CRBU 10-K & 10-Q changes, risk factors and insider trading
Caribou Biosciences, Inc. · Nasdaq · Biological Products, (No Diagnostic Substances) · CIK 1619856 · All filings on SEC.gov
At a glance
What changed in the latest 10-K
Risk Factors
New heading “We are not currently researching and developing additional product candidates; if we choose to do so in the future, we may not be successful in our efforts to identify and successfully research and develop additional product candidates.”
New heading “In the event that we are unable to continue to fund the clinical development of one or both of our product candidates, and/or if one or both of our clinical-stage product candidates is not clinically successful, does not receive regulatory approval, and/or is not commercially competitive, we currently do not have a research pipeline from which to generate new product candidates.”
New heading “Changes at the FDA may hinder the agency’s ability to hire and retain key leadership and other personnel, slow the time necessary for new product candidates to be reviewed and/or approved, or otherwise prevent these agencies from performing normal business functions on which our operations rely, which would adversely affect our business.”
New heading “Healthcare and other reform measures could hinder or prevent the commercial success of our product candidates, if approved.”
New heading “Our use of artificial intelligence and challenges with properly managing its use could adversely affect our business.”
New heading “Our stockholders have approved an amendment to our amended and restated certificate of incorporation to effect a reverse stock split at the discretion of our board of directors. We cannot provide any assurance that a reverse stock split, if implemented, will increase our stock price for a sustained period or will cause our stock to maintain compliance with Nasdaq continued listing requirements.”
New heading “The effective increase in the number of authorized shares of our common stock as a result of a reverse stock split may result in dilution to existing stockholders from future issuances of shares.”
Removed heading “We may not be successful in our efforts to identify and successfully research and develop additional product candidates and may expend our limited resources to pursue particular product candidates or indications while failing to capitalize on other product candidates or indications that may be more profitable, or for which there is a greater likelihood of commercial success.”
Removed heading “Enacted and future healthcare legislation may increase the difficulty and cost for us to obtain approval of and commercialize our product candidates and could adversely affect our business.”
Largest changes
“We incorporate certain artificial intelligence (“AI”) solutions into our business, and applications of AI may become more important in our operations over time. Although we scrutinize the AI platforms we use, there are significant risks involved in deploying AI. For example, any AI-related efforts, particularly those related to generative AI, could subject us to risks related to harmful content, inaccuracies, bias, discrimination, infringement of third-party intellectual property, or misappropriation, defamation, data privacy, cybersecurity, or sanctions and export controls, among others. …”see in full comparison
“In addition, although we do not currently utilize Chinese CMOs, certain Chinese biotechnology companies may become subject to trade restrictions, sanctions, other regulatory requirements, or proposed legislation by the U.S. government, which could restrict or even prohibit our ability to work with such entities, thereby potentially disrupting the supply of material to us. For example, the BIOSECURE Act, which was recently signed into law in December 2025, prohibits U.S. …”see in full comparison
“One of our CMOs that manufacturers our CAR-T cell therapy product candidates is a company that currently has ties to China, and we expect to continue to use this CMO for some of our manufacturing in the near future. U.S. lawmakers have urged the U.S. government to investigate CMOs and CROs that have ties to China to ensure that sensitive U.S. biotechnology intellectual property is not transferred to China. Any such investigations or other regulatory actions could affect the ability of this CMO to provide services to us in a timely manner. …”see in full comparison
“There can be no assurance that we will continue to be able to comply with the applicable Nasdaq Global Select listing requirements, or, if transferred, the Nasdaq Capital Market listing standards. If we fail to comply with the continued listing requirements of Nasdaq, Nasdaq may take steps to delist our common stock. …”see in full comparison
see in full comparisonIn the past, securitiesSecurities class action litigationhasis often been brought against a company following a decline in the market price of its securities,andand, in the past, werecentlyhavesettled onehad such securities class actionandlitigationare currently defending another in the U.S. District Court for the Northern District of California, filed by purported stockholdersbrought against usandascertainwellof our current and former officers. Additionally, aas shareholder derivativecomplaint has beencomplaints filed against our directors and certain of our current and former officersin the same courtrelating tothe pendingsecurities class action litigation.SeeInLegaltheProceedings in Item 3 of this Annual Report on Form 10-K for additional information. There is no guarantee thatfuture, wewill be able to settle this new securities class action litigation and, if we are able to settle, for what amount. Wemay face additional securities class action litigation, and our officers and directors may be subject to shareholder derivativesuits, in the future.suits. This risk is especially relevant for us because biotechnology and pharmaceutical companies have experienced significant stock price volatility in recent years, and we expect to experience continued stock price volatility. We cannot provide any assurance that we will be able to have such lawsuits dismissed or, if not, that we will be able to settle the cases. Defending againstthe current litigation and anyfuture litigation could result in substantial costs and a diversion of management’s attention and resources, which could harm our business.
“We cannot provide any assurance that our common stock will regain compliance with the Minimum Bid Price Rule by any compliance deadline. Even if the market price per post-reverse stock split share of our common stock remains in excess of $1.00 per share, we may be delisted due to a failure to meet other continued listing requirements, including Nasdaq requirements related to the minimum number of shares that must be in the public float, the minimum market value of the public float, and the minimum number of market makers, among others. …”see in full comparison
Full comparison: every changed paragraph (217)
Investing in shares of our common stock involves a high degree of risk. You should carefully consider the following risks and uncertainties, together with all of the other information contained in this Annual Report on Form 10-K, including our financial statements and related notes, before making an investment decision. TheThese disclosures reflect our beliefs and opinions as to factors that could materially and adversely affect our company and its securities in the future. References to past events are provided by way of example only and are not intended to be a complete listing or a representation as to whether or not such factors have occurred in the past or their likelihood of occurring in the future. Furthermore, the risks described below are not the only ones facing us. The occurrence of any of the following risks, or of additional risks and uncertainties not presently known to us or that we currently believe to be immaterial, could materially and adversely affect our business, financial condition, results of operations and prospects, and reputation. In such case, the trading price of shares of our common stock could decline, and you may lose all or part of your investment. This Annual Report on Form 10-K also contains forward-looking statements that involve risks and uncertainties. Our actual results could differ materially from those anticipated in the forward-looking statements as a result of a number of factors, including the risks described below. See Special Note Regarding Forward-Looking Statements in this Annual Report on Form 10-K.
We have incurred significant operating losses each year since our inception. For the years ended December 31, 2024,2025, and 2023,2024, we incurred net losses of $149.1$148.1 million and $102.1$149.1 million, respectively. As of December 31, 2024,2025, we had an accumulated deficit of $448.4$596.5 million. In addition, we have not commercialized any products and have never generated any revenue from product sales. We have devoted almost all of our financial resources to research and development, including our preclinical and clinical development activities.
We expect to continue to incur significant expenses and operating losses over the next several years and for the foreseeable future as we seek to advance product candidates through preclinicalclinical development, expand our research and development activities, develop new product candidates, initiate and complete clinical trials, seek regulatory approval and, if we receive approval from the FDA or foreign regulatory authorities, commercialize our products. Furthermore, the costs of advancing product candidates into each succeeding clinical phase with greater number of patients increase substantially over time and, if we areadvance tovispa-cel advanceinto our CAR-T cell therapy product candidates intoplanned pivotal clinical trials,trial, we will incur significant expenses inrunning runninga large, multicenter trialstrial in the United States and foreign jurisdictions. The total costs to advance any of our product candidates to marketing approval in even a single jurisdiction is substantial. Our prior losses, combined with expected future losses, will continue to have an adverse effect on our stockholders’ deficit and working capital. We anticipate that our expenses will increase substantially if and as we:
•progress our clinical trials for our CAR-Tvispa-cel and CB-011 product candidates, particularly as we advance our vispa-cel product candidatescandidate intoin succeedingour planned pivotal clinical phasestrial;
•continue our current research programs and our preclinical and clinical development of our other current product candidates and any other product candidates we identify and choose to develop;
•hire additional employeesemployees, as needed;
•seek to identify additional research programs and additional product candidates;
•further develop our genome-editing technologies;
•acquire or in-license intellectual property orproperty, new technologiestechnologies, and/or additional product candidates;
•seek regulatory and marketing approvals for anyour ofvispa-cel ouror CB-011 product candidates thatif they successfully complete clinical trials, if anytrials;
•establish and expand manufacturing capabilities and supply chain capacity for our vispa-cel and CB-011 product candidates;
•experience any delays, challenges or other issues associated with any of the above, including the failure of clinical trials meeting endpoints, the generation of unanticipated preclinical study results or clinical trial data subject to differing interpretations, or the occurrence of potential safety issues or other development or regulatory challenges;
•continue to operate as a public company, including defending against any future securities class action litigation.
We will need substantial additional financing to developconduct our productplanned candidatespivotal clinical trial for vispa-cel and to implement our operating plans. If we fail to obtain additional financing, we maywill be delayed or unable to complete the development and commercialization of our vispa-cel and/or CB-011 product candidates.
We will continue to need additional capital beyond the proceeds received from our initial public offering (“IPO”), follow-on public financing, at-the-market equity offering program, and other historical sources of proceeds. Because our allogeneic cell therapy product candidates are based on new technologies, they require extensive development. The costs to treat patients with our product candidates in clinical trials are significant. We expect to spend a substantial amount of capital in the research,clinical development,development and manufacture of our product candidates, particularly as we advance our CAR-T cell therapy product candidates through succeeding clinical phases with greater numbers of patients. We expectcurrently do not have sufficient funds to conduct our expensesplanned pivotal clinical trial for vispa-cel, and we will need to increaseraise additional funds in connectionorder withto do so. If we are unable to raise additional financing, we will be unable to initiate our ongoingplanned activities, particularly as we initiate additionalpivotal clinical trialstrial for,for andvispa-cel seekor marketingto approvaldevelop of,CB-011 ourbeyond productdose candidates.expansion. In addition, if we obtain marketing approval for any of our product candidates, we expect to incur significant commercialization expenses related to product sales, marketing, manufacturing, and distribution to the extent that we do not obtain commercialization partners who will bear the costs for such activities. We may also need to raise additional funds sooner if we choose to pursue additional indications or markets for our product candidates or otherwise expand more rapidly than we presently anticipate. Accordingly, we will need to obtain substantial additional funding in connection with our continuing operations. Because our allogeneic cell therapy product candidates are based on new technologies, they require extensive research and development and have substantial manufacturing costs. In addition, clinical costs to treat patients with our product candidates, including treatment of any potential side effects that may arise, will be significant.
As of December 31, 2024,2025, we had cash, cash equivalents, and marketable securities of $249.4$142.8 million. We expect our cash, cash equivalents, and marketable securities to be sufficient to fund our current operating plan through at least the next 12 months from the date theour consolidated financial statements included in this Annual Report on Form 10-K are issued.filed. Our expectation is based on assumptions that may prove to be wrong, and we could use our available capital resources sooner than we currently expect.
•costs, progress, and results of clinical trials for our vispa-cel and CB-011 product candidate preclinical studies and clinical trialscandidates;
•potential delays in our preclinical studies and clinical trials, whether current or planned, due to unforeseen events as well as other factors such as the economic or regulatory environment or pandemics or other public health crises;
•costs and prioritization of our research and development programs as well as costs to acquire or in-license technologies or other product candidates;
•costs of operating as a public company, including defending against any future class action securities litigation.
