Sangamo Therapeutics, Inc.
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ITEM 1 – BUSINESS
OVERVIEW
We are a genomic medicine company committed to translating ground-breaking science into medicines that transform the lives of patients and families afflicted with serious neurological diseases. We believe our zinc finger epigenetic regulators are ideally suited to potentially address devastating neurology disorders and our capsid engineering platform has demonstrated the ability to expand delivery beyond currently available intrathecal delivery capsids, including in the central nervous system, or CNS, in preclinical studies.
Our Core Neurology Programs
Our neurology development is focused on two innovative areas: (i) development of epigenetic regulation therapies to treat serious neurological diseases and (ii) development of 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. Following clearance in November 2024 of the investigational new drug application, or IND, from the U.S. Food and Drug Administration, or FDA, for ST-503 for the treatment of SFN, the first six Phase 1/2 clinical sites have been activated, and we have begun patient recruitment and enrollment. We have also begun to prepare a clinical trial application, or CTA, for our product candidate to treat prion disease.
In addition, we are party to collaboration and license arrangements pursuant to which our collaborators are developing preclinical product candidates in indications such as tauopathies, amyotrophic lateral sclerosis, or ALS, and other undisclosed neurology targets. These partners include Genentech, Inc., a member of the Roche Group, or Genentech, Astellas Gene Therapies, Inc., or Astellas, Alexion Pharmaceuticals, Inc., or Alexion, Eli Lilly and Company, or Lilly, and Takeda Pharmaceutical Company Limited, or Takeda.
We also are developing novel engineered AAV capsids enhanced for delivery to neurological targets and have identified a proprietary engineered neurotropic AAV capsid variant, STAC-BBB, that has demonstrated an ability to cross the blood-brain barrier, or BBB, in nonhuman primates, or NHPs, and in mice, and mediated robust transduction, transgene expression, and targeted epigenetic repression throughout the brain and spinal cord after intravenous, or IV, administration. We believe this novel capsid has the potential to unlock multiple wholly owned neurology epigenetic regulation programs, and is already the subject of multiple license agreements, including with Genentech, Astellas and Lilly.
Other Clinical Programs
Other clinical-stage product candidates include:
•Isaralgagene civaparvovec, also known as ST-920, our wholly owned gene therapy product candidate for the treatment of Fabry disease, was evaluated in our completed registrational Phase 1/2 STAAR clinical study and continues to be evaluated in the subsequent long term follow up study. We believe this product candidate has a clear regulatory pathway to Accelerated Approval from the FDA. Rolling submission of the Biologics License Application, or BLA, for isaralgagene civaparvovec was initiated in December 2025.
•Giroctocogene fitelparvovec, also known as SB-525, is a gene therapy product candidate for the treatment of moderately severe to severe hemophilia A, that we co-developed with, and licensed to Pfizer Inc., or Pfizer, and to which we regained development and commercialization rights in April 2025. We have completed the transition of the program back to Sangamo, and we currently are in business development negotiations with a potential collaboration partner for giroctocogene fitelparvovec.
Our Novel Science and Technologies
We are a leader in the research and development of zinc finger proteins, or ZFPs, which are abundantly occurring human proteins that have evolved to regulate the genome through interactions with DNA and regulatory proteins. Our strategy is to translate our differentiated and versatile zinc finger, or ZF, technology platform to create product candidates with best- or first-in-class clinical potential. We believe that the versatility and flexibility of our technology platforms enable us to design therapeutic approaches to resolve the underlying genetic or cellular causes of disease, using whichever technology is best suited to deliver that treatment. Our current area of focus is developing epigenetic regulation therapies with our ZF technology platform for serious neurological diseases.
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We are also evaluating several potential routes of administration for our neurology-targeted investigational therapies, as delivery of genomic medicines to the CNS is a significant obstacle to developing therapies treating neurological disorders. We have developed a proprietary AAV capsid engineering platform, Selecting In vivo For Transduction and Expression of RNA, or SIFTER, with the aim of engineering capsids with improved CNS transduction and have presented results from capsids for both IV and cerebrospinal fluid, or CSF, administration.
We are also developing next generation modular integrase technology, engineered to enable large-scale genome editing. Building on our deep expertise in protein-DNA interactions derived from our zinc finger platform, the Modular Integrase, or MINT, platform is a versatile, protein-guided genome editing method designed to integrate large sequences of DNA into the genome to potentially treat – with a single medicine – many different patients who have unique mutations in the same gene.
In the process of developing these technologies, we have additionally accrued significant scientific and development capabilities, as well as manufacturing know-how, that are broadly applicable to the field of gene therapy, which we have used to develop our genomic medicine product candidates.
Manufacturing
We expect to be substantially reliant on external partners to manufacture clinical supply for our neurology portfolio. We retain our in-house analytical and process development capabilities.
Collaborations and Licenses
Our collaborations with biopharmaceutical companies bring us important financial and strategic benefits and reinforce the potential of our research and development efforts, including our proprietary ZF technology and AAV capsid platforms. They leverage our collaborators’ therapeutic and clinical expertise and commercial resources with the goal of bringing our medicines more rapidly to patients. We believe these collaborations reflect the value of our technology and will potentially expand the addressable markets of our product candidates. To date, we have received approximately $911.0 million in upfront license fees, milestone payments and proceeds from sale of our common stock to collaborators and have the opportunity to earn up to $4.8 billion in potential future milestone payments and additional licensed target fees from our ongoing collaborations and licenses, in addition to potential product royalties.
Product Pipeline
Figure 1: Our pipeline, subject to adequate additional funding
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Core Neurology Pipeline
Chronic Neuropathic Pain – ST-503
We are developing ST-503, an investigational epigenetic regulator for the treatment of intractable pain due to SFN, a type of chronic neuropathic pain.
