Metagenomi Announces Corporate Name Change to Metagenomi Therapeutics, Inc. and Highlights Recent Business Milestones and 2026 Corporate Outlook

Rhea-AI Summary

Metagenomi Therapeutics (Nasdaq: MGX) completed a corporate name change and outlined 2025 achievements plus a 2026 outlook focused on advancing lead program MGX-001 for hemophilia A. Key reported facts: MGX-001 produced durable factor VIII (FVIII) activity in non-human primates over ~19 months, showed no identifiable off-target editing in orthogonal assays, and completed a pre-IND meeting in 4Q 2025. The company expects to submit an IND/CTA in 4Q 2026, initiate first-in-human studies in 2027, and anticipates cash runway into 4Q 2027.

Positive

• MGX-001 showed durable FVIII activity in NHPs (~19 months)
• Completed a pre-IND meeting for MGX-001 in 4Q 2025
• No identifiable off-target editing reported in orthogonal assays
• Targeting IND/CTA submission in 4Q 2026 for first-in-human 2027
• Cash runway extended into 4Q 2027 after pipeline reprioritization

Negative

• MGX-001 has no human clinical data; IND/CTA still pending
• Cash runway limited to 4Q 2027 without disclosed new financing

News Market Reaction – MGX

-2.23%

1 alert

-2.23% News Effect

On the day this news was published, MGX declined 2.23%, reflecting a moderate negative market reaction.

Data tracked by StockTitan Argus on the day of publication.

Key Figures

Pre-IND timing: 4Q 2025 IND/CTA submissions: 4Q 2026 First-in-human start: 2027 +3 more

6 metrics

Pre-IND timing 4Q 2025 Pre-IND meeting for MGX-001 held after NHP preclinical data

IND/CTA submissions 4Q 2026 Target timing for MGX-001 IND/CTA filings

First-in-human start 2027 Planned initiation of MGX-001 first-in-human study

Cash runway through 4Q 2027 Management’s stated operating runway after pipeline reprioritization

NHP study duration approximately 19 months Duration of early NHP study with durable FVIII activity

Wave 1 targets four targets Number of cardiometabolic targets in Wave 1 of Ionis collaboration

Market Reality Check

Price: $1.55 Vol: Volume 256,902 is below t...

normal vol

$1.55 Last Close

Volume Volume 256,902 is below the 20-day average of 338,247, suggesting no outsized trading interest pre-announcement. normal

Technical Shares at $1.79 are trading below the 200-day MA of $1.91 and 55.25% below the 52-week high of $4.00.

Peers on Argus

MGX was up 1.13% while close peers showed mixed moves (e.g., VANI up 2.31%, NRXP...

1 Up

MGX was up 1.13% while close peers showed mixed moves (e.g., VANI up 2.31%, NRXP down 5.53%). Momentum scanner flagged only one biotech peer (IMMX) moving up, indicating today’s action appears stock-specific rather than a sector-wide rotation.

Historical Context

5 past events · Latest: Dec 01 (Positive)

Pattern 5 events

Date	Event	Sentiment	Move	Catalyst
Dec 01	Collaboration data update	Positive	-8.6%	APOC3 collaboration preclinical data presentation at a Nature conference.
Nov 17	Conference appearance	Neutral	-2.3%	Jefferies Global Healthcare Conference presentation announcement by CEO.
Nov 11	Earnings and strategy	Neutral	+2.3%	Q3 2025 results, MGX-001 update, leadership changes, cost reductions.
Nov 11	Preclinical data update	Positive	+2.3%	MGX-001 NHP dose‑range data supporting advancement to clinical development.
Aug 12	Earnings and pipeline	Positive	+3.2%	Q2 2025 results and progress on MGX-001 with long‑term FVIII activity.

Pattern Detected

MGX has typically reacted positively to earnings and MGX-001 data, but conference‑style updates have seen weaker or negative follow‑through.

