Caribou Biosciences Inc

Previous wording

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, including our novel chRDNA (CRISPRh ybrid RNA-DNA, or “chRDNA,” pronounced “chardonnay”) technology, enables more precise genome editing to develop cell therapies that are armored to improve activity against diseases. We are advancing a pipeline of allogeneic, or off-the-shelf, cell therapies from our chimeric antigen receptor (“CAR”) T (“CAR-T”) cell and CAR-natural killer (“CAR-NK”) cell platforms as readily available therapeutic treatments for patients.

Current wording

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, including our novel chRDNA (CRISPRh ybrid RNA-DNA, or “chRDNA,” pronounced “chardonnay”) technology, enables more precise genome editing to develop cell therapies that are armored to improve activity against diseases. We are advancing a pipeline of allogeneic, or off-the-shelf, cell therapies from our chimeric antigen receptor (“CAR”) T (“CAR-T”) cell platform as readily available therapeutic treatments for patients.

Previous wording

We are currently focused on advancing our allogeneic cell therapies for the treatment of hematologic malignancies and autoimmune diseases. Our therapies are directed at established cell surface targets for 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,” also known as CD371). We use our chRDNA technologies to armor our cell therapies through multiple genome-editing strategies, such as checkpoint disruption, immune cloaking, or a combination of these two strategies to enhance activity against devastating diseases.

Current wording

We are currently focused on advancing our allogeneic cell therapies for the treatment of hematologic malignancies and autoimmune diseases. Our therapies are directed at established cell surface targets for which autologous CAR-T cell therapeutics have already demonstrated clinical proof of concept, including CD19 and B cell maturation antigen (“BCMA”), as well as other targets such as C-type lectin-like molecule-1 (“CLL-1,” also known as CD371). We use our chRDNA technologies to armor our cell therapies through multiple genome-editing strategies, such as checkpoint disruption, immune cloaking, or a combination of these two strategies to enhance activity against devastating diseases.

Previous wording

Our lead product candidate, CB-010, is an allogeneic CAR-T cell therapy that is, to our knowledge, the first anti-CD19 allogeneic, or off-the-shelf, CAR-T cell therapy to be evaluated in patients with second-line relapsed or refractory large B cell lymphoma (“r/r LBCL”). To our knowledge, CB-010 is also the first clinical-stage allogeneic anti-CD19 CAR-T cell therapy with programmed cell death protein 1 (“PD-1”) removed from the CAR-T cell surface by a genome-edited knockout of the PDCD1 gene. CB-010 is being evaluated in our ongoing ANTLER phase 1 clinical trial. In the dose escalation portion of our ANTLER clinical trial, three dose levels of CB-010 were evaluated: dose level 1 (40x106 viable CAR-T cells, n=8), dose level 2 (80x106 viable CAR-T cells, n=5), and dose level 3 (120x106 viable CAR-T cells, n=3). CB-010 was generally well-tolerated with adverse events as expected for anti-CD19 CAR-T cell therapies. Based on these encouraging dose escalation data, we are evaluating CB-010 in adult second-line LBCL patients in the dose expansion portion of the ongoing ANTLER trial. Additionally, based on a retrospective analysis of the ANTLER data, we intend to investigate partial human leukocyte antigen matching and donor-specific antibody screening for CB-010 and our other CAR-T programs. An abstract on the ANTLER phase 1 clinical trial has been accepted for a poster presentation on June 3, 2024, at the 2024 American Society of Clinical Oncology (“ASCO”) Annual Meeting, and we plan to present initial dose expansion data, the recommended phase 2 dose (“RP2D”) in second-line LBCL patients, and emerging translational data. CB-010 has received regenerative medicine advanced therapy (“RMAT”) designation for r/r LBCL, fast track designation for relapsed or refractory B cell non-Hodgkin lymphoma (“r/r B-NHL”), and orphan drug designation for follicular lymphoma (“FL”) from the U.S. Food and Drug Administration (“FDA”).

