Metagenomi Therapeutics, Inc.
For the period ended Mar 31, 2025. Filed May 13, 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.
Mar 31, 2025 · Filed May 13, 2025
What changed in Management’s Discussion and Analysis?
Read the current filingPrevious report: 10-Q
Period ended 2024-09-30; filed 2024-11-13.
0000950170-24-126273
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsCurrent report: 10-Q
Period ended 2025-03-31; filed 2025-05-13.
0000950170-25-070463
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 123 previous paragraphs and 115 current paragraphs.
62 unchanged; 30 reworded; 22 added; 31 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 (22)
Current 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.
Current report
Wholly Owned Programs
Current report
MGX-001 – novel, durable, knock-in approach for expression of Factor VIII (FVIII) in hemophilia A
Current report
In rodent and nonhuman primate 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 nonhuman primates 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 expect to conduct additional studies and/or 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”) submission 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.
Current report
Secreted Protein Deficiencies
Current report
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 report
Collaborations
Current report
Ionis Collaboration - investigational development programs focused on the cardiometabolic space
Current report
Affini-T collaboration – enabling development programs in the cell therapy space
Current 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.
Current report
Further Areas of Therapeutic Activity and Interest
Current 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.
Current 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.
Current report
RNA and DNA Mediated Integration Systems
Current 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.
Current report
CRISPR Associated Transposase (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.
Current report
ATM Program
Current report
On March 17, 2025, we entered into an Open Market Sale Agreement SM (the “ATM Sales Agreement”) with Jefferies LLC (“Jefferies”), pursuant to which we may issue and sell, from time to time and at our sole discretion, shares of our common stock, through Jefferies as the sales agent, by any method permitted by law deemed to be an “at the market” (“ATM”) offering as defined in Rule 415(a)(4) promulgated under the Securities Act, having an aggregate offering price of up to $75.0 million. We will pay Jefferies a commission fee of up to 3.0% of the gross proceeds of any shares sold through Jefferies under the ATM Sales Agreement, and have also provided Jefferies with customary indemnification and contribution rights. As of March 31, 2025, no shares have been sold under the ATM Sales Agreement.
Current report
Research and development expenses were $25.1 million for the three months ended March 31, 2025, compared to $31.4 million for the three months ended March 31, 2024. The decrease of $6.3 million was primarily due to a decrease of $2.7 million in stock-based compensation expense, a decrease of $2.3 million in research and development supplies and services, and a decrease of $1.3 million in other employee-related expenses.
Current report
Total other income, net, was $2.9 million for the three months ended March 31, 2025 compared to $3.9 million for the three months ended March 31, 2024. The $1.0 million decrease is primarily due to decreased interest income.
Current report
On March 17, 2025, we entered into the ATM Sales Agreement with Jefferies, pursuant to which we may issue and sell shares of our common stock from time to time and at our sole discretion, through Jefferies as our sales agent, by any method permitted that is deemed to be an “at the market offering” as defined in Rule 415(a)(4) promulgated under the Securities Act having an aggregate offering price of up to $75.0 million. As of March 31, 2025, we have not sold any shares under the ATM Sales Agreement.
Current report
Net cash used in financing activities for the three months ended March 31, 2025, was $0.1 million due to a payment of deferred financing costs.
Removed paragraphs (31)
Previous report
We are a precision genetic medicines company committed to developing curative therapeutics for patients using our proprietary, genome editing toolbox. We are harnessing the power of metagenomics, the study of genetic material recovered from the natural environment, to unlock four billion years of microbial evolution to discover and develop a suite of novel editing tools capable of correcting any type of genetic mutation found anywhere in the genome. Our platform combines artificial intelligence (“AI”), ancestral state reconstruction, and proprietary algorithms run on expansive cloud computing infrastructure to identify novel clustered regularly interspaced short palindromic repeat (“CRISPR”) nucleases and other effector enzymes at high speed.