Recent Company History

Over the last six months, MGX has highlighted steady progress in its MGX-001 hemophilia A program and its Ionis collaboration. On Aug 12, 2025, Q2 results emphasized a strong cash position and durable FVIII activity. On Nov 11, 2025, Q3 results and a strategic pipeline prioritization extended runway into 4Q 2027 and confirmed plans for IND/CTA submissions by 4Q 2026. Subsequent news in November and December focused on conference presentations and APOC3 data, which drew more muted or negative price reactions.

Market Pulse Summary

This announcement emphasizes MGX’s strategic focus on MGX-001 and related in vivo gene-editing progr...

Analysis

This announcement emphasizes MGX’s strategic focus on MGX-001 and related in vivo gene-editing programs, with pre-IND interactions completed in 4Q 2025 and IND/CTA submissions targeted for 4Q 2026. Management reiterates an anticipated cash runway through 4Q 2027 after pipeline reprioritization. Investors may track execution against the 2027 first-in-human goal, additional data in secreted protein disorders, and progress within the four cardiometabolic targets in the Ionis collaboration.

Key Terms

pre-IND, IND/CTA, gene-editing, in vivo, +4 more

8 terms

pre-IND regulatory

"Completed a pre-IND meeting for MGX-001 in 4Q 2025"

"Pre-ind" is short for "pre-indication" and refers to the period before a formal announcement or official signal that a significant change or event is about to happen, such as a company preparing to release important news. For investors, it can signal a time of increased activity or uncertainty, as market participants try to interpret hints and anticipate future developments. Recognizing pre-ind conditions helps investors make more informed decisions ahead of major shifts.

IND/CTA regulatory

"MGX-001 on track for IND/CTA submissions in 4Q 2026"

An IND/CTA is a formal regulatory submission that asks health authorities for permission to test an experimental drug or treatment in humans — IND is the U.S. route, CTA is the equivalent in many other countries. Think of it as the safety permit a developer must obtain before starting human trials; investors watch these filings because approval clears a major regulatory hurdle, changes development timelines and risk, and can materially affect a program’s value and funding needs.

gene-editing medical

"leveraging its most advanced, signature gene-editing capabilities"

Gene-editing is a set of laboratory techniques that change an organism’s DNA by adding, removing or altering specific genetic instructions—think of it as editing words in a document to fix a sentence. For investors, it matters because these tools can create new treatments, improve agricultural crops or reduce manufacturing costs, potentially driving product value, regulatory risk, research milestones and long-term revenue prospects for companies involved.

in vivo medical

"an in vivo genome editing company capitalizing on its proprietary technologies"

In vivo describes tests or experiments performed inside a living organism, such as an animal or human, to observe how a drug, device or biological process behaves in a real, functioning body. Investors care because in vivo results reveal safety, effectiveness and possible side effects that lab tests cannot, much like road-testing a prototype car in traffic rather than only on a bench — outcomes can strongly influence regulatory approval, clinical success and a company’s valuation.

non-human primates medical

"curative FVIII activity in non-human primates (NHPs)"

Non-human primates are members of the primate family other than people — such as monkeys and apes — that are used in biomedical research because their biology is closer to humans than rodents. For investors, results from studies in these animals can strongly influence a drug or vaccine’s safety profile, development timeline, regulatory chances and costs, acting like a high-fidelity dress rehearsal before human clinical trials.

off-target editing medical

"no identifiable off-target editing, representing a therapy with best-in-class"

Off-target editing is when a gene-editing technique changes DNA at locations other than the intended site, producing unintended genetic alterations. For investors this matters because those unexpected edits can create safety risks, reduce a therapy’s effectiveness, and lead to extra studies, regulatory delays or added costs — like a paint job that accidentally splashes neighboring surfaces and requires costly fixes.

orthogonal assays medical

"Demonstrated no identifiable off-target editing in a series of orthogonal assays"

Orthogonal assays are independent laboratory tests that use different methods to measure the same biological effect or signal, so one test verifies the findings of another — like checking a measurement with both a ruler and a laser. For investors, they matter because independent confirmation reduces the chance of false positives, strengthens the credibility of scientific claims, and lowers technical and regulatory risk that can affect a program's timeline and value.

proof-of-concept medical

"Demonstrated in vivo proof-of-concept in NHPs for Antithrombin (AT-III)"

A proof-of-concept is a demonstration that shows a new idea or method can work as intended, serving as a small-scale test before full development. For investors, it signals that a concept has been successfully tested in principle, reducing uncertainty about whether it can be practically implemented. This helps determine if further investment or effort is justified to develop the idea further.

