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Therapeutic Approach

Our Pipeline

Our Lead Program

MGX-001: Combating Hemophilia A

Stan­dard of care today man­ages bleeds — it does not pro­vide a cure. Every cur­rent­ly approved ther­a­py is chron­ic and non-cura­tive — the oppor­tu­ni­ty for a durable, cura­tive treat­ment remains open.

#01.

Patient Population

  • ~25,000

    patients in the U.S.1

  • ~500,000

    world­wide2

  • ~45%

    severe and mod­er­ate pre­sent­ing as severe (at great­est risk of life-threat­en­ing bleeds)3

Hemo­phil­ia A is the most com­mon X‑linked and de novo bleed­ing dis­or­der; onset is typ­i­cal­ly iden­ti­fied in infan­cy or ear­ly child­hood, requir­ing life­long dis­ease management.

1. CDC Sur­veil­lance, 2024.
2. Stone­brak­er, J.S., et al, 2010. Haemophil­ia 16:20 – 32. 
3. CDC Sur­veil­lance, 2024.

#02.

Unmet Medical Needs

Cur­rent stan­dard of care — FVIII replace­ment (IV, dosed 1 – 3x/​week) and bis­pe­cif­ic anti­body ​“mimet­ics” (SQ, dosed every 1 – 4 weeks) — requires chron­ic dos­ing, faces sig­nif­i­cant adher­ence chal­lenges, and does not elim­i­nate break­through bleed­ing risk.

While recent AAV-based gene ther­a­pies showed ear­ly promise for treat­ing hemo­phil­ia A, vari­able effi­ca­cy and declin­ing fac­tor VIII (FVIII) expres­sion have lim­it­ed their suc­cess. Most notably, these ther­a­pies are unsuit­able for pedi­atric patients as the non-inte­grat­ing vec­tor dilutes as the liv­er grows.

#03.

Annual Costs

No approved treat­ment deliv­ers a durable one time cure — despite annu­al costs of ~$565K – $750K4 and life­time costs of ~$18M – $24M per patient.5 

4. ICER. Gene Ther­a­py for Hemo­phil­ia B and A: Final Evi­dence Report, Dec 22, 2022. 
5. Cur­tis R et al. Poster, 65th ASH Annu­al Meet­ing & Expo­si­tion, Dec 11, 2023, San Diego, CA.

#04.

Our goal is to develop a durable one-time curative therapeutic

Our approach

Gene editing uniquely suited for Hemophilia A patients

  • Mono­genic and well-char­ac­ter­ized biol­o­gy with clear biomarker
  • Clear­ly defined tar­get thresh­old of cura­tive FVIII lev­el & wide safe­ty range
  • Robust pre­clin­i­cal mod­els and reg­u­la­to­ry familiarity
  • Strong advo­ca­cy and infrastructure
  • Clear oppor­tu­ni­ty for a durable, func­tion­al cure
Approach icon tech

Technology:

Proprietary Type V nuclease (MG29-1).

Approach icon durability

Durability:

Sustained, stable expression/activity of the therapeutic transgene over time without decline.

Approach icon status

Regulatory status:

IND-enabling stage.

Approach icon parental

Pediatric potential:

No transgene dilution as liver grows.

MGX-001 is a potentially durable, curative approach for adults but also for children who would benefit the most by avoiding all bleeding events and the associated cumulative joint damage.

Our scientific approach

Mechanism of action is to enable endogenous production of Factor VIII

Lever­ages body’s nat­ur­al albu­min promoter.

FVIII MOA illustration mobile

Two components of the therapy are administered separately by IV infusion to deliver the active ingredients bringing the Factor VIII gene into the liver.

The first infu­sion tar­gets the liv­er with the ther­a­peu­tic Fac­tor VIII gene, while the sec­ond deliv­ers the CRISPR tool to pre­cise­ly inte­grate the Fac­tor VIII gene into the albu­min locus, enabling endoge­nous Fac­tor VIII production.

Platform

Modular by design to go beyond Hemophilia A

Har­ness­ing the pow­er of one inte­gra­tion plat­form with inter­change­able trans­gene car­go: 

• Same inte­gra­tion site and deliv­ery approach across mul­ti­ple con­structs — only the trans­gene car­go changes
• Nor­mal human pro­tein expres­sion achieved con­sis­tent­ly, demon­strat­ing plat­form robust­ness across inde­pen­dent gene cargos.

  • Modular cargo — same chassis, different transgene:

One partner

Multiple ways
to gene edit

Metagenomi Technology

The Evolution of Genome Editing to Create Curative Genetic Medicines

Large Gene Integration

Protein replacement

Large Gene Integration for Protein Replacement

Gene editing in the presence of DNA templates to allow for site-specific gene integration.

Knockdown & Correction

CNS & muscle

Knockdown and Correction of Mutations in CNS/NM

Compact nucleases and base editors delivered by AAV to extrahepatic tissues.

Protein Knockdown

Cardiometabolic pathways

Protein Knockdown to Modulate Cardiometabolic Pathways

Programmable nucleases or base editors make deletions or change a single base to cause protein knockdown.

Multiplex Editing

Cell therapy

Multiplex Editing for Cell Therapy

Simultaneous multiplexed base editing for protein knockdown and site-specific transgene insertion.

Precision Corrections

Genetic disorders

Precision Corrections for Genetic Disorders

Gene correction by single-nucleotide changes and edits delivered as RNA.

Cur­rent Partners