Previous report
Lead Therapeutic Programs
Previous report
Hemophilia A
Previous report
We initiated manufacturing activities to support IND-enabling studies and clinical material supply. We are planning for additional engagement with the FDA and regulatory agencies outside the U.S. in preparation for a potential IND/Clinical Trial Application filing in 2026.
Previous report
Ionis Collaboration
Previous report
Transthyretin Amyloidosis
Previous report
Transthyretin amyloidosis is a disease of misfolded and aggregated transthyretin (“TTR”) protein that can deposit in tissues causing organ dysfunction, primarily in the heart and/or peripheral nerves. Our development program in TTR aims to provide efficient TTR knockdown and halt further deposition of amyloid fibrils. Along with our partner Ionis, we have achieved more than 90% knock-down of human TTR protein in a humanized TTR mouse model and have initiated NHP studies.
Previous report
Refractory Hypertension
Previous report
The refractory hypertension program is designed to knock-down the expression of AGT in the liver using one of our programmable nucleases to generate a durable reduction in blood pressure from a single treatment. Along with our partner Ionis, we have achieved greater than 85% knock-down of human AGT protein in a humanized AGT mouse model and have plans to initiate NHP studies.
Previous report
Ultra small (SMART) systems
Previous report
The ability to package our systems into a single AAV will enable more efficient targeting of organs and diseases beyond what is currently possible with LNP delivery. For example, at 429 aa in length, one of our SMART nucleases is a fraction of the size of the industry-standard SpCas9 system, which is 1,300 aa and exceeds the delivery capacity of standard AAV vectors. We recently achievedin vitro proof-of-concept for an undisclosed neuromuscular target with one of our compact editing systems. In October 2024, we presented a poster at the American Institute of Chemical Engineers’ 7th International Conference on CRISPR Technologies titled “Engineering of compact and efficient adenine base editors from metagenomic derived systems.” We showcased our SMART genome editing systems that are small enough to be packaged into standard AAV vectors, even when additional effector domains are included for base editing, potentially enabling a differentiated therapeutic approach for neuromuscular targets.
Previous report
Our ABEs triplex protein knockdowns exhibited excellent tolerability in cells, with no adverse effects on cell viability, expansion, or changes in stress-related gene expression observed post-editing.
Previous report
RNA-mediated Integration Systems (RIGS)
Previous report
RIGS are the systems we use for small genomic replacements such as transversions, transitions, insertions, and deletions, as well as the programmable integration of large transgenes delivered as RNA, with the potential to address any genetic disease requiring a large gene integration.
Previous report
We recently achievedin vitro proof-of-concept using our RIGS for a small gene correction in undisclosed liver targets. Additionally, 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. We consider this to be a major step forward in the genome editing space, as these systems could be delivered entirely as RNA, compatible with current LNP delivery technologies, and could enable large, targeted exogenous gene integrations.
Previous report
DNA-mediated Integrations
Previous report
CASTs are naturally occurring programmable transposase systems with the ability to site-specifically integrate large transgenes delivered as DNA, and with the potential to address any genetic disease requiring a large gene integration. Our CASTs are being developed in order to enable large, >10,000 base pairs, targeted genomic integrations for therapeutic applications. We recently achievedin vitro proof-of-concept for large gene integration using our potentially industry leading, compact CAST technology, with publication pending.
Previous report
Other Business Updates
Previous report
As a result of the acceleration of all four Wave 1 Ionis targets into lead optimization, as well as our decision to deprioritize Primary Hyperoxaluria Type 1 (“PH1”), we provided pipeline guidance through year-end 2025, including one to two DC nominations in 2025. We maintained our guidance for the planned IND filing for hemophilia A in 2026. We focused our internal efforts onin vivo genome editing therapeutic approaches and pursue technology out-licensing forex vivo cell therapy, where next generation genome editing systems are an important enabler of novel therapies. We determined that we would not pursue amyotrophic lateral sclerosis (“ALS”) based upon peer company clinical data regarding the lack of efficacy of Ataxin-2 as a therapeutic target for ALS. We revised our pipeline design to consolidate earlier stage programs and make it easier to identify our near term pipeline priorities focused onin vivo genome editing and liver indications.