AI-generated analysis. Not financial advice.

New name reflects the Company’s strategic evolution focused on later-stage preclinical assets leveraging its most advanced, signature gene-editing capabilities

Pre-IND meeting held in 4Q 2025 following MGX-001 preclinical data demonstrating curative FVIII activity in non-human primates (NHPs); MGX-001 on track for IND/CTA submissions in 4Q 2026

Cash runway anticipated through 4Q 2027

EMERYVILLE, Calif., Jan. 12, 2026 (GLOBE NEWSWIRE) -- Metagenomi Therapeutics, Inc. (Nasdaq: MGX) (the “Company”), an in vivo genome editing company capitalizing on its proprietary technologies to create curative genetic medicines for patients, today announced that it has completed its corporate name change to Metagenomi Therapeutics, Inc. to reflect the Company’s strategic evolution focused on driving forward the Company’s lead program in hemophilia A and other compelling programs and technologies that have the highest probability of near-term success. The Company also provided a summary of milestones achieved across its therapeutic development programs and technology platforms in 2025 as well as anticipated milestones in 2026.

“I am excited by the direction we are headed as we enter 2026, led by MGX-001, our wholly owned hemophilia A program. MGX-001 recently demonstrated curative factor VIII (FVIII) activity in non-human primates with clear dose-dependent activity and no identifiable off-target editing, representing a therapy with best-in-class treatment potential,” said Jian Irish, Ph.D., M.B.A., President and Chief Executive Officer of the Company. “The study informs our anticipated clinical dose regimen and based on recent regulatory engagement, we feel well positioned to file our IND/CTA submissions before year end 2026 and initiate a first-in-human study in 2027. The recent steps we took to strategically reprioritize our pipeline provide us a strengthened balance sheet with runway anticipated into the fourth quarter of 2027. Looking ahead, our new corporate name represents our focus and commitment to delivering curative genetic medicines to patients and accelerating development of these medicines by strategically deploying our most advanced, proprietary technologies built on our foundational science of metagenomics.”

Recent Pipeline Advancement and Corporate Updates

MGX-001 - Hemophilia A Program:

2025 Achievements

• New preclinical data from MGX-001 hemophilia A program demonstrated curative FVIII activity in NHPs and informed a clinical dose regimen strategy for a therapy with best-in-class treatment potential supporting advancement into clinical development.
• Achieved durable FVIII activity in an early NHP study over the approximately 19-month study duration using a cynomolgus surrogate FVIII gene (cFVIII).
• Demonstrated no identifiable off-target editing in a series of orthogonal assays employed to discover and validate potential off-target sites.
• Completed a pre-IND meeting for MGX-001 in 4Q 2025 and based on feedback remains on track to advance MGX-001 into clinical development.

2026 Anticipated Milestones

• Submit IND/CTA in 4Q 2026 to advance MGX-001 into first-in-human studies in 2027.
  

Secreted Protein Deficiencies

2025 Achievements

• Demonstrated in vivo proof-of-concept in NHPs for Antithrombin (AT-III) Deficiency evidencing the potential to expand the MGX-001 site-specific genome integration system into additional curative therapies for secreted protein disorders.
  
• The Company is evaluating optimal additional applications of the MGX-001 system, including AT-III and other secreted protein deficiencies, as it advances MGX-001 in hemophilia A toward proof-of-concept in humans.
  

