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Metagenomi Therapeutics is developing medicines that edit DNA inside the body to address disease. Its wholly owned lead program, MGX-001 for hemophilia A, aims to insert a gene so the body can produce a deficient protein. Its cardiometabolic programs with Ionis take a different approach: switching off genes to reduce particular proteins. None of its candidates has entered human clinical trials or been approved for sale.
The company earns collaboration revenue rather than selling medicines, and Ionis supplies nearly all of that revenue. Ionis has paid an upfront fee and reimburses exploratory research costs in exchange for research work and licensed gene-editing technology. Metagenomi can earn further payments as licensed products reach development, regulatory and sales milestones, plus royalties if those products sell.
That income depends on the programs advancing and on Ionis continuing to support them. Metagenomi does not control clinical development under the Ionis agreement, and any medicine must receive regulatory approval before it can be sold.
The risk section is the longest, dullest part of the filing. This keeps only what can really hurt you: where it hits, how hard, when it plays out.
Every company claims an edge. Each one is checked against hard numbers like margins and returns: which hold, and which line gives way first.
Metagenomi depends heavily on Ionis Pharmaceuticals, Inc., which pays to use its gene-editing technology and funds partnered drug research: Ionis provided $24.621 million of Metagenomi’s $25.210 million in 2025 collaboration revenue. Progress in their partnered programs matters for potential future milestone payments and royalties.
Intellia Therapeutics, Inc competes directly with Metagenomi’s Ionis-partnered effort to switch off the TTR gene to treat transthyretin disease. Watch Intellia’s nex-z, already in Phase 3 for transthyretin amyloidosis, while Metagenomi’s TTR program was in lead optimization in 2025.
Scribe Therapeutics Inc. challenges Metagenomi with engineered CRISPR systems for medicines that act inside the body to treat cardiovascular and metabolic diseases. Its triglyceride program is the closest overlap to watch alongside Metagenomi’s Ionis-partnered APOC3 gene-knockout program.
Beam Therapeutics Inc. competes with Metagenomi in gene-editing platforms and medicines that act inside the body, including cardiovascular and rare-disease programs. Beam changes individual DNA bases without making a double-stranded break—a method called base editing—and earns revenue mainly from technology licenses and collaborations rather than product sales.
Precision BioSciences, Inc. competes with Metagenomi in editing genes inside the body and inserting genes to treat genetic deficiencies, using its ARCUS platform. Its iECURE-partnered ECUR-506 program for neonatal-onset OTC deficiency is a concrete program to watch alongside Metagenomi’s gene-insertion approach to replacing missing proteins.
Prime Medicine, Inc. competes with Metagenomi in DNA-editing technologies for medicines that act inside the liver and treat genetic disease. Watch its wholly owned Wilson disease and alpha-1 antitrypsin deficiency programs, which use its DNA-rewriting approach, Prime Editing.
Editas Medicine, Inc. competes with Metagenomi in medicines that edit genes inside the body, including liver-directed and cardiovascular programs, using different DNA-cutting tools. Its cardiovascular candidate EDIT-401 is the program to watch against Metagenomi’s Ionis-partnered cardiometabolic work.
CRISPR Therapeutics AG competes with Metagenomi in CRISPR-based medicines, including treatments delivered directly into the body for common and rare diseases. Its in vivo programs are the relevant overlap; its approved CASGEVY instead edits cells outside the body before they are administered to patients.
Alnylam Pharmaceuticals, Inc. competes with Metagenomi’s Ionis-partnered TTR and AGT editing programs through its ATTR medicines and AGT-targeting zilebesiran. Watch AMVUTTRA and ONPATTRO in ATTR amyloidosis and the Roche-funded development of zilebesiran, which address the diseases or protein targets Metagenomi is pursuing.
Arrowhead’s approved REDEMPLO competes at the same APOC3 protein target as Metagenomi’s Ionis-partnered gene-knockout program, but suppresses protein production through RNA rather than editing DNA. REDEMPLO received U.S. approval in 2025 alongside diet to reduce triglycerides in adults with familial chylomicronemia syndrome—the patient group to watch.