Changing circumstances may cause us to consume capital significantly faster than we currently anticipate, and we may need to spend more money than expected because of circumstances beyond our control. We may also need to raise additional capital sooner if we choose to expand programs, personnel, and facilities more rapidly than planned. In any event, we will require additional capital for the further research, development,development and commercialization of our product candidates, including potentially establishing our own internal manufacturing capabilities. Any additional fundraising efforts may divert our management from their day-to-day activities, which may adversely affect our ability to research, develop,develop and commercialize our product candidates.
We cannot be certain that additional funding will be available when needed and on acceptable terms, or at all. If we are unable to obtain funding on a timely basis, we may be required to significantly delay initiation, curtail, delay, or discontinue one or more of our product candidate preclinical studies, clinical trials, or development and commercialization, or we may be unable to expand our operations or otherwise capitalize on our business opportunities, as desired. Any of the above could significantly harm our business, financial condition, results of operations, and prospects and cause the price of our common stock to decline.
Raising additional capital may cause dilution to our stockholders, restrict our operations, and/or require us to relinquish rights to our genome-editing technologies or product candidates.
Until such time, if ever, that we can generate substantial product revenues, we expect to finance our cash needs through a combination of equity offerings,offerings (including our at-the-market equity offering program), debt financings, andnew strategic collaborationcollaborations, andstructured or other non-dilutive financings, licensing arrangements.arrangements, and/or other sources. The terms of any financing may adversely affect the holdings or the rights of our stockholders and the issuance of additional securities, whether equity or debt, by us, or the possibility of such issuance, may cause the market price of our common stock to decline. Debt financing, if available, may involve agreements that include covenants limiting or restricting our ability to take specific actions, such as incurring additional debt, making capital expenditures, licensing or assigning our intellectual property rights, declaring dividends, and possibly other restrictions.
To the extent that we raise additional capital through the sale of equity or convertible debt securities, our existing stockholders’ interests will be diluted, perhaps substantially, and the terms of these securities may include liquidation or other preferences that adversely affect the rights of our common stockholders and new investors could gain rights superior to our existing investors. We may sell common stock, convertible securities, or other equity securities in one or more transactions at prices and in a manner we determine from time to time and existing investors may be substantially diluted by those issuances and sales. The terms of these securities may include liquidation or other preferences that adversely affect the rights of our common stockholders and new investors could gain rights superior to those of our existing investors.
Attempting to secure additional financing may also divert our management from our day-to-day activities, which could impair or delay our ability to develop our product candidates. Furthermore, if, in the future, one or more banks or financial institutions enter receivership or become insolvent in response to financial conditions affecting the banking system or financial markets, our ability to access our existing cash, cash equivalents, and marketable securities may be threatened and could have a material impact on our business and financial condition.
If we are unable to raise additional funds through equity or debt financings when needed, we may be required to delay, limit, reduce, or terminate our product development or future commercialization efforts. Alternatively, we could be required to seek collaboratorsfunding for our product candidates through collaborations or structured or other non-dilutive financings at an earlier stage than would otherwise be desirable or on terms that are less favorable than might otherwise be available. We might need to relinquish or license on unfavorable terms our rights to our product candidates in markets where we otherwise would seek to pursue development and commercialization ourselves, or to license our intellectual property to others who could develop products that will compete with our products. Any of these actions could have a material adverse effect on our business, financial condition, results of operations, and prospects.
We are a clinical-stage biotechnology company formed in 2011, with no products approved for commercial sale, and we have not generated any revenues from product sales. Our operations to date have been limited to financing and staffing our company, developing our technologies, and evaluating our CAR-T cell therapy product candidates in phase 1 clinical trials. Our prospects must be considered in light of the uncertainties, risks, expenses, and difficulties frequently encountered by companies in their early stages of operations. We have not yet demonstrated an ability to obtain marketing approval, manufacture at commercial scale, or conduct sales and marketing activities for our product candidates, which are all necessary for successful product commercialization. Consequently, predictions about our future success or viability may not be as accurate as they could be if we had a longer operating history or a history of successfully developing and commercializing cell therapy products. Our ability to generate product revenue or profits, which we do not expect to occur for many years, if ever, will depend heavily on the successful development and eventual commercialization of our product candidates, which may never occur.candidates. Unless we receive approval from the FDA or other regulatory authorities for our product candidates, we will not have product revenues. We may never be able to develop or commercialize a marketable cell therapy product.
We are early in our product development efforts. All of our programs will require clinical development, regulatory approval, manufacturing at commercial scale, distribution channels, a commercial organization, significant marketing efforts, and substantial investment before we generate any revenue from product sales. In addition, our product candidates must be approved for marketing by the FDA before we may commercialize our products in the United States and, if we wish to commercialize our products outside the United States, by foreign regulatory agencies. Furthermore, we will continue to incur costs associated with operating as a public company, including legal, accounting, insurance, investor relations, and other expenses.
Additionally, the rapidly evolving nature of the genome-editing and cell therapy fields may make it difficult to evaluate our technologies and product candidates as well as to predict our future performance. Our short history as an operating company makes any assessment of our future success or viability subject to significant uncertainty. We will encounter risks and difficulties, known and unknown, that are frequently experienced by early-stage companies in rapidly evolving fields. As we advance our product candidates, we must continue to transition from a company with a research focus to a company capable of supporting clinical development and, if successful, commercial activities. We may not be successful in such transitions. If we do not address these risks successfully, our business will suffer. Similarly, we expect that our financial condition and operating results may fluctuate significantly from quarter to quarter and year to year due to a variety of factors, many of which are beyond our control. As a result, you should not rely upon the results of any quarterly or annual period as an indicator of future operating performance.
WeOur CAR-T cell therapy product candidates are early in our productclinical development efforts and it will be many years before we commercialize a product candidate, if ever. If we are unable to advance our product candidates through clinical trials, obtain regulatory approval, and ultimately commercialize our product candidates, or if we experience significant delays in doing so, our business will be materially harmed.
We are early in the development of our cell therapy product candidates and haveinitially focused our research and development efforts to date on various CRISPR genome-editing technologies, including our chRDNA genome-editing technology, as well as identifying and developing our initial CAR-T cell product candidates. Our future success depends heavily on the successful clinical development of our vispa-cel and CB-011 product candidates. Our ability to generate product revenue, which we do not expect will occur for many years, if ever, will be a result of the successful development and eventual commercialization of ourthese product candidates, which may never occur. OurThese product candidates may have expected or unexpected adverse side effects or fail to demonstrate safety and efficacy. Additionally, our product candidates may have other characteristics that may make them impractical or prohibitively expensive for large-scale manufacturing. In certain cases, CROs and clinical trial sites may fail to conduct the clinical trials as planned, may fail to comply with applicable requirements, or may deviate from the clinical trial protocols. Furthermore, our vispa-cel and/or CB-011 product candidates may not receive regulatory approval or, if they do, they may not be accepted by the medical community or patients or may not be competitive with other products that become available. We currently have no product revenue and we may never be able to successfully develop or commercialize a marketable product.
We must submit an IND applicationsapplication to the FDA to initiate clinical trials in the United States. The filing of any future IND applicationsINDs for our product candidates iswe may develop will be subject to additional preclinicalnonclinical research, research-scale and clinical-scale manufacturing, exploration of possible other genome-editing systems, evaluation of potential targets, and other factors yet to be identified. In addition, commencing any new clinical trial is subject to review by the FDA based on the acceptability and sufficiency of our CMC, and preclinicalnonclinical information provided to support our IND applications.INDs. If the FDA or foreign regulatory authorities require us to complete additional preclinicalnonclinical studies or we are required to satisfy other requests for additional data or information, our clinical trials may be delayed. Even after we receive and incorporate guidance from the FDA or foreign regulatory authorities, these regulatory authorities could disagree that we have satisfied all requirements to initiate our clinical trials or they may change their position on the acceptability of our trial design or the clinical endpoints selected. They could impose a clinical hold, which may require us to complete additional preclinicalnonclinical studies or clinical trials. The FDA and foreign regulatory authorities may refuse to clear our IND applications.INDs. The success of our product candidates will depend on several factors, including the following:
•successful completion of preclinicalany additional nonclinical studies;
•clearance of IND applicationsINDs to initiate clinical trials;
•data from our clinical trials that support an acceptable risk-benefit profile of our product candidates for our intended patient populations and indications and demonstrate acceptable safety and efficacy;
•receipt of regulatory and marketing approvals from applicable regulatory authorities as well as receipt of nonpatent regulatory exclusivity for our product candidates;
•establishment of sales, marketing, and distribution capabilities for commercialization of our product candidates if and when approved, whether by us or in collaboration with third parties;
•legal and regulatory compliance by third parties that provide services to us or on our behalf, including but not limited to CMOs, suppliers, and clinicalcontract research organizations (“CROs”), some of which may be subject to regulatory investigations;
•acceptance of product candidates, if and when approved, by patients, the medical community, and third-party payors;
Our product candidates are cell therapies generated by our novel CRISPR chRDNA genome-editing technologies,technology, which makemakes it difficult to predict the time and cost of developing these product candidates and obtaining regulatory approval. To date, no other products that use these chRDNA genome-editing technologiestechnology have advanced into clinical trials or received marketing approval in the United States.
We are concentrating our initial research, development,development and manufacturing efforts on our allogeneic CAR-T cell therapies that are intended to treat patients with certain cancers. Before obtaining regulatory approval for the commercial sale of any of our product candidates, we must demonstrate through lengthy, complex, and expensive preclinicalnonclinical studies and clinical trials that our product candidates are both safe and effective for their intended use. The clinical trial requirements of the FDA and other regulatory authorities, and the criteria these regulators use to determine the safety and efficacy of a product candidate, vary substantially according to the type, complexity, novelty, intended use, and target population of our product candidates. The outcome of preclinicalnonclinical studies and clinical trials is inherently uncertain. PreclinicalNonclinical results in animals may not be predictive of safety or efficacy in humans. Failure can occur at any time during the preclinicalnonclinical study and clinical trial processes and because we have never successfully commercialized a product and our first product candidate is in an early stage of clinical development, there is a high risk of failure. We may never succeed in developing marketable products.