Previous report
Affini-T Collaboration
Previous report
In July 2024, pursuant to the terms of the Affini-T Agreement, we received equity consideration of 933,650 shares of Affini-T common stock upon the achievement of a regulatory milestone related to the submission of drug master files to the FDA in support of an IND for Affini-T’s T-cell receptor based therapy. In August 2024, in connection with Affini-T’s Series B extension, we purchased 33,383 shares of Affini-T’s Series B Preferred Stock and received warrants to purchase up to 16,691 shares of Affini-T’s common stock.
Previous report
Reorganization and Reverse Stock Split
Previous report
We previously operated as a Delaware limited liability company under the name Metagenomi Technologies, LLC (“Metagenomi LLC”). On January 24, 2024, we completed a series of transactions pursuant to which Metagenomi LLC merged with and into its wholly-owned subsidiary Metagenomi, Inc., a Delaware corporation, with Metagenomi, Inc. (“Metagenomi” or the “Company”) continuing as the surviving corporation (the “Reorganization”). In connection with the Reorganization, (i) all of the outstanding common unitholders of Metagenomi LLC received shares of common stock of Metagenomi, Inc., (ii) all of the outstanding redeemable convertible preferred unitholders of Metagenomi LLC received shares of redeemable convertible preferred stock of Metagenomi, Inc. with the same rights and privileges and (iii) certain holders of profits interests in Metagenomi LLC received shares of common stock or unvested restricted common stock in Metagenomi, Inc. as determined by the applicable provisions of the Amended and Restated Limited Liability Company Agreement in effect immediately prior to the Reorganization. In connection with the Reorganization, by operation of law, Metagenomi, Inc. acquired all assets of Metagenomi LLC, and assumed all of its liabilities and obligations. The Reorganization generally did not result in a taxable event to Metagenomi, Inc. for U.S. income tax purposes.
Previous report
On January 26, 2024, following the Reorganization, Metagenomi, Inc. effected a reverse stock splitof the shares of common stock at a ratio of 1-for-1.74692 (the “Reverse Stock Split”). Immediately prior to the closing of the IPO, each share of Metagenomi, Inc.’s redeemable convertible preferred stock then outstanding converted into 23,935,594 shares of common stock.
Previous report
Research and development expenses were $26.3 million for the three months ended September 30, 2024, compared to $26.8 million for the three months ended September 30, 2023. The decrease of $0.6 million was primarily due to a decrease of $1.6 million in laboratory materials and supplies partially as a result of the Termination Agreement, offset by an increase of $1.2 million in other research and development and consulting costs due to the timing of external research and development costs to support our research and preclinical development activities.
Previous report
Research and development expenses were $86.0 million for the nine months ended September 30, 2024, compared to $69.6 million for the nine months ended September 30, 2023. The increase of $16.4 million was primarily due to an increase of $10.2 million in employee-related expenses, including stock-based compensation expense, related to increased headcount and the issuance of annual equity awards to existing employees, in addition to a one-time acceleration of stock-based compensation expense related to profits interests units that were canceled without a concurrent grant of a replacement award during the Reorganization and were accounted for as a repurchase for no consideration during the nine months ended September 30, 2024, an increase of $2.4 million in facilities and allocated overhead, including rent, repairs and maintenance costs, common facilities and information technology related expenses allocated to research and development due to expansion of our research and development operations, an increase of $2.0 million in other research and development and consulting costs mainly due to the timing of external research and development costs to support our research and preclinical development activities, and an increase of $1.8 million in laboratory materials and supplies.
Previous report
General and administrative expenses were $7.6 million for the three months ended September 30, 2024, compared to $7.9 million for the three months ended September 30, 2023. The decrease of $0.3 million was primarily related to a decrease in professional service fees and other costs.