Neuropathic pain can be caused by a broad array of pathologies impacting the central or peripheral nervous systems, such as surgical trauma, spinal cord injury, nerve compression, neurological and infectious diseases, or metabolic and hereditary syndromes. ST-503 is not intended for sporadic or acute pain, but for chronic, intractable pain that completely dominates and often destroys the lives of patients over many years.
Following FDA clearance of the IND for SFN in November 2024, six clinical sites have been activated for the Phase 1/2 STAND study and patient recruitment and enrollment have commenced. In December 2025, the FDA granted Fast Track Designation to ST-503. Fast Track Designation aims to facilitate the development and expedite the review of new therapeutics that are intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs.
Figure 2: Our approach in SFN using ZFRs
The Phase 1/2 study is designed to assess the safety and efficacy of ST-503 in addressing SFN, a peripheral neuropathy that results in highly debilitating symptoms of burning, prickling, stabbing or “lightning-like” pain. SFN has an estimated prevalence of at least 180,000 patients in the U.S., and more broadly, peripheral neuropathies are estimated to affect nearly 40 million Americans. Antidepressants, anticonvulsants, opioids and topical therapies are potential treatment options, although no long-lasting or curative therapies are currently available for SFN patients, leading to a high unmet medical need for this patient population.
Subject to our ability to secure adequate additional funding, we intend to conduct a double-blind, randomized, sham-controlled dose escalation study to determine safety and tolerability of a single intrathecal dose of the ST-503 gene therapy for refractory pain due to small fiber neuropathy. The Phase 1/2 STAND study is designed to follow a dose escalation protocol with a 2:1 randomization of investigational product to sham, and consists of three ascending dose cohorts. The primary objectives of the Phase 1/2 study will be to assess the safety and tolerability of ST-503, with secondary objectives to assess
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preliminary efficacy based on the impact of ST-503 on refractory pain, and assessment of the multidimensional impact of ST-503 on sleep, mental health and quality of life.
A significant body of evidence implicates sodium channels in mediating the pathophysiology of neuropathic pain. ST-503 uses an AAV vector carrying an engineered zinc finger repressor, or ZFR, to specifically target the human gene, SCN9A, that encodes the Nav1.7 sodium channel and is critical for pain signaling. Developing small molecules that specifically target Nav1.7 is challenging due to the high structural similarities between different sodium channels, making it difficult to achieve selectivity and avoid off-target effects. By directly targeting the SCN9A gene, ST-503 was shown to selectively reduce the expression of Nav1.7 sodium channels in sensory neurons in animal models and significantly reduce pain hypersensitivity, following a single intrathecal administration of ST-503. Sangamo’s preclinical research has shown ST-503 to be well tolerated in NHPs, with substantial Nav1.7 reduction observed with no off-target effects, demonstrating the promise of ST-503 as a potential therapy for chronic neuropathic pain, regardless of cause.
Prion Disease
We are also developing our other lead wholly owned preclinical epigenetic regulation program in prion disease, a fatal and incurable neurodegenerative disease caused by the misfolding of the prion protein encoded by the gene PRNP.
This misfolding of the prion protein is acutely toxic to neurons, and our aim is to remove a portion of prion protein from neurons to protect them from the toxicity of the misfolded prion protein. We think that this may prevent the spread and propagation of misfolded prion, and may therefore slow or halt neurodegeneration and disease progression.
At least 1,500 new cases of prion disease are identified each year in the United States, Europe and Japan, with a similar presence globally.
CTA-enabling activities have commenced for ST-506, an investigational epigenetic regulator for the treatment of prion disease, leveraging STAC-BBB, our novel proprietary neurotropic AAV capsid. In 2025, we held productive interactions with the U.K. Medicines and Healthcare products Regulatory Agency, or MHRA, including alignment on nonclinical safety studies, the clinical study design and the Chemistry, Manufacturing and Controls, or CMC, strategy. The Good Laboratory Practice, or GLP, Toxicology study has been completed and analysis is in progress.
Figure 3: Our approach in prion disease using ZFRs
To address prion disease, we are developing ZFRs that target the PRNP gene, to be delivered by STAC-BBB, our IV-administered neurotropic AAV capsid. We presented updated preclinical data from this program in the prestigious Presidential Symposium at the 28th American Society of Gene & Cell Therapy, or ASGCT, Annual meeting, in May 2025 and at the Prion 2025 Conference in November 2025. The presented data demonstrated the profound survival extension observed in disease mouse models and the sustained widespread brain delivery and prion reduction in NHPs and mice treated with ST-506.
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The clinical study is expected to be a Bayesian optimal interval design, to assess safety and efficacy, while potentially enabling rapid escalation to the maximum tolerated dose. The clinical study plans to use the Medical Research Council prion disease rating scale to assess the efficacy of the ZFR and compare to matched historic controls. The aim of the planned clinical study is to delay the progression of prion disease, offering potential for a meaningful extension of survival in prion patients.
Clinical Programs
Isaralgagene civaparvovec – Fabry Disease
Isaralgagene civaparvovec, or ST-920, is our wholly owned gene therapy product candidate for the treatment of Fabry disease, a rare inherited metabolic disease. Isaralgagene civaparvovec was evaluated in our completed, registrational Phase 1/2 STAAR clinical study, and continues to be evaluated in the subsequent long term follow up study. We believe this product candidate has a clear regulatory pathway to Accelerated Approval from the FDA.
STAAR was a Phase 1/2 multicenter, open-label, dose-ranging clinical study designed to assess a single infusion of isaralgagene civaparvovec in symptomatic Fabry disease patients ≥ 18 years of age. Patients were infused intravenously with a single dose and followed for 52 weeks. A separate long-term follow-up study is underway to monitor the patients treated in this study for up to five years following treatment to further assess safety, durability and efficacy. Patients who were on stable Enzyme Replacement Therapy, or ERT, could withdraw ERT after treatment in a controlled and monitored fashion at the discretion of the patient and the investigator.