We use our CRISPR chRDNA genome-editing platformtechnology to generate our product candidates, and we believe our chRDNA guides significantly improve the specificity of CRISPR genome editing (e.g., by reducing the number of off-target events). CRISPR genome editing generally is relatively new; to date, only one cell therapy product using CRISPR-Cas9 genome editing has been approved in the United States although clinical trials of additional product candidates based on CRISPR-Cas9 and other genome-editing technologies are underway. As a result, the regulatory approval process for cell therapy product candidates such as ours is uncertain and may be more expensive and take longer than the approval process for product candidates based on better known or more extensively studied technologies. As such, it is difficult to accurately predict the developmental challenges we may face as we progress our product candidates through preclinical studies and clinical trials. There may be long-term adverse effects from treatment with our product candidates resulting from the use of our chRDNA genome-editing technologiestechnology that we cannot predict with the knowledge we have today. Also, animal models may not exist for some of the diseases we choose to pursue in ourthe programs,future, which may complicate and increase the cost of preclinicalnonclinical research. As a result of these factors, it is difficult for us to predict the time and cost of our product candidate development, and we cannot predict whether the application of our chRDNA technologies,technology, or other genome-editing technologies we may use in the future, will result in the identification, development, preclinicalnonclinical studies, and clinical trials to support regulatory approval of any of ourfuture cell therapy product candidates. ThereWe cancannot beprovide noany assurance that any development problems we experience in the future related to our chRDNA technologies or any of our research programstechnology will not cause significant delays or unanticipated costs, or that such development problems can be solved. We may not achieve the desired safety and efficacy of our product candidates. Also, we may not sufficiently improve genome-editing specificity and our genome editing may have off-target events. Moreover, we may not be able to achieve a high degree of on-target gene knockout and insertion efficiency in developing our product candidates. Any of these factors may prevent us from completing our clinical trials, delay or cause us to fail to meet our clinical trial endpoints, or lead us to fail to commercialize any of our cell therapy product candidates.
We may also experience delays in developing robust, reproducible, and scalable manufacturing processes and transferring those processes to CMOs, which may prevent us from completing our clinical trials or commercializing our products on a timely or profitable basis, if at all. Currently, we have only manufactured our CAR-T cell therapy product candidates for clinical trials.
We may also experience delays in developing robust, reproducible, and scalable manufacturing processes and transferring those processes to CMOs, which may prevent us from completing our clinical trials or commercializing our products on a timely or profitable basis, if at all. Currently, we have only manufactured our CAR-T cell therapy product candidates for clinical trials. In addition, since we are in the early stages of clinical development, we do not know the doses to be used in later phase 2 or pivotal phase 3 clinical trials necessary to evaluate the efficacy of our product candidates, which will affect the manufacturing requirements for our product candidates. Finding a suitable dose, such as a MTD or, as applicable, a RP2D, for our cell therapy product candidates may delay our anticipated clinical development timelines and prolong our clinical trials. Accordingly, our expectations regarding our costs of manufacturing may vary significantly as we develop our product candidates and understand these critical factors. Such factors may delay or keep us from bringing a product candidate to market and could decrease our ability to generate sufficient product revenue, which could harm our business, financial condition, results of operations, and prospects.
Manufacturing our product candidates is complex and we could experience manufacturing problems during our clinical trials, which could delay or limit development and/or commercialization of our product candidates.
The manufacturing processes used to produce our cell therapy product candidates are and will be complex, as our product candidates are new products.biologics. Several factors could cause production interruptions including facility contaminations; shortages or quality problems; contamination of healthy donor cells, chRDNA guides, Cas9 and Cas12a proteins, AAV6 viruses, iPSCand masterother cellmaterials banksused orin workingthe cellmanufacturing banksof our product candidates; natural disasters, including pandemics and other public health crises; labor shortages and strikes; lack or loss of experienced scientific,clinical, quality control, process development, and manufacturing personnel; human error; or other disruptions in the operations of our suppliers and CMOs. We conduct process development activities at our facilities and we may experience personnel and supply shortages. Problems with our manufacturing process, even minor deviations from the normal process, could result in product defects or manufacturing failures that result in lot failures, product recalls, product liability claims, or insufficient inventory. We may encounter problems achieving adequate quantities and quality of clinical grade materials that meet FDA or other applicable standards or specifications with consistent and acceptable production yields and costs.
As our product candidates proceed through preclinical studies to clinical trials to regulatory review, and potential marketing approval and commercialization, it is common that various aspects of our manufacturing methods will be altered along the way to optimize processes and results. Such changes carry the risk that intended objectives will not be achieved. If we make any such changes, our product candidates could perform differently and affect the results of clinical trials conducted with the altered materials. Such changes may also require additional testing as well as notification to or approval from the FDA or other regulatory authorities, which could delay completion of our clinical trials, require bridging clinical trials, require repetition of one or more clinical trials, increase clinical trial costs, delay approval of our product candidates, if any, and ultimately jeopardize commercialization.
If we receive marketing approval for a product candidate, the FDA and other regulatory authorities may require us to submit samples of any lot of any approved product together with the protocols showing the results of applicable tests at any time. Under some circumstances, the FDA or other regulatory authorities may require that we not distribute a lot until the relevant agency authorizes its release. Slight deviations in the manufacturing process, including those affecting quality attributes and stability, may result in unacceptable changes in the product that could result in lot failures or product recalls. Problems in our manufacturing processes could restrict our ability to meet market demand for our products. All these factors could be costly to us and otherwise harm our business, financial condition, results of operations, and prospects.
Problems in our manufacturing processes could restrict our ability to meet market demand for our products. All these factors could be costly to us and otherwise harm our business, financial condition, results of operations, and prospects.
Our business and future success depends on our ability to advance our product candidates through preclinical studies and clinical trials, obtain regulatory approval for, and successfully commercialize, our product candidates. The failure of our product candidates in clinical trials, or the failure of other companies’ allogeneic anti-CD19 CAR-T and allogeneic anti-BCMA CAR-T cell therapies, including for reasons due to safety, efficacy, or the durability of response, may impede our ability to develop our CAR-T cell therapy programs and product candidates and may significantly influence physicians’ and regulatory authorities’ opinions with regard to the viability of our entire pipeline of allogeneic cell therapies.therapies, Inwhich ordercould substantially impair our ability to submit IND applications for our other product candidates, we will need to complete many objectives, such as our preclinical research of product candidates still in discovery and advancement of cGMP conditions forcommercialize our product candidates. If we are unable to achieve any of these objectives, we may not be able to submit other IND applications in a timely manner or at all, which would significantly harm our business.
We are not currently researching and developing additional product candidates; if we choose to do so in the future, we may not be successful in our efforts to identify and successfully research and develop additional product candidates.
We may not be successful in our efforts to identify and successfully research and develop additional product candidates and may expend our limited resources to pursue particular product candidates or indications while failing to capitalize on other product candidates or indications that may be more profitable, or for which there is a greater likelihood of commercial success.
PartIn ofthe ourfuture, businesswe strategymay involveschoose identifyingto identify and developingdevelop new cell therapy product candidates.candidates, either internally or through acquisition from, or a partnership with, a third party. The process by which we identify product candidates may fail to yield successful product candidates for a number of reasons, including:
We have limited financial and managerial resources. We are currently focused initiallyon ontwo allogeneic CAR-T cell therapiestherapy product candidates and, as a result, we may forego or delay pursuit of opportunities with other product candidates or for other indications that later prove to have greater commercial potential. Our resource allocation decisions may cause us to fail to timely capitalize on viable commercial products or profitable market opportunities. Our spending on current and future product candidates for specific indications may not yield any commercially viable products. If we do not accurately evaluate the commercial potential or target market for a particular product candidate, we may relinquish valuable rights to that product candidate through collaboration, licensing, or structured or other royaltynon-dilutive arrangementsfinancings when it would have been more advantageous for us to retain sole development and commercialization rights to that product candidate.
In the event that we are unable to continue to fund the clinical development of one or both of our product candidates, and/or if one or both of our clinical-stage product candidates is not clinically successful, does not receive regulatory approval, and/or is not commercially competitive, we currently do not have a research pipeline from which to generate new product candidates.
On July 16, 2024, we announced that we had discontinued preclinical research activities associated with our allogeneic CAR-NK platform and reduced our workforce by 21 positions, or approximately 12% of our workforce, primarily in the research group and, on April 24, 2025, we announced a strategic pipeline prioritization, including reduction of our workforce by 47 positions, or approximately 32% of our workforce, primarily in the preclinical research group. Additionally, as part of the strategic pipeline prioritization announced in April 2025, we disclosed that we had discontinued our AMpLify phase 1 clinical trial of CB-012. In the event that we are unable to continue to fund the clinical development of our vispa-cel and/or CB-011 product candidates, and/or one or both of our clinical-stage product candidates are not clinically successful, do not receive regulatory approval, and/or are not commercially competitive, we do not have any product candidates other than vispa-cel and CB-011 to advance into the clinic. Additionally, we do not currently have a scientific workforce to research and develop such new product candidates, and we would need to either reestablish a preclinical research and development workforce to internally develop additional product candidates or acquire one or more new product candidates from a third party, both of which would be time-consuming and costly. We cannot provide any assurance that we would be able to research and develop new product candidates, or acquire such new product candidates from a third party.
The timely completion of clinical trials depends, among other things, on our ability to enroll a sufficient number of patients who remain in the trial until its conclusion. We may encounter delays in enrolling or be unable to enroll a sufficient number of patients to complete any of our clinical trials and, even if patients are enrolled, they may withdraw from our clinical trials before completion. For our current clinical trials, we have entered into contracts with CROs, as well as clinical trial agreements with the sites participating in our clinical trials. Patient selection and enrollment may be challenging; additionally, the protocols for our ongoing clinical trials specifically exclude patients with certain prior treatments as well as other conditions. Additionally, even after the FDA clears an IND for one of our product candidates, our clinical trials may not commence immediately if we are negotiating clinical trial agreements with clinical sites, conducting site initiation visits, or waiting for the sites to receive IRB approval. Due to competition from other clinical trials within the same therapeutic area at clinical sites, particularly with autoimmune diseases, sites may drop out or take longer to start up and enroll trials. Thus, we may not treat the first patient in a clinical trials for several months, or even for a year, after IND clearance.
Our current and future clinical trials, will compete for enrollment of patients with other clinical trials for product candidates that are in the same cell therapeutic areas with the same or similar study populations as our product candidates. Our clinical trials will also compete for enrollment of patients with other clinical trials for product candidates based on non-cellular modalities, such as small molecules and antibodies, that are intended for the same or similar study populations as our product candidates. This competition will reduce the number and types of patients available to us because some patients who might opt to enroll in our trials may instead opt to enroll in a trial being conducted by one of our competitors. Additionally, since the number of qualified and experienced clinical investigators for therapeutic areas is limited, some of our clinical trial sites may be also conducting clinical trials for some of our competitors, which may reduce the number of patients who are available for our clinical trials at that clinical trial site. Moreover, because our product candidates represent a departure from more commonly used methods for cancer treatment, potential patients and their doctors may be inclined to use conventional therapies, such as chemotherapy, HSC transplantation, or autologous CAR-T cell therapies, rather than refer patients to our clinical trials. Because our cell therapy product candidates are edited with CRISPR chRDNA guides, our products may be perceived to have additional or greater safety risks. Patients eligible for allogeneic CAR-T cell therapies but ineligible for autologous CAR-T cell therapies may be difficult to treat due to advanced and aggressive cancers and may fail to experience improved outcomes and be at greater risk for complications and death from our product candidates. If patients are unwilling to participate in our cell therapy trials, the timeline for recruiting patients, conducting clinical trials, and obtaining regulatory approval of any of our product candidates may be delayed.
