and
deletions), while Big RIGS describes systems capable of making
large targeted genomic integrations.
About base editing, including ultra-small (SMART)
systems
Our base editors are highly active and progressing towards in
vivo therapeutic applications. Using PAM interacting domain
engineering, we expanded the genome targetability of our base
editors by 5-fold compared to SpCas9 base editors.
We identified a series of novel, highly efficient ultra-small
nucleases and demonstrated our ability to engineer these systems
into ultra-small base editors and prime editors. We believe our
SMART base editors which are as small as 623 amino acids, represent
some of the smallest editing systems in the industry. As the size
of these systems is well-within the packaging limits of AAV
vectors, our SMART base editors could greatly expand both the
delivery and targetability options of these systems for disease
targets outside the liver.
About Metagenomi
Metagenomi is a precision genetic medicines company committed to
developing curative therapeutics for patients using its
proprietary, comprehensive metagenomics-derived toolbox. Metagenomi
is harnessing the power of metagenomics, the study of genetic
material recovered from the natural environment, to unlock four
billion years of microbial evolution to discover and develop a
suite of novel editing tools capable of correcting any type of
genetic mutation found anywhere in the genome. Its comprehensive
genome editing toolbox includes programmable nucleases, base
editors, and RNA and DNA-mediated integration systems (including
prime editing systems and clustered regularly interspaced short
palindromic repeat associated transposases). Metagenomi believes
its diverse and modular toolbox positions the company to access the
entire genome and select the optimal tool to unlock the full
potential of genome editing for patients. For more information,
please visit https://metagenomi.co.
Cautionary Note Regarding
Forward‐Looking
Statements
This press release contains“forward-looking statements” within
the meaning of Section 27A of the Securities Act of 1933 and
Section 21E of the Securities Exchange Act of 1934, each as
amended. Such statements, which are often indicated by terms such
as“anticipate,” “believe,” “could,” “estimate,” “expect,”
“goal,” “intend,” “look forward to,” “may,” “plan,”
“potential,” “predict,” “project,” “should,” “will,” “would”
and similar expressions, include, but are not limited to, any
statements relating to our growth strategy and product development
programs, including the timing of and our ability to conduct
IND-enabling studies, make regulatory filings such as INDs and
other applications and to obtain regulatory approvals for our
product candidates, statements concerning the potential of
therapies and product candidates, and any other statements that are
not historical facts. Forward looking statements are based on
management’s current expectations and are subject to risks and
uncertainties that could negatively affect our business, operating
results, financial condition, and stock value. Factors that could
cause actual results to differ materially from those currently
anticipated include: risks relating to our growth strategy; our
ability to obtain, perform under, and maintain financing and
strategic agreements and relationships; risks relating to the
results of research and development activities; risks relating to
the timing of starting and completing clinical trials;
uncertainties relating to preclinical and clinical testing; our
dependence on third party suppliers; our ability to attract,
integrate and retain key personnel; the early stage of products
under development; our need for substantial additional funds;
government regulation; patent and intellectual property matters;
competition; as well as other risks described in“Risk Factors,” in
our most recent Form 10-K on file with the SEC. We expressly
disclaim any obligation or undertaking to release publicly any
updates or revisions to any forward-looking statements contained
herein to reflect any change in our expectations or any changes in
events, conditions or circumstances on which any such statement is
based, except as required by law, and we claim the protection of
the safe harbor for forward-looking statements contained in the
Private Securities Litigation Reform Act of 1995.
Investor Contact:
Simon Harnest — CIO, SVP Investor
Relationssimon@metagenomi.co
Media Contact:
Ashlye Hodge — Communications Managerashlye@metagenomi.co
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