Previous report
Total other income, net, decreased by $2.3 million, from $3.8 million for the three months ended September 30, 2023 to $1.5 million for the three months ended September 30, 2024 and decreased by $5.3 million, from $14.6 million for the nine months ended September 30, 2023 to $9.3 million for the nine months ended September 30, 2024. The decrease in other income, net, for both the three and nine months ended September 30, 2024 was primarily due to a net change in the fair value of our long term investment in Affini-T.
Previous report
Benefit (Provision) for Income Taxes
Previous report
Provision for income taxes decreased by $3.3 million and $9.6 million for the three and nine months ended September 30, 2024, respectively, from a provision for income taxes of $1.2 million and $5.3 million for three and nine months ended September 30, 2023, respectively, to a benefit for income taxes of $2.1 million and $4.3 million for the three and nine months ended September 30, 2024, respectively. The benefit for income taxes for the three and nine months ended September 30, 2024 was primarily due to our intention to elect to carry back the 2024 research and development credit to the prior year, in addition to a return to provision adjustment related to the prior year return. The provision for income taxes for the three and nine months ended September 30, 2023 was mainly due to our taxable income related to an upfront payment received under the Ionis Agreement and capitalization of research and development expenses under the Internal Revenue Code Section 174 (“Section 174”).
Previous report
Net cash provided by financing activities for the nine months ended September 30, 2023 was $2.3 million due to net proceeds from the issuance of our Series B-1 redeemable convertible preferred stock of $4.3 million, offset by $1.9 million payments of initial public offering costs.
Reworded paragraphs (30)
Previous wording
You should read the following discussion and analysis of our financial condition and results of operations together with our unaudited condensed consolidated financial statements and the related notes thereto included elsewhere in this Quarterly Report on Form 10-Q and our audited consolidated financial statements and notes and 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, 2023, filed with the SEC on March 27, 2024. As discussed in the section titled “Special Note Regarding Forward-Looking Statements,” the following discussion and analysis contains forward-looking statements that involve risks and uncertainties, such as statements regarding our plans, objectives, expectations, intentions and projections. Our actual results could differ materially from those discussed in these forward-looking statements. Factors that could cause or contribute to such differences include, but are not limited to, those discussed in the “Risk Factors” section under Part II, Item 1A below.
Current wording
You should read the following discussion and analysis of our financial condition and results of operations together with our unaudited condensed financial statements and the related notes thereto included elsewhere in this Quarterly Report on Form 10-Q and our audited consolidated financial statements and notes and 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. As discussed in the section titled “Special Note Regarding Forward-Looking Statements,” the following discussion and analysis contains forward-looking statements that involve risks and uncertainties, such as statements regarding our plans, objectives, expectations, intentions and projections. Our actual results could differ materially from those discussed in these forward-looking statements. Factors that could cause or contribute to such differences include, but are not limited to, those discussed in the “Risk Factors” section under Part II, Item 1A below.
Previous wording
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 – gene knock-down, gene knock-in as well as small and large genetic changes. In addition, our toolbox includes ultra small editing systems that are small enough 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. We anticipate that our toolbox will continue to expand as we discover, interrogate, and optimize our novel editing systems.
Current wording
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 wording
We are taking a stepwise approach deploying our genome editing toolbox to prosecute a genetic medicine therapeutic development strategy across a broad array of diseases and target organs including liver, central nervous system, muscle, kidney, and lung, matching the right tool to each specific target.
Current wording
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 wording
Our investigational development program in hemophilia A is a potentially curative therapy designed to provide life-long protection from bleeding events and joint damage in adults and children.
Current 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.
Previous wording
Our program is designed to insert a Factor VIII (“FVIII”) DNA cassette into a “safe harbor location,” within an intron of the albumin gene that is not expected to have deleterious effects. FVIII expression is then driven off the strength of the native albumin promoter. 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 that 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.
Current wording
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 wording
In an ongoing 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 FVIII activity levels over a twelve-month period, as of the September 2024 data cutoff. 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 or adverse clinical observations.
Current wording
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 wording
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, the company selected a bioengineered FVIII construct.
