Metagenomi, Inc.
For the period ended Sep 30, 2025. Filed Nov 12, 2025.
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A company's report for each of its first three quarters. The financial statements are not audited. It shows how the business has moved since the last annual report, and what management says changed.
Sep 30, 2025 · Filed Nov 12, 2025
MGX$0.71-2.9%Stored close · as of Nov 6, 2026
What changed in Management’s Discussion and Analysis?
Read the current filingPrevious report: 10-Q
Period ended 2025-06-30; filed 2025-08-12.
0000950170-25-107308
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsCurrent report: 10-Q
Period ended 2025-09-30; filed 2025-11-12.
0001193125-25-276150
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsThe baseline is this issuer’s latest earlier reporting period of the same form that was filed before the current report. A previous 10-Q is a different quarter, not a year-over-year comparison. Amendments are excluded.
Compared 121 previous paragraphs and 107 current paragraphs.
68 unchanged; 26 reworded; 12 added; 27 removed; 0 previous and 1 current paragraphs rearranged.
Tables are not compared: 4 previous and 4 current tables excluded. Dates, whitespace, punctuation and numbering changes are ignored; changes to financial figures remain significant.
A changed paragraph is not evidence that risk increased or decreased. An unchanged paragraph does not establish that risk stayed the same.
Added paragraphs (12)
Current report
We are an in vivo genome editing company capitalizing on our proprietary technologies to create curative genetic medicines for patients. We were 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. We focus on high value programs in disease indications with well-understood biology and clearly defined clinical development and regulatory pathways. We are committed to advancing our most compelling programs that have the highest probability of success, and potential to address unmet needs and create near-term value.
Current report
MGX-001 – Novel, Durable, Site-specific Genome Integration for Expression of Factor VIII (FVIII) and Other Secreted Protein Deficiencies
Current report
New dose range finding data presented in November 2025 demonstrated curative factor VIII (FVIII) activity in non-human primates (NHPs). The data also demonstrated clear dose dependency across both the AAV and LNP components of MGX-001, informing our clinical dose-regimen strategy. The new data builds upon earlier data which demonstrated durable and stable FVIII activity in NHPs over an approximately 19-month study. The new study also confirmed the encouraging preclinical safety profile of MGX-001. Additionally, MGX-001 has shown no identifiable off-target editing in a series of orthogonal assays employed to discover and validate potential off-target sites.
Current report
We plan to advance MGX-001 into clinical development, conduct pre-IND regulatory meetings by the end of 2025, submit investigational new drug (“IND”) and clinical trial application (“CTA”) submissions in the fourth quarter of 2026, and initiate clinical trials in 2027.
Current report
Ionis Collaboration
Current report
Future Programs
Current report
We are exploring opportunities to pursue neuromuscular disease targets and liver disease targets such as A1AT and Wilson Disease, as well as partnerships including cell therapy applications.
Current report
Going forward, we intend to continue to expand our pipeline by exploiting our most advanced, signature technologies for site specific deletion, integration and correction. This includes:
Current report
Our novel nucleases, where we have demonstrated our capabilities across our MGX-001, secreted protein deficiencies and Ionis programs, including precise and specific genetic editing in vivo with no off-target editing; and our SMall Arginine-Rich sysTems (“SMART”) genome editing systems, which are small enough to be packaged within an adeno associated virus (“AAV”) for delivery.
Current report
Total Other Income (Expense), Net
Current report
Total Other Income (Expense), Net
Current report
Total other income (expense), net increased $0.7 million for thethree months ended September 30, 2025, as compared to the corresponding prior year period, primarily related to a higher loss from the change in fair value of our long-term investment in Affini-T in the prior year period, partially offset by a decrease in interest income.
Removed paragraphs (27)
Previous report
We are a precision gene editing company committed to developing curative therapeutics for patients using our proprietary, comprehensive genome editing toolbox, which has the potential to target any genetic mutation found across the entire human genome. By harnessing the power of metagenomics, the study of genetic material recovered from the natural environment, combined with high-throughput screening, artificial intelligence (“AI”), and proprietary algorithms, our platform rapidly and effectively mines through billions of novel proteins from our genome-resolved database to select genome editing tools that we develop into our next generation genetic medicines.