The dose escalation phase included males with classic Fabry disease. The study was subsequently expanded to enroll both males and females, including patients with Fabry-associated cardiac or renal disease. The study’s primary endpoint was the incidence of treatment-emergent adverse events, or AEs. Secondary endpoints included change from baseline at specific time points over the one-year study period in alpha‑galactosidase A, or α‑Gal A, activity, globotriaosylceramide, or Gb3, and lyso‑Gb3 levels in plasma; frequency of ERT infusion; changes in renal function and cardiac function (left ventricular mass) measured by cardiac magnetic resonance imaging, or MRI, and rAAV2/6 vector clearance. Key exploratory endpoints included change from baseline in disease severity (Fabry Outcome Survey adaptation of the Mainz Severity Score Index, or FOS-MSSI, score), quality of life, or QoL, gastrointestinal, or GI, symptoms and neuropathic pain scores; and immune response to AAV6 capsid and α‑Gal A.
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Figure 4: Our approach in Fabry disease
The goal of the study was to abrogate the need for ERT with a recombinant AAV2/6 vector encoding cDNA for human α‑Gal A, resulting in long-term expression of α‑Gal A. As a liver-directed gene therapy, isaralgagene civaparvovec is designed to be delivered by a one-time IV infusion that does not require any preconditioning regimen for patients.
The FDA has granted Orphan Drug, Fast Track and regenerative medicine advanced therapy, or RMAT, designations to isaralgagene civaparvovec, which has also received Orphan Medicinal Product designation and PRIME eligibility from the European Medicines Agency, or EMA, and Innovative Licensing and Access Pathway, or ILAP, from the MHRA.
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Financial statements
data from SEC XBRL filings. Values are as-reported; restatements supersede originals. Values reported in .
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ITEM 7 – MANAGEMENT’S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS
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 included elsewhere in this report.
In addition, the “Results of Operations” section of this “Management’s Discussion and Analysis of Financial Condition and Results of Operations” generally discusses 2025 and 2024 items and year-to-year comparisons between 2025 and 2024.
Overview
We are a genomic medicine company committed to translating ground-breaking science into medicines that transform the lives of patients and families afflicted with serious neurological diseases. We believe our zinc finger epigenetic regulators are ideally suited to potentially address devastating neurology disorders and our capsid engineering platform has demonstrated the ability to expand delivery beyond currently available intrathecal delivery capsids, including in the central nervous system, or CNS, in preclinical studies. For additional information regarding our business, see “Business” in Part I, Item 1 of this Annual Report on Form 10‑K.
Corporate Updates
Underwritten Offering
In February 2026, we issued and sold in an underwritten offering an aggregate of 35.2 million shares of our common stock and pre-funded warrants to purchase up to an aggregate of 17.8 million shares of common stock, together with accompanying warrants to purchase up to an aggregate of 53.0 million shares of common stock, or the February 2026 Offering.
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The net proceeds from the February 2026 Offering were approximately $23.1 million, after deducting underwriting discounts and other offering costs. We are using the proceeds from this offering for working capital and general corporate purposes.
Financial Position – Going Concern
Based on our current operating plan, we estimate that our cash and cash equivalents as of December 31, 2025, together with net proceeds of approximately $23.1 million from the February 2026 Offering, a $4.6 million research tax credit received from the French government in February 2026, and $3.7 million generated through our at-the-market offering program since December 31, 2025, will be sufficient to meet our liquidity requirements only into the third quarter of 2026. This estimate regarding our cash resources is based on assumptions that are inherently uncertain, and actual results could differ materially from those estimates. In this regard, we could use our available capital resources sooner than we currently expect and changing circumstances, some of which may be beyond our control, could cause us to consume capital significantly faster than we currently anticipate. Although we believe our cash and cash equivalents could fund our planned operations into the third quarter of 2026, unless we secure substantial upfront funding through a significant partnership or other transaction for our programs in the very near term, we expect that we will need to significantly scale back our operations and focus substantially all of our efforts on pursuing strategic alternatives to maximize the value of our assets for our stockholders and creditors. In particular, at any time we may determine that it is in the best interest of our stockholders and creditors 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 Sangamo and our stakeholders and the most advantageous time for such filing in order to preserve sufficient resources to undertake an appropriate bankruptcy process.
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, 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. 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 clinical-stage programs. In 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 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 program, 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 Fabry disease program, we will be unable to secure the substantial additional capital needed to support our operations and to continue to operate as a going concern. If adequate funds are not available to us in the very near 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. In this regard, we have periodically, including recently, reduced our headcount, and we are actively considering a
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variety of additional significant 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 would be materially and adversely affected, and 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 in the very near term, and you may lose all or part of your investment. See “—Liquidity and Capital Resources.”
Core Preclinical Neurology Programs and Technology
Chronic Neuropathic Pain – ST-503
•Since our last update in November 2025, six clinical sites are now active for the Phase 1/2 STAND study evaluating ST-503, an investigational epigenetic regulator for the treatment of intractable pain due to small fiber neuropathy, or SFN, a type of chronic neuropathic pain.
•In December 2025, the FDA granted Fast Track Designation to ST-503. Fast Track Designation aims to facilitate the development and expedite the review of new therapeutics that are intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs. Companies granted this designation are given the opportunity for more frequent interactions with the FDA. These clinical programs may also be eligible to apply for Accelerated Approval and Priority Review if relevant criteria are met.
•In March 2026, a manuscript was published in Science Translational Medicine detailing the preclinical safety and pharmacology of ST-503 in human neurons, mice and nonhuman primates.
Prion Disease – ST-506
•Clinical Trial Application, or CTA, enabling activities have commenced for ST-506, an investigational epigenetic regulator for the treatment of prion disease, leveraging STAC-BBB, our novel proprietary neurotropic adeno-associated virus, or AAV, capsid.