Current wording

In our ANTLER phase 1 clinical trial, 16 patients with multiple subtypes of aggressive r/r B-NHL were enrolled in the dose escalation portion of the clinical trial, and three dose levels of CB-010 were evaluated: dose level 1 (40x106 viable CAR-T cells, n=8), dose level 2 (80x106 viable CAR-T cells, n=5), and dose level 3 (120x106 viable CAR-T cells, n=3). Following the conclusion of the dose escalation portion of our ANTLER clinical trial, we began enrolling second-line (“2L”) large B cell lymphoma (“LBCL”) patients in the dose expansion phase of our ANTLER clinical trial, which is still ongoing. Dose level 2 (80x106 viable CAR-T cells) was selected as the recommended phase 2 dose (“RP2D”). In early June 2024, we presented safety, efficacy, and translational data for the first 46 patients evaluated in our ANTLER phase 1 clinical trial during a poster presentation at the 2024 American Society of Clinical Oncology (“ASCO”) Annual Meeting. CB-010 was generally well-tolerated with adverse events as expected for anti-CD19 cell therapies. A retrospective analysis of the ANTLER clinical trial data showed that patients who received a dose of CB-010 manufactured from a healthy donor who shared four or more matching human leukocyte antigen (“HLA”) alleles with the patient (referred to as “partial HLA matching”) demonstrated the potential for improved efficacy. Based on these data, we have begun dosing a cohort of approximately 20 2L LBCL patients to prospectively evaluate whether partial HLA matching improves patient outcomes. In addition, we are enrolling approximately 10 patients who have relapsed following any prior CD19-targeted therapy in a proof-of-concept cohort, which will also incorporate partial HLA matching. We plan to report initial data from both cohorts in the first half of 2025. Upon confirmation of improved outcomes in 2L LBCL patients receiving a partially HLA matched dose of CB-010, we plan to initiate a pivotal phase 3 clinical trial in the second half of 2025, following agreement with the U.S. Food and Drug Administration (“FDA”) on a pivotal trial design. CB-010 has received regenerative medicine advanced therapy (“RMAT”) designation for relapsed or refractory LBCL, fast track designation for r/r B-NHL, and orphan drug designation for follicular lymphoma (“FL”) from the FDA.

Previous wording

We are expanding our clinical development of CB-010 to include autoimmune diseases, and we recently received clearance of our investigational new drug (“IND”) application from the FDA for CB-010 to be evaluated in adult patients with lupus nephritis (“LN”) and extrarenal lupus (“ERL”) in our multicenter, open label, GALLOP phase 1 clinical trial, which we expect to initiate by year-end 2024.

Current wording

We are expanding our clinical development of CB-010 to include autoimmune diseases, and we plan to evaluate CB-010 in a multicenter, open label, phase 1 clinical trial (“GALLOP”) in adult patients with lupus nephritis (“LN”) and extrarenal lupus (“ERL”), which will incorporate partial HLA matching. We expect to initiate our GALLOP clinical trial by year-end 2024.

Previous wording

Our second product candidate, CB-011, is an allogeneic CAR-T cell therapy that is, to our knowledge, the first anti-BCMA CAR-T cell therapy incorporating an immune cloaking approach that includes both the removal of the endogenous beta-2 microglobulin (“B2M”) protein and insertion of a beta-2-microglobulin–human-leukocyte-antigen-E–peptide transgene (“B2M–HLA-E”). This strategy is designed to reduce CAR-T cell rejection by both patient T cells and natural killer (“NK”) cells to potentially enable more durable antitumor activity. CB-011 is being evaluated in our CaMMouflage phase 1 clinical trial in adult patients with relapsed or refractory multiple myeloma (“r/r MM”), and we are currently enrolling patients in the dose escalation portion of the CaMMouflage trial. CB-011 was granted fast track and orphan drug designations for r/r MM from the FDA.

Current wording

Our second product candidate, CB-011, is an allogeneic CAR-T cell therapy that is, to our knowledge, the first anti-BCMA CAR-T cell therapy incorporating an immune cloaking approach that includes both the removal of the endogenous beta-2 microglobulin (“B2M”) protein and insertion of a beta-2-microglobulin–human-leukocyte-antigen-E–peptide transgene (“B2M–HLA-E”). This strategy is designed to reduce CAR-T cell rejection by both patient T cells and natural killer (“NK”) cells to potentially enable more durable antitumor activity. CB-011 is being evaluated in our CaMMouflage phase 1 clinical trial in adult patients with relapsed or refractory multiple myeloma (“r/r MM”), and we are currently enrolling patients in the dose escalation portion of the CaMMouflage trial. We plan to report initial dose escalation data by year-end 2024. CB-011 was granted fast track and orphan drug designations for r/r MM from the FDA.