Previous report
Our comprehensive genome editing toolbox includes systems for making small edits such as programmable nucleases, base editors, and small RNA-mediated integration systems (“RIGS”), as well as large gene integration systems including large template RIGS and CRISPR-associated transposases (“CASTs”). These tools form a toolbox that can potentially make any desired gene modification ranging from gene knock-down, gene knock-in as well as small and large genetic changes. In addition, our toolbox includes ultra small editing systems which have the potential to be packaged into a single adeno-associated virus (“AAV”) to address extrahepatic therapeutic indications. All elements of our toolbox are wholly-owned, and we have constructed a broad patent estate that protects our intellectual property, which we expect will continue to expand as we discover, interrogate, and optimize our novel editing systems.
Previous report
We are taking a stepwise approach, deploying our genome editing toolbox to develop potentially curative therapies for patients across a broad array of diseases and target organs including the liver, central nervous system, muscle, kidney, and lung, matching the right tool to each specific target. Our lead programs are selected to both address important diseases and establish new standards in targetability, precision, efficiency, and scope of editing capabilities. We are focused onin vivo gene editing for our wholly owned pipeline, while pursuing technology out-licensing forex vivo cell therapy, where next generation genome editing systems are an important enabler of novel therapies. We believe our therapeutic programs are well-positioned to leverage the clinical, regulatory, and manufacturing advancements made to date across gene therapy, gene editing, and delivery modalities to accelerate progression to clinical trials and potential approval.
Previous report
MGX-001 – novel, durable, knock-in approach for expression of Factor VIII (FVIII) in hemophilia A
Previous report
Our lead hemophilia A genome editing strategy has two components. A lipid nanoparticle (“LNP”) is designed to deliver mRNA along with a gRNA to the liver in order to produce a highly efficient and specific nuclease that creates a precise cut at the albumin safe harbor gene locus. Additionally, an adeno-associated virus (“AAV”) vector is designed to deliver the donor template FVIII DNA that becomes inserted into the nuclease cut site by a naturally occurring DNA repair process called non-homologous end joining.
Previous report
In a non-human primate (“NHP”) study, we demonstrated integration of a surrogate cynomolgus-FVIII cassette (used to avoid immune response that would occur with a foreign human FVIII protein) and confirmed durable and therapeutically relevant FVIII activity levels over an approximately nineteen-month period. Plasma FVIII activity levels correlated with gene integration frequency measured in the liver at termination. Treatment was generally well tolerated with findings limited to moderate transient elevation of liver transaminases following AAV and LNP administration. There were no notable findings in total bilirubin or albumin levels.
Previous report
This early NHP study was conducted without the benefit of several subsequent optimizations of the therapeutic candidate designed to enhance safety and efficacy in the clinic. Following our meeting with the FDA to align on key aspects of our planned IND submission, in August 2024, we nominated a lead development candidate, MGX-001. For the development candidate MGX-001, we selected a bioengineered B-domain deleted FVIII construct (a “bioengineered FVIII construct”).
Previous report
In rodent and NHP studies designed to support MGX-001, we demonstrated significantly higher FVIII activity levels using this bioengineered FVIII construct compared to wild type B-domain deleted FVIII construct, despite similar integration frequencies. These data suggested that incorporating the bioengineered FVIII construct into MGX-001 may enable therapeutic levels of FVIII activity at lower AAV doses, potentially resulting in MGX-001 having improved safety characteristics. Emerging data relating to MGX-001 from additional NHPs using this bioengineered FVIII construct has, to date, demonstrated FVIII activity higher than expected. While we have not received all of the data from the studies, we have commenced additional studies and may consider alternatives to this bioengineered FVIII construct. Based on the current information, we plan to conduct pre-IND and ex-U.S. regulatory meetings in the second half of 2025 and submit IND and clinical trial application (“CTA”) submissions in 2026. For more information regarding the risks related to our IND submission and timelines, see “Risks Related to Business, Technology, and Industry” described in Part II, Item 1A, Risk Factors herein.