Our allogeneic CAR-T cell therapy product candidates can be administered not only in academic centers of excellence but also in community hospital settings; however, recruitment of community sites for participation in the trial may be challenging due to hesitancy to adopt a CAR-T cell therapy in a center that has not previously administered this type of therapy or to administer our clinical trial protocol. Moreover, because our product candidates represent a departure from more commonly used methods for cancer treatment, potential patients and their doctors may be inclined to use conventional therapies, such as chemotherapy, HSC transplantation, or autologous CAR-T cell therapies, rather than refer patients to our clinical trials. Additionally, because our cell therapy product candidates are edited with CRISPR chRDNA guides, our products may be perceived to have additional or greater safety risks. Patients eligible for allogeneic CAR-T cell therapies but ineligible for autologous CAR-T cell therapies may be difficult to treat due to advanced and aggressive cancers and may fail to experience improved outcomes and be at greater risk for complications and death from our product candidates. If patients are unwilling to participate in our cell therapy trials, the timeline for recruiting patients, conducting clinical trials, and obtaining regulatory approval of any of our product candidates may be delayed.
Management's Discussion & Analysis (MD&A)
New heading “Impairment Charges”
New heading “Impairment Charges”
New heading “Shelf Registration Statements”
New heading “Impairment of Long-Lived Assets”
Removed heading “Follow-on Public Offering”
Removed heading “MSKCC Agreement Success Payments”
Largest changes
Because of the numerous risks and uncertainties associated withsee in full comparisonthetherapeuticdevelopmentproductof human therapeutics,development, we may never achieve profitability and, unless and until we are able to develop and commercialize our product candidates, we will need to continue to raise additionalcapital;capital.however,Untilfundingwemaycannotgeneratebesignificantavailablerevenue from product sales, if ever, we expect tous on acceptable terms, or at all. If we are unable to obtain adequate financing when needed, we may have to delay, reduce the scope of, or suspend one or more offinance ourpreclinical studies, clinical trials, research and development programs, and/or commercialization efforts. We may seek to raise any necessary additional capitaloperations througha combination ofequity offerings (including our at-the-market equity offering program), debt financings,collaborationsnewandcollaborations,strategicstructuredalliances,or other non-dilutive financings, licensing arrangements, and/or other sources. We cannot provide any assurance that we will be successful in obtaining an adequate level of financing to support our business plans as needed on acceptable terms, or at all. If we raise additionalcapitalfunds throughdebtnewfinancing,strategic collaborations or licensing arrangements with third parties, we maybe subjecthave tocovenantsrelinquishlimitingvaluable rights to our intellectual property, future revenue streams, orrestrictingproductourcandidatesabilityor grant licenses on terms that may not be favorable totakeus.specific actions, such as incurring additional debt, making capital expenditures, or declaring dividends. The disruptionDisruptions and volatility in the global and domestic capital markets resulting from heightened inflation, tariffs, capital market volatility, interest rate and currency rate fluctuations, artificialintelligence,intelligence (“AI”), political and geopolitical tensions, government agencychanges under the new Administration,changes, any potential economic slowdown or recession, including trade wars or civil or political unrest (such as the ongoing war between Ukraine and Russia,conflictconflicts in the Middle East,andincluding the recent hostilities involving Iran, tension between China andTaiwanTaiwan, geopolitical tensions in Europe, South America, and elsewhere) may increase the cost of capital and limit our ability to access capital. If we are unable to raiseadditionalcapitalthroughastheandsalewhenof equityneeded orconvertibleondebtattractivesecurities, the issuance of these securities could result in dilution to our stockholders. If we raise additional capital through marketing and distribution arrangements or other collaborations, strategic alliances, or licensing arrangements with third parties or other sources,terms, we may have torelinquishsignificantlycertaindelay,valuablereduce,rightsortodiscontinue the development and commercialization of our productcandidates, technologies, future revenue streams,candidates orresearchscaleprogramsback orgrantterminatelicensesouronpursuittermsofthatnewmayin-licensesnotandbe favorable to us.acquisitions.
“With the effectiveness of the 2025 Shelf Registration Statement, we refreshed our at-the-market equity offering program under the ATM Sales Agreement. We may, from time to time, sell and issue shares of our common stock, through Jefferies as sales agent under the ATM Sales Agreement, having an aggregate offering price of up to $100.0 million in gross proceeds under the 2025 Shelf Registration Statement, by any method permitted by law deemed to be an “at the market offering” as defined in Rule 415(a)(4) of the Securities Act of 1933, as amended (“Securities Act”). …”see in full comparison
On August 9, 2022, we entered into an at-the-market Open Market Sale AgreementSM (see in full comparisonthe“ATM Sales Agreement”) with Jefferies LLC (“Jefferies”), pursuant to which,uponon the terms and subject to the conditions and limitations set forth in the ATM Sales Agreement,we may,from time to time,inweourcouldsolehavediscretion, issueissued andsell,sold, through Jefferies, acting as sales agent, up to $100.0 million of our shares of commonstock,stockby any method permitted by law deemed to be an “atunder themarket2022offering”ShelfasRegistrationdefinedStatement.in Rule 415(a)(4) of the Securities Act of 1933, as amended (“Securities Act”). Jefferies uses commercially reasonable efforts consistent with its normal sales and trading practices to sell shares from time to time, based upon our instructions (including any price or size limits or other customary parameters or conditions we may impose). We pay Jefferies a commission equal to 3.0% of the aggregate gross proceeds of any shares sold through Jefferies pursuant toUnder the ATM SalesAgreement.AgreementThroughandDecemberthe31,20222024,Shelf Registration Statement, we issued and sold an aggregate of 3,588,696 shares of our common stockunder the ATM Sales Agreementat an average price per share of $4.71 for aggregate gross proceeds of $16.9 million ($16.2 million net of offering expenses).
Full comparison: every changed paragraph (99)
We are a clinical-stage Clustered Regularly Interspaced Short Palindromic Repeats (“CRISPR”) genome-editing biopharmaceutical company dedicated to developing transformative therapies for patients with devastating diseases. Our genome-editing platform,platform includingis based on our novel chRDNA (CRISPR hybrid RNA-DNA, or “chRDNA,” pronounced “chardonnay”) genome-editing technology, which enables more precise genome editing of allogeneic cell therapies.
Our allogeneic chimeric antigen receptor (“CAR”) -T (“CAR-T”) cell therapy product candidates are manufactured in advance with cells from healthy donors, with the goal of enabling broad patient access, rapid patient treatment, and increased manufacturing scale. Our allogeneic CAR-T cell therapy product candidates in clinical development are directed at established cell surface targets against which autologous CAR-T cell therapeutics have already demonstrated clinical proof of concept, including CD19 and B cell maturation antigen (“BCMA”), as well as targets such as C-type lectin-like molecule-1 (“CLL-1”). We use our chRDNA technologiestechnology to armor our cell therapiestherapy product candidates through multiple genome-editing strategies, such as checkpoint disruption,disruption and immune cloaking, or a combination of these two strategies, to enhance allogeneic CAR-T cell therapy activity against diseases.hematologic malignancies.
We are advancing ourtwo pipeline ofclinical-stage allogeneic CAR-T cell therapiestherapy withproduct thecandidates following four clinical development programs targetingfor the treatment of patients with hematologic malignancies and autoimmune diseases:
•Vispacabtagene regedleucel (“vispa-cel,” formerly CB-010): an allogeneic anti-CD19 CAR-T cell therapy,therapy beingthat has been evaluated in patients with relapsed or refractory B cell non-Hodgkin lymphoma (“r/r B-NHL”) in our ANTLER phase 1 clinical trial
•CB-010: also being evaluated in patients with lupus nephritis (“LN”) and in patients with extrarenal lupus (“ERL”) in our GALLOP phase 1 clinical trial
•CB-011: an allogeneic anti-BCMA CAR-T cell therapy, being evaluated in patients with relapsed or refractory multiple myeloma (“r/r MM”) in our CaMMouflage phase 1 clinical trial
•CB-012CB-011: an allogeneic anti-CLL-1anti-BCMA CAR-T cell therapy,therapy that is being evaluated in patients with relapsed or refractory acutemultiple myeloid leukemiamyeloma (“r/r AMLMM”) in our AMpLifyCaMMouflage phase 1 clinical trial Since our founding in 2011, we have devoted substantially all of our resources to organizing and staffing, business planning, raising capital, expanding our genome-editing platform technologies, developing our product candidates and building our pipeline, creating and maintaining our intellectual property portfolio, and establishing arrangements with third parties for the manufacture, testing, and clinical trial evaluations of our product candidates. We do not have any products approved for commercial sale and have not generated any revenue from product sales. We have incurred operating losses since commencement of our operations.
•advanceprogress our clinical trials for our CAR-Tvispa-cel and CB-011 cell therapy product candidatescandidates, particularly as we advance vispa-cel in our planned pivotal clinical trial;
•continue our current research programs and our preclinical and clinical development of our other current product candidates and any other product candidates we identify and choose to develop;
•seek to identify additional research programs and additional product candidates;
•further develop our genome-editing technologies;
•acquire or in-license intellectual property orproperty, new technologiestechnologies, and/or additional product candidates;
•seek regulatory and marketing approvals for anyour ofvispa-cel ourand CB-011 product candidates thatif successfully completeour clinical trials,trials ifare anysuccessful;
•expand manufacturing capabilities and supply chain capacity for our vispa-cel and CB-011 product candidates;
•experience any delays, challenges, or other issues associated with any of the above, including the failure of clinical trials meeting endpoints, unanticipatedgeneration preclinical results, orof clinical trial data subject to differing interpretations, or the occurrence of potential safety issues or other development or regulatory challenges;
•make royalty, milestone, or other payments under current, and any future, in-license or assignment agreements with third parties;
•continue to operate as a public company, including defending against any future class action securities litigation.
We do not own or operate any manufacturing facilities. We use multiple contract manufacturing organizations (“CMOs”) to individually manufacture, under current good manufacturing processes, our chRDNA guides, Cas9 and Cas12a proteins, plasmids, and adeno-associated virus serotype 6 (“AAV6”) vectors used in the manufacture of our cell therapy product candidates as well as the CAR-T cell therapy product candidates themselves. We expect to continue to rely on our CMOs for manufacturing our preclinical study and clinical trial materials, and most of these CMOs have capabilities for commercial manufacturing. Additionally, we may decide to build our own manufacturing facility in the future to provide greater flexibility and control over our clinical or commercial manufacturing needs.
Because of the numerous risks and uncertainties associated with therapeutic product development, we may never achieve profitability and, unless and until we are able to develop and commercialize our product candidates, we will need to continue to raise additional capital. Until such time, if ever, that we can generate significant revenue from product sales, if ever, we expect to finance our operations through equity offerings (including our at-the-market equity offering program), debt financings, collaborations andnew strategic alliances,collaborations, structured or other non-dilutive financings, licensing arrangements, and/or other sources. ThereWe arecannot noprovide assurancesany assurance that we will be successful in obtaining an adequate level of financing to support our business plans as needed on acceptable terms, or at all. If we raise additional funds through collaborations, new strategic alliances,collaborations, structured or other non-dilutive financings, or licensing arrangements with third parties, we may have to relinquish valuable rights to our intellectual property, future revenue streams, research programs, or product candidates or grant licenses on terms that may not be favorable to us. If we are unable to raise capital as and when needed or on attractive terms, we may have to significantly delay, reduce, or discontinue the development and commercialization of our product candidates or scale back or terminate our pursuit of new in-licenses and acquisitions.