Alnylam Pharmaceuticals, Inc. earningsALNY
Alnylam’s report can show how its ATTR medicines and AGT-targeting drug are faring, giving context for Metagenomi Therapeutics’ partnered programs aimed at those same diseases or targets.
Read the two lines separately. Use of AMVUTTRA and ONPATTRO shows how ATTR patients and clinicians are responding to existing medicines; progress in Roche-funded zilebesiran would show an AGT-targeting rival moving forward. Stronger use or faster progress raises the bar for Metagenomi Therapeutics’ eventual editing programs. Weak results would lessen that signal, but would not establish that patients will choose gene editing.
Precision BioSciences, Inc. earningsDTIL
Precision BioSciences’ report can show how its iECURE-partnered OTC deficiency program is progressing, a concrete comparison for Metagenomi Therapeutics’ gene-insertion approach.
Watch ECUR-506’s progress, not as a direct comparison between different diseases, but as evidence of whether another company can advance a gene-insertion treatment. Continued progress would deepen competition in this approach and leave Metagenomi Therapeutics needing to show that its own therapy can safely and reliably replace a missing protein. A stall would weaken that competitive signal, but would not validate Metagenomi’s approach. The report tests progress in a related treatment path, not Metagenomi’s own program.
Ionis Pharmaceuticals, Inc. earningsIONS
Ionis Pharmaceuticals’ report can shed light on partnered-program progress; Ionis is Metagenomi Therapeutics’ largest collaboration-revenue source, and those programs could also lead to milestone and royalty payments.
Read Ionis’s comments on partnered-program resources and progress alongside Metagenomi Therapeutics’ reliance on Ionis collaboration revenue. Continued work keeps open a path to development or regulatory milestones, and potentially sales royalties; a slowdown or abandonment would narrow that path and make collaboration funding less dependable for Metagenomi’s research. The conclusion is whether its largest partner is still advancing a potential source of future value.
Intellia Therapeutics, Inc earningsNTLA
Intellia Therapeutics’ report can show how its Phase 3 TTR program is progressing, a direct benchmark for Metagenomi Therapeutics’ partnered editing program against the same target.
Compare Intellia’s clinical progress with Metagenomi Therapeutics’ TTR program, which was in lead optimization in 2025. If Intellia’s Phase 3 program advances, it moves closer to testing whether editing the same target can treat patients, increasing the development and timing pressure on Metagenomi. A setback would narrow that lead, but would not show that Metagenomi’s therapy works or is safer. The question is whether the clinical gap between programs is widening.
Editas Medicine, Inc. earningsEDIT
Editas Medicine’s report can show how its cardiovascular candidate EDIT-401 is progressing, a competitive reference for Metagenomi Therapeutics’ Ionis-partnered cardiometabolic work.
Watch whether EDIT-401 advances, treating it as competition in the broad cardiovascular and metabolic disease area shared with Metagenomi Therapeutics’ Ionis partnership—not as proof the programs target the same condition. Progress would show another editing program moving forward and raise the need for Metagenomi to demonstrate progress of its own. A stall would weaken that competitive signal, but would not predict Metagenomi’s outcome. The conclusion is whether editing competition in this disease area is intensifying.
Metagenomi Therapeutics, Inc. earningsThis company
Metagenomi Therapeutics has no product-sales revenue, so this report will show whether Ionis collaboration income continues to help fund its research spending.
Read collaboration revenue alongside cash used in operations. If revenue remains supported mainly by Ionis and operating cash use does not accelerate, the partnership is still helping fund research. If collaboration revenue weakens while cash use continues, funding pressure rises and Metagenomi may need other financing or to adjust its research plans. The question is whether partner payments still support the work.
Arrowhead Research Corporation earningsARWR
Arrowhead’s report can show how REDEMPLO is being used by people with familial chylomicronemia syndrome, a market reference for Metagenomi Therapeutics’ partnered APOC3 program.
Watch REDEMPLO’s use in this patient group. Rising use would show patients, clinicians, and payors engaging with treatment for the same disease and protein target, raising the efficacy, convenience, and coverage bar for Metagenomi Therapeutics’ future APOC3 editing program. Limited use could reflect reimbursement, acceptance, or other hurdles; it would not prove patients have no need for another treatment. The question is whether need in this patient group is translating into use.
