SGMOQ 10-K & 10-Q changes, risk factors and insider trading

We have a history of recurring net losses, including $97.9$122.9 million and $257.8$97.9 million for the years ended December 31, 20242025 and 2023,2024, respectively, and we have otherwise generated operating losses since we began operations in 1995. The extent of our future losses and the timing of profitability are uncertain, and we expect to incur losses for the foreseeable future. We have been engaged in developing our ZF technology since inception, which has and will continue to require significant research and development expenditures. To date, we have generated our funding from issuance of equity securities, revenues derived from collaboration agreements, licenses to our capsid technology, other strategic partnerships in non-therapeutic applications of our technology, federal government research grants and grants awarded by research foundations. We expect to continue to incur additional operating losses for the next several years as we continue, subject to our ability to raise substantial additional capital, to develop our preclinical core neurology therapeutic programs and capsid engineering platform. IfIn any event, we will need substantial additional funding in the timevery requirednear term to generateexecute significantour productoperating revenuesplan and achieveto profitabilitycontinue to operate as a going concern, which funding is longerdependent thanon our ability to secure collaboration partners for our programs. There can be no assurance that we currentlywill anticipatebe able to secure a commercialization partner for our Fabry disease program or partner or sell any other programs in a timely manner, on acceptable terms, or at all, and if we are unable to generateexecute liquiditysuch throughan equityagreement financingproviding orus otherwith sourcessignificant ofupfront funding,funding in the very near term, we maywill not be forcedable to secure sufficient capital to continue to operate as a going concern and we will further curtail or suspend, or entirely cease, our operations.

There is substantial doubt about our ability to continue to operate as a going concern. We will need substantial additional funding in the very near term to execute our operating plan and to continue to operate as a going concern. If adequate funds are not available to us on a timely basis, or at all, we will be required to take additional actions to address our liquidity needs, including additional cost reduction measures such as further reducing operating expensesexpenses, including through additional workforce reductions, and delaying, reducing the scope of, discontinuing or altering our research and development activities, which would have a material adverse effect on our business and prospects, or at any time we may elect to or may be required to cease operations entirely, liquidate all or a portion of our assets, and/or seek protection under the U.S. Bankruptcy Code,Code in the very near term, and you may lose all or part of your investment. Future sales and issuances of equity securities would also result in substantial dilution to our stockholders.

Moreover, we have historically relied in part on collaboration partners to provide funding for and otherwise advance our preclinical and clinical programs. However, in June 2023, our collaboration agreements with Biogen and Novartis terminated, our collaboration agreement with Kite expired pursuant to its terms in April 2024, and in December 2024, Pfizer notified us of its termination for convenience, effective April 21, 2025, of its collaboration agreement with us. Further, while we may identify new collaboration partners who can progress some of the programs that were the subject of these collaborations, as well as our Fabry disease program, our hemophilia A program and our STAC-BBB capsid and modular integrase platform,platforms, we have not yet been, and may never be, successful in doing so in a timely manner, or on acceptable terms or at all, and we may otherwise fail to raise sufficient additional capital in order to progress these and our other programs ourselves, in which case, we will not receive any return on our investments in these programs. Although we have received $50.0an aggregate of $88.0 million in upfront license fees and/or milestone payments and are eligible to earn future licensed target fees and development and commercial milestone payments in connection with our license agreementagreements with GenentechGenentech, Astellas and we also received $20.0 million in an upfront license fee and are eligible to earn future additional licensed target fees and milestone payments in connection with our license agreement with Astellas,Lilly, we may never receive any further payments under eitherany agreement.of these agreements. In any event, we need substantial additional funding in order to advance our core neurology programs, including to make planned regulatory submissions and commence planned clinical trials as described in this Annual Report, as well as our Fabry disease and hemophilia A programs, capsid engineering efforts and modular integrase platform, and to otherwise execute on our current operating plan.

If we raise additional capital through public or private equity offerings, including sales pursuant to our at-the-market offering program with Jefferies LLC, theyour ownership interest of our existing stockholders will be diluted, and such dilution may be substantial given our current stock price decline. In addition, the terms of any new equity securities we may issue may have a preference over, and include rights superior to, our common stock. If we raise additional capital through royalty financings or other collaborations, strategic alliances or licensing arrangements with third parties, we may need to relinquish certain valuable rights to our product candidates, technologies, future revenue streams or research programs or grant licenses on terms that may not be favorable. If we raise additional capital through debt financing, we may be subject to specified financial covenants or covenants limiting or restricting our ability to take specific actions, such as incurring additional debt, making capital expenditures or pursuing certain transactions, any of which could restrict our ability to commercialize our product candidates or operate as aour business.

In addition, as we focus our efforts on proprietary human therapeutics, we will need to seek regulatory approvals of our product candidates from the FDA or other comparable foreign regulatory authorities, a process that could cost in excess of hundreds of millions of dollars per product. We may experience difficulties in accessing the capital markets due to external factors beyond our control, such as volatility in the equity markets for emerging biotechnology companies and general economic and market conditions both in the United States and abroad. In particular, our ability to raise the substantial additional capital we need in order to fund our business may be adversely impacted by global economic conditions and disruptions to and volatility in the credit and financial markets in the United States and worldwide, such as has been experienced recently, including as a result of the imposition of tariffs and escalating trade tensions. We cannot be certain that we will be able to obtain financingthe onsubstantial termsadditional acceptablecapital that we need to us,support orour atoperations all.and to continue to operate as a going concern. Our failure to obtain adequate andfunding timelyin fundingthe very near term will adversely affect our ability to continue to operate as a going concern and our ability to develop our technology and products candidates, and at any time we may elect to or may be required to cease operations.

Based on our current operating plan we expect to meet our liquidity requirements only into the middle of the secondthird quarter of 2025.2026. We continue to actively seek substantial additional capital, including through public or private equity or debt financing, royalty financing or other sources, such as strategic collaborations and other direct investments in our programs. In addition, we could determine at any time that it is in the best interest of our stakeholders to cease operations entirely, liquidate all or a portion of our assets, and/or seek protection under the U.S. Bankruptcy Code in the very near term. We have explored, and will continue to explore, whether filing for bankruptcy protection is in the best interest of our companySangamo and our stakeholders.stakeholders and the most advantageous time for such filing in order to preserve sufficient resources to undertake an appropriate bankruptcy process. In the event we file for relief under the U.S. Bankruptcy Code, our operations, our ability to develop our product candidates and execute on our operating plan, and our ability to continue as a going concern will be subject to the risks and uncertainties associated with bankruptcy proceedings, including, among others: our ability to execute, confirm and consummate a plan of reorganizationreorganization, including the sufficiency of our cash resources to facilitate such a plan; the additional, significant costs of bankruptcy proceedings and related fees; our ability to obtain sufficient financing to allow us to emerge from bankruptcy and execute our business plan thereafter, and our ability to comply with terms and conditions of any such financing; our ability to continue our operations in the ordinary course; our ability to maintain our relationships with our collaborators, counterparties, employees and other third parties; our ability to obtain, maintain or renew contracts that are critical to our operations on reasonably acceptable terms and conditions or at all; our ability to attract, motivate and retain key employees; the ability of third parties to use certain provisions of the U.S. Bankruptcy Code to terminate contracts without first seeking Bankruptcy Court approval; the ability of third parties to seek and obtain court approval to terminate or shorten the exclusivity period for us to propose and confirm a plan of reorganization, to appoint a trustee, or to convert a proceeding under Chapter 11 of the U.S. Bankruptcy Code to a proceeding under Chapter 7 of the U.S. Bankruptcy Code; and the actions and decisions of our stakeholders and other third parties who have interests in our bankruptcy proceedings that may be inconsistent with our operational and strategic plans. Any delays in our bankruptcy proceedings would increase the risks that we may not be able to reorganize our business and emerge from bankruptcy proceedings and may increase our costs associated with the bankruptcy process or result in prolonged operational disruption. In addition, we would need the prior approval of the Bankruptcy Court for transactions outside the ordinary course of business during the course of any bankruptcy proceedings, which may limit our ability to respond timely to certain events or take advantage of certain opportunities. Because of the risks and uncertainties associated with any bankruptcy proceedings, we cannot accurately predict or quantify the ultimate impact of events that could occur during any such proceedings. There can be no guarantees that if we seek protection under the U.S. Bankruptcy Code, we will emerge from any such proceedings as a going concern or that holders of our common stock will receive any recovery from any bankruptcy proceedings.

We have fully impaired our goodwill and indefinite-lived intangible assets, have recorded significant impairment of our long-lived assets, and may be required to record significant additional charges if our long-lived assets become further impaired in the future.

We evaluate the carrying value of long-lived assets, which include property and equipment, leasehold improvements and right-of-use assets, for impairment whenever events or changes in circumstances indicate that the carrying amounts of the asset may not be fully recoverable. Factors that may indicate potential impairment and trigger an impairment test include, but are not limited to, general macroeconomic conditions, conditions specific to the industry and market, an adverse change in legal factors, business climate or operational performance of the business, and sustained decline in our stock price and market capitalization compared to the net book value. During the years ended December 31, 20242025 and 2023,2024, we recognized impairment charges of $5.5$13.2 million and $155.0$5.5 million, respectively.respectively, We have fully impaired our goodwill and indefinite-lived intangible assets in 2023 and have significantly impairedon our long-lived assets in both 2023 and 2024.assets. We will continue to assess whether our long-lived assets are impaired in future periods. We have completed the wind-down of our France researchperiods and development activities and corresponding reduction in force of all France employees, and expect to close our Brisbane, California facility in the near future, and we have recognized related impairments in both 2023 and 2024. Itit is reasonably possible that additional impairment charges will be recognized, for example, if sublease rates of leased facilities are less than those estimated.recognized. For additional information regarding these impairment charges, see Note 5 – Impairment of Goodwill, Indefinite-lived Intangible Assets and Long-lived Assets and Write-Down of Assets Held For Sale in the accompanying notesNotes to the Consolidated Financial Statements included in Part II, Item 8, “Financial Statements and Supplementary Data” of this Annual Report on Form 10-K.