•Since the last update in November 2025, the Good Laboratory Practice, or GLP, toxicology study has been completed and analysis is ongoing.
Clinical Programs
Fabry Disease
•On February 3, 2026, we announced the presentation of clinical data from our registrational Phase 1/2 STAAR study evaluating isaralgagene civaparvovec, or ST-920, a wholly owned gene therapy product candidate for the treatment of Fabry disease, in four clinical and nonclinical platform and poster presentations at the 22nd Annual WORLDSymposiumTM that took place in San Diego, CA, February 2-6, 2026. The data showed that as of the April 10, 2025 data cutoff date, the totality of data demonstrates the potential of isaralgagene civaparvovec as a one-time, well-tolerated and durable gene therapy treatment option for Fabry disease to provide meaningful, multi-organ clinical benefits that could fundamentally shift the Fabry treatment paradigm. A positive mean annualized estimated glomerular filtration rate, or eGFR, slope of 1.965 mL/min/1.73m2/year (95% confidence interval, or CI: -0.153, 4.083) at 52-weeks was observed across all 32 dosed patients, indicating an improvement in renal function. Furthermore, a mean annualized eGFR slope of 1.747 mL/min/1.73m2/year (95% CI: -0.106, 3.601) was observed for the 19 patients who had achieved 104-weeks of follow-up. Stable cardiac function was observed over one year, including consistent cardiac structural stability across clinical and demographic subgroups. Durability of effect was demonstrated with elevated expression of alpha-galactosidase A, or α-Gal A, activity maintained for up to 4.5 years for the longest treated patient, alongside statistically significant Quality of Life improvements and other clinical benefits. Isaralgagene civaparvovec demonstrated a favorable safety and tolerability profile in the study, without the requirement for preconditioning.
•The Phase 1/2 STAAR study of isaralgagene civaparvovec is complete, and all 32 patients with 52 weeks follow-up have successfully rolled into the long-term follow-up study. We believe that the U.S. Food and Drug Administration, or FDA, has provided a clear regulatory pathway for isaralgagene civaparvovec, agreeing that data from the ongoing Phase 1/2 STAAR study can serve as the primary basis for approval under the Accelerated Approval Program, using mean annualized eGFR slope at 52 weeks as an intermediate clinical endpoint. In October 2025, we held a meeting with the FDA to discuss the proposed efficacy and safety data package for isaralgagene civaparvovec where, in the
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meeting minutes, among other things, the FDA reiterated its October 2024 agreement to use eGFR slope as an endpoint to support an accelerated approval pathway.
•In December 2025, we initiated a rolling submission of a Biologics License Application, or BLA, to the FDA seeking approval of isaralgagene civaparvovec under an Accelerated Approval pathway. We have submitted the preclinical and clinical modules to the FDA for review. In addition, the antibody assay companion diagnostic, which is designed to screen patients for eligibility with isaralgagene civaparvovec, has been submitted to, and accepted by, the FDA’s Center for Devices and Radiological Health, or CDRH, seeking Premarket Approval, or PMA.
•We continue to develop the Chemistry, 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 summer of 2026 subject to our ability to secure adequate additional funding, while we continue early stage business development discussions for a potential Fabry commercialization agreement.
Certain Components of Results of Operations
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 2025, we entered into license agreements for STAC-BBB 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 STAC-BBB with Eli Lilly and 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 Notes 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.
Subject 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.
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 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 and advancing our product candidates from research stage through clinical trials.
Critical Accounting Policies and Estimates
Our Consolidated Financial Statements and the related disclosures have been prepared in accordance with generally accepted accounting principles in the United States. The preparation of these Consolidated Financial Statements requires us to make estimates, assumptions and judgments that affect the reported amounts in our Consolidated Financial Statements and accompanying notes. We base our estimates on historical experience and on various other assumptions that we believe to be reasonable under the circumstances, the results of which form the basis for making judgments about the carrying values of assets and liabilities that are not readily apparent from other sources. Actual results may differ from these estimates under different assumptions or conditions. We believe the following policies to be the most critical to an understanding of our financial condition and results of operations because they require us to make estimates, assumptions and judgments about matters that are inherently uncertain.
We believe our critical accounting policies and estimates relating to revenue recognition and valuation of long-lived assets are the most significant estimates and assumptions used in the preparation of our Consolidated Financial Statements.
For a complete description of our significant accounting policies, see Note 1 – Organization, Basis of Presentation and Summary of Significant Accounting Policies in the accompanying Notes to the Consolidated Financial Statements included in Part II, Item 8, “Financial Statements and Supplementary Data” of this Annual Report on Form 10-K.
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Revenue Recognition
Our revenues are primarily derived from collaboration agreements, including licensing arrangements and research services. Research and license agreements typically include nonrefundable upfront signing or license fees, payments at negotiated rates for time incurred by our researchers, third-party cost reimbursements, additional target selection fees, sublicense fees, milestone payments tied to ongoing development and product commercialization, and royalties on future licensees’ product sales. All funds received from our collaboration partners are generally not refundable. Non-refundable upfront fees are fixed at the commencement of the contract. All other fees represent variable consideration in contracts. For contracts that contain a provision where we reimburse our customer for certain costs they incur and where we do not acquire any distinct goods or services in exchange for such payments, we account for it as a reduction to the contract transaction price. Deferred revenue primarily represents the portion of nonrefundable upfront fees received but not earned.
For a further description of our revenue recognition, see Note 1 – Organization, Basis of Presentation and Summary of Significant Accounting Policies and Note 4 – Major Customers, Partnerships and Strategic Alliances in the accompanying Notes to the Consolidated Financial Statements included in Part II, Item 8, “Financial Statements and Supplementary Data” of this Annual Report on Form 10-K.
Valuation of Long-lived Assets
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 their 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.
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 the stock price and market capitalization compared to the net book value.