To date, all of our revenue consists of licensing and collaboration revenue earned from collaboration and/or licensing agreements entered into with third parties, including related parties. Under these agreements, we license rights to certain intellectual property controlled by us. The terms of these arrangements typically include payments to us of one or more of the following: nonrefundable, upfront license fees or exclusivity fees; annual maintenance fees; regulatory and/or commercial milestone payments; research and development payments; and royalties on the net sales of products and/or services. Each of these payments results in licensing and collaboration revenue. Revenue under such licensing and collaboration agreements was $10.0$11.2 million and $34.5$10.0 million for the years ended December 31, 2024,2025, and 2023,2024, respectively. See NoteNotes 45 and 7 to our consolidated financial statements included elsewhere in this Annual Report on Form 10-K for additional information.
Our research and development expenses consist of internal and external expenses incurred in connection with the development of our product candidates and our genome-editing platform technologies, and our in-licensing, assignment, and other third-party agreements.
•costs incurred in connection with the preclinical and clinical development and manufacturing of our product candidates, including under agreements with CMOs, suppliers, clinicalcontract research organizations (“CROs”), and clinical sites; and
•other research and development costs, including laboratory materials andlab supplies, and consulting services.
We expense research and development costs as incurred. Costs of certain activities are recognized based on an evaluation of the progress to completion of specific tasks. However, payments made prior to the receipt of goods or services that will be used or rendered for future research and development activities are deferred and capitalized as prepaid expenses and other current assets on our consolidated balance sheets. The capitalized amounts are recognized as expenses as the goods are delivered or as related services are performed. We separately track certain external costs on a program-by-program basis; however, we do not track costs that are deployed across multipleour programs. We do not allocate internal costs as several of our departments support multipleour programs and our payroll and other personnel expenses are not tracked on a program-by-program basis.
Research andClinical development activities are central to our business model. Product candidates in later stages of clinical development generally have higher development costs than those in earlier stages of clinical development, primarily due to the increased size and duration of later-stage clinical trials. We expect that our research and development expenses will increase substantially for the foreseeable future as we continue to implement our business strategy; advance our product candidates through clinical trials and commercialization; conduct preclinicaltranslational studiesresearch andto clinical trials forsupport our other product candidates; seek regulatory approvals for anyour product candidates that successfully complete clinical trials; expand our research and development efforts and incur expenses associated with hiringhire additional personnel to support our research andclinical development efforts; and seek to identify, in-license, acquire, and/or develop additional product candidates.efforts.
The successful development of our CAR-T product candidates, as well as other potential future product candidates,candidates is highly uncertain. Accordingly, at this time, we cannot reasonably estimate or know the nature, timing, and costs of the efforts that will be necessary to complete the development of our product candidates. We are also unable to predict when, if ever, we will generate revenue and material net cash inflows from the commercialization and sale of any of our product candidates for which we may obtain marketing approval. We may never succeed in achieving regulatory approval for any of our product candidates. The duration, costs, and timing of preclinical studies, clinical trials, and development of our product candidates will depend on a variety of factors, including:
•completion of preclinical studies;
•timely clearance of IND applications to initiate clinical trials of new product candidates;
•entry into collaborations to further the development of our product candidates or for the development of new product candidates;
•successful development of our internal process development and transfer to larger-scale facilitiesCMOs;
•establishment and maintenance of agreements with CMOs and suppliers for clinical and commercial supplies and scaling up manufacturing processes and capabilities to support our clinical trials;
•grant of nonpatent regulatory exclusivity for our product candidates;
•establishment of sales, marketing, and distribution capabilities necessary for commercialization of our product candidates if and when approved, whether by us or in collaboration with third parties;
•acceptance of our product candidates, if and when approved by the applicable regulatory authorities, by patients, the medical community, and third-party payors;
Our general and administrative expenses consist primarily of personnel-related costs, intellectual property costs, consulting costs, and allocated overhead, including rent, equipment depreciation, and utilities. Personnel-related costs consist of salaries, benefits, and stock-based compensation expense for our general and administrative personnel. Intellectual property costs include expenses for filing, prosecuting, and maintaining patents and patent applications, including certain patents and patent applications that we license from third parties. We are entitled to receive reimbursement from third parties of a portion of the costs for filing, prosecuting, and maintaining certain patents and patent applications. We accrue for these reimbursements as the respective expenses are incurred and classify such reimbursements as a reduction of general and administrative expenses. During each of the years ended December 31, 2024,2025, and 2023,2024, we recorded $1.2 million and $1.5 million, respectively, of patent cost reimbursements as a reduction to general and administrative expenses.
We expect that our general and administrative expenses will increase in the future as a result of expandingif our operations,clinical includingtrials hiringare personnel,successful preparingand if we prepare for potential commercialization of our product candidates, and additional facility occupancy costs, as well as other expenses necessary to support the growth and operations of a clinical-stage public company.company with late-stage clinical programs and potential commercial products.
Impairment Charges
Impairment charges consist of charges related to the strategic pipeline prioritization with workforce and cost reduction initiatives announced on April 24, 2025, and include impairment of our leasehold improvements, right of use assets, and lab equipment. See Note 15 to our consolidated financial statements included elsewhere in this Annual Report on Form 10-K for additional information.
Other Income (Expense) Income
Other income (expense) income consists primarily of impairment of an equity investment, interest income earned on cash and marketable securities and the change in fair value of the Memorial Sloan Kettering Cancer Center (“MSKCC”) success payments liability under ourthe now-terminated Exclusive License Agreement, dated November 13, 2020, with MSKCC (as amended, “MSKCC Agreement”).
Licensing and collaboration revenue decreased by $24.5 million to $10.0 million for the year ended December 31, 2024, from $34.5 million for the year ended December 31, 2023. This decrease primarily relates to a $24.8 million decrease in revenue recognized under the now-terminated Collaboration and License Agreement (as amended, “AbbVie Agreement”) with AbbVie Manufacturing Management Unlimited Company (“AbbVie”). In connection with the termination of the AbbVie Agreement, we recognized the remaining deferred revenue of $20.8 million during the year ended December 31, 2023.
(1)Pfizer ceased to be a related party as of December 31, 2025.
Licensing and collaboration revenue increased by $1.2 million to $11.2 million for the year ended December 31, 2025, from $10.0 million for the year ended December 31, 2024. This increase is primarily due to a $2.8 million increase related to other licensees, which was partially offset by a $1.6 million decrease in revenue related to the issuance of additional shares of convertible preferred stock received as consideration to us under the Exclusive License Agreement for Veterinary Therapeutics (as amended, “Edge chRDNA License Agreement”) with Edge Animal Health (“Edge”) in the year ended December 31, 2024.
Research and development expenses increaseddecreased by $18.1$20.7 million to $109.4 million for the year ended December 31, 2025 from $130.2 million for the year ended December 31, 2024 from $112.1 million for the year ended December 31, 2023.2024. This increasedecrease was primarily relateddue to (i) ana increasedecrease of $6.6$8.8 million in other research and development expenses to advance preclinical and clinical development for our programs, as well as other consulting servicesprimarily related to researchthe reduction in workforce and development;strategic pipeline prioritization, (ii) ana increasedecrease of $4.1$5.5 million in personnel-related expenses, including an increase in salary and benefit expense of $2.5 million, an increase in stock-based compensation expense of $1.1 million, and $0.5 million of one-time expenses associatedrelated withto the reduction in forceworkforce thatand occurredstrategic duringpipeline the third quarter of 2024;prioritization, (iii) a net increasedecrease of $3.5$3.1 million in external CMO and CRO activities for our clinical CAR-T cell therapy product candidates,activities, driven by (a) an increase of $9.8 million in CRO activities for clinical trials; partially offset by (b) a decrease of $6.3(a) $3.5 million due to timing of CMO activities, and an increase of (b) $0.4 million in CRO activities; for our clinical trials, (iv) ana increasedecrease of $2.1 million in expenses related to licenses, sublicensing revenue, and milestones;milestones, and (v) ana increasedecrease of $1.8$1.2 million in other facilities and allocated expenses.
General and administrative expenses increaseddecreased by $8.0$8.5 million to $37.9 million for the year ended December 31, 2025, from $46.5 million for the year ended December 31, 2024, from $38.5 million for the year ended December 31, 2023.2024. This increasedecrease was primarily related to increasesa decrease of $5.7$4.7 million in legal and other service-related expenses, including $3.9 million of costs related to the accrual of a securities class action litigation settlement,settlement expense in 2024 and $2.3a decrease of $3.3 million in personnel-related expenses, including an increase in salary and benefit expense of $0.4 million, an increase in stock-based compensation expense of $1.8 million, and $0.1 million of one-time expenses associatedrelated withto the reduction in forceworkforce thatand occurredstrategic duringpipeline the third quarter of 2024.prioritization.
Impairment Charges
Impairment charges consist of charges related to the strategic pipeline prioritization with workforce and cost reduction initiatives announced on April 24, 2025, and include impairment of our leasehold improvements, right of use assets, and lab equipment. See Note 15 to our consolidated financial statements included elsewhere in this Annual Report on Form 10-K for additional information.
Total Other (Expense) Income
Total other (expense) income increaseddecreased by $3.3$17.8 million for the year ended December 31, 2024,2025, as compared to the year ended December 31, 2023.2024.
Impairment of equity investment was $9.2 million related to our equity investment in Edge for the year ended December 31, 2025, compared to zero for the year ended December 31, 2024. See Note 3 to our consolidated financial statements included elsewhere in this Annual Report on Form 10-K for additional information.
Other income, net decreased by $8.7 million for the year ended December 31, 2025, compared to December 31, 2024. This decrease was primarily related to a $7.2 million decrease in interest income earned from marketable securities and a $1.4 million decrease in gain recognized related to the change in the fair value of the MSKCC success payments liability.
We recognized a gain related to the change in the fair value of the MSKCC success payments liability in the amount of $2.2 million for the year ended December 31, 2024. We recognized a loss related to the change in the fair value of the MSKCC success payments liability in the amount of $1.3 million for the year ended December 31, 2023.
Benefit From Income TaxTaxes
An income tax benefit of $0.6 million was recognized for the year ended December 31, 2025, which was primarily related to deferred federal and state taxes. An income tax benefit of less than $0.1 million was recognized for the year ended December 31, 2024, which was primarily related to deferred state taxes. An income tax expense of $0.2 million was recognized for the year ended December 31, 2023, which was primarily related to deferred state taxes.
Since our inception through December 31, 2024,2025, we have raised an aggregate net proceeds of $836.2$849.0 million to fund our operations through our initial public offering (“IPO”); sales of convertible preferred stock; a follow-on public offering; proceeds from our licensing, licensing and collaboration, service, and patent assignment agreements, including sales of Intellia stock; private placements; at-the-market equity offerings; and government grants.
Shelf Registration Statements
On August 9, 2022, we filed a universal shelf registration statement on Form S-3 (“2022 Shelf Registration Statement”) with the U.S. Securities and Exchange Commission (“SEC”),SEC, which allowsallowed us to,to sell, from time to time, sell up to $400.0 million of common stock, preferred stock, debt securities, warrants, rights, or units comprised of any combination thereof (including the $100.0 million of common stock reserved under the 2022 Shelf Registration Statement for our at-the-market equity offering program described below). The Shelf Registration Statement was declared effective by the SEC on August 16, 2022, and will expire after three years.