Previous wording

The third product candidate from our CAR-T cell platform is CB-012, an allogeneic CAR-T cell therapy targeting CLL-1 (also known as CD371). CB-012 is, to our knowledge, the first allogeneic CAR-T cell therapy with both checkpoint disruption and immune cloaking strategies. We believe that CLL-1 is an attractive target for acute myeloid leukemia (“AML”) due to its expression on myeloid cancer cells, its enrichment on leukemic stem cells, and its absence on hematopoietic stem cells (“HSCs”). CB-012 is being evaluated in our AMpLify phase 1 clinical trial in adult patients with relapsed or refractory AML (“r/r AML”), and we are currently enrolling patients in the dose escalation portion of the AMpLify trial.

Current wording

The third product candidate from our CAR-T cell platform is CB-012, an allogeneic CAR-T cell therapy targeting CLL-1 (also known as CD371). CB-012 is, to our knowledge, the first allogeneic CAR-T cell therapy with both checkpoint disruption and immune cloaking strategies. We believe that CLL-1 is an attractive target for acute myeloid leukemia (“AML”) due to its expression on myeloid cancer cells, its enrichment on leukemic stem cells, and its absence on hematopoietic stem cells (“HSCs”). CB-012 is being evaluated in our AMpLify phase 1 clinical trial in adult patients with relapsed or refractory AML (“r/r AML”), and we are currently enrolling patients in the dose escalation portion of the AMpLify trial. We plan to provide updates on dose escalation as the AMpLify phase 1 clinical trial in r/r AML advances.

Previous wording

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.

Current wording

Since our founding in 2011, we have devoted substantially all 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.

Previous wording

Our net losses for the three months ended March 31, 2024, and 2023 were $41.2 million and $28.0 million, respectively. We had an accumulated deficit of $340.5 million as of March 31, 2024. 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 nonclinical studies and our other research and development expenses. We anticipate that our expenses will increase substantially as we:

Current wording

Our net losses for the three months ended June 30, 2024, and 2023, were $37.7 million and $29.5 million, respectively. Our net losses for the six months ended June 30, 2024, and 2023, were $78.9 million and $57.6 million, respectively. We had an accumulated deficit of $378.2 million as of June 30, 2024. 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 nonclinical studies and our other research and development expenses. We anticipate that our expenses will increase substantially as we:

Previous wording

•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;

Current wording

•continue our current research programs and our preclinical and clinical development of our current product candidates and any other product candidates we identify and choose to develop;

Previous wording

•add operational, legal, financial, and management information systems and personnel;

Current wording

•add operational, legal, financial, and management information systems to support growth initiatives;

Previous wording

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 and CAR-NK cell therapy product candidates themselves. We expect to continue to rely on our CMOs for the manufacturing of our preclinical study and clinical trial materials, and most of these CMOs have capabilities for commercial manufacturing. Additionally, we may decide to bring certain manufacturing functions in house and/or build our own manufacturing facility in the future to provide greater flexibility and control over our clinical and commercial manufacturing needs.

Current wording

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 the manufacturing of our preclinical study and clinical trial materials, and most of these CMOs have capabilities for commercial manufacturing. Additionally, we may decide to bring certain manufacturing functions in house and/or build our own manufacturing facility in the future to provide greater flexibility and control over our clinical and commercial manufacturing needs.

Previous wording

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 $2.4 million and $3.5 million for the three months ended March 31, 2024, and 2023, respectively. See Notes 4, 5, and 7 to our unaudited condensed consolidated financial statements included elsewhere in this Form 10-Q for additional information.

Current wording

To date, all 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 $3.5 million and $3.8 million for the three months ended June 30, 2024, and 2023, respectively, and $5.9 million and $7.3 million for the six months ended June 30, 2024, and 2023, respectively. See Notes 4, 5, and 7 to our unaudited condensed consolidated financial statements included elsewhere in this Form 10-Q for additional information.

Previous wording

Research and 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 later stages of development; conduct preclinical studies and clinical trials for our other product candidates; seek regulatory approvals for any product candidates that successfully complete clinical trials; expand our research and development efforts and incur expenses associated with hiring additional personnel to support our research and development efforts; and seek to identify, in-license, acquire, and/or develop additional product candidates.

Current wording

Research and 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 later stages of development; conduct preclinical studies and clinical trials for our other product candidates; seek regulatory approvals for any product candidates that successfully complete clinical trials; and seek to identify, in-license, acquire, and/or develop additional product candidates.