Previous report
Collaborations
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Ionis Collaboration - investigational development programs focused on the cardiometabolic space
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Affini-T collaboration – enabling development programs in the cell therapy space
Previous report
In June 2022, we entered into a Development, Option and License Agreement (the “Affini-T Agreement”) with Affini-T Therapeutics, Inc. (“Affini-T”) to develop and commercialize gene edited TCR-based therapeutic products exclusively for the treatment, prevention or diagnosis of any human cancer using products with any engineered primary TCR alpha/beta T cells and non-exclusively for the treatment, prevention or diagnosis of any human cancer using products with certain other engineered immune cells worldwide. Our collaboration with Affini-T is consistent with our strategy for out-licensing our technology for ex vivo cell therapy applications where next generation genome editing systems are an important enabler of novel therapies. In 2024, we achieved a development milestone related to establishing cGMP gene editing reagents for cell therapy and filing related Drug Master Files with the FDA to support an IND for Affini-T’s T-cell receptor-based therapy. On April 14, 2025, Affini-T exercised an option to a non-exclusive license for one of its pre-specified targets triggering an exercise payment to us of $0.3 million.
Previous report
Further Areas of Therapeutic Activity and Interest
Previous report
Building on our experience delivering our nucleases to the liver via LNP systems, we are extending that experience with the goal to deliver novel RIGS to the liver to potentially correct ATP7B mutations in Wilson’s disease and PiZ mutations in alpha-1-antitrypsin deficiency (“A1AT deficiency”). Both of these liver diseases have well-defined biology, readily available translational biomarkers for early proof-of-concept, established development pathways based on prior drug approvals, and important unmet medical needs.
Previous report
In parallel with our translation efforts in our lead programs focused on liver-associated targets, we are developing editing systems to allow gene editing beyond the liver. Using our small editing systems designed to be amenable to viral vector delivery, which includes novel type II and type V programmable nucleases, we intend to pursue extrahepatic indications in neuro-muscular and lung and kidney diseases.
Previous report
Generative AI and Structure-Guided Engineering
Previous report
We believe our metagenomics-based, AI-enhanced, discovery approach will allow us to achieve our goal of having highly effective genome editing tools to address any genetic disease by editing anywhere in the human genome. We have demonstrated how our metagenomics-informedde novo sequence generation approach, which includes using ancestral sequence reconstruction and generative AI techniques, can be used together with structure-guided engineering methods to potentially expand therapeutic applications.
Previous report
Nucleases
Previous report
To date, we have identified thousands of novel CRISPR nucleases, expanding the collection of known programmable nucleases by mining our proprietary metagenomics database. This allows us to potentially select the ideal nuclease for targeting any given gene in a site-specific manner, overcoming a major limitation of first-generation CRISPR/Cas9 systems. We recently demonstrated technological advancements to address unmet needs for therapeutic applications ofin vivo gene editing beyond the liver using our novel nucleases, including single-AAV delivery to a central nervous system (“CNS”) target, a key milestone towards addressing neurological disorders. We achievedin vivo proof-of-concept for CNS editing with a single blood-brain-barrier penetrating AAV, which may enable treatments with systemic injections and provide a more tolerable treatment for patients. Furthermore, based on data generated to date, we believe our SMall Arginine-Rich sysTems(“SMART”) genome editing systems, which are small enough to be packaged into standard AAV vectors, even when additional effector domains are included for base editing, potentially enable a differentiated therapeutic approach for extrahepatic targets.
Previous report
Base Editors
Previous report
RIGS are genome editing systems that make programmable genomic modifications that are encoded in RNA templates. Because of the modifications are encoded in RNA, these systems have the ability to repair diverse mutations, including insertions, deletions and all types of point mutations. Using proprietary reverse transcriptases we demonstrated what we believe to be the first-ever targeted integration of >900 bp in human cells with our RIGS using all-RNA delivery.
Previous report
CAST systems are a class of genome editing systems that provide directed and programmable genomic integration of large DNA templates (>10,000 base pairs). We recently achievedin vitro proof-of-concept for large gene integration using our potentially industry leading, compact CAST technology. In March 2025, we published our latest findings in Nature Communications describing our novel, compact CAST systems, which demonstrated integration of a large, therapeutically relevant gene into the genome of human cells.
Previous report
Recent Developments
Previous report
In July 2025, Sarah Noonberg, M.D., Ph.D., tendered her resignation as the Company’s Chief Medical Officer, effective August 22, 2025.
Previous report
Additionally, in July 2025, the Board approved the formation of a new committee of the Board, the Research and Development Committee (the “R&D Committee”), to assist with the Board’s oversight of the Company’s research and development activities, advise the Board with respect to strategic and tactical scientific and clinical considerations, and periodically report to the Board on matters related to the Company’s research and development initiatives. The R&D Committee is currently comprised of Willard Dere, Jian Irish, Laurence Reid, and Brian Thomas.