Calculating the fair value of a reporting unit, an asset group and an individual asset involves significant estimates and assumptions. These estimates and assumptions include, among others, projected future cash flows, risk-adjusted discount rates, future economic and market conditions, and the determination of appropriate market comparables. Changes in these factors and assumptions used can materially affect the amount of impairment loss recognized in the period the asset was considered impaired.
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 Long-lived Assets and Write-Down of Assets Held For Sale in the accompanying Notes to the Consolidated Financial Statements included in Part II, Item 8, “Financial Statements and Supplementary Data” of this Annual Report on Form 10-K.
Recent Accounting Pronouncements
For a summary of recent accounting pronouncements and the anticipated effects on our Consolidated Financial Statements, see Note 1 – Organization, Basis of Presentation and Summary of Significant Accounting Policies in the accompanying Notes to the Consolidated Financial Statements included in Part II, Item 8, “Financial Statements and Supplementary Data” of this Annual Report on Form 10-K.
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Results of Operations
Years Ended December 31, 2025 and 2024
Revenues
| Year Ended December 31, | |||||||||||||||||||||||||||||||||||||||
| (in thousands, except percentage values) | |||||||||||||||||||||||||||||||||||||||
| 2025 | 2024 | Change | % | ||||||||||||||||||||||||||||||||||||
| Revenues | $ | 39,552 | $ | 57,800 | $ | (18,248) | (32) | % | |||||||||||||||||||||||||||||||
Revenues in 2025 primarily consisted of revenues from the collaboration agreements with Lilly, Astellas and Pfizer, Inc., or Pfizer, and royalties from our license agreements with Sigma-Aldrich Corporation, or Sigma, and Open Monoclonal Technology, Inc. (now Ligand Pharmaceuticals Incorporated), 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 $18.2 million in revenues in 2025 compared to 2024 was primarily attributable to a decrease of $49.9 million in revenue relating to our collaboration agreement with Genentech. This decrease was offset by $18.4 million in revenue relating to our capsid license agreement with Lilly, $6.0 million in revenue relating to Pfizer’s exercise of its option to obtain a license pursuant to the terms of the 2008 licensing agreement for certain zinc finger modified cell lines, $5.0 million in revenue relating to our collaboration agreement with Pfizer upon transfer of a specified sublicense, an increase of $1.4 million in revenue relating to our license agreement with Sigma, and an increase of $1.0 million in revenue relating to our collaboration agreement with Astellas.
Operating Expenses
| Year Ended December 31, | |||||||||||||||||||||||||||||||||||||||
| (in thousands, except percentage values) | |||||||||||||||||||||||||||||||||||||||
| 2025 | 2024 | Change | % | ||||||||||||||||||||||||||||||||||||
| Operating expenses: | |||||||||||||||||||||||||||||||||||||||
| Research and development | $ | 112,670 | $ | 111,521 | $ | 1,149 | 1 | % | |||||||||||||||||||||||||||||||
| General and administrative | 34,886 | 44,727 | (9,841) | (22) | % | ||||||||||||||||||||||||||||||||||
| Impairment of long-lived assets | 13,235 | 5,521 | 7,714 | 140 | % | ||||||||||||||||||||||||||||||||||
| Total operating expenses | $ | 160,791 | $ | 161,769 | $ | (978) | (1) | % | |||||||||||||||||||||||||||||||
Research and Development Expenses
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 $1.1 million in research and development expenses in 2025 compared to 2024 was attributable to an increase of $19.2 million in clinical and manufacturing expenses, primarily due to BLA readiness activities for our Fabry disease program which was partially offset by 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 $14.6 million due to changes in variable compensation and lower headcount, lower facilities, infrastructure related expenses and allocated overhead costs of $4.5 million, and lower licensing and patent related expenses of $2.1 million. Stock-based compensation expense included in research and development expenses was $4.2 million and $5.7 million for the years ended December 31, 2025 and 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 disease program increased by $39.9 million in 2025 as compared to 2024, primarily driven by BLA readiness activities and the commencement of the rolling BLA submission in December 2025. Chronic neuropathic pain clinical program expenses decreased by $9.1 million in 2025 as compared to 2024, primarily because the program has advanced from preclinical activities and patient enrollment and recruitment activities have commenced. Expenses related to preclinical and early research programs decreased by $19.5 million
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and other research and development programs decreased by $10.1 million in 2025 as compared to 2024, primarily driven by deferral and reprioritization of certain programs.
| Year Ended December 31, | |||||||||||||||||
| (in thousands) | |||||||||||||||||
| Programs | 2025 | 2024 | |||||||||||||||
| Fabry disease program | $ | 67,591 | $ | 27,725 | |||||||||||||
| Chronic neuropathic pain clinical program | 10,148 | 19,295 | |||||||||||||||
| Wholly owned preclinical programs and early research activities | 29,455 | 48,960 | |||||||||||||||
| Other research and development programs | 5,476 | 15,541 | |||||||||||||||
| Total research and development expenses | $ | 112,670 | $ | 111,521 | |||||||||||||
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. 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-BBB 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.
Our potential therapeutic products are subject to a lengthy and uncertain regulatory process that may not result in our receipt of any necessary regulatory approvals. Failure to receive the necessary regulatory approvals would prevent us from commercializing the product candidates affected. In addition, clinical trials of our product candidates may fail to demonstrate safety and efficacy, which could prevent or significantly delay regulatory approval. A discussion of the risks and uncertainties with respect to our research and development activities, including completing the development of our product candidates, and the consequences to our business, financial position and growth prospects can be found in “Risk Factors” in Part I, Item 1A of this Annual Report on Form 10-K.
General and Administrative Expenses
General and administrative expenses consist primarily of compensation-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 $9.8 million in general and administrative expenses in 2025 compared to 2024 was primarily attributable to lower compensation and other personnel costs of $8.4 million due to changes in variable compensation and lower headcount, lower external professional services expenses of $2.1 million, and lower facilities and infrastructure related expenses of $1.2 million. These decreases were partially offset by an increase of $2.0 million in allocated overhead costs. Stock-based compensation expense included in general and administrative expenses was $4.9 million and $6.7 million for the years ended December 31, 2025 and 2024, respectively.