Previous wording

The successful development of our CAR-T and CAR-NK product candidates, as well as other potential future product candidates, is highly uncertain. Accordingly, 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 regulatory 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:

Current wording

The successful development of our CAR-T cell therapy product candidates, as well as other potential future product candidates, is highly uncertain. Accordingly, 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 regulatory 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:

Previous wording

•acceptance of our CRISPR chRDNA genome-editing technology;

Current wording

•acceptance of our CRISPR chRDNA genome-editing technology and other new technologies we may develop or in-license;

Previous wording

•ability to develop differentiating features so that our products have a competitive edge;

Current wording

•the ability to develop differentiating features so that our products have a competitive edge;

Previous wording

•our ability to not infringe, misappropriate, or otherwise violate third-party intellectual property rights;

Current wording

•the ability to not infringe, misappropriate, or otherwise violate third-party intellectual property rights;

Previous wording

•data from our clinical trials that support an acceptable risk-benefit profile of our product candidates for the intended patient populations and that demonstrate safety and efficacy;

Current wording

•generation of data from our clinical trials that support an acceptable risk-benefit profile of our product candidates for the intended patient populations and that demonstrate safety and efficacy;

Previous wording

•establishment of agreements with CMOs and suppliers for clinical and commercial supplies and scaling up manufacturing processes and capabilities to support our clinical trials;

Current wording

•maintenance of, and compliance with, our agreements with CMOs and suppliers for clinical and commercial supplies and scaling up manufacturing processes and capabilities to support our clinical trials;

Previous wording

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 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 the three months ended March 31, 2024, and 2023, we recorded $0.1 million and $0.4 million, respectively, of patent cost reimbursements as a reduction to general and administrative expense.

Current wording

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 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 the three months ended June 30, 2024, and 2023, we recorded $0.4 million and $0.7 million, respectively, of patent cost reimbursements as a reduction to general and administrative expense. During the six months ended June 30, 2024, and 2023, we recorded $0.5 million and $1.1 million, respectively, of patent cost reimbursements as a reduction to general and administrative expense.

Previous wording

We expect that our general and administrative expenses will increase in the future as a result of expanding our operations, including hiring personnel, preparing 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.

Current wording

We expect that our general and administrative expenses will increase in the future as a result of expanding our operations, including preparing 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.

Previous wording

Licensing and collaboration revenue decreased by $1.1 million to $2.4 million for the three months ended March 31, 2024, from $3.5 million for the three months ended March 31, 2023. This decrease primarily relates to a decrease of $1.6 million recognized under the now-terminated Collaboration and License Agreement, dated February 9, 2021 (as amended, “AbbVie Agreement”) with AbbVie Manufacturing Management Unlimited Company (“AbbVie”), partially offset by an increase of $0.6 million in revenues recognized under our Information Rights Agreement with Pfizer, Inc. (“Pfizer”), dated June 29, 2023.

Current wording

Licensing and collaboration revenue decreased by $0.3 million to $3.5 million for the three months ended June 30, 2024, from $3.8 million for the three months ended June 30, 2023. This decrease primarily relates to a decrease of $1.4 million of revenue recognized under the now-terminated Collaboration and License Agreement, dated February 9, 2021 (as amended, “AbbVie Agreement”) with AbbVie Manufacturing Management Unlimited Company (“AbbVie”), partially offset by (i) an increase of $0.6 million in revenue recognized under our Information Rights Agreement with Pfizer Inc. (“Pfizer”), dated June 29, 2023; and (ii) a net increase of $0.5 million in revenue recognized under our agreements with Edge Animal Health (“Edge”), driven by (a) an increase of $1.6 million related to the Exclusive License Agreement for Veterinary Therapeutics (as amended, the “Edge chRDNA License Agreement”) with Edge, partially offset by (b) a decrease of $1.2 million related to the Exclusive License Agreement for Veterinary Therapeutics (CRISPR-Cas9) (the “Edge Cas9 License Agreement”) with Edge.

Previous wording

Research and development expenses increased by $8.1 million to $33.8 million for the three months ended March 31, 2024, from $25.7 million for the three months ended March 31, 2023. This increase was primarily related to increases of $3.2 million in expenses related to licenses and milestones; $2.0 million in personnel-related expenses, including stock-based compensation, due to headcount increases; and $1.8 million of net increase in external CMO and CRO activities for our clinical CAR-T cell therapy product candidates, driven by an increase of $5.1 million in CRO activities for clinical trials, and partially offset by a decrease of $3.6 million due to timing of CMO activities.