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Total Other Income, Net
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Total Other Income, Net
Reworded paragraphs (26)
Previous wording
Our investigational development program in hemophilia A, MGX-001, is a potentially curative therapy designed to provide life-long protection from bleeding events and joint damage in adults and children. Our approach is designed to insert a Factor VIII (“FVIII”) DNA cassette into a “safe harbor location” within an intron of the albumin gene, leveraging the strong native albumin promoter to express FVIII in an approach that is not expected to have deleterious effects. This unique design differentiates from gene therapies that deliver the FVIII gene in an episomal location thereby increasing the risk of dilution from cell division, cell death, and episomal transcriptional silencing. Our FVIII knock-in approach is designed to provide stable expression and clinically relevant circulating levels of FVIII, even at low integration rates because of the strength of the albumin promoter.
Current wording
MGX-001 is designed to provide life-long protection from bleeding events and joint damage in adults and children with hemophilia A. MGX-001 is designed to insert a Factor VIII (“FVIII”) DNA cassette into a “safe harbor location” within an intron of the albumin gene, leveraging the strong native albumin promoter to express FVIII.
Previous wording
Building on our lead hemophilia A program, we are advancing additional wholly owned therapeutic candidates targeting secreted protein deficiencies, leveraging the MGX-001 editing system as a blueprint, with the goal of achieving targeted and durable protein replacement. We plan to demonstrate NHP proof-of-concept for our lead secreted protein deficiency target in 2025 and nominate our development candidate in 2026.
Current wording
Leveraging the MGX-001 site-specific genome integration approach, we are advancing additional wholly owned therapeutic candidates targeting secreted protein deficiencies with the goal of achieving targeted and durable protein replacement. We plan to demonstrate NHP proof-of-concept for our lead secreted protein deficiency target in 2025.
Previous wording
In November 2022, we entered into a Collaboration and License Agreement with Ionis Pharmaceuticals, Inc. (“Ionis”) to research, develop and commercialize investigational medicines using our genome editing technologies. All four therapeutic targets in Wave 1 of our collaboration with Ionis focus on cardiometabolic diseases and are in lead optimization. In vivo rodent proof-of-concept was achieved in all four wave 1 genetic targets in 2024, including transthyretin (“TTR”) for transthyretin amyloidosis and angiotensinogen (“AGT”) for refractory hypertension, as well as two undisclosed programs. In partnership with Ionis, we plan to nominate one to two development candidates from the Wave 1 targets and disclose the remaining two therapeutic indications in 2025. In 2026, we plan to initiate IND-enabling activities for the development candidates nominated in 2025, and nominate additional development candidates from the remaining Wave 1 targets.
Current wording
In November 2022, we entered into a Collaboration and License Agreement with Ionis Pharmaceuticals, Inc. (“Ionis”) to research, develop and commercialize investigational medicines using our genome editing technologies. Wave 1 of the collaboration is comprised of four cardiometabolic targets including transthyretin (“TTR”) for transthyretin amyloidosis and angiotensinogen (“AGT”) for refractory hypertension, as well as two undisclosed programs.
Previous wording
Our nucleases can be leveraged as a chassis by adding on additional effector enzymes to create base editors capable of single nucleotide changes in the genome. In October 2024, we demonstrated that our novel Adenine Base Editors (“ABEs”) are potentially targetable to over 95% of the human genome’s base pairs, a significantly wider range of sites than first-generation SpCas9 base editors. Our ABE platform achieved greater than 90% editing efficiency simultaneously at seven genomic targets in primary T-cells, confirming its highly efficient application for multiplex gene editing. The ABE platform demonstrated highly specific on-target deamination with no detectable translocations and no significant genome composition differences, and the platform demonstrated no adverse effects on cell viability, expansion or other measures of cell health.
Current wording
Our novel Adenine Base Editors (“ABEs”), which achieved greater than 90% editing efficiency simultaneously at seven genomic targets in primary T-cells, with high specificity and no adverse effects on cell health.
Previous wording
RNA and DNA Mediated Integration Systems
Current wording
Our RNA and DNA Mediated Integration Systems, which leverage RNA and DNA templates to repair diverse mutations.