Impairment
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 Long-lived Assets and Write-Down of Assets Held For Sale in the accompanying Notes to the Consolidated Financial Statements included in Part II, Item 8, “Financial Statements and Supplementary Data” of this Annual Report on Form 10-K.
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During the year ended December 31, 2024, we recognized impairment charges of $5.5 million. In 2024, our Board of Directors approved the wind-down of research and development activities in France and a corresponding reduction in workforce, including closure of our cell therapy manufacturing facility and research labs in Valbonne, France, or the France Restructuring. Additionally, we initiated actions to commence the closure of our facility in Brisbane, California, and we faced a sustained decline in our stock price and related market capitalization. There was also a decline in the market rates for facility subleases, indicating the carrying values of right-of-use and leasehold improvement assets could be impaired. As a result of these factors, we concluded certain long-lived assets, primarily comprising right-of-use assets, related leasehold improvements, and certain manufacturing and laboratory equipment, were impaired. For more information see Note 5 – Impairment of Long-lived Assets and Write-Down of Assets Held For Sale in the accompanying Notes to the Consolidated Financial Statements included in Part II, Item 8, “Financial Statements and Supplementary Data” of this Annual Report on Form 10-K.
Other (expense) income, net
The decrease of $7.9 million in 2025 compared to 2024 was primarily attributable to a decrease of $5.3 million related to fluctuations in foreign currency exchange rates, and a decrease of $2.5 million in research income tax credits.
Income tax benefit
The income tax benefit was $0.6 million and $0.2 million for the years ended December 31, 2025 and 2024, respectively. The income tax benefits for the years ended December 31, 2025 and 2024 were primarily driven by the reversal of the Company’s United Kingdom subsidiary’s long-term payable as a result of the statute of limitations lapsing and foreign income taxes.
On July 4, 2025, the current administration signed the One Big Beautiful Bill Act, or OBBBA, which includes comprehensive U.S. corporate tax legislation. The legislation includes the modification and permanent extension of prior tax law under the 2017 Tax Cuts and Jobs Act and the introduction of new provisions such as permanently reinstating the immediate deduction of domestic specified research and experimental expenditures, permanent changes in the limitations for deducting business interest expense, and permanently restoring bonus depreciation allowances. Due to our valuation allowance on deferred tax assets, this tax law change did not result in a material impact to our consolidated financial statements.
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, 2025 and 2024, which is fully offset with a valuation allowance.
As of December 31, 2025, we had net operating loss carryforwards for federal and state income tax purposes of approximately $980.4 million and $470.3 million, respectively. The federal net operating loss generated before 2018 will begin to expire in 2026 and will keep expiring through 2037, if not utilized. Federal net operating loss generated from 2018 will carry forward indefinitely. If not utilized, the state net operating loss carryforwards will begin to expire in 2029. Our French net operating loss carryforward balance is $129.3 million, which carries over indefinitely. We also have federal and state research tax credit carryforwards of $53.2 million and $35.2 million, respectively. The federal research credits will begin to expire in 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.
Liquidity and Capital Resources
Liquidity
Since inception, we have incurred significant net losses, and we have funded our operations primarily through the issuance of equity securities, payments from corporate collaborators and strategic partners and research grants.
As of December 31, 2025, we had cash and cash equivalents of $20.9 million compared to $41.9 million as of December 31, 2024. Our most significant use of capital during the year was for external research and development expenses, such as manufacturing, clinical trials, regulatory and preclinical activity related to our therapeutic programs, and employee compensation.
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We are party to an Open Market Sale Agreement℠, as amended, or the sales agreement, with Jefferies LLC, providing for the sale of up to $325.0 million of our common stock from time to time in “at-the-market” offerings under an existing shelf registration statement. Approximately $133.3 million remained available under the sales agreement as of December 31, 2025. We sold 84.7 million and 3.6 million shares of our common stock under the sales agreement for net proceeds of $52.2 million and $7.1 million, respectively, during the years ended December 31, 2025 and 2024. Additionally, in May 2025, we issued 12.2 million shares of common stock, pre‑funded warrants to purchase 34.4 million shares of common stock and accompanying warrants to purchase an aggregate of 46.6 million shares of common stock and accompanying warrant of $0.50 per share (or $0.49 per pre-funded warrant and accompanying warrant), for total net proceeds of approximately $21.1 million, after deducting underwriting discounts and commissions and other offering costs. Additionally, in March 2024, we issued 24.8 million shares of common stock, pre‑funded warrants to purchase 3.8 million shares of common stock and accompanying warrants to purchase an aggregate of 28.6 million shares of common stock at a price per share of common stock and accompanying warrant of $0.84 per share (or $0.83 per pre-funded warrant and accompanying warrant), for total net proceeds of approximately $21.9 million, after deducting placement agent fees and other offering costs. Subsequent to December 31, 2025, we sold 9.3 million shares of our common stock under the sales agreement for net proceeds of approximately $3.7 million. Additionally, in February 2026, we issued 35.2 million shares of common stock, and pre‑funded warrants to purchase up to an aggregate of 17.8 million shares of common stock, together with accompanying warrants to purchase up to an aggregate of 53.0 million shares of common stock, for net proceeds of approximately $23.1 million, after deducting underwriting discounts and commissions and other offering costs. In connection with this offering, we entered into a warrant amendment, or the Warrant Amendment, pursuant to which we agreed to reduce the exercise price of outstanding common stock warrants issued on March 26, 2024 and held by the investor to purchase 23.8 million shares of common stock from $1.00 to $0.4719 (the “Repriced Warrants”). The Repriced Warrants will become exercisable six months from the closing date of the offering. In connection with the reduction in exercise price, we also agreed to extend the expiration date of the Repriced Warrants to be five and a half years from the closing of the offering.