Although a certain amount of our federal net operating loss carryforwards carry forward indefinitely (but are subject to a percentage limitation), a significant amount of our federal and all of our state net operating loss carryforwards will begin to expire, if not utilized, beginning in 20252026 and 2029, respectively. The net operating loss carryforwards subject to expiration could expire unused and be unavailable to offset future income tax liabilities. In addition, under Sections 382 and 383 of the Internal Revenue Code of 1986, as amended, and corresponding provisions of state law, if a corporation undergoes an “ownership change,” which is generally defined as a greater than 50 percentage point change in its equity ownership value over a three-year period, the corporation’s ability to use its pre-change net operating loss carryforwards and other pre-change tax attributes to offset its post-change income or taxes may be limited. We have experienced an ownership change in the past and we may also experience additional ownership changes in the future as a result of subsequent shifts in our stock ownership, some of which may be outside of our control. If an ownership change occurs and our ability to use our net operating loss carryforwards is materially limited, it would harm our future operating results by effectively increasing our future tax obligations. In addition, at the state level, there may be periods during which the use of net operating loss carryforwards is suspended or otherwise limited, which could accelerate or permanently increase state taxes owed. For example, California recently enacted legislation that, with certain exceptions, suspends the ability to use California net operating loss carryforwards to offset California income and limits the ability to use California business tax credits to offset California taxes, for taxable years beginning after 2023 and before 2027. As a result, if we earn net taxable income, we may be unable to use all or a material portion of our net operating loss carryforwards and other tax attributes, which could potentially result in increased future tax liability to us and adversely affect our future cash flows.

Our core preclinical neurology programs, which are the current focus of our research and development efforts, are in the early stages of development. We intend to advance our core neurology program product candidates from research programs through preclinical development and to submit new INDs, applications for clinical trial approval and equivalent filings in other jurisdictions necessary to conduct human clinical trials evaluating our product candidates. While the FDA cleared our IND application for ST-503 for the treatment of intractable pain due to idiopathic small fiber neuropathy in November 2024 and we expecthave to commenceactivated the Phasefirst 1/2six clinical studysites inand mid-2025,have subjectbegun topatient ourrecruitment receiptand of substantial additional capital,enrollment, we have not yet demonstrated our ability to successfully commence any clinical trialsdosing of patients with any product candidates from our core preclinical neurology programs. The preparation and submission of applications to conduct clinical trials require us to conduct rigorous and time-consuming preclinical testing and studies and prepare documentation relating to, among other things, the toxicity, safety, manufacturing, chemistry and clinical protocols of our product candidates. We may experience unforeseen difficulties that could delay or otherwise prevent us from executing this strategy successfully. For example, we may encounter problems in the manufacturing of a product candidate and may fail to demonstrate consistency in the formulation of a product candidate. Our preclinical tests may produce negative or inconclusive results, which may lead us to decide, or which may lead regulators to require us, to conduct additional preclinical testing. If we cannot obtain positive results in preclinical testing, we may decide to abandon a product candidate altogether. In addition, our ability to complete and submit such applications to conduct clinical trials may depend on the support of collaborators and the timely performance of their obligations under relevant collaboration agreements. If our collaborators are not able to perform such obligations or if they choose to slow down or delay the development of a product candidate, we may not be able to submit the clinical trial applications on a timely basis or at all. Furthermore, the submission of applications to conduct clinical trials involves significant cost and labor, and we may not have sufficient resources and personnel to complete the filing of all intended applications, which may force us to scale back the number of applications or forego potential applications that we believe are promising. Any delay, suspension or reduction of our efforts to pursue our preclinical and clinical development strategy could have an adverse effect on our business and cause the market price of our common stock to decline.

We have received RMAT designation for our product candidates to treat severe hemophilia A and Fabry disease. Additionally, some of our product candidates, including our product candidate to treat Fabry disease, have also been granted Orphan Drug Designation by the FDA and PRIME eligibility by the EMA, and some have also been designated Orphan Medicinal Products by the European Commission. Regulatory authorities in some jurisdictions, including the United States and the EU, may designate drugs for relatively small patient populations as orphan drugs. In addition, our product candidate to treat Fabry disease was granted FDA Fast Track Designation in May 2023.2023 and ST-503 received fast track designation for the treatment of small fiber neuropathy in December 2025. In October 2024, we announced that the FDA agreed that the ongoing Phase 1/2 STAAR study can serve as the primary basis for seeking Accelerated Approval for our product candidate to treat Fabry disease.disease, and in December 2025 we initiated a rolling BLA submission with the FDA. For additional information regarding these special regulatory designations, see “Business—Government Regulation.”

In addition to uncertainty about the potential pricing structure for certain of our product candidates, cost containment is a recurrent trend in the healthcare industry. Government authorities and third-party payors have attempted to control costs by limiting coverage and the amount of reimbursement for certain medications. For example, the HHS imposes rebates on many Medicare Part B and Medicare Part D products to penalize price increases that outpace inflation on an annual basis. HHS has also been empowered to negotiate the price of certain single-source biologics that have been on the market for at least 11 years covered under Medicare as part of the Medicare Drug Price Negotiation Program. Each year up to twenty (20) products will be selected by HHS for the Medicare Drug Price Negotiation Program. Products subject to the Medicare Drug Price Negotiation Program are expected to experience a significant reduction in reimbursement from the Medicare program on a per unit basis. We cannot be sure that coverage and adequate reimbursement will be available for any product that we commercialize and, if reimbursement is available, what the level of reimbursement will be. If reimbursement is not available or is available only at limited levels, we may be unable to successfully commercialize any product candidate for which we obtain regulatory approval. Our inability to promptly obtain coverage and profitable reimbursement rates from both government-funded and private payors for any approved products that we develop could have an adverse effect on our operating results, our ability to raise capital needed to commercialize products and our overall financial condition.

Moreover, in order to obtain reimbursement for our products in some European countries, including some EU Member States, we may be required to compile additional data comparing the cost-effectiveness of our products to other available therapies. HTA of medicinal products is becoming an increasingly common part of the pricing and reimbursement procedures in some EU Member States, including those representing the larger markets. The outcome of an HTA will often influence the pricing and reimbursement status granted to these medicinal products by the competent authorities of individual EU Member States. The extent to which pricing and reimbursement decisions are influenced by the HTA of the specific medicinal product currently varies between EU Member States. InOn DecemberJanuary 2021,12, 2025, the EU HTA Regulation was adopted, which will enterentered into application in 2025 and is intended to harmonize the clinical benefit assessment of HTA across the EU. However, individual EU Member States will continue to be responsible for assessing non-clinical (e.g., economic, social and ethical) aspects of health technologies and making decisions on pricing and reimbursement. If we are unable to maintain favorable pricing and reimbursement status in EU Member States for product candidates that we may successfully develop and for which we may obtain regulatory approval, any anticipated revenue from and growth prospects for those products in the EU could be negatively affected.2025.

The regulations that govern, among other things, regulatory approvals, coverage, pricing and reimbursement for new drug products vary widely from country to country. In the United States and some foreign jurisdictions, there have been a number of legislative and regulatory changes and proposed changes regarding the healthcare system that could prevent or delay regulatory approval of our product candidates, restrict or regulate post-approval activities and affect our ability to successfully sell any product candidates for which we obtain regulatory approval. Also, there has been heightened governmental scrutiny recently over biopharmaceutical pricing practices in light of the rising cost of prescription drugs and biologics. Such scrutiny has resulted in several recent Presidential executive orders, Congressional inquiries and proposed and enacted federal and state legislation designed to, among other things, bring more transparency to product pricing, review the relationship between pricing and manufacturer patient programs, and reform government program reimbursement methodologies for biopharmaceutical products. At the state level, legislatures have increasingly passed legislation and implemented regulations designed to control pharmaceutical and biological product pricing, including price or patient reimbursement constraints, discounts, restrictions on certain product access and marketing cost disclosure and transparency measures, and, in some cases, have been designed to encourage importation from other countries and bulk purchasing. For a discussion of health reform activity and the current pricing framework, see “Business—Government Regulation—Healthcare ReformReform,” “Business—Government Regulation—Regulation of Our Operations,” and “Business—Government Regulation—Pricing, Coverage and Reimbursement.”

We have implemented several strategic decisions to reallocate resources among our various clinical and preclinical development programs, including the decision to defer new investments in our Fabry disease gene therapy program unless and until we are able to successfully secure a commercialization partner or external investment in this program.programs. There can be no assurance that such efforts will be successful in a timely manner, or at all, in which case, we will not receive any return on our investments in this program. If we are not able to secure a commercialization partner for our Fabry disease program in the very near term, our ability to raise additional capital needed to support our operations will be substantially impaired. As part of our restructurings and the related strategic reprioritization, we have determined to focus substantially all of our efforts on our core preclinical neurology programs. Investment in preclinical programs is highly speculative, as it entails substantial upfront capital expenditures and significant risk that any potential product candidate will fail to demonstrate adequate effect and/or an acceptable safety profile. In addition, we are developing, and may in the future develop, certain product candidates designed to treat neurological diseases using our novel capsid delivery technology. If our capsid development efforts are not successful, we may be required to defer, or cease entirely, development of such product candidates. For additional details on our collaboration terminations, see “Risk Factors—Risks Relating to our Industry.” As a result of these strategic decisions, we could miss valuable opportunities to capitalize on the potential of our discontinued and halted programs. We may also allocate internal resources to a product candidate in a therapeutic area in which it would have been more advantageous to enter into a collaboration or that does not prove to have viable commercial opportunities. Any failure to use our financial and human resources efficiently could harm our business and operations.

There are significant risks associated with manufacturing, storing and transporting our product candidates including, among others, cGMP compliance, cost overruns, technical problems with process scale-up, specialized facilities and equipment, process reproducibility, stability issues, lot consistency, yields and timely availability of highly specific raw materials. Even though product batches released for use in clinical trials undergo sample testing, some defects may only be identified following release. In addition, process deviations or unanticipated effects of approved process changes may result in these intermediate products not complying with stability requirements or specifications. Also, our product candidates must be stored and transported at temperatures within a certain range. If these environmental conditions deviate, our product candidates’ remaining shelf-lives could be impaired or their efficacy and safety could be adversely affected, making them no longer suitable for use. Moreover, product candidates that are biologics involve complex processes, including the development of cell lines or cell systems to produce the biologic, with the challenge of significant variability. There are difficulties in growing large quantities of such cells, consistently and sufficiently isolating certain types of cells and harvesting and purifying the biologic produced by them. The cost to manufacture biologics is generally far higher than traditional small molecule chemical compounds, and the manufacturing process can be difficult to reproduce.

