What Metagenomi does

Metagenomi Therapeutics is a clinical-stage in vivo genome editing company that uses metagenomics — the sequencing and analysis of genetic material recovered from uncultivated microorganisms in natural environments — combined with proprietary AI/machine learning algorithms to discover novel gene editing systems. Founded in 2018 and headquartered in Emeryville, California, the company has built a proprietary discovery platform that has sequenced 385 trillion base pairs and identified over 20,000 editing systems, including programmable nucleases, ultra-small systems compatible with single-AAV delivery for extrahepatic targets, the smallest known CRISPR base editors, RIGS (RNA-mediated integration / prime editing), and CRISPR-associated transposases (CASTs) for large gene insertions. Its core technical differentiation is sourcing entirely new CRISPR and CRISPR-adjacent systems from nature rather than engineering variants of SpCas9, enabling extrahepatic delivery and edits beyond the reach of first-generation CRISPR.

Metagenomi's lead wholly-owned therapeutic program is MGX-001 for hemophilia A, which has demonstrated curative factor VIII activity in non-human primates with no identifiable off-target editing, with IND/CTA filing expected in Q4 2026 and first-in-human dosing targeted for Q1 2027. The company also has a flagship strategic partnership with Ionis Pharmaceuticals signed November 2022 covering up to eight genetic targets, with an $80 million upfront payment plus milestone and royalty economics. Revenue is derived almost entirely from licensing/collaboration upfronts and milestone payments rather than product sales. Metagenomi completed an initial public offering on Nasdaq under ticker MGX in 2024, raising approximately $100 million; the stock subsequently declined roughly 85% from its IPO price, with market capitalization falling to approximately $46-52 million by mid-2026.

In November 2025 the company underwent a major defensive restructuring: founder/CEO Brian Thomas departed (remaining on the board), President/COO Jian Irish was elevated to CEO, and the workforce was reduced by 25% to extend cash runway to Q4 2027 and concentrate resources on advancing MGX-001 into the clinic. Strategic focus has shifted from broad pipeline build-out toward a single-asset clinical execution posture, with collaboration revenue and one-time licensing fees supplementing — but not yet replacing — the need for additional capital or partnership events.

Name

Metagenomi

Legal name

Metagenomi Therapeutics, Inc.

Website

https://metagenomi.co

Company type

Public

Founded year

2019

Operating status

Operating

Headcount range

101–250 employees

Short description

Metagenomi Therapeutics is a clinical-stage biotech that uses metagenomics and AI/ML to discover novel gene editing systems from uncultivated microorganisms, advancing a lead hemophilia A gene therapy program (MGX-001) and earning milestone and royalty revenue through a flagship Ionis Pharmaceuticals partnership.

Ownership category

akta.pro rank

Product category

Gene Editing Therapeutics

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714), Scientific Research and Development Services (5417)

SIC

Biological Products, (No Disgnostic Substances) (2836), Pharmaceutical Preparations (2834)

akta.pro primary industry

AI/ML-Enabled Biologic Discovery Platforms (computational design, in silico screening) (HLAAAIAE)

akta.pro secondary industries

Synthetic Biology & Genetic Circuit Design Platforms (pathway engineering, chassis development) (HLAAAIAD), Protein Engineering & Directed Evolution Platforms (enzyme/therapeutic protein optimization) (HLAAAIAC), Target Identification & Validation Platforms (omics-driven, chemoproteomics, genetic validation) (HLAAAIAI)

Keywords

• Gene editing therapeutics
• Metagenomics discovery platform
• CRISPR nucleases
• Genome engineering systems
• In vivo gene therapy

Headquarters

HQ city

Emeryville

HQ country

United States

HQ region

North America

Offices4 records

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Operations, Infrastructure, Marketing or Sales

Revenue model

1. Strategic Partnerships and Collaborations: Revenue from upfront payments, milestone payments, and royalties from pharmaceutical partnerships such as the Ionis collaboration ($80M upfront plus potential additional milestones)
2. Clinical Development and Commercialization: Future revenue from advancement of proprietary therapeutic programs into clinical development and potential commercialization of gene editing therapies

Go-to-market motion2 records

Distribution channels1 record

Marketing channels4 records

Core offering

Metagenomi operates a metagenomics-powered discovery platform that mines uncultivated microorganisms to identify novel gene editing systems, producing a modular toolbox of programmable nucleases, base editors, ultra-small systems, RIGS, and CASTs. The company advances these discoveries into in vivo gene editing therapeutics (lead program MGX-001 for hemophilia A) and monetizes them through strategic pharmaceutical collaborations such as the Ionis partnership ($80M upfront plus milestones and royalties).