Cardiometabolic Programs:

2025 Achievements

• Presented preclinical data supporting APOC3 as a new collaboration target with Ionis Pharmaceuticals (“Ionis”) at the Nature Conference, “Cracking the Code: Nucleic Acid Medicines Coming of Age”
  • APOC3 is a part of Wave 1 of the Ionis collaboration, which includes four targets in significant cardiometabolic indications.

Corporate Highlights

• Completed a strategic pipeline prioritization to focus on the advancement of the Company’s leading in vivo therapeutics including the MGX-001 program in hemophilia A, other secreted protein disorders leveraging the MGX-001 approach, and cardiometabolic indications in collaboration with Ionis.
  
• In conjunction with the strategic evolution, the Company appointed Jian Irish, Ph.D., M.B.A., as Chief Executive Officer. Based on a revised capital allocation strategy, the Company extended its anticipated cash runway into 4Q 2027.
  

About Metagenomi Therapeutics, Inc.

Metagenomi Therapeutics, Inc. is an in vivo genome editing company capitalizing on its proprietary technologies to create curative genetic medicines for patients. The Company was founded on the science of metagenomics, the study of genetic materials recovered from the natural environment, to discover and develop a suite of novel editing tools potentially capable of correcting any type of genetic mutation found anywhere in the human genome. The Company focuses on high value programs in disease indications with well-understood biology and clearly defined clinical development and regulatory pathways. Going forward, the Company intends to continue to expand its pipeline by leveraging its proprietary genetic editing capabilities in site specific deletion, integration and correction.

MGX-001, the Company’s lead, wholly-owned development program in hemophilia A, has demonstrated a preclinical profile potentially competitive with best-in-class treatment options, including targeted genome editing and durable gene expression in a one-time treatment. MGX-001 is designed to provide curative, life-long protection from bleeding events and joint damage in adults and children with hemophilia A. The Company is also currently pursuing other secreted protein deficiencies leveraging the MGX-001 site-specific genome integration system and partnered assets targeting cardiometabolic diseases. 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 product development programs, including the timing of and our ability to conduct IND-enabling studies and make regulatory filings such as INDs, expectations regarding MGX-001 including the preclinical profile being potentially competitive with best-in-class treatment options and timing to submit the IND/CTA package, statements regarding the Company’s plans to prioritize its preclinical pipeline and potential for value creation and sustainable growth, statements regarding upcoming milestones including the nomination of development candidates, statements concerning the potential of therapies and product candidates, statements concerning the impact of the organizational restructuring, statements concerning our anticipated cash runway, 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 IND submissions and 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 and the current regulatory environment; patent and intellectual property matters; competition; the volatility of capital markets and other adverse macroeconomic factors; as well as other risks described in ​“Risk Factors,” in our most recent Form 10-K and other risk factors set forth from time to time in our filings with the Securities and Exchange Commission made pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934, as amended. 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:

Stephen Jasper
Gilmartin Group
stephen@gilmartinir.com

FAQ

What does Metagenomi Therapeutics (MGX) announce about its corporate name?

The company changed its corporate name to Metagenomi Therapeutics to reflect focus on later-stage preclinical gene-editing assets.

What were the key 2025 MGX-001 results reported by Metagenomi (MGX)?

MGX-001 produced durable FVIII activity in NHPs over ~19 months and showed no identifiable off-target editing in orthogonal assays.

When does Metagenomi (MGX) plan to submit an IND/CTA for MGX-001?

The company expects to submit an IND/CTA in 4Q 2026 and to initiate first-in-human studies in 2027.

What is Metagenomi's (MGX) cash runway timeline?

Based on the revised capital allocation strategy, cash runway is anticipated into 4Q 2027.

Did Metagenomi (MGX) report any safety or off-target findings for MGX-001?

The company reported no identifiable off-target editing detected across the orthogonal assays used.

Are there other programs highlighted by Metagenomi (MGX) besides MGX-001?

Yes; the company cited expansion of the MGX-001 integration system to secreted protein deficiencies (e.g., AT-III) and cardiometabolic targets via an Ionis collaboration.