Natural disasters could severely disrupt our operations and our facilities and the manufacturing facilities of our CMOs, and any disruption would likely have a negative impact on our business, financial condition, results of operations and prospects. If a natural disaster, pandemic or epidemic, geopolitical crisis, including the ongoing conflict between Russia and Ukraine and conflicts in the Middle East, conflict between Russia and Ukraine, power outage or any other event that is out of our control occurred that prevented us or third parties on which we rely from using all or a significant portion of our or their facilities, that damaged critical infrastructure or that otherwise disrupted our or their operations, it may be difficult or, in certain cases, impossible for us to continue our business and operations for a substantial period of time. The disaster recovery and business continuity plans we have in place currently are limited and may not prove adequate in the event of a serious disaster or similar event. We may incur substantial expenses as a result of the limited nature of our disaster recovery and business continuity plans, which could have an adverse effect on our business, financial condition, results of operations and prospects. Such disasters or events occurring at facilities of third parties on which we rely could also negatively impact our business and operations.

Certain jurisdictions have enacted data localization and cross-border data transfer laws, which could make it more difficult to transfer information across jurisdictions. In particular, the EEA and the U.K. have significantly restricted the transfer of personal data to the United States and other countries whose privacy and data security laws they believe to be inadequate. Other jurisdictions may adopt or have already adopted similarly stringent interpretations of data localization and cross-border data transfer laws. Although there are currently various mechanisms that may be used to transfer personal data from the EEA and United Kingdom to the United States in compliance with law, such as the EEA standard contractual clauses, the U.K.’s International Data Transfer Agreement / Addendum and the EU-U.S. Data Privacy Framework and the U.K. extension thereto (which allows for transfers to relevant U.S.-based organizations who self-certify compliance and participate in the Framework), these mechanisms are subject to legal challenges, and there is no assurance that we can satisfy or rely on these measures to lawfully transfer personal data to the United States. If we are unable to implement a legal mechanism to ensure that our transfers of personal data from the EEA or the U.K. are lawful, or if the requirements for a legally-compliant transfer are too onerous, we could face adverse consequences, including the interruption or degradation of our operations, increased exposure to regulatory actions, substantial fines and penalties and injunctions against processing or transferring personal data, and could be required to increase our data processing capabilities in the EEA, the U.K. or elsewhere at significant expense. Restrictions on our ability to transfer personal data from the EEA, the U.K. or elsewhere could impact our clinical trial activities in the EEA or the U.K. and limit our ability to collaborate with CROs and other third parties. Additionally, companies that transfer personal data out of the EEA and U.K. to other jurisdictions, particularly to the United States, are subject to increased scrutiny from regulators, individual litigants, and activist groups. Some European regulators have ordered certain companies to suspend or permanently cease certain transfers out of Europe for allegedly violating the GDPR’s cross-border data transfer limitations. Additionally, the U.S. Department of Justice issued a rule entitled the Preventing Access to U.S. Sensitive Personal Data and Government-Related Data by Countries of Concern or Covered Persons, which places additional restriction on certain data transactions involving countries of concern (e.g., China, Russia, Iran) and covered persons that may impact certain business activities such as vendor engagements, sale or sharing of data, employment of certain individuals, and investor agreements. Violations of the rule could lead to significant civil and criminal fines and penalties. The rule applies regardless of whether data is anonymized, key-coded, pseudonymized, de-identified or encrypted, which presents particular challenges for companies like ours and may impact our ability to transfer data in connection with certain transactions or agreements. For more information regarding these regulations, see “Business—Government Regulation—Privacy Regulation.”

Our obligations related to privacy and data security (and consumers’ expectations regarding them) are quickly changing and becoming increasingly stringent, creating uncertainty. These obligations may be subject to differing applications and interpretations, which may be inconsistent or in conflict among jurisdictions. Preparing for and complying with these obligations requires us to devote significant resources. These obligations may also necessitate changes to our information technologies, systems and practices and those of any third parties that process personal data on our behalf. In addition, these obligations may require us to change our business model. We may at times fail, or be perceived to have failed, in our efforts to comply with our data privacy and security obligations. Moreover, despite our efforts, our personnel or third parties upon which we rely may fail to comply with such obligations, which could negatively impact our business operations and compliance posture.

Financial instability and a general decline in economic conditions in the United States and other countries caused by political instability and conflict, including the ongoing conflict between Russia and Ukraine and conflicts in the Middle East, conflict between Russia and Ukraine, geopolitical challenges arising from the imposition of tariffs and escalating trade tensions, and economic or financial challenges caused by recent and potential future bank failures or by general health crises, have led to market disruptions, including significant volatility in commodity prices, credit and capital markets instability, including disruptions in access to bank deposits and lending commitments, supply chain interruptions, rising interest rates and global inflationary pressures. These macroeconomic factors could materially and adversely affect our ability to continue to operate as a going concern and could otherwise have a material adverse effect on our business, operations, operating results and financial condition as well as the price of our common stock. The failure of any bank in which we deposit our funds could reduce the amount of cash we have available for our operations or delay our ability to access such funds. Any such failure may increase the possibility of a sustained deterioration of financial market liquidity, or illiquidity at clearing, cash management and/or custodial financial institutions. In the event we have a commercial relationship with a bank that has failed or is otherwise distressed, we may experience delays or other issues in meeting our financial obligations. If other banks and financial institutions fail or become insolvent in the future in response to financial conditions affecting the banking system and financial markets, our ability to access our cash and cash equivalents may be threatened and our ability to raise additional capital when needed could be substantially impaired, which could have a material adverse effect on our business, operations, operating results and financial condition as well as the price of our common stock. In particular, failure to secure any necessary financing in a timely manner and on favorable terms could require us to delay or abandon clinical development plans or we may be forced to further curtail or suspend, or entirely cease, our operations. In addition, any or all of these factors could disrupt our and our collaborators’ supply chains and adversely affect our and our collaborators’ ability to conduct ongoing and future clinical trials of our product candidates.

Our ability to continue funding our operations, advance development of our product candidates and ultimately commercialize our technologies depends on our ability to secure collaboration partners for our programs. If we are not able to find collaborators, or if our collaborators do not diligently pursue product development efforts, we maywill not be able to secure sufficient capital to continue to operate as a going concern or to develop our technologies or product candidates.concern.

If we are unable to secure additionalone or more significant collaborations in the very near term or if our collaborators are unable or unwilling to diligently advance the development, regulatory approval and commercialization of our product candidates, whether due to internal portfolio decisions or otherwise, ourwe growthwill maynot slowbe and adversely affect our abilityable to generatesecure fundingsufficient for development of our technologies and product candidates as well as our abilitycapital to continue to operate as a going concern, and we maywill be required to cease operations. AlthoughIn particular, we are activelyengaged seekingin partnersearly stage business development discussions with potential counterparties concerning a commercialization agreement for our Fabry disease program, therebut have been unsuccessful in consummating any such transaction to date despite our efforts to do for many months. There can be no assurance ourthat efforts to secure a collaborationwe will be successful in a timely manner, or at all, in which case, we may not receive any return on our investments in these programs and our ability to operate as a going concern may be materially and adversely affected. Moreover, if we are unableable to secure a commercialization partner for our Fabry disease program or partner or sell any other programs in a timely manner, on acceptable terms, or at all, and if we are unable to execute such an agreement providing us with significant upfront funding in the very near term, ourwe abilitywill not be able to raisesecure additionalsufficient capital necessaryto continue to fundoperate ouras operationsa willgoing be substantially impaired.concern. While we are also seeking additional partnerships for our capsid delivery technology, there can be no assurance that we will be successful in doing so, and such partnerships, including the Genentech AgreementAgreement, the Astellas Agreement, and the AstellasLilly Agreement, do not generally provide for upfront license and near-term milestone payments in amounts sufficient to fully fund our ongoing and planned operations. In addition, our ongoing collaborators may sublicense or abandon development programs with little advance notice, or we may have disagreements or disputes with our collaborators, which would cause associated product development to slow or cease. In addition, the business or operations of our collaborators may change significantly through restructurings, acquisitions, other strategic transactions that may negatively impact their ability to advance our programs.

Defense of these claims, regardless of their merit, would involve substantial litigation expense, could expose proprietary information and would be a substantial diversion of employee resources from our business. Furthermore, recent policy changes at the U.S. PTO have limited the ability to use inter partes review, or IPR, as a means to challenge U.S. patents, which may reduce our ability to mitigate the risk of third-party patents prior to or during litigation. In the event of a successful claim of infringement against us, we may have to pay substantial damages, including treble damages and attorneys’ fees for willful infringement, pay royalties, redesign our infringing products or obtain one or more licenses from third parties, which may be impossible or require substantial time and monetary expenditure.

The legal systems of many foreign countries do not favor the enforcement of patents and other intellectual property protections, which could make it difficult for us to stop the infringement of our patents or marketing of competing products in violation of our proprietary rights. For example, some foreign countries have compulsory licensing laws under which a patent owner must grant licenses to third parties. In addition, some countries limit the enforceability of patents against third parties, including government agencies or government contractors. In these countries, patents may provide limited or no benefit. Further, the complexity and uncertainty of European patent laws have increased in recent years. In Europe, the new unitary patent system that came into effect in June 2023 would significantly impact European patents, including those granted before the introduction of such a system. Under the unitary patent system, European applications will have the option, upon grant of a patent, of becoming a Unitary Patent which will be subject to the jurisdiction of the newly formed Unified Patent Court, or UPC. As the UPC is a relatively new court system, there is nolittle precedent or established body of case law on which the court can base its decisions, thus increasing the uncertainty of any litigation before the UPC. Patents granted before the implementation of the UPC will have the option of opting out of the jurisdiction of the UPC and remaining as national patents in the UPC countries. Patents that remain under the jurisdiction of the UPC will be potentially vulnerable to a single UPC-based revocation challenge that, if successful, could invalidate the patent in all countries who are signatories to the UPC. We cannot predict with certainty the long-term effects of any potential changes. Proceedings to enforce our patent rights in foreign jurisdictions could result in substantial costs and divert our efforts and attention from other aspects of our business, could put our patents at risk of being invalidated or interpreted narrowly and our patent applications at risk of not issuing and could provoke third parties to assert claims against us. We may not prevail in any lawsuits that we initiate, and the damages or other remedies awarded, if any, may not be commercially meaningful. Accordingly, our efforts to enforce our intellectual property rights around the world may be inadequate to obtain a significant commercial advantage from the intellectual property that we develop or license.

