Metagenomi Presents Novel, Highly Specific and Efficient Adenine Base Editors for Broad Genome Editing at ESGCT
October 24 2024 - 7:04AM
Metagenomi, Inc. (Nasdaq: MGX), a precision genetic medicines
company committed to developing curative therapeutics for patients
using its proprietary gene editing toolbox, today presented a
poster (P0533 Poster Session III) titled “Efficient and specific
genome editing with metagenomics-derived base editor for human
therapeutic applications” at the European Society of Gene and Cell
Therapy (ESGCT) 31st Annual Congress in Rome, Italy.
“Base editors are powerful gene editing tools that are able to
modify genes through single nucleotide changes, including
multiplexed gene editing applications without risk of
translocations. Our ABEs display critical attributes necessary for
successful multiplexed gene editing with remarkable targetability,
efficiency, precision and tolerability,” said Brian C. Thomas, PhD,
CEO and founder of Metagenomi. “We remain focused on advancing our
base editors for in vivo genome editing programs, while pursuing
partnerships to advance ex vivo cell therapy applications.”
Today’s poster presentation described the following key
advantages of the Metagenomi ABE platform:
Targetability: Metagenomi’s ABE is targetable
to over 95% of the human genome's base pairs, a significantly wider
range of sites than first-generation SpCas9 base editors. This
expanded scope provides unparalleled flexibility in addressing
complex gene target locations.
Efficiency: The ABE platform achieved over 95%
reproducible and durable triplex protein knockdown in primary
T-cells, confirming its highly efficient application for multiplex
gene editing. This platform is also compatible with various
non-viral methods of site-specific template-integration in a
simultaneous step, including CAR, engineered TCR, and/or regulatory
genes leveraging a Metagenomi nuclease. This enables a potent
all-in-one editing solution for the development of next-generation
cell therapies.
Specificity: The ABE demonstrated highly
specific on-target deamination with minimum-to-no indel formation.
Genome-wide analyses confirmed no detectable translocations and no
significant genomic composition differences when compared to
unedited cells. This precision minimizes the risk of unintended
genetic alterations, positioning the company’s ABE for long-term
therapeutic applications.
Tolerability: Metagenomi’s ABE triplex protein
knockdowns exhibited excellent tolerability in cells, with no
adverse effects on cell viability, expansion, or changes in
stress-related gene expression observed post-editing. Tolerability
is especially critical in multiplex editing of primary T-cells,
where maintaining cell health is vital for therapeutic
efficacy.
About MetagenomiMetagenomi 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
StatementsThis 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,
statements concerning the potential of therapies and product
candidates, statements concerning the timing of data presentations
and publications, 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
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described in “Risk Factors,” in our most recent Form 10-K and our
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Commission. We expressly disclaim any obligation or undertaking to
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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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