Product overview

Metagenomi is an in vivo genome editing company offering a proprietary modular toolbox of next-generation gene editing systems discovered through metagenomics. The platform includes Programmable Nucleases for site-specific gene targeting, Ultra-Small Systems for AAV delivery beyond the liver, Base Editors for single nucleotide changes, RIGS (RNA Mediated Integration Systems) for prime editing and larger edits, and CRISPR Associated Transposases (CASTs) for large gene integrations. The core discovery platform utilizes AI-enabled screening of millions of nuclease and protein libraries. Lead therapeutic programs include MGX-001 for hemophilia A and the APOC3 collaboration with Ionis for hypertriglyceridemia.

Differentiator

Problem solved

Functional benefit

Products and services

• Metagenomics Discovery Platform Proprietary metagenomics-powered discovery platform combining AI/ML with proprietary algorithms to screen metagenomics database for novel CRISPR nucleases and effector enzymes. Used internally to generate gene editing tools for therapeutic development and forms the basis for strategic partnerships.
• MGX-001 Hemophilia A Program Lead in vivo gene editing therapeutic program targeting hemophilia A, demonstrating curative factor VIII activity in non-human primates with no identifiable off-target editing. IND filing expected Q4 2026 with patient dosing in Q1 2027.
• APOC3 Program (Ionis Collaboration) Collaboration with Ionis Pharmaceuticals targeting APOC3 for severe hypertriglyceridemia, using CRISPR/Cas-mediated knockout as a one-time treatment approach. Part of broader partnership covering up to four genetic targets with potential for four additional.

Quantifiable outcome

• 64% knockdown of Atxn2 protein in mice after direct AAV injection
• +3 more outcomes

Named customers1 record

Segments2 records

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

AI capability3 records

Feature7 records

Partnerships

One partnership is on record.

• Ionis PharmaceuticalsflagshipStrategic or Co-development Partner · 14 November 2022Collaboration to develop gene editing therapies combining Ionis' RNA-targeted technology with Metagenomi's gene editing systems. Initially targeting up to four genetic targets with potential for four additional targets upon achievement of predetermined development milestones. Ionis paid $80 million upfront with potential for additional milestone payments and royalties.

Scale indicators12 records

Recent moves6 records

Expansion highlights5 records

Direct peers

• Intellia Therapeutics: Intellia is a clinical-stage gene editing company developing in vivo CRISPR/Cas9 and in vivo/ex vivo cell therapies. Comparable as a public peer developing in vivo gene editing therapeutics with platform-based discovery and a pipeline of wholly-owned and partnered programs.
• Sangamo Therapeutics: Sangamo Therapeutics develops genomic medicines using zinc finger nucleases (ZFNs) and other genome editing platforms, including in vivo programs in hemophilia and other indications. Comparable as an established public gene editing peer pursuing similar therapeutic areas.
• Beam Therapeutics: Beam Therapeutics is a clinical-stage biotech developing base editing and prime editing therapies. Directly comparable as a public gene editing company pioneering next-generation editing modalities that overlap with Metagenomi's base editor and RIGS platforms.
• Verve Therapeutics: Verve Therapeutics develops in vivo base editing therapies for cardiovascular disease, including VERVE-101 for heterozygous familial hypercholesterolemia. Directly comparable in pursuing in vivo gene editing for liver/cardiovascular indications with a similar clinical-stage profile.
• Editas Medicine: Editas Medicine develops CRISPR-based gene editing medicines including EDIT-101 and EDIT-301. Directly comparable as a public, clinical-stage gene editing company with a similar scientific founding history and therapeutic focus.
• Prime Medicine: Prime Medicine develops prime editing therapies for a range of genetic diseases. Comparable as a public, clinical-stage next-generation gene editing company whose prime editing technology overlaps with Metagenomi's RIGS platform.
• CRISPR Therapeutics: CRISPR Therapeutics is a clinical-stage gene editing company developing CRISPR/Cas9-based therapies including Casgevy (approved for sickle cell disease and beta-thalassemia). Directly comparable as a public, gene-editing therapeutics company pursuing in vivo and ex vivo indications.

Emerging players

• Precision BioSciences: Precision BioSciences develops in vivo gene editing using ARCUS meganucleases for genetic diseases and allogeneic CAR-T for oncology. Comparable as a gene editing platform company with both in vivo and ex vivo programs but smaller scale.
• Caribou Biosciences: Caribou Biosciences is a clinical-stage company developing allogeneic CAR-T cell therapies using CRISPR Cas12a chRDNA technology. Comparable as a gene editing company but with a primary focus on oncology cell therapy rather than in vivo therapeutics.
• Tessera Therapeutics: Tessera Therapeutics is developing Gene Writing technology for precise genomic modifications, including large-gene integrations. Comparable as an emerging gene writing/editing platform pursuing similar next-generation capabilities like CAST-mediated integration.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat4 records

Key risks6 records

Key highlights7 records

Customer concentration

• LinkedInlinkedin.com/company/metagenomi
• Xx.com/metagenomi

Revenue estimate

Valuation estimate

Number of profiles

Profiles11 records

Funding overview

Funding rounds6 records

Investors26 records

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Metagenomi