We are increasingly dependent on information technology systems and infrastructure to operate our business, which are large and complex. In the ordinary course of our business, we and the third parties upon which we rely, may collect, receive, store, process, generate, use, transfer, disclose, make accessible, protect, secure, dispose of, share and transmit large amounts of proprietary, confidential and sensitive information, including intellectual property, trade secrets and personal data (such as health-related information). It is critical that we do so in a secure manner to maintain the confidentiality, integrity and availability of such sensitive information. We have also outsourced elements of our operations (including elements of our information technology infrastructure) to third parties, and as a result, we manage a number of third-party vendors who may have access to our computer networks or our confidential information. Many of those third parties in turn subcontract or outsource some of their responsibilities to other third parties. Our ability to monitor third parties’ information security practices is limited, and these third parties may not have adequate information security measures in place. If the third parties upon which we rely experience a security incident or other interruption, we could experience adverse consequences. In addition, supply-chain attacks have increased in frequency and severity, and we cannot guarantee that third parties’ infrastructure in our supply chain or that of the third parties with whom we work have not been compromised.

While all information technology operations are inherently vulnerable to inadvertent or intentional security breaches, incidents, attacks and exposures, the size, complexity, accessibility and distributed nature of our information technology systems, and the large amounts of sensitive information stored on those systems, make such systems potentially vulnerable to unintentional or malicious, internal and external attacks on our technology environment. Attacks of this nature are increasing in their frequency, levels of persistence, sophistication and intensity. Threats to information systems and data are increasingly difficult to detect and come from a variety of sources, including traditional computer “hackers,” threat actors, “hacktivists,” organized criminal threat actors, personnel (such as through theft or misuse), sophisticated nation-states and nation-state-supported actors. Some actors now engage and are expected to continue to engage in cyber-attacks, including nation-state actors for geopolitical reasons and in conjunction with military conflicts and defense activities. During times of war and other major conflicts, we and the third parties on which we rely may be vulnerable to a heightened risk of these attacks, including retaliatory cyber-attacks, that could materially disrupt our systems, operations and supply chain. We and the third parties upon which we rely may be subject to a variety of evolving threats, including but not limited to social-engineering attacks (including through deep fakes, which may be increasingly more difficult to identify as fake, and phishing attacks), malicious code (such as viruses and worms), malware (including as a result of advanced persistent threat intrusions), denial-of-service attacks (such as credential stuffing), credential harvesting, personnel misconduct or error, ransomware attacks, supply-chain attacks, software bugs, server malfunctions, software or hardware failures, loss of data or other information technology assets, adware, telecommunications failures, natural disasters (such as earthquakes, fires, floods), war, terrorism, attacks enhanced or facilitated by artificial intelligence, or AI, and other similar threats. Ransomware attacks are becoming increasingly prevalent and severe and can lead to significant interruptions in our operations, loss of data and income, reputational harm and diversions of funds. Extortion payments may alleviate the negative impact of a ransomware attack, but we may be unwilling or unable to make such payments due to, for example, applicable laws or regulations prohibiting such payments. In addition, our updated work from home policies have intensified our dependence on information technology systems and could increase our cybersecurity risk as many of our critical business activities are currently being conducted remotely utilizing network connections, computers and devices outside our premises or network and our increased reliance on personnel working from home, while in transit and in public locations. Future or past business transactions (such as acquisitions or integrations) could expose us to additional cybersecurity risks and vulnerabilities, as our systems could be negatively affected by vulnerabilities present in acquired or integrated entities’ systems and technologies. Furthermore, we may discover security issues that were not found during due diligence of such acquired or integrated entities, and it may be difficult to integrate companies into our information technology environment and security program. Any of the previously identified or similar threats have in the past and may in the future cause a security incident or other interruption that have in the past and may in the future result in unauthorized, unlawful, or accidental acquisition, modification, destruction, loss, alteration, encryption, disclosure of, or access to our sensitive information or our information technology systems, or those of the third parties upon which we rely.

While we have implemented security measures designed to protect against data security incidents, there can be no assurance that these measures will be effective. We have not always been able in the past and may be unable in the future to detect vulnerabilities in our information technology systems. We take steps designed to detect, mitigate and remediate vulnerabilities in our information security systems (such as our hardware and/or software, including that of third parties upon which we rely), but we may not be able to detect, mitigate and remediate all such vulnerabilities on a timely basis. For example, in April 2018, we announced a data security incident involving the compromise of a senior executive’s company email account. Our investigation of the incident did not reveal any evidence that our systems were otherwise compromised in connection with the incident or that personal data about patients or other individuals besides the executive were accessed or disclosed. However, proprietary, confidential and other sensitive information of ours and that of other entities was accessed and may have been compromised as a result of the incident. Unforeseen developments related to this incident could occur, which could have a further adverse impact on us. Any litigation or regulatory review or investigation arising from this incident could result in significant legal exposure to us. A security incident or other interruption could also result in a material disruption of our development programs and our business operations. For example, the loss of clinical trial data from completed or future clinical trials could result in delays in our regulatory approval efforts and significantly increase our costs to recover or reproduce the data.

While we have no reason to believe we have experienced any recent security incidents, attackers have become very sophisticated in the way they conceal access to systems, and many companies that have been attacked are not aware that they have been attacked. Any delay in the discovery of an attack may result in increased expense and may harm our reputation. Any security incident or interruption that we, or a third-party upon which we rely, experience or are perceived to have experienced could lead to material adverse consequences, including government enforcement actions (for example, investigations, fines, penalties, audits and inspections), additional reporting requirements and/or oversight, restrictions on processing sensitive data (including personal data), litigation (including class claims), indemnification obligations, negative publicity, harm to our reputation, monetary fund diversions, diversion of management attention, interruptions in our operations (including availability of data) and financial loss.loss, and other similar harms. Certain data privacy and security obligations have required us to implement and maintain specific security measures or industry-standard or reasonable security measures to protect our information technology systems and sensitive information. Applicable data privacy and security obligations may require usus, or we may voluntarily choose, to notify relevant stakeholders, including affected individuals, customers, regulators and investors, of security incidents. Such disclosures are costly, and the disclosures or the failure to comply with such requirements could lead to adverse consequences. In addition, failure to maintain effective internal accounting controls related to security breaches and cybersecurity in general could impact our ability to produce timely and accurate financial statements and subject us to regulatory scrutiny. We may expend significant resources or modify our business activities (including our clinical trial activities) in an effort to protect against security incidents or other interruptions. Despite our efforts, there can be no assurance that these vulnerability mitigation measures will be effective. Further, we may experience delays in developing and deploying remedial measures designed to address any such identified vulnerabilities. Vulnerabilities could be exploited and result in a security incident. Our contracts may not contain limitations of liability, and even where they do, there can be no assurance that limitations of liability in our contracts are sufficient to protect us from liabilities, damages or claims related to our data privacy and security obligations. While we may be entitled to damages if our third-party partners fail to satisfy their privacy or data security-related obligations to us, any award may be insufficient to cover our damages, or we may be unable to recover such award. Additionally, we cannot be sure that our insurance coverage, if any, will be adequate or sufficient to protect us from or mitigate liabilities arising out of our privacy and security practices, that such coverage will continue to be available on commercially reasonable terms, or at all, or that such coverage will pay future claims.

•natural disasters, political and economic instability, including wars, terrorism and political unrest, including the conflicts between Russia and Ukraine and in the Middle East, and conflict between Russia and Ukraine, outbreak of health epidemics and the resulting global economic and social impacts;

There currently is a shortage of skilled individuals with substantial experience discovering, developing and manufacturing genomic medicines, which is likely to continue. As a result, competition for these individuals is intense and the turnover rate can be high. We have experienced, and may continue to experience, difficulty hiring, integrating and retaining employees with these skills on acceptable terms given the uncertainty regarding our ability to obtain sufficient additional funding and to continue to operate as a going concern as well as the competition among numerous biopharmaceutical companies and academic institutions for individuals with these skills. In this regard, as a result of the April 2023 and November 2023 Restructurings, approximately 272 roles at our companyCompany were eliminated, and as a result of the France Restructuring, 93 roles were eliminated. Accordingly, we have been and are operating with a shortage of resources and may not be able to effectively conduct our operations with our substantially reduced number of employees. In addition, our history of implementing significant workforce reductions, along with the potential for future workforce reductions, may negatively affect our ability to retain or attract talented employees. Moreover, any negative or unexpected results in our preclinical studies or clinical trials or applications for marketing approval would make it more challenging to hire and retain qualified skilled employees. If we do not obtain sufficient additional funding in the very near term so that we can continue to operate as a going concern and to potentially achievedevelop our growthproduct objectives,candidates, the progress of our research, development, manufacturing and regulatory efforts will slowbe down or halt altogether, which would materially and adversely affect our business, financial condition, results of operations and prospects,halted, and we maywill be required to cease operations.

We are dependent on certain key members of our executive team and certain of our scientific, clinical development and manufacturing personnel, the loss of whose services may impede the progress of our research, development and regulatory efforts. For example, in 2025, our former Senior Vice President and Chief Financial Officer, and in 2024, our former Senior Vice President and Chief People Officer, our former Chief Medical Officer and our former Vice President, Head of Research resigned from their positions. In connection with our restructurings, the employment of each of our former Executive Vice President, Technical Operations, Executive Vice President, Chief Operating Officer, and Senior Vice President, Chief Scientific Officer, was terminated. We could experience resignations of other executives and employees in the future given the uncertainty regarding our ability to obtain sufficient additional funding and to continue to operate as a going concern as well as the intensity of the competition for talent in the biotechnology industry, particularly in the San Francisco Bay Area. Additional resignations or workforce reductions could result in more significant disruptions and threats to our stability and potential growth. While we have entered into employment agreements with each of our executive officers, any of them could leave our employment at any time, as all of our employees are “at will” employees. We do not have “key person” insurance on any of our employees.

We havecurrently indo thenot past,meet, and maydo innot the future, be unableexpect to complyregain withcompliance with, the listing standards of the Nasdaq StockCapital Market LLC,Market, or Nasdaq.Nasdaq, prior to the April 27, 2026 compliance deadline. If we faildo not regain compliance prior to comply with listing standards in the future,April 27, 2026 compliance deadline, our common stock maywill be delisted.delisted from Nasdaq. Delisting from Nasdaq could adversely affect the liquidity of our common stock and the market price of our common stock could decrease, and our ability to obtain sufficient additional capital to fund our operations and to continue to operate as a going concern would be substantially impaired.

