Metagenomi To Present New Preclinical Hemophilia A Data at American Society of Hematology (ASH) 66th Annual Meeting

Rhea-AI Summary

Metagenomi (Nasdaq: MGX) announced that its abstract on Factor VIII gene insertion has been accepted for oral presentation at the American Society of Hematology (ASH) 66th Annual Meeting in December 2024. The presentation will showcase preclinical proof-of-concept data for their lead candidate MGX-001, which aims to provide a single, curative treatment for hemophilia A.

The research demonstrates durable Factor VIII expression in nonhuman primates, addressing current therapy limitations that fail to prevent breakthrough bleeding. The presentation, led by VP Preclinical Alan Brooks, PhD, is scheduled for December 9, 2024, at 5:30 PM in San Diego Convention Center.

EMERYVILLE, Calif., Nov. 05, 2024 (GLOBE NEWSWIRE) -- 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 Primates
Presenting Author: Alan Brooks, PhD, VP Preclinical, Metagenomi
Date/Time: Monday, December 9, 2024, 5:30 PM
Session Name: 801. Gene Therapies: Gene Therapies for Hemophilia, Cancer and Immunodeficiencies
Location: San Diego Convention Center, Room 33
Publication 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://​metageno​mi​.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 Relations
simon@​metagenomi.​co

Media Contact:
Ashlye Hodge - Communications Manager
ashlye@​metagenomi.​co

FAQ

What is Metagenomi's MGX-001 treatment designed to achieve for hemophilia A patients?

MGX-001 is designed to provide a single, curative treatment for both adults and children with hemophilia A, aiming to overcome limitations of existing therapies that don't prevent breakthrough bleeding.

When will Metagenomi (MGX) present their hemophilia A research at ASH 2024?

Metagenomi will present their hemophilia A research on Monday, December 9, 2024, at 5:30 PM during the ASH 66th Annual Meeting in San Diego.

What are the key findings of Metagenomi's (MGX) Factor VIII gene research?

The research demonstrates successful site-specific insertion of Factor VIII gene resulting in durable Factor VIII expression in nonhuman primates, showing promising preclinical proof-of-concept data.