Metagenomi To Present New Preclinical Hemophilia A Data at American Society of Hematology (ASH) 66th Annual Meeting
06 November 2024 - 8:05AM
Metagenomi, Inc. (Nasdaq: MGX), a precision genetic medicines
company committed to developing curative therapeutics for patients
using its proprietary gene editing toolbox, today announced that
the abstract titled “Site-Specific Insertion of Factor VIII Gene
Results in Durable Factor VIII Expression in Nonhuman Primates“ has
been accepted for an oral presentation at the American Society of
Hematology (ASH) 66th Annual Meeting and Exposition being held
December 7-10, 2024, in San Diego, CA, and online.
“We are advancing our lead development candidate MGX-001 toward
the clinic, with the goal to provide a single, curative treatment
for adults and children with hemophilia A. Our upcoming oral
presentation at ASH will showcase our preclinical proof-of-concept
data in rodents and nonhuman primates. This program is designed to
overcome a significant challenge of existing therapies that are not
curative and do not prevent breakthrough bleeding, leaving a
significant unmet need in the hemophilia A community,” said Brian
C. Thomas, PhD, CEO and Founder of Metagenomi.
ASH Oral Presentation
Details:Title: Site-Specific Insertion of
Factor VIII Gene Results in Durable Factor VIII Expression in
Nonhuman PrimatesPresenting Author: Alan Brooks,
PhD, VP Preclinical, MetagenomiDate/Time: Monday,
December 9, 2024, 5:30 PMSession Name: 801. Gene
Therapies: Gene Therapies for Hemophilia, Cancer and
ImmunodeficienciesLocation: San Diego Convention
Center, Room 33Publication Number: 1055
The abstract can be accessed on the ASH website here.
About Hemophilia A
Hemophilia A is the most common X-linked inherited bleeding
disorder, caused by a large variety of mutations in the FVIII gene
leading to a loss of functional FVIII protein. Intracranial
bleeding is of greatest concern as this can lead to major morbidity
and mortality. Bleeding into joints leads to cumulative joint
damage and is a major cause of morbidity. Diagnosis of severe
disease typically occurs in infancy due to exaggerated bleeding in
response to minor injury or routine medical procedures. Prevalence
is estimated to be up to 26,500 patients in the US and more than
500,000 patients globally according to the World Federation of
Hemophilia (WFH), with the vast majority of patients being
male.
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 (CAST)). 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,
statements concerning the potential of therapies and product
candidates, including our development candidate, MGX-001,
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
intellectual property matters; competition; as well as other risks
described in “Risk Factors,” in our most recent Form 10-K and our
most recent 10-Qs on file with the Securities and Exchange
Commission. 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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