Our common stock is currently listed on the Nasdaq Capital Market, which has minimum requirements that a company must meet in order to remain listed. These requirements include maintaining a minimum closing bid price of $1.00 per share, which closing bid price cannot fall below $1.00 per share for a period of more than 30 consecutive trading days, or the Bid Price Requirement. WeOn haveApril previously30, 2025, we received a deficiency noticesnotice from the Listing Qualifications Staff, or the Staff, of Nasdaq,Nasdaq notifying us thatthat, wefor havethe failedlast 30 consecutive business days, the bid price of our common stock had closed below $1.00 per share, thereby failing to satisfy the bid price requirement set forth in the continued listing requirements of Nasdaq Listing Rule 5550(a)(2), or the Minimum Bid Price Requirement. WeOn haveOctober in29, 2025, we received a letter, or the pastExtension beenNotice, ablefrom Nasdaq advising that we were granted a 180-day extension, or until April 27, 2026, to regain compliance with the Minimum Bid Price Requirement,Requirement. howeverIf closingat pricesany time prior to April 27, 2026, the bid price of our common stock oncloses Nasdaq have fluctuated to belowat $1.00 per share or more recentlyfor and,a accordingly,minimum thereof can10 beconsecutive notrading assurance thatdays, we will be able to maintain compliance with the Bid Price Requirement. In the event we fail to comply with listing standards in the future, and we do not regain compliance with the Minimum Bid Price Requirement prior to the expiration of the compliance period, unless Nasdaq exercises its discretion to extend this period, our common stock may be subject to a delisting action by Nasdaq.Requirement.

If we again fail to satisfy the Bid Price Requirement,While a reverse stock split maycould ultimately allow us to meet the Minimum Bid Price Requirement, butwe are at this time unable to implement a reverse stock split within the time required to regain compliance with the Minimum Bid Price Requirement prior to the April 27, 2026 deadline. In addition, we cannot assure you that a reverse stock split willwould be approved by our stockholders or that any reverse stock split, if implemented, willwould be sufficient to enable us to maintain or regain our Nasdaq listing. Additionally, if a reverse stock split is implemented, there can be no assurance that the market price per new share of our common stock following the reverse stock split willwould remain unchanged or willwould increase in proportion to the reduction in the number of shares of our common stock outstanding before the reverse stock split. The liquidity of the shares of our common stock maycould be affected adversely by any reverse stock split given the reduced number of shares of our common stock that will be outstanding following such reverse stock split. Furthermore, following any reverse stock split, the resulting market price of our common stock maymight not attract new investors and may not satisfy the investing requirements of those investors.

Additionally,In ifthe event that our common stock is delisted from Nasdaq as a result of our failure to regain compliance with the Minimum Bid Price Requirement, as a result of the panel not granting us a favorable decision or due to our failure to continue to comply with any other requirement for continued listing on Nasdaq, we expect to seek transfer to an over-the-counter trading market such as the OTCQB Venture Market or an electronic bulletin board established for unlisted securities, but there can be no assurance that our common stock will be approved for trading on such alternative exchange or market. Even if we are approved for trading on an alternative exchange or market, the liquidity of our common stock would be adversely affected, the market price of our common stock could decrease, our ability to obtain sufficient additional capital to fund our operations and to continue to operate as a going concern would be substantially impaired and transactions in our common stock could lose federal preemption of state securities laws. Furthermore, there could also be a further reduction in our coverage by securities analysts and the news media and broker-dealers may be deterred from making a market in or otherwise seeking or generating interest in our common stock, which could cause the price of our common stock to decline further. Moreover, delisting may also negatively affect our collaborators’, vendors’, suppliers’ and employees’ confidence in us and employee morale.

The discussion in “Management’s Discussion and Analysis of Financial Condition and Results of Operations” contains trend analysis, estimates and other forward-looking statements within the meaning of Section 27A of the Securities Act and Section 21E of the Exchange Act. These forward-looking statements include, without limitation, statements containing the words “anticipates,” “believes,” “continues,” “could,” “estimates,” “expects,” “intends,” “may,” “plans,” “potential,” “seeks,” “should,” “will,” and other words of similar import or the negative of those terms or expressions. Such forward-looking statements are subject to known and unknown risks, uncertainties, estimates and other factors that may cause our actual results, performance or achievements, or industry results, to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. Actual results could differ materially from those set forth in such forward-looking statements as a result of, but not limited to, the “Risk Factors” described in Part I, Item 1A of this Annual Report on Form 10-K. You should read the following discussion and analysis along with the Consolidated Financial Statements and accompanying notes attached to those statements included elsewhere in this report.

Based on our current operating plan, which includes our estimates that our cash and cash equivalents as of December 31, 2024, together with $10.1 million generated through our at-the-market offering program in 2025 and the $5.0 million payment expected from Pfizer by the end of March 2025, we expect to meet our liquidity requirements only into the middle of the second quarter of 2025. Our history of significant losses, negative cash flows from operations, negative working capital, limited liquidity resources currently on hand and dependence on our ability to obtain additional financing to fund our operations have resulted in management’s assessment that there is substantial doubt about our ability to continue as a going concern for at least the next 12 months from the date the financial statements included in this Annual Report are issued. Our ability to continue to operate as a going concern is dependent upon our ability to raise substantial additional capital to fund our operations and support our research and development endeavors, including to progress our preclinical and clinical programs as described in this Annual Report. We need substantial additional capital in order to continue to operate as a going concern and fund our operations. We have been actively seeking, and will continue to actively seek, substantial additional capital, including through additional strategic collaborations and other direct investments in our programs, public or private equity or debt financing, royalty financing and other sources. AdditionalThe substantial additional capital needed to support our operations and to continue to operate as a going concern may not be available on acceptable terms or at all. In particular, the perception of our ability to continue to operate as a going concern mayhas made and will continue to make it more difficult to obtain financing for the continuation of our operations, particularly in light of currently challenging macroeconomic and market conditions. Moreover, we currently are not in compliance with the listing standards of Nasdaq, and we do not expect to regain compliance by the April 27, 2026 deadline. If we are unable to regain compliance prior to the April 27, 2026 compliance deadline, our common stock will be delisted from Nasdaq, which would substantially impair our ability to access the capital markets and raise additional funds. See “Risk Factors—We currently do not meet, and do not expect to regain compliance with, the listing standards of the Nasdaq Capital Market, or Nasdaq, prior to the April 27, 2026 compliance deadline. If we do not regain compliance prior to the April 27, 2026 compliance deadline, our common stock will be delisted from Nasdaq. Delisting from Nasdaq could adversely affect the liquidity of our common stock and the market price of our common stock could decrease, and our ability to obtain sufficient additional capital to fund our operations and to continue to operate as a going concern would be substantially impaired.” Further, we have been and may continue to be unable to attract new investments as a result of the speculative nature of our newly reprioritized core neurology preclinical programs and the absence of partners to progress our more advanced clinicalclinical-stage programs. In particular,this ifregard, our ability to fund our current operations and to advance the development of our technologies and product candidates and to extend our cash resources beyond the second quarter of 2026 will remain wholly dependent on our ability to secure collaborations or other transactions for our more advanced clinical-stage programs that provide significant upfront funding in the very near term. If we are not able to execute such collaborations or transactions for these more advanced clinical-stage programs, we will not be able to secure sufficient capital to continue to operate as a going concern and to advance the development of our technologies and product candidates. In particular, despite an extensive, long-term process to secure a commercialization partner for our Fabry disease programprogram, we are currently only in the early stages of discussions with potential counterparties. There can be no assurance that such current or potential future discussions will meaningfully advance at all or ultimately result in transactions that provide us with the substantial capital we need, and if we are unable to execute one or more such transactions for our more advanced clinical-stage programs in the very near term, particularly our abilityFabry disease program, we will be unable to raisesecure the substantial additional capital needed to support our operations willand beto substantiallycontinue impaired.to operate as a going concern. If adequate funds are not available to us onin athe timelyvery basis,near or at all,term, we will be required to take significant additional actions to address our liquidity needs, including substantial additional cost reduction measures such as further reducing operating expenses and further delaying, reducing the scope of, discontinuingaltering or alteringdiscontinuing entirely our research and development activities,activities. whichIn wouldthis regard, we have periodically, including recently, reduced our headcount, and we are actively considering a materialvariety adverseof effectadditional onsignificant cost-cutting measures designed to preserve our cash resources and the value of our assets including, among others, further reductions in our workforce. Moreover, in light of our current financial position, we have deferred many investments in our programs until adequate capital becomes available. Accordingly, we do not expect significant progress with respect to any of our programs unless and until substantial additional funding is obtained. If we are unable to consummate one or more transactions to provide for, or enable, the substantial additional funding needed to operate our business in the very near term, our business and prospects,prospects would be materially and adversely affected, and at any time we may elect to or we may be required to cease operations entirely, liquidate all or a portion of our assets, and/or seek protection under the U.S. Bankruptcy Code,Code in the very near term, and you may lose all or part of your investment. WeSee have explored,“—Liquidity and willCapital continue to explore, whether filing for bankruptcy protection is in the best interest of our Company and our stakeholders.Resources.”

•Since our last update in November 2024,2025, thesix U.S.clinical Foodsites andare Drugnow Administration, or FDA, cleared the investigational new drug application, or IND,active for the ST-503Phase program,1/2 STAND study evaluating ST-503, an investigational epigenetic regulator for the treatment of intractable pain due to idiopathic small fiber neuropathy, or iSFN,SFN, a type of chronic neuropathic pain.

Prion Disease – ST-506

•Clinical trialTrial authorisation,Application, or CTA, enabling activities continuehave to advancecommenced for ourST-506, an investigational epigenetic regulationregulator productfor candidatethe totreatment treatof prion disease, leveraging STAC-BBB, our novel proprietary neurotropic adeno-associated virus, or AAV, capsid variant, known as STAC-BBB, which demonstrated industry-leading blood-brain barrier, or BBB, penetration in nonhuman primates, or NHPs, following a single intravenous administration.capsid.