Current wording
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 wording
All four therapeutic targets in Wave 1 of our collaboration with Ionis Pharmaceuticals, Inc. (“Ionis”) focus on high value cardiometabolic diseases and are advancing in lead optimization. In vivo rodent proof-of-concept was achieved in all four wave 1 genetic targets, including transthyretin (“TTR”) for transthyretin amyloidosis and angiotensinogen (“AGT”) for refractory hypertension, as well as two undisclosed programs in significant cardiometabolic indications. Along with our partner Ionis, we are conducting preclinical activities with the plan to nominate one to two development candidates in 2025.
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. 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.
Previous wording
We believe our metagenomics-based, AI-enhanced discovery approach will allow us to achieve our goal of having the most effective genome editing tools to address any genetic disease anywhere in the human genome. In August 2024, we presented a poster at the CRISPR Frontiers conference at Cold Spring Harbor Laboratory titled “Unlocking the Therapeutic Potential of Compact SMART Nucleases through Ancestral Reconstruction, Structure-Guided Engineering, and Generative AI” which demonstrated how our metagenomics-informed synthetic sequence generation, including ancestral sequence reconstruction and generative AI techniques, potentially enable rapid development of novel programmable CRISPR nuclease- and base editing systems.
Current wording
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 wording
To date, we have identified thousands of novel CRISPR type II, type V and highly efficient ultra-small (SMall Arginine-Rich sysTems or SMART) 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.
Current wording
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 wording
The chassis of our nucleases can be leveraged by adding on additional effector enzymes to create base editors capable of single nucleotide changes in the genome. In October 2024, we presented a poster at the European Society of Gene and Cell Therapy’s 31st Annual Congress titled “Efficient and specific genome editing with metagenomics-derived base editor for human therapeutic applications.” We showcased that our novel Adenine Base Editor (“ABE”) was 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 over 95% reproducible and durable triplex protein knockdown in primary T-cells, confirming its highly efficient application for multiplex gene editing. Our ABE demonstrated highly specific on-target deamination with minimum-to-no indel formation, and genome-wide analyses confirmed no detectable translocations and no significant genomic composition differences when compared to unedited cells.
Current 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 over 95% reproducible and durable triplex protein knockdown 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.
Previous wording
Unfavorable conditions in the economy in the United States and abroad may negatively affect the growth of our business and our results of operations. For example, macroeconomic events, including, rising inflation, tensions in U.S.-China relations, the COVID-19 pandemic, the U.S. Federal Reserve raising interest rates, recent and potential future disruptions in access to bank deposits and lending commitments due to bank failures and the effects of the ongoing geopolitical conflict in Ukraine and the Israel-Hamas war, have led to economic uncertainty and volatility globally. The effect of macroeconomic conditions may not be fully reflected in our results of operations until future periods. To date, the macroeconomic trends discussed above have not had a material adverse impact on our business, financial condition or results of operations. If, however, economic uncertainty increases or the global economy worsens, our business, financial condition and results of operations may be harmed. For further discussion of the potential impacts of macroeconomic events on our business, financial condition, and operating results, refer to the section titled “Risk Factors” included elsewhere in this Quarterly Report on Form 10-Q.
Current wording
Unfavorable conditions in the economy in the United States (“U.S.”) and abroad may negatively affect the growth of our business and our results of operations. For example, macroeconomic events, including, inflationary pressures, interest rate and currency rate fluctuations, increased U.S. trade tariffs, reciprocal and retaliatory tariffs from other countries, and trade disputes with other countries, economic slowdown or recession, banking instability, monetary policy changes, geopolitical tensions or the outbreak of hostilities or war, including from the ongoing Russia-Ukraine conflict, the current conflict in Israel and Gaza (including any escalation or expansion) and increasing tensions between China and Taiwan, have led to economic uncertainty and volatility globally. The effect of macroeconomic conditions may not be fully reflected in our results of operations until future periods. To date, the macroeconomic trends discussed above have not had a material adverse impact on our business, financial condition or results of operations, but we continue to monitor these developments closely. If, however, economic uncertainty increases or the global economy worsens, our business, financial condition and results of operations may be harmed. For further discussion of the potential impacts of macroeconomic events on our business, financial condition, and operating results, refer to the section titled “Risk Factors” included elsewhere in this Quarterly Report on Form 10-Q.