On May 8, 2025, in anticipation of the expiration of the 2022 Shelf Registration Statement on August 16, 2025, we filed a new shelf registration statement on Form S-3 (“2025 Shelf Registration Statement”), which was declared effective by the SEC on May 14, 2025. Upon the effectiveness of the 2025 Shelf Registration Statement, the offering of securities under the 2022 Shelf Registration Statement was deemed terminated. Pursuant to the 2025 Shelf Registration Statement, we may, from time to time, sell up to $300.0 million of common stock, preferred stock, debt securities, warrants, rights, or units comprised of any combination thereof (including the $100.0 million of common stock reserved under the 2025 Shelf Registration Statement for our at-the-market equity offering program described below). As of December 31, 2025, we had $295.7 million available for sale under the 2025 Shelf Registration Statement.
On August 9, 2022, we entered into an at-the-market Open Market Sale AgreementSM (the “ATM Sales Agreement”) with Jefferies LLC (“Jefferies”), pursuant to which, uponon the terms and subject to the conditions and limitations set forth in the ATM Sales Agreement, we may, from time to time, inwe ourcould solehave discretion, issueissued and sell,sold, through Jefferies, acting as sales agent, up to $100.0 million of our shares of common stock,stock by any method permitted by law deemed to be an “atunder the market2022 offering”Shelf asRegistration definedStatement. in Rule 415(a)(4) of the Securities Act of 1933, as amended (“Securities Act”). Jefferies uses commercially reasonable efforts consistent with its normal sales and trading practices to sell shares from time to time, based upon our instructions (including any price or size limits or other customary parameters or conditions we may impose). We pay Jefferies a commission equal to 3.0% of the aggregate gross proceeds of any shares sold through Jefferies pursuant toUnder the ATM Sales Agreement.Agreement Throughand Decemberthe 31,2022 2024,Shelf Registration Statement, we issued and sold an aggregate of 3,588,696 shares of our common stock under the ATM Sales Agreement at an average price per share of $4.71 for aggregate gross proceeds of $16.9 million ($16.2 million net of offering expenses).
With the effectiveness of the 2025 Shelf Registration Statement, we refreshed our at-the-market equity offering program under the ATM Sales Agreement. We may, from time to time, sell and issue shares of our common stock, through Jefferies as sales agent under the ATM Sales Agreement, having an aggregate offering price of up to $100.0 million in gross proceeds under the 2025 Shelf Registration Statement, by any method permitted by law deemed to be an “at the market offering” as defined in Rule 415(a)(4) of the Securities Act of 1933, as amended (“Securities Act”). Jefferies has agreed to use commercially reasonable efforts consistent with its normal sales and trading practices to sell shares from time to time, based on our instructions (including any price or size limits or other customary parameters or conditions we may impose).
What changed in the latest 10-Q
Risk Factors
There have been no material changes to the Risk Factors previously disclosed in Item 1A. to Part I of our Form 10-K. The risks described in our Form 10-K are not the only risks facing our company. Additional risks and uncertainties not currently known to us or that we currently deem to be immaterial also may materially adversely affect our business, financial condition, and/or operating results.
No wording changes found in this section.
Full comparison: every changed paragraph (0)
Management's Discussion & Analysis (MD&A)
New heading “Comparison of the Six Months Ended June 30, 2026, and June 30, 2025”
New heading “Licensing and Other Third-party Revenue”
New heading “Research and Development Expenses”
New heading “General and Administrative Expenses”
New heading “Total Other Income (Expense)”
Largest changes
“Comparison of the Six Months Ended June 30, 2026, and June 30, 2025”see in full comparison
see in full comparisonWeTo our knowledge, vispa-cel is the first clinical-stage allogeneic CAR-T cell therapy with a genome-edited knockout of the PDCD1 gene to prevent PD-1 expression on the CAR-T cell surface. On May 7, 2026, we announced that we reached alignment with the FDAregardingon the design of ANTLER-3, ourvispa-celplanned pivotal phase 3 clinical trialdesign following interactions to date with the agency enabled by the RMAT designationforvispa-cel.We plan to evaluate vispa-cel in thevispa-cel. ANTLER-3trial,will be a randomized, controlledpivotal phase 3clinical trialinexpected to enroll approximately 250 CD19-naïve second-line (“2L”) large B cell lymphoma (“LBCLCD19-naïve”) patients who are not eligible for transplant andwho arenot candidatesfor,orwho arenot eligiblefor,for autologous CAR-T cell therapy based on access challenges or medical criteria, including the urgent need forurgenttherapy. Patientsrandomized toin thestudyinvestigational armwouldwill receive a single dose ofvispa-cel80x106 viable CAR-T cells following a lymphodepletion (“LD”) regimen of cyclophosphamide atthe60RP2Dmg/kg/dayfollowingforLDtwo days andpatientsfludarabinerandomizedatto25 mg/m²/day for five days. Patients in the comparator armwouldwill be treated withthean investigator’s choice of a standard-of-careregimen, such as polatuzumab vedotin (“Pola”), bendamustine,regimen: rituximab (”“R”)(“Pola-BR”); R,, gemcitabine, and oxaliplatin (“R-GemOx”);Pola-R-GemOxpolatuzumab vedotin (“Pola”) and R-GemOx (“Pola-RGO”); or tafasitamab andlenalidomide,lenalidomide.with crossoverCrossover to the vispa-cel arm will be permitted after progressive disease. The primary endpointwouldwill bePFS. Secondary endpoints would be ORR, overallprogression-free survival (“OSPFS”), event-free survival (“EFS”), duration of response (“DoR”), duration of CR, quality of life (“QoL”), and safety.. Clinical trial siteswouldwill include both academic and sophisticated community centers in the United States and globally.
Full comparison: every changed paragraph (53)
You should read the following discussion and analysis of our financial condition and results of operations together with our unaudited condensed consolidated financial statements and the related notes included in Part I, Item 1, of this Quarterly Report on Form 10-Q for the fiscal quarter ended MarchJune 31,30, 2026 (“Form 10-Q”) and with the audited consolidated financial statements and the related notes for the fiscal year ended December 31, 2025, included in our Annual Report on Form 10-K (“Form 10-K”) filed with the U.S. Securities and Exchange Commission (“SEC”) on March 5, 2026.
This Form 10-Q contains “forward-looking” statements within the meaning of Section 27A of the Securities Act of 1933, as amended (“Securities Act”), and Section 21E of the Securities Exchange Act of 1934, as amended (“Exchange Act”). All statements, other than statements of historical facts, contained in this Form 10-Q are forward-looking statements, including statements regarding our business strategy, plans, and objectives; expectations regarding our clinical-stage CAR-T cell therapy product candidates, including our expectations about the development timelines for such product candidates; the expected timing of disclosure of clinical data; expectations regarding the safety, efficacy, and potential advantages of our CAR-T cell therapy product candidates; expectations about our future regulatory filings and interactions with regulatory authorities, including expectations about ANTLER-3, our planned pivotal phase 3 pivotalclinical trial design for vispa-celvispacabtagene regedleucel (“vispa-cel,” formerly CB-010); our results of operations and financial position; plans and objectives of management for future operations; and the like. In some cases, you can identify forward-looking statements by terms such as “may,” “will,” “should,” “expect,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplate,” “believe,” “estimate,” “predict,” “potential,” or “continue” or the negative of these terms or other similar expressions, although not all forward-looking statements contain these words.
•Vispacabtagene regedleucel (“vispa-cel,” formerly CB-010)Vispa-cel: an allogeneic anti-CD19 CAR-T cell therapy that has been evaluated in our multicenter, open-label ANTLER phase 1 clinical trial in patients with relapsed or refractory B cell non-Hodgkin lymphoma (“r/r B-NHL”) in our ANTLER phase 1 clinical trial
•CB-011: an allogeneic anti-BCMA CAR-T cell therapy that is being evaluated in our multicenter, open-label CaMMouflage phase 1 clinical trial in patients with relapsed or refractory multiple myeloma (“r/r MM”) in our CaMMouflage phase 1 clinical trial Vispa-cel has received regenerative medicine advanced therapy (“RMAT”) designation for relapsed or refractory large B cell lymphoma (“r/r LBCL”), fast track designation for r/r B-NHL, and orphan drug designation for follicular lymphoma (“FL”) from the U.S. Food and Drug Administration (“FDA”). CB-011 has received RMAT, fast track, and orphan drug designations for r/r MM from the FDA.
To our knowledge, vispa-cel is the first clinical-stage allogeneic CAR-T cell therapy with a genome-edited knockout of the PDCD1 gene to prevent PD-1 expression on the CAR-T cell surface. On November 3, 2025, we announced that, as of the September 2, 2025, safety data cutoff date, 84 patients with r/r B-NHL had been enrolled in the ANTLER phase 1 clinical trial. We selected a single dose of 80x10⁶ viable CAR-T cells following lymphodepletion (“LD”) with 60 mg/kg/day for two days of cyclophosphamide and 25 mg/m2/day for five days of fludarabine as the recommend phase 2 dose (“RP2D”).
By leveraging our allogeneic CAR-T cell therapy clinical data set of more than 140 patients dosed across multiple clinical trials, we identified key factors that we believe are linked to successful patient outcomes, and we have incorporated these learnings to develop an optimized vispa-cel profile. Two of the key attributes of the optimized vispa-cel profile are donor age (young donors drive enhanced outcomes relative to older donors) and partial human leukocyte antigen (“HLA”) matching (matching two or more alleles correlates with enhanced patient outcomes). As of the September 2, 2025, 84 patients had been treated in our ANTLER phase 1 clinical trial, of which 35 CD19-naïve LBCL patients received vispa-cel with the optimized profile. As of the September 29, 2025 efficacy data cutoff, the results for the 35-patient optimized profile cohort included an 86% overall response rate (“ORR”), a 63% complete response (“CR”) rate, and 53% progression-free survival (“PFS”) at 12 months. Median follow up for the optimized profile cohort was 11.8 months.
Vispa-cel is generally well tolerated. As of the September 2, 2025, safety data cutoff date, treatment emergent adverse events (“TEAEs”) at any grade in 25% or greater of all 84 patients who had received vispa-cel were thrombocytopenia (62%), cytokine release syndrome (“CRS”) (55%), anemia (52%), neutropenia (39%), hypokalemia (26%), and leukopenia (26%). In the optimized profile cohort (N=35), there were no cases of graft versus host disease (“GvHD”) or grade 3 or greater immune effector cell-associated neurotoxicity syndrome (“ICANS”), 1% of patients experienced grade 3 or greater CRS, and 28% (out of 80 patients) experienced prolonged cytopenias.