•We arecontinue executingto develop the Chemistry, Manufacturing and Controls, or CMC, module, ahead of completion of the rolling BLA readiness activitiessubmission for isaralgagene civaparvovec, whilecurrently continuingexpected to engageoccur inas early as the summer of 2026 subject to our ability to secure adequate additional funding, while we continue early stage business development negotiationsdiscussions for a potential Fabry commercialization agreement.

Our revenues have consisted primarily of revenues from collaboration agreements, including upfront license fees, reimbursements for research services, and milestone achievements, and research grant funding. In 2023, our collaboration agreements with Biogen MA, Inc. and Biogen International GmbH, which we refer to together as Biogen, and Novartis Institutes for BioMedical Research, Inc., or Novartis, were terminated. In April 2024, the collaboration agreement with Kite Pharma, Inc., a Gilead Sciences, Inc. subsidiary, or Kite, expired pursuant to its terms, and in December 2024, Pfizer notified us of its termination for convenience of the Pfizer Collaboration Agreement effective April 21, 2025. In 2024,2025, we entered into revenue-generatinglicense partnershipsagreements for STAC-BBB with Genentech Inc., a member of the Roche Group, or Genentech, and Astellas.Astellas InGene 2024,Therapies, Inc., or Astellas, and in April 2025, we entered into revenue-generatinga partnershipslicense agreement for STAC-BBB with GenentechEli Lilly and Astellas.Company, or Lilly. Under these license agreements, we earned upfront license fees and are eligible to earn potential future payments for additional license targets or upon successful achievement of certain development and/or commercial milestones. We expect revenues to continue to fluctuate from period to period and there can be no assurance that our collaborations or partner reimbursements will continue beyond their initial terms or that we are able to meet the milestones specified in these agreements, or that we will be able to secure additional collaborations. For additional information concerning the terms of our ongoing collaboration agreements, see Note 4 – Major Customers, Partnerships and Strategic Alliances in the accompanying notesNotes to the Consolidated Financial Statements included in Part II, Item 8, “Financial Statements and Supplementary Data” of this Annual Report on Form 10-K. We have historically incurred net losses since inception and expect to incur losses for at least the next several years as we continue our research and development activities. To date, we have funded our operations primarily through the issuance of equity securities and revenues from collaborations and research grants.

WeSubject to our ability to secure adequate additional funding to continue to operate as a going concern and progress our programs, we expect research and development expenses to increase in the near-term due to Fabry disease program BLA readiness activities and we expect to continue to devote substantial resources to research and development in the future and expect research and development expenses to increase in the next several years if we are successful in raising substantial additional capital and advancing our product candidates from research stage through clinical trials.

We evaluate the carrying value of long-lived assets, which include property and equipment, leasehold improvements and right-of-use assets, for impairment whenever events or changes in circumstances indicate that the carrying amounts of the asset may not be fully recoverable. If a change in circumstance occurs that indicates long-lived assets may be impaired, we perform a test of recoverability by comparing the carrying value of the asset or asset group to its undiscounted expected future cash flows. The long-lived asset evaluation is performed at the asset group level, i.e., the lowest level for which identifiable cash flows are largely independent of the cash flows of other assets and liabilities. We reassess the composition of our asset groups whenever there are changes in itstheir operations that affect whether the cash flows associated with assets included in asset groups are largely independent. If the impairment review indicates that the carrying amount of an asset group is not recoverable, an impairment loss is measured as the amount by which the carrying amount of an asset group exceeds its fair value. Any impairment loss is allocated to the long-lived assets of the group on a pro rata basis using the relative carrying amounts of those assets, except that the carrying amount of an individual asset shall not be reduced below its fair value.

For a further description of our valuation of long-lived assets, see Note 1 – Organization, Basis of Presentation and Summary of Significant Accounting Policies and Note 5 – Impairment of Goodwill, Indefinite-lived Intangible Assets and Long-lived Assets and Write-Down of Assets Held For Sale in the accompanying notesNotes to the Consolidated Financial Statements included in Part II, Item 8, “Financial Statements and Supplementary Data” of this Annual Report on Form 10-K.

Revenues in 20242025 primarily consisted of revenues from the collaboration agreements with GenentechLilly, Astellas and Astellas,Pfizer, Inc., or Pfizer, and royalties from our license agreements with Sigma-Aldrich Corporation, or Sigma, and Open Monoclonal Technology, Inc. (now Ligand Pharmaceuticals Inc.Incorporated), or Ligand. We anticipate revenues in the future will be derived primarily from our license agreements. The terminations of our collaboration agreements with Biogen and Novartis became effective in June 2023, following which we are not entitled to any further milestone payments or royalties from either Biogen or Novartis, nor does either Biogen or Novartis have any further obligations to develop or to reimburse us the costs of any of the programs previously subject to the Biogen and Novartis collaborations. Further, our collaboration agreement with Kite expired pursuant to its terms in April 2024. Moreover, inIn December 2024, Pfizer notified us of its termination for convenience of the collaboration agreement effective April 21, 2025, and we are not entitled to receive any further milestone payments or royalties from Pfizer.

The decreaseincrease of $122.5$1.1 million in research and development expenses in 20242025 compared to 20232024 was primarily attributable to loweran preclinical,increase of $19.2 million in clinical and manufacturing expenses of $52.6 millionexpenses, primarily relateddue to theBLA terminationreadiness ofactivities collaborationfor agreementsour withFabry Biogendisease andprogram Novartiswhich andwas deferralpartially andoffset reprioritizationby a decrease due to wind-down of certain non-neurology programs, and $3.2 million due to a decrease in reimbursements of certain research and development expenses by a collaboration partner. These increases were partially offset by lower compensation and other personnel costs of $43.1 million due to lower headcount as a result of restructurings of operations and corresponding reductions in workforce announced during 2023 and restructuring charges, lower allocated overhead costs of $14.5$14.6 million due to changes in thevariable pool of allocable costs as a result of restructuring of operations,compensation and lower facilitiesheadcount, andlower facilities, infrastructure related expenses and allocated overhead costs of $12.1$4.5 million, includingand depreciation.lower licensing and patent related expenses of $2.1 million. Stock-based compensation expense included in research and development expenses was $5.7$4.2 million and $15.2$5.7 million for the years ended December 31, 20242025 and 2023,2024, respectively.

The table below shows research and development expenses related to our clinical, preclinical and other research and development programs. As shown in the table below, expenses related to the Fabry clinicaldisease program contributedincreased $19.2by $39.9 million of the decrease in our research and development expenses in 20242025 as compared to 2023,2024, primarily driven by favorableBLA outcomereadiness activities and the commencement of ourthe interactionrolling withBLA submission in December 2025. Chronic neuropathic pain clinical program expenses decreased by $9.1 million in 2025 as compared to 2024, primarily because the FDA,program whichhas resultedadvanced infrom thepreclinical decreased spend on the program. Preclinicalactivities and patient enrollment and recruitment activities have commenced. Expenses related to preclinical and early research programs decreased by $47.3$19.5 million and other research and development programs decreased by $56.0$10.1 million in 2025 as compared to 2024, primarily driven by termination of collaboration agreements with Biogen and Novartis and deferral and reprioritization of certain programs.

We expect research and development expenses to increase in the near-term due to Fabry disease program BLA readiness activities.activities and advancement of our other research and development programs. We expect to continue to devote substantial resources to research and development in the future and expect research and development expenses to increase in the next several years if we are successful in raising substantial additional capital andto advancingadvance our product candidates from research stage throughand clinical trials.pipeline.

General and administrative expenses consist primarily of compensation relatedcompensation-related expenses including restructuring charges and stock-based compensation for executive, legal, finance and administrative personnel, professional fees, allocated facilities and information technology expenses, and other general corporate expenses.

The decrease of $16.4$9.8 million in general and administrative expenses in 20242025 compared to 20232024 was primarily attributable to lower compensation and other personnel costs of $12.1$8.4 million due to lowerchanges headcountin asvariable a result of restructurings of operationscompensation and corresponding reductions in workforce announced during 2023 and restructuring charges, lower facilities and infrastructure related costs of $8.1 million,headcount, lower external professional services expenses of $8.0$2.1 million, and Biogenlower contractfacilities costand assetinfrastructure amortizationrelated expenses of $2.6$1.2 million recorded in 2023 due to the termination of the collaboration agreement.million. These decreases were partially offset by higheran increase of $2.0 million in allocated overhead costs of $14.5 million due to changes in the pool of allocable costs as a result of restructuring of operations.costs. Stock-based compensation expense included in general and administrative expenses was $6.7$4.9 million and $12.2$6.7 million for the years ended December 31, 20242025 and 2023,2024, respectively.

During the year ended December 31, 2025, we recognized impairment charges of $13.2 million on the right-of-use asset and related leasehold improvements at our facility in Brisbane, California. In 2025, based on current and forecasted real estate market conditions, we identified impairment indicators for this asset group and concluded that the carrying value of the asset group was not recoverable. For more information see Note 5 – Impairment of Goodwill, Indefinite-lived Intangible Assets and Long-lived Assets and Write-Down of Assets Held For Sale in the accompanying notesNotes to the Consolidated Financial Statements included in Part II, Item 8, “Financial Statements and Supplementary Data” of this Annual Report on Form 10-K.

InterestOther and other(expense) income, net

Interest and other income, net was $5.9 million and $11.1 million for the years ended December 31, 2024 and 2023, respectively. The decrease of $5.2$7.9 million in 20242025 compared to 20232024 was primarily drivenattributable byto a decrease of $5.2 million in interest income due to a decrease in marketable securities and a decrease of $3.4 million in research tax credits, partially offset by $2.9$5.3 million related to fluctuations in foreign currency exchange ratesrates, and $0.6a decrease of $2.5 million fromin gainresearch onincome saletax of investments.credits.

The income tax benefit was $0.2$0.6 million and $5.1$0.2 million for the years ended December 31, 20242025 and 2023,2024, respectively. The income tax benefitbenefits for the yearyears ended December 31, 2025 and 2024 waswere dueprimarily todriven by the reversal of the Company’s United Kingdom subsidiary’s long-term payable as a result of the statute of limitations lapsing.lapsing Theand foreign income tax benefit for the year ended December 31, 2023 was primarily driven by the reduction of the foreign deferred tax liability due to the impairment of the indefinite-lived intangible asset and was offset by the setup of the valuation allowance for the Company’s United Kingdom subsidiary.taxes.