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. (“Affini-T”) (the “Affini-T Agreement”) to develop and commercialize gene edited 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 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 in our condensed consolidated 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 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 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 $11.5 million and $42.7 million for the three and nine months ended September 30, 2024, respectively, and $12.4 million and $32.4 million for the three and nine months ended September 30, 2023, respectively.
Current 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 $4.1 million for the three months ended March 31, 2025 and $11.2 million for the three months ended March 31, 2024.
Previous wording
The largest component of our total operating expenses since our inception has been research and development activities. Research and development expenses consist primarily of compensation and benefits for research and development employees, including stock-based compensation; the costs of acquiring research and development supplies and services; manufacturing process development costs; the research and development expenses that we share with our collaboration partners for co-development programs; other outside services and consulting costs; and allocated facilities, information technology and overhead expenses. Research and development costs are expensed as incurred.
Current wording
The largest component of our total operating expenses since our inception has been research and development activities. Research and development expenses consist primarily of personnel costs for research and development employees, including stock-based compensation; the costs of acquiring research and development supplies and services; manufacturing process development costs; the research and development expenses that we share with our collaboration partners for co-development programs; other outside services and consulting costs; and allocated facilities, information technology and overhead expenses. Research and development costs are expensed as incurred.
Previous wording
Comparison of the Three and Nine Months Ended September 30, 2024 and 2023
Current wording
Comparison of the Three Months Ended March 31, 2025 and 2024
Previous wording
Our revenue consists of collaboration revenue recognized under our agreements with Moderna (prior to the termination of the Moderna Agreement), Affini-T and Ionis. We recognize revenue as the performance obligations are satisfied. Collaboration revenue decreased by $0.8 million, from $12.4 million for the three months ended September 30, 2023 to $11.5 million for the three months ended September 30, 2024 and increased by $10.3 million, from $32.4 million for the nine months ended September 30, 2023 to $42.7 million for the nine months ended September 30, 2024. The decrease in collaboration revenue for the three months ended September 30, 2024 was primarily driven by a $5.3 million decrease in revenue related to the Termination Agreement, offset by a $3.9 million increase in revenue related to the Ionis Agreement as we performed more services and a $0.5 million increase in revenue related to the Affini-T Agreement. The increase in collaboration revenue for the nine months ended September 30, 2024 was primarily driven by a $6.6 million increase in revenue related to the Ionis Agreement as we performed more services and a $4.5 million increase in revenue related to the Moderna Agreement mainly due to the recognition of all remaining deferred revenue during the nine months ended September 30, 2024, offset by a $0.8 million decrease in revenue related to the Affini-T Agreement.
Current wording
Collaboration revenue decreased by $7.0 million, from $11.2 million for the three months ended March 31, 2024 to $4.1 million for the three months ended March 31, 2025. The decrease in collaboration revenue for the three months ended March 31, 2025, was primarily driven by a $2.8 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, a $4.1 million decrease in revenue related to the Ionis Agreement and a $0.1 million decrease in revenue related to the Affini-T Agreement.
Previous wording
General and administrative expenses were $24.9 million for the nine months ended September 30, 2024, compared to $21.0 million for the nine months ended September 30, 2023. The increase of $3.9 million was primarily related to an increase of $4.6 million in employee-related expenses, including stock-based compensation expense, as a result of increased headcount and the issuance of annual equity awards to existing employees and an increase of $0.8 million in facilities and allocated overhead, offset by a decrease of $1.5 million in professional service fees and other costs.
Current wording
General and administrative expenses were $6.8 million for the three months ended March 31, 2025, compared to $8.8 million for the three months ended March 31, 2024. The decrease of $1.9 million was primarily related to a decrease of $1.3 million in professional services and consulting costs, a decrease of $0.7 million in other employee-related expenses, and a decrease of $0.5 million in facilities and allocated overhead, offset by an increase of $0.6 million in stock-based compensation expense.