WeTo our knowledge, vispa-cel is the first clinical-stage allogeneic CAR-T cell therapy with a genome-edited knockout of the PDCD1 gene to prevent PD-1 expression on the CAR-T cell surface. On May 7, 2026, we announced that we reached alignment with the FDA regardingon the design of ANTLER-3, our vispa-celplanned pivotal phase 3 clinical trial design following interactions to date with the agency enabled by the RMAT designation for vispa-cel.We plan to evaluate vispa-cel in thevispa-cel. ANTLER-3 trial,will be a randomized, controlled pivotal phase 3 clinical trial inexpected to enroll approximately 250 CD19-naïve second-line (“2L”) large B cell lymphoma (“LBCL CD19-naïve”) patients who are not eligible for transplant and who are not candidates for, or who are not eligible for,for autologous CAR-T cell therapy based on access challenges or medical criteria, including the urgent need for urgent therapy. Patients randomized toin the studyinvestigational arm wouldwill receive a single dose of vispa-cel80x106 viable CAR-T cells following a lymphodepletion (“LD”) regimen of cyclophosphamide at the60 RP2Dmg/kg/day followingfor LDtwo days and patientsfludarabine randomizedat to25 mg/m²/day for five days. Patients in the comparator arm wouldwill be treated with thean investigator’s choice of a standard-of-care regimen, such as polatuzumab vedotin (“Pola”), bendamustine,regimen: rituximab (”“R”)(“Pola-BR”); R,, gemcitabine, and oxaliplatin (“R-GemOx”); Pola-R-GemOxpolatuzumab vedotin (“Pola”) and R-GemOx (“Pola-RGO”); or tafasitamab and lenalidomide,lenalidomide. with crossoverCrossover to the vispa-cel arm will be permitted after progressive disease. The primary endpoint wouldwill be PFS. Secondary endpoints would be ORR, overallprogression-free survival (“OSPFS”), event-free survival (“EFS”), duration of response (“DoR”), duration of CR, quality of life (“QoL”), and safety.. Clinical trial sites wouldwill include both academic and sophisticated community centers in the United States and globally.
On June 11, 2026, we announced that 85 patients with r/r B-NHL were treated in the ANTLER phase 1 clinical trial. As of the March 6, 2026, data cutoff date, 27 2L LBCL patients received a single dose of vispa-cel manufactured from a donor younger than 30 years old with at least two matched human leukocyte antigen (“HLA”) alleles between patient and donor. This 27-patient subgroup best represents the treatment regimen and patient population for ANTLER-3. The results for this subgroup included an 82% overall response rate (“ORR”), a 67% complete response (“CR”) rate, and 17.1-month median PFS. Vispa-cel is generally well tolerated. In the 27-patient subgroup, there were no reports of graft-versus-host disease (“GvHD”) or grade 3 or higher immune effector cell-associated neurotoxicity syndrome (“ICANS”), and there was one (4%) grade 3 or higher cytokine release syndrome (“CRS”). Other adverse events of special interest included six (22%) grade 3 or higher infections, five (21%; 5/24) grade 3 or higher prolonged cytopenias, and one (4%) grade 3 or higher immune effector cell-associated hemophagocytic lymphohistiocytosis-like syndrome (“IEC-HS”). In the 27-patient subgroup, one vispa-cel-related death occurred due to IEC-HS and one possibly related death occurred due to progressive multifocal leukoencephalopathy.
To our knowledge, CB-011 is the first clinical-stage allogeneic CAR-T cell therapy incorporating an immune cloaking approach that includes both the removal of the endogenous beta-2 microglobulinbeta-2-microglobulin (“B2M”) protein and insertion of a beta-2-microglobulin–human-leukocyte-antigen-E–peptide transgene (“B2M–HLA-E”). On November 3, 2025, we announced results from the dose escalation portion of our ongoing CaMMouflage phase 1 trial evaluating CB-011 in r/r MM patients. Forty-eight fourth-line or later (“4L+”) patients were enrolled in the dose escalation portion of theour trial;CaMMouflage enrollmentphase in1 clinical trial, which evaluated two different LD regimens and multiple CAR-T cell dose escalationlevels. isThe completed.eligibility Patientscriteria required that patients had been treated with three or more prior lines of therapy including a proteasome inhibitor, an immunomodulatory drug, and an anti-CD38 antibody. PriorPatients who received treatment with a BCMA-targeted therapy, but not an autologous CAR-T cell therapytherapy, wasmore notthan permittedthree and no BCMA-targeted therapy was permitted within themonths prior threeto months.enrollment Wein evaluateddose twoescalation differentwere LDpermitted. regimensThe andCB-011 multiplerecommended dose for expansion (“RDE”) is 450x106 viable CAR-T cellcells dose levels. Thirty-five patients were treated withfollowing an LD regimen of 500 mg/m2 cyclophosphamide and 30 mg/m2 fludarabine daily for three daysdays. (“In the selected LD regimen”). A single dose escalation portion of CB-011our precededCaMMouflage byphase 1 clinical trial, 12 patients were treated with the selected LD regimen resulted in responses at all dose levels evaluated (150x106 viable CAR-T cells, N=6; 300x106 viable CAR-T cells, N=13; 450x106 viable CAR-T cells, N=13; and 800x106 viable CAR-T cells, N=3).RDE.
On June 11, 2026, we announced longer follow-up data from the dose escalation portion of our CaMMouflage clinical trial. As of the May 26, 2026, efficacy data cutoff date, the 12-patient, BCMA-naïve cohort treated with the RDE had the following outcomes: an ORR of 92%, a complete response or stringent complete response (“≥CR”) rate of 83%, and 91% of evaluable patients achieved minimal residual disease (“MRD”) negativity (≤10-5). Fifty percent of patients were in ≥CR at 15 months. The median follow-up for these 12 patients was 17.7 months. As of the April 20, 2026, safety data cutoff date, CB-011 showed a manageable safety profile across all patients with no cases of GvHD, immune effector cell-associated enterocolitis (“IEC-EC”), parkinsonism, or cranial nerve palsies (N=48). In all patients treated with the selected LD regimen (N=35), there was one CB-011-related death due to immune effector cell-associated hematotoxicity and three unrelated deaths due to pneumonia, respiratory syncytial virus, and respiratory acidosis, respectively. In the 12-patient BCMA-naïve RDE cohort, there were no reports of grade 3 or higher ICANS and one (8%) grade 3 or higher CRS. Other adverse events of special interest in the RDE cohort included three (25%) grade 3 or higher infections, one (8%) grade 3 or higher IEC-HS, and five (42%; 5/12) grade 3 or higher prolonged cytopenias. We initiated the dose expansion portion of the CaMMouflage phase 1 clinical trial in late 2025, which is ongoing and includes enrollment of BCMA-naive and BCMA-exposed patient cohorts.
CB-011 had a manageable safety profile across all dose levels and LD regimens (N=48), with no cases of GvHD, immune effector cell-associated enterocolitis (“IEC-EC”), parkinsonism, or cranial nerve palsies as of the September 24, 2025 data cutoff date. TEAEs in 25% or greater of all patients treated with CB-011 following the selected LD regimen (N=35) were as follows: neutropenia (80%), anemia (60%), thrombocytopenia (49%), infections (49%), dizziness (31%), CRS (31%), fatigue (31%), leukopenia (29%), decreased appetite (29%), constipation (26%), and pyrexia (26%). Notable adverse events in the recommend dose for expansion (“RDE”) cohort as of the data cutoff date included one CB-011-related grade 5 immune effector cell-associated hematotoxicity (“ICAHT”) on day 90, one grade 5 pneumonia not related to CB-011 on day 50, and one grade 4 CB-011-related Guillain-Barré Syndrome on day 129, which is resolving. In the cohort evaluating the 300x106 viable CAR-T cell dose level following the selected LD regimen, there was one grade 5 respiratory syncytial virus not related to CB-011 on day 73. Prophylactic measures for cytopenias and infections and early intervention for certain immune effector cell disorders have been successfully implemented in our clinical protocol.
We also announced on November 3, 2025, that we have selected the 450x106 viable CAR-T cell dose with the selected LD regimen as our RDE. As of the September 24, 2025, data cutoff date, the 12-patient, BCMA-naïve cohort treated with the selected LD regimen and a single dose of 450x106 viable CAR-T cells had the following outcomes: an ORR of 92%, a CR/stringent CR (“sCR”) rate of 75%, and 91% of evaluable patients achieved minimal residual disease (“MRD”) negativity (≤10-5). Seven of the 12 patients remained on study as of the data cutoff date in very good partial response (“VGPR”) or better six months or longer following receipt of a single dose of CB-011. The median follow up for these 12 patients was 8.3 months. We initiated the dose expansion portion of the CaMMouflage phase 1 clinical trial in late 2025, which includes enrollment of BCMA-naive and BCMA-exposed patient cohorts.
Our net losses for the three months ended MarchJune 31,30, 2026, and MarchJune 31,30, 2025, were $25.1$24.3 million and $40.0$54.1 million, respectively. Our net losses for the six months ended June 30, 2026, and June 30, 2025, were $49.4 million and $94.1 million, respectively. We had an accumulated deficit of $621.6$645.9 million as of MarchJune 31,30, 2026. Our net losses and operating losses may fluctuate from quarter to quarter and year to year depending primarily on the timing of expenses associated with our clinical trials and development of our product candidates. We anticipate that our expenses will increase substantially as we:
•progress our clinical trials for our vispa-cel and CB-011 CAR-T cell therapy product candidates, particularly as we advance vispa-cel in ANTLER-3, our planned pivotal phase 3 clinical trial;
To date, all of our revenue has been earned from licensing, collaboration, and other third-party agreements, including agreements with related parties. Under these agreements, we may license rights to certain intellectual property controlled by us. The terms of these arrangements typically include payments to us of one or more of the following: nonrefundable, upfront license fees or exclusivity fees; annual maintenance fees; regulatory and/or commercial milestone payments; research and development payments; and royalties on the net sales of products and/or services. Each of these payments results in licensing and other revenue. Revenue under such agreements was $2.4$1.5 million and $2.7 million for each of the three-monththree periodsmonths ended MarchJune 31,30, 2026, and MarchJune 31,30, 2025.2025, respectively, and $3.9 million and $5.0 million for the six months ended June 30, 2026, and June 30, 2025, respectively. See Notes 5 and 7 to our unaudited condensed consolidated financial statements included elsewhere in this Form 10-Q for additional information.
•entry into new collaborationscollaboration or other agreements to further the development of our CAR-T cell therapy product candidates;
Our general and administrative expenses consist primarily of personnel-related costs, intellectual property costs, consulting costs, and allocated overhead, including rent, equipment depreciation, and utilities. Personnel-related costs consist of salaries, benefits, and stock-based compensation expense for our general and administrative personnel. Intellectual property costs include expenses for filing, prosecuting, and maintaining patents and patent applications, including certain patents and patent applications that we license from third parties. We are entitled to receive reimbursement from third parties of a portion of the costs for filing, prosecuting, and maintaining certain patents and patent applications. We accrue for these reimbursements as the respective expenses are incurred and classify such reimbursements as a reduction ofin general and administrative expenses.
Impairment charges consist of charges related to the strategic pipeline prioritization with workforce and cost reduction initiatives announced on April 24, 2025, and include impairment of our leasehold improvements, right of use assets, and lab equipment. See Note 13 to our unaudited condensed consolidated financial statements included elsewhere in this Form 10-Q for additional information.
Other Income (Expense)
Other income (expense) consists primarily of impairment of an equity investment and interest income earned on cash and marketable securities.