Previous wording
As part of our strategy, we have entered into collaborations with third parties for one or more of our programs or product candidates we may develop. For example, in June 2022, we entered into a Development, Option and License Agreement with Affini-T Therapeutics, Inc. to develop and commercialize gene edited T-cell receptor (“TCR”)-based therapeutic products exclusively in the field of treatment, prevention or diagnosis of any human cancer using products with any engineered primary TCR alpha/beta T cells and non-exclusively in the field of treatment, prevention or diagnosis of any human cancer using products with certain other engineered immune cells worldwide, and in November 2022, we entered into a Collaboration and License Agreement with Ionis to research, develop and commercialize investigational medicines using our genome editing technologies. In October 2021, we entered into a Strategic Collaboration and License Agreement (the “Moderna Agreement”) with ModernaTX, Inc. (“Moderna”), focused on advancing new genome editing system forin vivo human therapeutic applications, and in April 2024, we and Moderna mutually terminated the Moderna Agreement. Refer to Note 7, “Significant Agreements,” in our condensed financial statements included elsewhere in this Quarterly Report on Form 10-Q for additional information related to the terms of the agreements between us and our collaborators.
Current wording
As part of our strategy, we have entered into license and collaboration agreements with third parties for one or more of our programs or product candidates we may develop. For example, in September 2025, we entered into the Acuitas License Agreement to license certain Acuitas LNP technology, including LNP patents, formulation processes, and analytical characterization methods for manufacturing products that incorporate the Company's proprietary genome editing constructs formulated with Acuitas LNP technology, to research, develop, sell, and commercialize certain licensed products in connection with a single target. In November 2022, we entered into a Collaboration and License Agreement with Ionis to research, develop and commercialize investigational medicines using our genome editing technologies. Refer to Note 7, “Significant Agreements,” in our condensed financial statements included elsewhere in this Quarterly Report on Form 10-Q for additional information related to the terms of the agreements between us and our collaborators.
Previous wording
To date, all of our revenue consists of collaboration revenue, earned from collaboration agreements with Moderna (prior to the termination of the Moderna Agreement), Ionis and Affini-T. These agreements may include the following types of promised goods or services: (i) grants of licenses; (ii) performance of research and development services and (iii) participation on joint research and/or development committees. They also may include options to obtain licenses to our intellectual property or to extend the term of the research activities. Our revenues under such collaboration agreements were $8.5 million and $12.6 million for the three and six months ended June 30, 2025, respectively, and $20.0 million and $31.2 million for the three and six months ended June 30, 2024, respectively.
Current wording
To date, all of our revenue consists of collaboration revenue, earned from collaboration agreements. These agreements may include the following types of promised goods or services: (i) grants of licenses; (ii) performance of research and development services and (iii) participation on joint research and/or development committees. They also may include options to obtain licenses to our intellectual property or to extend the term of the research activities. Our revenues under such collaboration agreements were $8.7 million and $21.3 million for the three and nine months ended September 30, 2025, respectively, and $11.5 million and $42.7 million for the three and nine months ended September 30, 2024, respectively.
Previous wording
Total other income, net, includes interest income from our investments in available-for-sale marketable securities and changes in the fair value of our investments in Affini-T.
Current wording
Total other income (expense), net, includes interest income from our investments in available-for-sale marketable securities and changes in the fair value of our investments in Affini-T.
Previous wording
Comparison of the Three and Six Months Ended June 30, 2025 and 2024
Current wording
Comparison of the Three and Nine Months Ended September 30, 2025 and 2024
Previous wording
Collaboration revenue decreased by $11.5 million for the three months ended June 30, 2025, as compared to the corresponding prior year period, primarily due to a $15.9 million decrease in revenue related to the Moderna Agreement due to the recognition of all remaining deferred revenue during the year ended December 31, 2024 as a result of the Termination Agreement and a $0.7 million decrease in revenue related to the Affini-T Agreement, partially offset bya $5.1 million increase in revenue related to the Ionis Agreement.
Current wording
Collaboration revenue decreased by $2.9 million for the three months ended September 30, 2025, as compared to the corresponding prior year period, primarily due to a $1.5 million decrease in revenue related to the Affini-T Agreement anda $1.4 million decrease in revenue related to the Ionis Agreement.
Previous wording
Collaboration revenue decreased by $18.5 million for the six months ended June 30, 2025, as compared to the corresponding prior year period, primarily due to a $18.7 million decrease in revenue related to the Moderna Agreement due to the recognition of all remaining deferred revenue during the year ended December 31, 2024 as a result of the Termination Agreement and a $0.8 million decrease in revenue related to the Affini-T Agreement, partially offset bya $1.0 million increase in revenue related to the Ionis Agreement.
