Market reactionShares rose 11% on Nov 3.
Metagenomi Therapeutics is a preclinical gene editing company developing MGX-001, a one-time treatment designed to insert a working factor VIII gene in people with hemophilia A.
Metagenomi's case rests on MGX-001, which uses an AAV vector and a CRISPR nuclease to insert a factor VIII gene at the albumin site in the liver. In monkeys it produced factor VIII in the normal range, and activity lasted about 19 months in an earlier study. No off-target edits were found at 623 candidate sites in human liver cells. Metagenomi plans to submit an IND in the fourth quarter of 2026 and dose the first patient in 2027. Cash was $120.7 million at June 30.
Thesis checklist
1 risk needs attention
Why it is in this state
The evidence, what would weaken it and the source filing
Why it is in this state
The evidence, what would weaken it and the source filing
Why it is in this state
The evidence, what would weaken it and the source filing
Next decision point
Tests: The MGX-001 IND is filed and cleared
Does Metagenomi file the MGX-001 IND on time and receive clearance to dose patients?
Strong, inconclusive and weak outcomes
What to look for in the result and how each would move the checklist
Price and material events
Select an event marker to read the update below. Marker colors show Thesis status.
Metagenomi aims to file the MGX-001 IND for hemophilia A by year-end
Curative NHP FVIII data fuels hemophilia A gene-editing bet, but human proof is still a 2027 question
MGX-001's hemophilia A bet hinges on whether preclinical FVIII durability translates to safe, curative editing in humans
MGX-001 carries curative NHP data into a 2026 IND; the thesis turns on clinical translation against established hemophilia A gene therapies
About this history: every entry shows when the Thesis moved and what it said. The reasoning behind each move (the evidence, the analysis and the source filing) and the earnings write-ups are part of a subscription.
Dates, trials and completed outcomes are open to everyone. What each upcoming milestone hinges on is part of a subscription.
Disease areas: Blood disorders, Obesity, diabetes and heart disease
Catalyst calendar · Upcoming FDA decisions · Thesis changes
Competitive landscape
vs. direct competitors
Cash: Market-data provider estimate; measurement period unavailable.
Debt: Market-data provider estimate; a complete comparable filing balance is unavailable.
Quarterly burn: Estimated from trailing free cash flow; measurement period unavailable.
Net cash and estimated runway require verified matching measurement periods. Missing debt components are not assumed to be zero.
Runway: No underlying guidance available.