Current wording

Research and development expenses increased by $9.0 million to $35.5 million for the three months ended June 30, 2024, from $26.5 million for the three months ended June 30, 2023. This increase was primarily related to (i) an increase of $5.1 million in other research and development expenses to advance preclinical and clinical research for our programs, as well as other consulting services related to research and development; (ii) a net increase of $1.9 million in external CMO and CRO activities for our clinical CAR-T cell therapy product candidates, driven by (a) an increase of $3.6 million in CRO activities for clinical trials, partially offset by (b) a decrease of $1.7 million due to timing of CMO activities; (iii) an increase of $1.6 million in personnel-related expenses, including stock-based compensation, due to headcount increases; and (iv) an increase of $0.8 million in other facilities and allocated expenses; partially offset by a decrease of $0.4 million in expenses related to licenses, sublicensing revenue, and milestones.

Previous wording

Total other income increased by $1.7 million for three months ended March 31, 2024, as compared to the three months ended March 31, 2023.

Current wording

Total other income increased by $2.5 million for three months ended June 30, 2024, as compared to the three months ended June 30, 2023.

Previous wording

We recognized a gain related to the change in the fair value of the MSKCC success payments liability in the amount of $0.3 million, for each of the three-month periods ended March 31, 2024, and 2023.

Current wording

We recognized a gain related to the change in the fair value of the MSKCC success payments liability in the amount of $1.8 million and $0.3 million, for the three months ended June 30, 2024, and 2023, respectively.

Previous wording

Other income, net increased by $1.6 million during the three months ended March 31, 2024, compared to March 31, 2023. The increase primarily relates to a $1.7 million increase in interest income earned from marketable securities.

Current wording

Other income, net increased by $1.1 million during the three months ended June 30, 2024, compared to June 30, 2023. The increase primarily relates to a $1.0 million increase in interest income earned from marketable securities.

Previous wording

General and administrative expenses increased by $5.7 million to $14.6 million for the three months ended March 31, 2024, from $8.9 million for the three months ended March 31, 2023. This increase was primarily related to increases of $4.6 million in legal, and other service-related expenses, including $3.9 million of accrued litigation settlement costs; and $1.3 million in personnel-related expenses, including stock-based compensation, due to headcount increases; partially offset by a decrease of $0.4 million in patent prosecution and maintenance fees.

Current wording

General and administrative expenses increased by $7.1 million to $26.1 million for the six months ended June 30, 2024, from $19.0 million for the six months ended June 30, 2023. This increase was primarily related to increases of $5.7 million in legal, and other service-related expenses, including $3.9 million of accrued securities litigation settlement costs and $2.6 million in personnel-related expenses, including stock-based compensation, due to headcount increases; partially offset by a decrease of $1.1 million patent prosecution and maintenance costs.

Previous wording

Since our inception, we have not generated any revenue from product sales and have incurred significant operating losses and negative cash flows from our operations. We have funded our operations through sales of our capital stock, including sales of our convertible preferred stock, which generated approximately $150.1 million in aggregate net proceeds through 2021, from our initial public offering (“IPO”) in 2021, which generated approximately $321.0 million in net proceeds and from an underwritten follow-on public offering in 2023, which generated approximately $134.4 million in net proceeds. We have also received approximately $88.4 million in net proceeds from the sale of Intellia common stock through 2020. Additionally, we received a $25.0 million equity investment from Pfizer through a private placement transaction in June 2023. Through March 31, 2024, we received approximately $96.5 million from licensing agreements, licensing and collaboration agreements, a service agreement, patent assignments, and government grants, including $36.7 million that was received from AbbVie under the now-terminated AbbVie Agreement.

Current wording

Since our inception, we have not generated any revenue from product sales and have incurred significant operating losses and negative cash flows from our operations. We have funded our operations through sales of our capital stock, including sales of our convertible preferred stock, which generated approximately $150.1 million in aggregate net proceeds through 2021, from our initial public offering (“IPO”) in 2021, which generated approximately $321.0 million in net proceeds and from an underwritten follow-on public offering in 2023, which generated approximately $134.4 million in net proceeds. We have also received approximately $88.4 million in net proceeds from the sale of Intellia common stock through 2020. Additionally, we received a $25.0 million equity investment from Pfizer through a private placement transaction in June 2023. Through June 30, 2024, we received approximately $98.2 million from licensing agreements, licensing and collaboration agreements, a service agreement, patent assignments, and government grants, including $36.7 million that was received from AbbVie under the now-terminated AbbVie Agreement.