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 $68.3 million and $43.6 million for the years ended December 31, 2023 and 2022, respectively, and $54.7 million and $49.0 million for the nine months ended September 30, 2024 and 2023, respectively. As of September 30, 2024, we had an accumulated deficit of $199.6 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 $25.0 million and $25.1 million for the three months ended March 31, 2025 and 2024, respectively. As of March 31, 2025, we had an accumulated deficit of $248.0 million.
Previous wording
the impact of the COVID-19 pandemic, as well as other factors, including inflation, economic uncertainty and geopolitical tensions, which may exacerbate the magnitude of the factors discussed above.
Current wording
the impact of health epidemics, pandemics and other widespread outbreaks of contagious disease, as well as other factors, including inflation, economic uncertainty and geopolitical tensions, which may exacerbate the magnitude of the factors discussed above.
Previous wording
As of September 30, 2024, we had $274.6 million in cash, cash equivalents and available-for-sale marketable securities. We believe that our existing cash, cash equivalents and available-for-sale marketable securities will be sufficient to fund our current operating plan for at least the next 12 months. 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 into 2027. 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 March 31, 2025, we had $226.0 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.
Previous wording
Net cash used in operating activities was $82.8 million for the nine months ended September 30, 2024 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 net change in 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 charge 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.
Current wording
Net cash used in operating activities was $22.8 million for the three months ended March 31, 2025, and consisted primarily of our net loss of $25.0 million and changes in our net operating assets and liabilities of $2.3 million, offset by net non-cash charges of $4.5 million. The net change in operating assets and liabilities consisted primarily of a decrease of $3.0 million in deferred revenue as we recognized revenue under our collaboration agreements, a decrease of $2.6 million in accrued expenses and other current liabilities, a decrease of $1.4 million in operating lease liabilities and a decrease of $2.2 million in prepaid expenses and other current assets, offset by an increase of $2.2 million in accounts payable due to the timing of payments to our vendors. The net non-cash charges consisted primarily of $2.9 million in stock-based compensation expense, $1.4 million of depreciation expense, $1.3 million in non-cash lease expense, partially offset by $1.0 million in accretion of discounts on available-for-sale marketable securities and $0.1 million in amortization of non-cash collaboration revenue related to the Affini-T Agreement.
Previous wording
Net cash used in operating activities for the nine months ended September 30, 2023 was $70.8 million and consisted primarily of our net loss of $49.0 million and changes in our net operating assets and liabilities of $22.1 million, partially offset by net non-cash charges of $0.2 million. The changes in our net operating assets and liabilities consisted primarily of a decrease of $25.0 million in deferred revenue and collaboration advances as we recognized revenue under our collaboration agreements, an increase of $3.9 million in accounts receivable related to the Affini-T Agreement and the Moderna Agreement and a $1.0 million decrease in operating lease liabilities due to recurring payments under the existing lease agreements, partially offset by a $2.4 million increase in accounts payable due to the timing of payments to our vendors, a $2.4 million increase in other non-current liabilities, a $1.3 million decrease in contract assets related to the Affini-T Agreement, a $0.7 million increase in accrued expenses and other liabilities and a $0.6 million increase in income tax payable due to additional tax expense. The net non-cash charges consisted primarily of $4.5 million in stock-based compensation expense, $3.1 million in non-cash lease expense and $3.0 million of depreciation expense, offset by $6.9 million credit related to amortization of the discount on our available-for-sale marketable securities, $2.9 million increase in the fair value of our investments in Affini-T and $0.7 million in amortization of non-cash collaboration revenue related to the Affini-T Agreement.