Current wording
Collaboration revenue decreased by $21.4 million for thenine months ended September 30, 2025, as compared to the corresponding prior year period, primarily due to a $18.7 million decrease in revenue related to the Moderna Agreement due to the recognition of all remaining deferred revenue during the year ended December 31, 2024 as a result of the Termination Agreement and a $2.3 million decrease in revenue related to the Affini-T Agreement.
Previous wording
Research and development expenses decreased $12.1 million for the six months ended June 30, 2025, as compared to the corresponding prior year period, primarily due to decreases of $4.6 million in research and development supplies and services, $3.6 million in stock-based compensation expense, and $3.5 million in employee-related expenses.
Current wording
Research and development expenses decreased $1.0 million for thethree months ended September 30, 2025, as compared to the corresponding prior year period, primarily due to decreases of $1.6 million in employee-related expenses and $0.5 million in facilities and overhead costs, partially offset by increases of $0.7 million in research and development supplies and services and $0.2 million in stock-based compensation expense.
Previous wording
Research and development expenses decreased $5.8 million for the three months ended June 30, 2025, as compared to the corresponding prior year period, primarily due to decreases of $2.4 million in research and development supplies and services, $2.2 million in employee-related expenses, and $0.9 million in stock-based compensation expense.
Current wording
Research and development expenses decreased $13.1 million for the nine months ended September 30, 2025, as compared to the corresponding prior year period, primarily due to decreases of $5.1 million in employee-related expenses, $3.9 million in research and development supplies and services and $3.4 million in stock-based compensation expense.
Previous wording
General and administrative expenses decreased $1.6 million for the three months ended June 30, 2025, as compared to the corresponding prior year period, primarily related to decreases of $0.4 million in professional services and consulting costs, $0.4 million in employee-related expenses, $0.4 million in stock-based compensation expense, and $0.3 million in facilities and overhead costs.
Current wording
General and administrative expenses decreased $1.4 million for thethree months ended September 30, 2025, as compared to the corresponding prior year period, primarily related to decreases of $0.6 million in professional services and consulting costs, $0.4 million in stock-based compensation expense, $0.3 million in employee-related expenses, and $0.1 million in facilities and overhead costs.
Previous wording
General and administrative expenses decreased $3.5 million for the six months ended June 30, 2025, as compared to the corresponding prior year period, primarily related to decreases of $1.7 million in professional services and consulting costs, $1.1 million in employee-related expenses, and $0.9 million in facilities and overhead costs.
Current wording
General and administrative expenses decreased $4.9 million for the nine months ended September 30, 2025, as compared to the corresponding prior year period, primarily related to decreases of $2.3 million in professional services and consulting costs, $1.4 million in employee-related expenses, and $1.0 million in facilities and overhead costs.
Previous wording
Total other income, net, decreased $2.8 million and $3.8 million for the three and six months ended June 30, 2025, respectively, as compared to the corresponding prior year periods, primarily due to the $1.3 million loss recorded for the three and six months ended June 30, 2025, for the change in fair value of our long term investment in Affini-T and a decrease in interest income.
Current wording
Total other income (expense), net decreased $3.1 million for the nine months ended September 30, 2025, as compared to the corresponding prior year period, primarily related to a decrease in interest income, partially offset by higher loss from the change in fair value of our long-term investment in Affini-T in the prior year period.
Previous wording
We have incurred significant operating losses since inception and we expect to continue to incur substantial losses for the foreseeable future. Our net losses were $78.1 million and $68.3 million for the years ended December 31, 2024 and 2023, respectively, and $44.9 million and $35.9 million for the six months ended June 30, 2025 and 2024, respectively. As of June 30, 2025, we had an accumulated deficit of $267.9 million.
Current wording
We have incurred significant operating losses since inception and we expect to continue to incur substantial losses for the foreseeable future. Our net losses were $78.1 million and $68.3 million for the years ended December 31, 2024 and 2023, respectively, and $65.3 million and $54.7 million for the nine months ended September 30, 2025 and 2024, respectively. As of September 30, 2025, we had an accumulated deficit of $288.3 million.