Beginning in 2022, the 2017 Tax Cuts and Jobs Act amended Section 174 to eliminate current-year deductibility of research and experimentation, or R&E, expenditures and software development costs, collectively, R&E expenditures, and instead require taxpayers to charge their R&E expenditures to a capital account amortized over five years (15 years for expenditures attributable R&E activity performed outside the United States). Following the enactment of the OBBBA, we are no longer capitalizing domestic research and experimental expenditures as of December 31, 2025. We generated a deferred tax asset for capitalized R&E expenditures for the years ended December 31, 20242025 and 2023,2024, which is fully offset with a valuation allowance.

As of December 31, 2024,2025, we had net operating loss carryforwards for federal and state income tax purposes of approximately $818.5$980.4 million and $349.8$470.3 million, respectively. The federal net operating loss generated before 2018 will begin to expire in 20252026 and will keep expiring through 2037, if not utilized. Federal net operating lossesloss generated from 2018 will carry forward indefinitely. If not utilized, the state net operating loss carryforwards will begin to expire in 2029. The Company’sOur French net operating loss carryforward balance is $120.4$129.3 million, which carries over indefinitely. We also have federal and state research tax credit carryforwards of $50.0$53.2 million and $33.2$35.2 million, respectively. The federal research credits will begin to expire in 2025,2026 and will keep expiring through 2045, while the state research credits have no expiration date. Utilization of our net operating loss carryforwards and research tax credit carryforwards may be subject to substantial annual limitations due to the ownership change limitations provided by the Internal Revenue Code and similar state provisions. The annual limitation could result in the expiration of the net operating loss carryforwards and research tax credit carryforwards before use. Due to the carryforwards related to the net operating losses and research and development tax credits, we do not expect to pay any U.S. federal taxes related to income in the near future.

As of December 31, 2024,2025, we had cash and cash equivalents of $41.9$20.9 million compared to cash, cash equivalents and marketable securities of $81.0$41.9 million as of December 31, 2023.2024. Our most significant use of capital during the year was for external research and development expenses, such as manufacturing, clinical trialstrials, regulatory and preclinical activity related to our therapeutic programs, and employee compensation. Cash in excess of immediate requirements is invested in accordance with our investment policy with a view toward capital preservation and liquidity.

We currently do not meet, and may not regain compliance with, the listing standardsTrading of the Nasdaq Capital Market, or Nasdaq, and as a result our common stock on The Nasdaq Capital Market was suspended on May 5, 2026 due to our failure to meet Nasdaq’s minimum bid price requirement, and our common stock now trades on the OTCQB Venture Market. This transition has resulted, and may becontinue delisted.to Delistingresult, in a decrease in the market price of our common stock and could adversely affect the liquidity of our common stock and the market price of our common stock could decrease, and our ability to obtain sufficient additional capital to fund our operations and to continue to operate as a going concern would be substantially impaired.concern.

Additionally,On ifMay 5, 2026, our common stock iscommenced delistedtrading from Nasdaq,on the liquidityOTCQB ofVenture Market, an over-the-counter market operated by OTC Markets Group, under our commonexisting stocksymbol would“SGMO.” beThis adverselytransition affected,has resulted, and may continue to result, in downward pressure on the market price of our common stock and could decrease,adversely affect the liquidity of our common stock. In turn, this may decrease the number of institutional and other investors willing to hold or acquire our common stock and, as a result, our ability to obtainraise sufficient additional capital to fund our operations and to continue to operate as a going concern wouldmay be substantially impairedimpaired. and transactions in our common stock could lose federal preemption of state securities laws. Furthermore,Moreover, there could also be a further reduction in our coverage by securities analysts and the news mediamedia, and broker-dealers may be deterred from making a market in or otherwise seeking to execute trades in or generatinggenerate interest in our common stock, which could cause the price of our common stock to decline further. Moreover,In addition, in the event our common stock is ultimately delisted from Nasdaq, we will be subject to additional regulation in the states in which we offer our securities. Furthermore, delisting may also negatively affect our collaborators’, vendors’,vendors’ and suppliers’ and employees’ confidence in us and could have a detrimental effect on employee morale.

The discussion in “Management’s Discussion and Analysis of Financial Condition and Results of Operations” contains trend analysis, estimates and other forward-looking statements within the meaning of Section 27A of the Securities Act and Section 21E of the Exchange Act. These forward-looking statements include, without limitation, statements containing the words “anticipates,” “believes,” “continues,” “could,” “estimates,” “expects,” “intends,” “may,” “plans,” “seeks,” “should,” “will,” and other words of similar import or the negative of those terms or expressions. Such forward-looking statements are subject to known and unknown risks, uncertainties, estimates and other factors that may cause our actual results, performance or achievements, or industry results, to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. Actual results could differ materially from those set forth in such forward-looking statements as a result of, but not limited to, the “Risk Factors” described in Part I, Item 1A our Annual Report on Form 10-K for the year ended December 31, 20242025 as filed with the Securities and Exchange Commission on March 17,30, 2025,2026, or the 20242025 Annual Report, as supplemented by the risks described under “Risk Factors” in Part II, Item 1A of this Quarterly Report on Form 10-Q. All forward-looking statements about our future plans and expectations are subject to our ability to secure adequate additional funding. You should also read the following discussion and analysis in conjunction with our Condensed Consolidated Financial Statements and accompanying notes included in this Quarterly Report and the Consolidated Financial Statements and accompanying notes thereto included in our 20242025 Annual Report. See “Special Note Regarding Forward-Looking Statements.”

Based on our current operating plan, we estimate that our cash and cash equivalents as of September 30, 2025, together with the $6.0 million license fee we received from Pfizer in October 2025, and $9.1 million generated through our at-the-market offering program since September 30, 2025, will be sufficient to meet our liquidity requirements only into the first quarter of 2026. Our history of significant losses, negative cash flows from operations, negative working capital, limited liquidity resources currently on hand and dependence on our ability to obtain substantial additional financing to fund our operations have resulted in management’s assessment that there is substantial doubt about our ability to continue as a going concern for at least the next 12 months from the date the financial statements included in this Quarterly Report are issued. Our ability to continue to operate as a going concern is dependent upon our ability to raise substantial additional capital to fund our operations and support our research and development endeavors, including to progress our preclinical and clinical programs as described in our 2024 Annual Report and in this Quarterly Report. We need substantial additional capital in order to continue to operate as a going concern and fund our operations. We have been actively seeking, and will continue to actively seek, substantial additional capital, including through additional strategic collaborations and other direct investments in our programs, public or private equity or debt financing, and other sources. The substantial additional capital needed to support our operations and to continue to operate as a going concern may not be available on acceptable terms or at all. In particular, the perception of our ability to continue to operate as a going concern has made and will continue to make it more difficult to obtain financing for the continuation of our operations, particularly in light of currently challenging macroeconomic and market conditions. WeMoreover, the trading of our common stock on The Nasdaq Capital Market has been suspended, and our common stock now trades on the OTCQB Venture Market. This transition has resulted, and may continue to result, in downward pressure on the market price of our common stock, and could adversely affect the liquidity of our common stock and ultimately our ability to raise sufficient additional capital to fund our operations and to continue to operate as a going concern. See “Risk Factors—Trading of our common stock on The Nasdaq Capital Market was suspended on May 5, 2026 due to our failure to meet Nasdaq’s minimum bid price requirement, and our common stock now trades on the OTCQB Venture Market. This transition has resulted, and may continue to result, in a decrease in the market price of our common stock and could adversely affect the liquidity of our common stock and our ability to obtain sufficient additional capital to fund our operations and to continue to operate as a going concern.” Further, we have been and may continue to be unable to attract new investments as a result of the speculative nature of our newly reprioritized core neurology preclinical programs and the absence of partners to progress our more advanced clinicalclinical-stage programs. In particular,this regard, our ability to fund our current operations and to advance the development of our technologies and product candidates and to extend our cash resources beyond the third quarter of 2026 will remain wholly dependent on our ability to secure collaborations or other transactions for our more advanced clinical-stage programs that provide significant upfront funding in the very near term. If we are engagednot able to execute such collaborations or transactions for these more advanced clinical-stage programs in businessthe developmentvery discussionsnear with potential counterparties concerning a commercialization agreement for our Fabry disease program, but have been unsuccessful in consummating any such transaction to date. There can be no assurance thatterm, we will not be able to secure sufficient capital to continue to operate as a going concern and to advance the development of our technologies and product candidates. In particular, despite an extensive, long-term process to secure a commercialization partner for our Fabry disease programprogram, we are currently only in athe timelyearly manner,stages onof acceptablediscussions terms,with potential counterparties. There can be no assurance that such current or potential future discussions will meaningfully advance at all,all or ultimately result in transactions that provide us with the substantial capital we need, and if thewe Company isare unable to execute one or more such antransactions agreementfor our more advanced clinical-stage programs in the very near term, particularly our abilityFabry disease program, we will be unable to raisesecure the substantial additional capital needed to support our operations willand beto substantiallycontinue impaired.to operate as a going concern. If adequate funds are not available to us onin athe timelyvery basis,near or at all,term, we will be required to take significant additional actions to address our liquidity needs, including substantial additional cost reduction measures such as further reducing operating expenses and further delaying, reducing the scope of, altering or discontinuing entirely our research and development activities,activities. whichIn wouldthis regard, we have periodically, including recently, reduced our headcount, and we are actively considering a materialvariety adverseof effectadditional onsignificant cost-cutting measures designed to preserve our cash resources and the value of our assets including, among others, further reductions in our workforce. Moreover, in light of our current financial position, we have deferred many investments in our programs until adequate capital becomes available. Accordingly, we do not expect significant progress with respect to any of our programs unless and until substantial additional funding is obtained. If we are unable to consummate one or more transactions to provide for, or enable, the substantial additional funding needed to operate our business in the very near term, our business and prospects,prospects would be materially and adversely affected, and at any time we may elect to or we may be required to cease operations entirely, liquidate all or a portion of our assets, and/or seek protection under the U.S. Bankruptcy Code,Code in the very near term, and you may lose all or part of your investment. WeSee have explored,“—Liquidity and willCapital continue to explore, whether filing for bankruptcy protection is in the best interest of our Company and our stakeholders.Resources.”