Current wording
Net cash used in operating activities for the three months ended March 31, 2024, was $29.9 million and consisted primarily of our net loss of $25.1 million and changes in our net operating assets and liabilities of $10.9 million, partially offset by net non-cash charges of $6.2 million. The changes in our net 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 $3.0 million decrease in accrued expenses and other liabilities primarily related to payout of the 2024 annual bonus, and a $0.9 million increase in prepaid expenses and other assets, offset by an increase of $3.2 million in accounts payable due to the timing of payments to our vendors. The net non-cash charges consisted primarily of $5.1 million in stock-based compensation expense, $1.1 million in non-cash lease expense and $1.3 million of depreciation expense, partially offset by $1.2 million in amortization of discounts on available-for-sale marketable securities.
Previous 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.
Current wording
Net cash provided by investing activities for the three months ended March 31, 2025, was $24.7 million primarily due to net maturities of available-for-sale marketable securities of $25.0 million, partially offset purchases of property and equipment of $0.3 million.
Previous wording
Net cash used in investing activities for the nine months ended September 30, 2023 was $14.9 million due to net purchases of available-for-sale marketable securities of $6.8 million and purchases of property and equipment of $8.1 million.
Current wording
Net cash used in investing activities for the three months ended March 31, 2024, was $94.7 million due to net purchases of available-for-sale marketable securities of $93.8 million and purchases of property and equipment of $0.9 million.
Previous 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.
Current wording
Net cash provided by financing activities for the three months ended March 31, 2024, was $86.5 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 nine months ended September 30, 2024, 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, 2023, filed with the SEC on March 27, 2024.
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 three months ended March 31, 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
A description of recently issued accounting pronouncements that may potentially impact our financial position, results of operations or cash flows is disclosed in Note 2 to our condensed consolidated financial statements included elsewhere in this Quarterly Report on Form 10-Q.
Current wording
A description of recently issued accounting pronouncements that may potentially impact our financial position, results of operations or cash flows is disclosed in Note 2 to our condensed financial statements included elsewhere in this Quarterly Report on Form 10-Q.
Previous wording
Our management’s discussion and analysis of our financial condition and results of operations is based on our financial statements, which have been prepared in accordance with generally accepted accounting principles (“GAAP”). The preparation of these financial statements requires us to make estimates and assumptions that affect the reported amounts of assets and liabilities and the disclosure of contingent assets and liabilities at the date of the financial statements, as well as thereported revenue generated and expenses incurred during the reporting periods. On an ongoing basis, we evaluate our estimates and judgments, including but not limited to those related to revenue recognition under our collaboration agreements, accrued research and development costs, the fair value of common stock and stock-based compensation expense, the valuation of deferred tax assets, and uncertain income tax positions. These estimates and assumptions are monitored and analyzed by us for changes in facts and circumstances, and material changes in these estimates and assumptions could occur in the future. Our estimates are based on our historical experience and on various other factors that we believe are reasonable underthe circumstances, the results of which form the basis for making judgments about the carrying value of assets and liabilities that are not readily apparent from other sources. Changes in estimates are reflected in reported results for the period in which they become known. Actual results may differ from these estimates under different assumptions or conditions.
Current wording
On an ongoing basis, we evaluate our estimates and judgments, including but not limited to those related to revenue recognition under our collaboration agreements, stock-based compensation expense, the valuation of deferred tax assets, and uncertain income tax positions. These estimates and assumptions are monitored and analyzed by us for changes in facts and circumstances, and material changes in these estimates and assumptions could occur in the future. Our estimates are based on our historical experience and on various other factors that we believe are reasonable underthe circumstances, the results of which form the basis for making judgments about the carrying value of assets and liabilities that are not readily apparent from other sources. Changes in estimates are reflected in reported results for the period in which they become known. Actual results may differ from these estimates under different assumptions or conditions.
Previous wording
During the nine months ended September 30, 2024, 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, 2023.
Current wording
During the three months ended March 31, 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
Our management’s discussion and analysis of our financial condition and results of operations is based on our financial statements, which have been prepared in accordance with generally accepted accounting principles (“GAAP”). The preparation of these financial statements requires us to make estimates and assumptions that affect the reported amounts of assets and liabilities and the disclosure of contingent assets and liabilities at the date of the financial statements, as well as thereported revenue generated and expenses incurred during the reporting periods.