Previous wording
As of June 30, 2025, we had $205.0 million in cash, cash equivalents and available-for-sale marketable securities. Based on our current operating plan, we estimate that our existing cash, cash equivalents and available-for-sale marketable securities will be sufficient to fund our projected operating expenses and capital expenditure requirements for at least the next 12 months from the date of issuance of this Quarterly Report on Form 10-Q. We have based this estimate on assumptions that may prove to be wrong, and we could exhaust our available capital resources sooner than we expect. See “Liquidity and Capital Resources” and “Risk Factors—Risks Related To Our Financial Position and Need for Additional Capital.” We expect that we will require additional funding to: continue our current research development activities; develop, maintain, expand and protect our intellectual property portfolio; further develop our platform; and hire additional research, clinical and scientific personnel. If we receive regulatory approval for any of our product candidates, we expect to incur significant commercialization expenses related to product manufacturing, sales, marketing and distribution, depending on where we choose to commercialize our products.
Current wording
As of September 30, 2025, we had $184.1 million in cash, cash equivalents and available-for-sale marketable securities. Based on our current operating plan, we estimate that our existing cash, cash equivalents and available-for-sale marketable securities will be sufficient to fund our projected operating expenses and capital expenditure requirements for at least the next 12 months from the date of issuance of this Quarterly Report on Form 10-Q. We have based this estimate on assumptions that may prove to be wrong, and we could exhaust our available capital resources sooner than we expect. See “Liquidity and Capital Resources” and “Risk Factors—Risks Related To Our Financial Position and Need for Additional Capital.” We expect that we will require additional funding to: continue our current research development activities; develop, maintain, expand and protect our intellectual property portfolio; further develop our platform; and hire additional research, clinical and scientific personnel. If we receive regulatory approval for any of our product candidates, we expect to incur significant commercialization expenses related to product manufacturing, sales, marketing and distribution, depending on where we choose to commercialize our products.
Previous wording
Net cash used in operating activities was $44.1 million for the six months ended June 30, 2025, and consisted primarily of our net loss of $44.9 million and changes in our net operating assets and liabilities of $10.2 million, offset by net non-cash charges of $11.1 million. The net change in operating assets and liabilities consisted primarily of a decrease of $10.4 million in deferred revenue as we recognized revenue under our collaboration agreements, a decrease of $2.5 million in accrued expenses and other current liabilities, and a decrease of $2.8 million in operating lease liabilities, partially offset by a decrease of $2.7 million in prepaid expenses and other current assets and an increase of $2.3 million in accounts payable due to the timing of payments to our vendors. The net non-cash charges consisted primarily of $6.1 million in stock-based compensation expense, $2.7 million of depreciation expense, $2.7 million in non-cash lease expense,$1.3 million change in fair value related to our investment in Affini-T, partially offset by $1.8 million in accretion of discounts on available-for-sale marketable securities.
Current wording
Net cash used in operating activities for the nine months ended September 30, 2025, was $65.3 million and consisted primarily of our net loss of $65.3 million and changes in our net operating assets and liabilities of $15.8 million, offset by net non-cash charges of $15.8 million. The net change in operating assets and liabilities consisted primarily of decreases of $18.0 million in deferred revenue as we recognized revenue under our collaboration agreements and $4.1 million in operating lease liabilities, partially offset by a decrease of $3.8 million in prepaid expenses and other current assets and an increase of $1.5 million in accrued expenses and other current liabilities. The net non-cash charges consisted primarily of $9.2 million in stock-based compensation expense, $4.0 million of depreciation expense, $3.9 million in non-cash lease expense, $1.3 million change in fair value related to our investment in Affini-T, partially offset by $2.5 million in accretion of discounts on available-for-sale marketable securities.
Previous wording
Net cash used in operating activities for the six months ended June 30, 2024, was $56.2 million and consisted primarily of our net loss of $35.9 million and changes in our net operating assets and liabilities of $31.7 million, partially offset by net non-cash charges of $11.4 million. The changes in our net operating assets and liabilities consisted primarily of a decrease of $29.4 million in deferred revenue as we recognized revenue under our collaboration agreements, a $0.3 million increase in accrued expenses and other liabilities primarily related to payout of the 2024 annual bonus, and a $0.3 million increase in prepaid expenses and other assets, offset by an increase of $1.7 million in accounts payable due to the timing of payments to our vendors. The net non-cash charges consisted primarily of $9.6 million in stock-based compensation expense, $2.3 million in non-cash lease expense, and $2.6 million of depreciation expense, partially offset by $3.2 million in amortization of discounts on available-for-sale marketable securities.