Under Accounting Standard Codification Topic 205-40, Presentation of Financial Statements—Going Concern, or ASC Topic 205-40, we have the responsibility to evaluate whether conditions and/or events raise substantial doubt about our ability to meet our future financial obligations as they become due within one year after the date that the Consolidated Financial Statements included in this Annual Report on Form 10-K are issued. As required under ASC Topic 205-40, management’s evaluation should initially not take into consideration the potential mitigating effects of management’s plans that have not been fully implemented as of the date the Consolidated Financial Statements are issued. When substantial doubt exists, management evaluates whether the mitigating effects of its plans sufficiently alleviate the substantial doubt about the company’s ability to continue as a going concern. The mitigating effects of management’s plans, however, are only considered if both (i) it is probable that the plans will be effectively implemented within one year after the date that the financial statements are issued, and (ii) it is probable that the plans, when implemented, will mitigate the relevant conditions or events that raise substantial doubt about the entity’s ability to continue as a going concern within one year after the date that the financial statements are issued. Generally, to be considered probable of being effectively implemented, the plans must have been approved by the company’s Board of Directors before the date that the financial statements are issued.
Based on our current operating plan, we estimate that our cash and cash equivalents as of December 31, 2025, together with net proceeds of approximately $23.1 million from the February 2026 Offering, a $4.6 million research tax credit received from the French government in February 2026, and $3.7 million generated through our at-the-market offering program since December 31, 2025, will be sufficient to meet our liquidity requirements only into the third quarter of 2026. This estimate regarding our cash resources is based on assumptions that are inherently uncertain, and actual results could differ materially from those estimates. In this regard, we could use our available capital resources sooner than we currently expect and changing circumstances, some of which may be beyond our control, could cause us to consume capital significantly faster than we currently anticipate. Although we believe our cash and cash equivalents could fund our planned operations into the third quarter of 2026, unless we secure substantial upfront funding through a significant partnership or other transaction for our programs in the very near term, we expect that we will need to significantly scale back our operations at that time and focus substantially all of our efforts on pursuing strategic alternatives to maximize the value of our assets for our stockholders and creditors. In particular, at any time we may determine that it is in the best interest of our stockholders and creditors 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 Sangamo and our stakeholders and the most advantageous time for such filing in order to preserve sufficient resources to undertake an appropriate bankruptcy process.
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
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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, 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 in a timely manner 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. Moreover, we currently are not in compliance with the listing standards of Nasdaq and do not expect to regain compliance prior to the April 27, 2026 compliance 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 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 substantial 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 clinical-stage programs. In 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 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 secure such collaborations or other 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 program, 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 Fabry disease program, we will be unable to secure the substantial additional capital needed to support our operations and to continue to operate as a going concern. If adequate funds are not available to us in the very near 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. In this regard, we have periodically, including recently, reduced our headcount, and we are actively considering a variety of significant 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 would be materially and adversely affected, and 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 in the very near term, and you may lose all or part of your investment.
Moreover, we have historically relied in part on our collaboration partners to provide funding for and otherwise advance our preclinical and clinical programs, however, several of our prior collaborations expired or were terminated in the last several years. 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 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. In any event, we need substantial additional funding in order to execute on our current operating plan, and our ability to raise such funding and to continue our operations will be substantially impaired if we are not able to secure a commercialization partner for our Fabry disease program in the very near term. If we raise additional capital through public or private equity offerings, including sales pursuant to our at-the-market offering program with Jefferies LLC, the ownership interest of our existing stockholders will be diluted, and such dilution may be substantial given our current stock price decline, and the terms of any new equity securities may have a preference over, and include rights superior to, our common stock. If we raise additional capital through 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
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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 our 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 due in part to, among other things, the ongoing conflicts in the Middle East, conflict between Russia and Ukraine, and geopolitical challenges arising from the imposition of tariffs and escalating trade tensions. 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 in a timely manner, or at all.
Cash Flows
Operating activities
Net cash used in operating activities was $97.2 million in 2025, primarily due to:
•a net loss of $122.9 million, adjusted for non-cash long-lived asset impairment charges of $13.2 million, other non-cash expenses related to stock-based compensation of $9.1 million, depreciation and amortization of $4.0 million, and amortization of operating lease right-of-use assets of $3.6 million; and
•an increase in accrued compensation and employee benefits of $3.1 million, an increase in accounts payable and other accrued liabilities of $2.8 million, a decrease in prepaid expenses and other assets of $0.8 million, and a decrease in accounts receivable of $0.2 million. These were partially offset by a decrease in deferred revenues of $6.7 million, a decrease in lease liabilities of $4.0 million, and a decrease in other non-current liabilities of $0.4 million.
Net cash used in operating activities was $67.1 million in 2024, primarily due to:
•a net loss of $97.9 million, adjusted for non-cash long-lived asset impairment charges of $5.5 million, other non-cash expenses related to stock-based compensation of $12.4 million, depreciation and amortization of $5.1 million, amortization of operating lease right-of-use assets of $4.3 million, and other non-cash adjustments of $0.3 million, offset by gain on sale of assets classified as held for sale of $1.0 million, and accretion of discounts and impairment of marketable securities of $0.3 million; and
•a decrease in accounts payable and other accrued liabilities of $14.2 million, a decrease in lease liabilities of $5.6 million, and a decrease in other non-current liabilities of $0.3 million. These were partially offset by an increase in deferred revenues of $13.4 million, an increase in accrued compensation and employee benefits of $5.7 million, a decrease in prepaid expenses and other assets of $4.4 million, a decrease in refundable research income tax credits of $0.6 million, and a decrease in accounts receivable of $0.4 million.
Investing activities
Net cash used in investing activities was not material in 2025.