Comparison of the Three Months Ended MarchJune 31,30, 2026, and MarchJune 31,30, 2025
Licensing and other third-party revenue increaseddecreased by less than $0.1$1.2 million to $2.4$1.5 million for the three months ended MarchJune 31,30, 2026, from $2.4$2.7 million for the three months ended MarchJune 31,30, 2025. This decrease primarily relates to the recognition of a $1.0 million milestone payment under a license agreement for the three months ended June 30, 2025, with no comparable milestone revenue recognized for the three months ended June 30, 2026.
Research and development expenses decreased by $14.9$8.8 million to $20.6$18.9 million for the three months ended MarchJune 31,30, 2026, from $35.5$27.7 million for the three months ended MarchJune 31,30, 2025. This decrease was primarily related to decreases of (i) $7.7$3.3 million in external CMO and CRO activities for our clinical CAR-T cell therapy product candidates, driven by decreases of (a) $4.5$2.4 million in CRO activities for our clinical trials and (b) $3.2$0.9 million due to timing of CMO activities,activities; (ii) $3.0$2.6 million in personnel-related expenses related to the reduction in workforce and strategic pipeline prioritization; (iii) $2.0 million in other research and development expenses primarily related to the reduction in workforce and strategic pipeline prioritization, (iii) $2.8 million in personnel-related expenses related to the reduction in workforce and strategic pipeline prioritization,prioritization; (iv) $0.9$0.6 million in other facilities and allocated expenses,expenses; and (v) $0.5$0.3 million in expenses related to licenses, sublicensing revenue, and milestones.
General and administrative expenses decreased by $1.7$2.5 million to $8.1$7.9 million for the three months ended MarchJune 31,30, 2026, from $9.7$10.4 million for the three months ended MarchJune 31,30, 2025. This decrease was primarily related to a decreasedecreases of $1.2(i) $0.9 million in personnel-related expenses related to the reduction in workforce and strategic pipeline prioritization; and(ii) a decrease of $0.5$0.8 million in legal and other service-related expenses; and (iii) $0.7 million in other facilities and allocated expenses.
Impairment charges were zero for the three months ended June 30, 2026, compared to $12.2 million for the three months ended June 30, 2025, in conjunction with the previously announced strategic pipeline prioritization. These charges include $7.4 million related to tenant improvements, $2.6 million for the right-of-use asset, and $2.2 million for lab equipment.
Total Other Income (Expense)
Total other income decreased(expense) increased by $1.7$7.6 million for the three months ended MarchJune 31,30, 2026, as compared to the three months ended MarchJune 31,30, 2025. This decrease was primarily related to a $1.4 million decrease in interest income earned from marketable securities.
Impairment of equity investment was zero for the three months ended June 30, 2026, compared to $9.2 million related to our equity investment in Edge for the three months ended June 30, 2025. See Note 3 to our unaudited condensed consolidated financial statements included elsewhere in this Form 10-Q for additional information.
Other income, net decreased by $1.6 million for the three months ended June 30, 2026, compared to the three months ended June 30, 2025. This decrease was primarily related to a $1.1 million decrease in interest income earned from marketable securities.
Comparison of the Six Months Ended June 30, 2026, and June 30, 2025
The following table summarizes our results of operations for the periods indicated:
Licensing and Other Third-party Revenue
Licensing and other third-party revenue decreased by $1.1 million to $3.9 million for the six months ended June 30, 2026, from $5.0 million for the six months ended June 30, 2025. This decrease primarily relates to the recognition of a $1.0 million milestone payment under a license agreement for the six months ended June 30, 2025, with no comparable milestone revenue recognized for the six months ended June 30, 2026.
Research and Development Expenses
Research and development expenses decreased by $23.7 million to $39.6 million for the six months ended June 30, 2026, from $63.2 million for the six months ended June 30, 2025. This decrease was primarily related to decreases of (i) $11.0 million in external CMO and CRO activities for our clinical CAR-T cell therapy product candidates, driven by decreases of (a) $6.8 million in CRO activities for our clinical trials and (b) $4.2 million due to CMO activities; (ii) $5.4 million in personnel-related expenses related to the reduction in workforce and strategic pipeline prioritization; (iii) $4.9 million in other research and development expenses primarily related to the reduction in workforce and strategic pipeline prioritization; (iv) $1.6 million in other facilities and allocated expenses; and (v) $0.8 million in expenses related to licenses, sublicensing revenue, and milestones.
General and Administrative Expenses
General and administrative expenses decreased by $4.2 million to $16.0 million for the six months ended June 30, 2026, from $20.1 million for the six months ended June 30, 2025. This decrease was primarily related to decreases of (i) $2.1 million in personnel-related expenses related to the reduction in workforce and strategic pipeline prioritization; (ii) $1.2 million in legal and other service-related expenses; and (iii) $0.8 million in other facilities and allocated expenses.
Impairment charges were zero for the six months ended June 30, 2026, compared to $12.2 million for the six months ended June 30, 2025, in conjunction with the previously announced strategic pipeline prioritization. These charges include $7.4 million related to tenant improvements, $2.6 million for the right-of-use asset, and $2.2 million for lab equipment.
Total Other Income (Expense)
Total other income (expense) increased by $5.9 million for the six months ended June 30, 2026, as compared to the six months ended June 30, 2025.
Impairment of equity investment was zero for the six months ended June 30, 2026, compared to $9.2 million for the six months ended June 30, 2025, related to our equity investment in Edge. See Note 3 to our unaudited condensed consolidated financial statements included elsewhere in this Form 10-Q for additional information.
Other income, net decreased by $3.3 million for the six months ended June 30, 2026, compared to the six months ended June 30, 2025. This decrease was primarily related to a $2.5 million decrease in interest income earned from marketable securities.
Since our inception through MarchJune 31,30, 2026, we have raised an aggregate net proceeds of $852.8$871.4 million to fund our operations through our initial public offering (“IPO”); sales of convertible preferred stock; a follow-on public offering; proceeds from our licensing, licensing and collaboration, service, and patent assignment agreements, including sales of Intellia stock; private placements; at-the-market equity offerings; and government grants.
As of MarchJune 31,30, 2026, we had cash, cash equivalents, and marketable securities of $118.6$113.8 million.
We expect that our existing cash, cash equivalents, and marketable securities will be sufficient to fund our operations for at least the next 12 months from the date this Form 10-Q is filed.filed with the SEC. We have based these estimates on our current assumptions, which may require future adjustments based on our ongoing business decisions.
We will continue to be dependent on equity financing, debt financing, licensing arrangements, and/or other forms of capital raises, including structured or other non-dilutive financings, to fund operating expenses, including to fully fund ANTLER-3, our planned vispa-cel pivotal phase 3 trial,trial for vispa-cel, at least until we are able to generate significant positive cash flows from our operations. We have no current ongoing material financing commitments, such as lines of credit or guarantees, that are expected to affect our liquidity over the next five years, except for our lease commitments and payments under certain of our license agreements as described in Notes 4 and 8 to our unaudited condensed consolidated financial statements included elsewhere in this Form 10-Q.
•whether we enter into any collaboration or other agreements and the terms of any such agreements;
Because of the numerous risks and uncertainties associated with therapeutic product development, we may never achieve profitability and, unless and until we are able to develop and commercialize our CAR-T cell therapy product candidates, we will need to continue to raise additional capital. Until we can generate significant revenue from product sales, if ever, we expect to finance our operations through equity offerings (including our at-the-market equity offering program), debt financings, new collaborations, structured or other non-dilutive financings, licensing arrangements, and/or other sources. We cannot provide any assurance that we will be successful in obtaining an adequate level of financing to support our business plans as needed on acceptable terms, or at all. If we raise additional funds through new collaborations or licensing arrangements with third parties, we may have to relinquish valuable rights to our intellectual property, future revenue streams, or product candidates or grant licenses on terms that may not be favorable to us. Disruptions and volatility in the global and domestic capital markets resulting from heightened inflation, tariffs, capital market volatility, interest rate and currency rate fluctuations, artificial intelligence (“AI”), political and geopolitical tensions, government agency changes, any potential economic slowdown or recession, including trade wars or civil or political unrest (such as the ongoing war between Ukraine and Russia, conflicts in the Middle East, including the recent hostilities involving Iran, tension between China and Taiwan, geopolitical tensions in Europe, South America, and elsewhere) may increase the cost of capital and limit our ability to access capital. If we are unable to raise capital as and when needed or on attractive terms, we may have to significantly delay, reduce, or discontinue the development and commercialization of our CAR-T cell therapy product candidates or scale back or terminate our pursuit of new in-licenses and acquisitions.
Comparison of the ThreeSix Months Ended MarchJune 31,30, 2026, and MarchJune 31,30, 2025
Net cash used in operating activities was $27.0$49.8 million for the threesix months ended MarchJune 31,30, 2026, compared to $36.7$65.0 million for the threesix months ended MarchJune 31,30, 2025. The decrease was primarilydue drivento by a decreasedecreases in netresearch lossand fordevelopment expenses and general and administrative expenses, excluding the threeeffect monthsof endednon-cash March 31, 2026items; partially offset by a decrease in net changes in our operating assets and liabilities primarily related to a decrease in net changes in accounts payable.
Net cash provided by investing activities was $24.0$42.9 million for the threesix months ended MarchJune 31,30, 2026, compared to $49.4$73.4 million for the threesix months ended MarchJune 31,30, 2025. The decrease was primarily driven by a decrease in proceeds from maturities of marketable securities and higher cash utilized for purchases of marketable securities.
Net cash provided by financing activities was $2.7$20.6 million for the threesix months ended MarchJune 31,30, 2026, compared to $0.5 million for the threesix months ended MarchJune 31,30, 2025. The increase was primarily driven by proceeds from the issuance of common stock under the ATM Sales Agreement, net of offering expenses, duringfor the threesix months ended MarchJune 31,30, 2026. We did not sell any common stock pursuant to the ATM Sales Agreement during the threesix months ended MarchJune 31,30, 2025.
CRBU insider buying and selling (Form 4)
Since 2026-04-11, insiders reported open-market purchases in 0 Form 4 filings and open-market sales in 0 filings. Totals add up every open-market (code P and S) line in those filings, using the prices reported in the filings. Awards, option exercises, tax withholding and gifts are listed below but not counted.
No Form 4 stock transactions in this period.
Well-known investors holding CRBU (13F)
| Investor | Quarter | Shares | Reported value | % of their 13F | Change vs prior quarter |
|---|---|---|---|---|---|
| Renaissance Technologies | 2026-06-30 | 2,773,545 | $4.9M | 0.01% | Added 7% |
| Two Sigma Investments | 2026-06-30 | 1,046,279 | $1.8M | 0.0% | Reduced 41% |
| Millennium Management (Israel Englander) | 2026-06-30 | 51,171 | $90.1K | 0.0% | Reduced 95% |
| Citadel Advisors (Ken Griffin) | 2026-06-30 | 40,415 | $71.1K | 0.0% | Reduced 75% |
| AQR Capital Management (Cliff Asness) | 2026-06-30 | 15,678 | $29.8K | — | Sold out |
| Point72 Asset Management (Steve Cohen) | 2026-06-30 | 15,769 | $27.8K | 0.0% | Reduced 92% |