Current wording
Net cash used in operating activities for the nine months ended September 30, 2024, was $82.8 million and consisted primarily of our net loss of $54.7 million and changes in our net operating assets and liabilities of $44.2 million, partially offset by net non-cash charges of $16.1 million. The changes in our net operating assets and liabilities consisted primarily of a decrease of $38.4 million in deferred revenue as we recognized revenue under our collaboration agreements, a decrease of $3.3 million in income tax payable due to payment of our 2023 income tax liability, a decrease of $2.2 million in operating lease liabilities, a decrease of $2.0 million in other non-current liabilities and an increase of $1.2 million in prepaid expenses and other current assets, offset by a decrease of $1.9 million in accounts receivable related to the Affini-T Agreement and the Moderna Agreement and an increase of $1.5 million in accounts payable due to the timing of payments to our vendors. The net non-cash charges consisted primarily of $12.8 million in stock-based compensation expense, $3.9 million of depreciation expense, $3.4 million in non-cash lease expense, and a $2.1 million change related to the fair value of our investments in Affini-T, partially offset by $4.8 million in amortization of discounts on available-for-sale marketable securities and $1.6 million in amortization of non-cash collaboration revenue related to the Affini-T Agreement.
Previous wording
Net cash provided by investing activities for the six months ended June 30, 2025, was $43.8 million primarily due to net maturities of available-for-sale marketable securities of $44.2 million.
Current wording
Net cash provided by investing activities for the nine months ended September 30, 2025, was $71.2 million primarily due to net maturities of available-for-sale marketable securities of $71.6 million.
Previous wording
Net cash used in investing activities for the six months ended June 30, 2024, was $107.8 million due to net purchases of available-for-sale marketable securities of $106.2 million and purchases of property and equipment of $1.6 million.
Current wording
Net cash used in investing activities for the nine months ended September 30, 2024, was $116.7 million primarily due to net purchases of available-for-sale marketable securities of $113.6 million and purchases of property and equipment of $2.8 million.
Previous wording
Net cash used in financing activities for the six months ended June 30, 2025, was $0.2 million primarily due to a payment of deferred financing costs.
Current wording
Net cash used in financing activities for the nine months ended September 30, 2025, was $0.4 million primarily due to a payment of deferred financing costs.
Previous wording
Net cash provided by financing activities for the six months ended June 30, 2024, was $84.5 million due to net proceeds from the issuance of our common stock in our IPO, net of issuance costs paid during the period.
Current wording
Net cash provided by financing activities for the nine months ended September 30, 2024, was $84.0 million due to net proceeds from the issuance of our common stock in our IPO, net of issuance costs paid during the period.
Previous wording
Our material cash requirements include our contractual obligations for our leased office and laboratory space under three lease agreements and one vivarium lease agreement. During the six months ended June 30, 2025, there were no material changes outside the ordinary course of our business to our material cash requirements described under “Management’s Discussion and Analysis of Financial Condition and Results of Operations” included in our Annual Report on Form 10-K for the year ended December 31, 2024, filed with the SEC on March 17, 2025.
Current wording
Our material cash requirements include our contractual obligations for our leased office and laboratory space under three lease agreements and one vivarium lease agreement. During the nine months ended September 30, 2025, there were no material changes outside the ordinary course of our business to our material cash requirements described under “Management’s Discussion and Analysis of Financial Condition and Results of Operations” included in our Annual Report on Form 10-K for the year ended December 31, 2024, filed with the SEC on March 17, 2025.
Previous wording
During the six months ended June 30, 2025, there were no material changes to our critical accounting estimates or in the methodology used for estimates from those described in “Management’s Discussion and Analysis of Financial Condition and Results of Operations” included in our Annual Report on Form 10-K for the year ended December 31, 2024.
Current wording
During the nine months ended September 30, 2025, there were no material changes to our critical accounting estimates or in the methodology used for estimates from those described in “Management’s Discussion and Analysis of Financial Condition and Results of Operations” included in our Annual Report on Form 10-K for the year ended December 31, 2024.
Rearranged current paragraphs (1)
Current report — wording carried from elsewhere
In partnership with Ionis, we plan to nominate a development candidate from the Wave 1 targets in 2025. In 2026, we plan to initiate IND-enabling activities for the development candidate nominated in 2025 and nominate additional development candidates from the remaining Wave 1 targets.