Our neurology development is focused on two innovative areas: (i) development of epigenetic regulation therapies to treat serious neurological diseases and (ii) development ofusing novel engineered adeno-associated virus, or AAV, capsids to deliver our therapies to the intended neurological targets. Initial indications for our wholly owned preclinical programs include small fiber neuropathy, or SFN, a type of chronic neuropathic pain, and prion disease. As we estimate that our cash and cash equivalents as of SeptemberMarch 30,31, 2025,2026, together withincluding the $6.0implementation millionof licensepotential feeadditional wecost receivedreduction from Pfizer in October 2025, and $9.1 million generated through our at-the-market offering program since September 30, 2025,measures, will be sufficient to meet our liquidity requirements only into the firstthird quarter of 2026, our plans and expectations discussed below are subject to our ability to secure adequate additional funding, which we may be unable to do in a timely manner or at all.

•Patient enrollment and recruitment have now commenced following the activation of the first twoSix clinical sites are activated for the Phase 1/2 STAND study evaluating ST-503, an investigational epigenetic regulator for the treatment of intractable pain due to SFNSFN, a type of chronic neuropathic pain.

•Clinical Trial Application, or CTA, enabling activities continuehave to advancecommenced for ST-506, an investigational epigenetic regulator for the treatment of prion disease, leveraging STAC-BBB, our novel proprietary neurotropic AAV capsid.

•WeThis hadquarter, we held a productive interaction with the U.K.’s Medicines and Healthcare products Regulatory Agency, or MHRA, including alignment on thediagnostic Chemistry,testing, Manufacturinganalytical validation and Controls,nonclinical orsafety CMC, strategy.matters.

•We arecontinue preparingto fordevelop anthe anticipatedChemistry, Manufacturing and Controls, or CMC, module, ahead of completion of the rolling BLA submission for isaralgagene civaparvovec, currently expected to occur as early as the first quartersummer of 2026,2026 subject to our ability to secure adequate additional funding, while continuingwe continue business development discussions for a potential Fabry commercialization agreement.

Our business and operations and those of our collaborators may be affected by financial instability and declining economic conditions in the United States and other countries, whether caused by political instability and conflict, including the ongoing conflicts in the Middle East, conflict between Russia and Ukraine, the current and potential future government shutdowns, by general health crises, or by global trade issues and changes inin, and ongoing uncertainties with respect toto, tariffs and international trade disputes,disputes. whichThe macroeconomic environment has led to market disruptions, including significant volatility in commodity prices, credit and capital markets instability, including disruptions in access to bank deposits and lending commitments, supply chain interruptions, fluctuations in interest rates, the imposition of tariffs and global inflationary pressures. These macroeconomic factors could materially and adversely affect our ability to continue to operate as a going concern and could otherwise have a material adverse effect on our business, operations, operating results and financial condition as well as the price of our common stock. In particular, our ability to raise the substantial additional capital we need in order to fund our business and to continue to operate as a going concern may be adversely impacted by these macroeconomic factors, and we cannot be certain that we will be able to obtain the substantial additional capital that we need to support our operations and to continue to operate as a going concern on terms acceptable to us, or at all.

Our revenues have consisted primarily of revenues from collaboration agreements, including upfront license fees, reimbursements for research services, and milestone achievements, and research grant funding. In April 2024, the collaboration agreement with Kite Pharma, Inc., a Gilead Sciences, Inc. subsidiary, or Kite, expired pursuant to its terms, and in December 2024, Pfizer notified us of its termination for convenience of the global collaboration and license agreement effective April 21, 2025. In 2024, we entered into license agreements for our STAC-BBB capsid with Genentech Inc., a member of the Roche Group, or Genentech, and Astellas Gene Therapies, Inc., or Astellas, and in April 2025, we entered into a license agreement for our STAC-BBB capsid with Eli Lilly and Company, or Lilly. We are also party to a license agreement with Miltenyi Biotec B.V. & Co. KG, or Miltenyi, pursuant to which we granted Miltenyi certain rights to use our proprietary technology and related know-how in connection with the research, development, manufacturing and commercialization activities in the cell therapy field. Under these license agreements, we earned upfront license fees and royalties and are eligible to earn potential future payments for additional license targets or upon successful achievement of certain development and/or commercial milestones. We expect revenues to continue to fluctuate from period to period and there can be no assurance that our collaborations or partner reimbursements will continue beyond their initial terms or that we are able to meet the milestones specified in these agreements, or that we will be able to secure additional collaborations. For additional information concerning the terms of our ongoing collaboration agreements, see Note 5 – Major Customers, Partnerships and Strategic Alliances in the accompanying notes to the Condensed Consolidated Financial Statements included in Part I, Item 1 of this Quarterly Report on Form 10-Q.

WeSubject to our ability to secure adequate additional funding to continue to operate as a going concern and progress our programs, we expect research and development expenses to increase in the near-term due to Fabry disease program BLA readiness activities,activities and we expect to continue to devote substantial resources to research and development in the future and expect research and development expenses to increase in the next several years if we are successful in raising substantial additional capital toand advanceadvancing our product candidates from research andstage through clinical pipeline.trials.

General and administrative expenses consist primarily of salaries and personnel related expenses for executive, finance and administrative personnel, stock-based compensation expense, professional fees, allocated facilities and information technology expenses, patent prosecution expenses and other general corporate expenses. Although we expect general and administrative expenses to remain relatively consistent in the near term, we expect the growth of our business to require increased general and administrative expenses if we are successful in raising substantial additional capital toand advanceadvancing our product candidates from research andstage through clinical pipeline.trials.

There have been no significant changes in our critical accounting policies and estimates during the three and nine months ended SeptemberMarch 30,31, 2025,2026, as compared to the critical accounting policies and estimates disclosed in “Management’s Discussion and Analysis of Financial Condition and Results of Operations” included in Part II, Item 7 of the 20242025 Annual Report.

Results of Operations for the Three and Nine Months Ended SeptemberMarch 30,31, 20252026 and 20242025

Revenues in 20252026 primarily consisted of revenues from the collaborationresearch agreementsagreement with LillyLilly, andour Pfizer,license agreement with Miltenyi, and royalties from our license agreements with Sigma-Aldrich Corporation, or Sigma, and Open Monoclonal Technology, Inc. (now Ligand Pharmaceuticals Inc.), or Ligand. We anticipate revenues in the future will be derived primarily from our license agreements. In December 2024, Pfizer notified us of its termination for convenience of the collaboration agreement effective April 21, 2025, and we are not entitled to receive any further milestone payments or royalties from Pfizer.

The decrease of $24.9$5.0 million in revenues for the ninethree months ended SeptemberMarch 30,31, 2025,2026, compared to the same period in 2024,2025, was primarily attributable to a decrease of $49.2 million in revenue relating to our collaboration agreement with Genentech. This decrease was offset by $18.0 million in revenue relating to our capsid license agreement with Lilly, $5.0 million in revenue relating to our collaboration agreement with Pfizer upon transfer of a specified sublicense in 2025, and ana increasedecrease of $1.1$0.8 million in revenue relating to our license agreement with Sigma. These decreases were partially offset by $0.5 million in revenue relating to our license agreement with Miltenyi.

Research and development expenses consisted primarily of compensation related expenses, including restructuring charges and stock-based compensation, laboratory supplies, preclinical and clinical studies, manufacturing clinical supply, contracted research and development, and allocated facilities and information technology expenses.

The increase of $0.4$0.6 million in research and development expenses for the three months ended SeptemberMarch 30,31, 2025,2026, compared to the same period in 2024,2025, was primarily attributable to an increase of $8.3$3.7 million in clinical and manufacturing expensesexpenses, primarily due to BLA readiness activities for our Fabry disease program.program, which was partially offset by a decrease due to deferral of certain program expenses. These increases were partially offset by lower compensation and other personnel costs of $5.9$1.5 million due to changes in variable compensation and lower headcount, and lower licensingfacilities, and patentinfrastructure related expenses and allocated overhead costs of $1.7$1.6 million. Stock-based compensation expense included in research and development expenses was $0.9$0.6 million and $1.5$1.2 million for the three months ended SeptemberMarch 30,31, 20252026 and 2024,2025, respectively.

We expect research and development expenses to increase in the near-term due to Fabry disease program BLA readiness activities and advancement of our other research and development programs.programs, subject to our ability to secure adequate additional funding to continue to operate as a going concern. We expect to continue to devote substantial resources to research and development in the future and expect research and development expenses to increase in the next several years if we are successful in raising substantial additional capital to advance our research and clinical pipeline.

The length of time required to complete our development programs and our development costs for those programs may be impacted by the results of preclinical testing, scope and timing of enrollment in clinical trials for our product candidates, our decisions to pursue development programs in other therapeutic areas, whether we pursue development of our product candidates with a partner or collaborator or independently and our ability to secure the necessary funding to progress the development of our programs. In addition, we are actively seeking commercialization and collaboration partners or a direct external investment, as applicable, to progress our Fabry disease and hemophilia A programs, STAC-BBBand capsid and modular integrase platforms. Furthermore, the scope and number of clinical trials required to obtain regulatory approval for each pursued therapeutic area is subject to the input of the applicable regulatory authorities, and we have not yet sought such input for all potential therapeutic areas that we may elect to pursue, and even after having given such input, applicable regulatory authorities may subsequently require additional clinical studies prior to granting regulatory approval based on new data generated by us or other companies, or for other reasons outside of our control. As a condition to any regulatory approval, we may also be subject to post-marketing development commitments, including additional clinical trial requirements. As a result of the uncertainties discussed above, we are unable to determine the duration of or complete costs associated with our development programs.

General and administrative expenses consist primarily of compensation related expensesexpenses, including restructuring charges and stock-based compensation for executive, legal, finance and administrative personnel, professional fees, allocated facilities and information technology expenses, and other general corporate expenses.

The decrease of $7.7$3.2 million in general and administrative expenses for the ninethree months ended SeptemberMarch 30,31, 2025,2026, compared to the same period in 2024,2025, was primarily attributable to lower external professional services expenses of $1.6 million, and lower compensation and other personnel costs of $6.2$1.4 million due to changes in variable compensation and lower headcount, and lower external professional services expenses of $3.8 million. These decreases were partially offset by an adjustment of $2.2 million to expense relating to settlement of obligations under a manufacturing agreement in 2024.headcount. Stock-based compensation expense included in general and administrative expenses was $3.7$0.4 million and $4.9$1.4 million for the ninethree months ended SeptemberMarch 30,31, 20252026 and 2024,2025, respectively.