Net cash provided by investing activities was $37.5 million in 2024, primarily related to sales of marketable securities of $34.7 million, proceeds from sale of assets classified as held for sale of $2.0 million, and maturities of marketable securities of $1.1 million, partially offset by purchases of property and equipment of $0.3 million.
Financing activities
Net cash provided by financing activities was $70.7 million in 2025, primarily related to $52.2 million of proceeds from our at-the-market offering, net of offering expenses of $1.4 million, $21.5 million of proceeds from issuance of common stock, net of offering expenses of $1.9 million, and proceeds from issuance of common stock under our employee stock purchase plan of $0.3 million, partially offset by taxes paid related to net share settlement of equity awards of $3.3 million.
Net cash provided by financing activities was $28.4 million in 2024, primarily related to $21.9 million of proceeds from issuance of common stock, net of offering expenses of $2.1 million, $7.1 million of proceeds from the at-the-market offering, net of offering expenses of $0.4 million, and proceeds from issuance of common stock under employee stock purchase plan of $0.3 million, partially offset by taxes paid related to net share settlement of equity awards of $0.9 million.
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Operating Capital and Capital Expenditure Requirements
We anticipate continuing to incur operating losses for at least the next several years and need to raise substantial additional capital in order to continue operating as a going concern. The effects of the current macroeconomic and regulatory environment, including evolving staff and policy changes at the FDA, the current and potential future government shutdowns, the effects of the ongoing conflicts in the Middle East, conflict between Russia and Ukraine, global trade issues and changes in and uncertainties with respect to tariffs and international trade disputes, inflation, climate change, fluctuations in interest rates and other economic uncertainty and volatility, has resulted and may continue to result in significant disruption of global financial markets, which could continue to impair our ability to access substantial additional capital on terms that are acceptable or at all, and in turn could negatively affect our liquidity and our ability to continue to operate as a going concern. Future capital requirements beyond the period into which we expect our existing cash and cash equivalents will be sufficient to fund our planned operations will be substantial, and we otherwise need to raise substantial additional capital to continue to operate as a going concern and to fund the development, manufacturing and potential commercialization of our product candidates (see “–Financial Position–Going Concern” and “–Liquidity and Capital Resources–Liquidity” above).
As we focus our efforts on proprietary human therapeutics, we will need to seek FDA approvals of our product candidates, a process that could cost in excess of hundreds of millions of dollars per product. Our future capital requirements will depend on many forward-looking factors, including the following:
•the results of preclinical testing of our early-stage core neurology program product candidates;
•the initiation, progress, timing and completion of clinical trials for our product candidates and potential product candidates;
•the outcome, timing and cost of regulatory approvals;
•the success of our existing collaboration agreements and our ability to secure additional collaborations;
•delays that may be caused by changing regulatory requirements, including the evolving staff and policy changes at the FDA;
•the number of product candidates that we pursue;
•the costs involved in filing and prosecuting patent applications and enforcing and defending patent claims;
•the timing and terms of future in-licensing and out-licensing transactions;
•the cost and timing of establishing sales, marketing, manufacturing and distribution capabilities;
•the cost of procuring clinical and commercial supplies of our product candidates;
•the extent to which we acquire or invest in businesses, products or technologies, including the costs associated with such acquisitions and investments; and
•the costs of potential disputes and litigation.
Contractual Obligations
Our contractual obligations as of December 31, 2025 relate primarily to (i) operating leases consisting of base rents for facilities in Brisbane, California and Richmond, California, (ii) purchase obligations related to manufacturing, facilities, and equipment, and (iii) license obligations for ongoing license maintenance fees associated with cancellable in-licensed patent agreements. These agreements, including amendments, are enforceable and legally binding and specify all significant terms, including fixed or minimum services to be used, fixed, minimum or variable price, and the approximate timing of the actions under the contracts. For more information regarding our contractual obligations and commitments as of December 31, 2025, see Note 7 – Commitments and Contingencies in the accompanying Notes to the Consolidated Financial Statements included in Part II, Item 8, “Financial Statements and Supplementary Data” of this Annual Report on Form 10-K.
Recent insider activity
| Date | Insider | Role | Action | Shares | Price | Value |
|---|---|---|---|---|---|---|
| 2026-04-22 | Davis Gregory D | Head of Research & Technology | Sell | -69,827 | $0.25 | -$17,694 |
| 2026-04-21 | Dubois-Stringfellow Nathalie | SVP-CHIEF DEVELOPMENT OFFICER | Sell | -345,942 | $0.26 | -$89,184 |
Source: SEC Form 4 filings.
Next expected filings
- ~2026-08-09 10-Q expected by 2026-08-11 (in 76 days)
- ~2026-11-08 10-Q expected by 2026-11-10 (in 167 days)
- ~2027-03-30 10-K expected by 2027-03-31 (in 309 days)
- ~2027-05-16 10-Q expected by 2027-05-18 (in 356 days)
Predicted from historical filing cadence; not an SEC commitment.
Recent SEC filings
- 2026-05-14 10-Q Quarterly Report
- 2026-05-14 8-K Earnings Release; Financial Statements and Exhibits
- 2026-04-30 10-K/A Annual Report (Amended)
- 2026-04-29 8-K Delisting Notice
- 2026-03-30 10-K Annual Report
- 2026-03-30 8-K Earnings Release; Financial Statements and Exhibits
- 2026-02-04 8-K Material Agreement Entered; Unregistered Equity Sale; Financial Statements and Exhibits
- 2026-02-03 8-K Other Events
- 2026-02-03 8-K Earnings Release; Officer/Director Change
- 2025-11-06 10-Q Quarterly Report
- 2025-11-06 8-K Earnings Release; Financial Statements and Exhibits
- 2025-10-29 8-K Delisting Notice
- 2025-09-30 8-K Officer/Director Change
- 2025-09-11 8-K Officer/Director Change
- 2025-09-04 8-K Other Events