Previous wording

As of March 31, 2024, we had cash, cash equivalents, and marketable securities of $345.9 million. We will continue to be dependent upon equity financing, debt financing, collaboration and licensing arrangements, and/or other forms of capital raises 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 Note 8 and in Notes 4 and 9, respectively, to our unaudited condensed consolidated financial statements included elsewhere in this Form 10-Q.

Current wording

As of June 30, 2024, we had cash, cash equivalents, and marketable securities of $311.8 million. We will continue to be dependent upon equity financing, debt financing, collaboration and licensing arrangements, and/or other forms of capital raises 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 Note 8 and in Notes 4 and 9, respectively, to our unaudited condensed consolidated financial statements included elsewhere in this Form 10-Q.

Previous wording

•the clinical development plans we establish for these product candidates;

Current wording

•the clinical development plans we establish for our product candidates;

Previous wording

•the number and characteristics of the product candidates that we develop;

Current wording

•the number and characteristics of the new product candidates that we develop;

Previous wording

•the extent to which we acquire or in-license other product candidates and technologies;

Current wording

•the extent to which we acquire or in-license other product candidates and/or new technologies;

Previous wording

•the cost and timing of completion of commercial-scale outsourced manufacturing activities or the cost and timing of completion of clinical-scale and commercial-scale internal manufacturing activities;

Current wording

•the cost and timing of completion of clinical-scale and commercial-scale internal manufacturing activities, if we elect to conduct these activities ourselves;

Previous wording

Net cash used in operating activities was $37.2 million and $27.9 million for the three months ended March 31, 2024, and 2023, respectively.

Current wording

Net cash used in operating activities was $70.1 million and $43.1 million for the six months ended June 30, 2024, and 2023, respectively.

Previous wording

Cash used in operating activities for the three months ended March 31, 2024, was primarily due to our net loss of $41.2 million, adjusted by non-cash charges of $4.9 million and net changes in our operating assets and liabilities of $0.8 million. Our non-cash charges were primarily comprised of $4.0 million of stock-based compensation, $0.7 million of depreciation and amortization expense, and non-cash lease expense of $0.5 million, which were partially offset by change in the fair value of the MSKCC success payments liability of $0.3 million, and accretion of discounts on our marketable securities investments of $1.5 million. The changes in our operating assets and liabilities were primarily due to an increase in accounts receivable of $0.8 million, and prepaid expenses and other current assets of $0.8 million and decreases of $2.2 million in accrued expenses and other current liabilities, and $0.7 million in deferred revenue, current and long-term, partially offset by decreases in other receivables of $0.6 million, and $0.5 million in contract assets, and an increase of $2.7 million in accounts payable.

Current wording

Cash used in operating activities for the six months ended June 30, 2024, was primarily due to our net loss of $78.9 million, adjusted by non-cash charges of $6.9 million and net changes in our operating assets and liabilities of $2.0 million. Our non-cash charges were primarily comprised of (i) $8.7 million of stock-based compensation, (ii) $1.6 million of depreciation and amortization expense, (iii) $1.6 million of acquired in-process research and development, and (iv) non-cash lease expense of $1.0 million; which were partially offset by (i) accretion of discounts on our marketable securities investments of $2.5 million, (ii) change in the fair value of the MSKCC success payments liability of $2.1 million, and (iii) non-cash consideration for licensing and collaboration revenue of $1.6 million. The changes in our operating assets and liabilities were primarily due to (i) decreases in other receivables of $0.6 million and $0.5 million in contract assets, and (ii) increases of $2.8 million in accrued expenses and other current liabilities and $0.4 million in accounts payable; partially offset by (i) an increase in prepaid expenses and other current assets of $0.2 million and (ii) decreases of $1.5 million in deferred revenue, current and long-term and $0.3 million in operating lease liabilities.

Previous wording

Cash used in operating activities for the three months ended March 31, 2023, was primarily due to our net loss of $28.0 million, adjusted by non-cash charges of $2.5 million and net changes in our operating assets and liabilities of $2.4 million. Our non-cash charges were primarily comprised of $3.1 million of stock-based compensation, $0.6 million of depreciation and amortization expense, and non-cash lease expense of $0.5 million, which were partially offset by amortization of investment premiums of $1.5 million, and change in the fair value of the MSKCC success payments liability of $0.3 million. The changes in our operating assets and liabilities were due to increases of $1.3 million in accounts receivable, and decreases of $4.1 million in accrued expenses and other current liabilities, $1.1 million in deferred revenue, and $0.3 million in operating lease liabilities, partially offset by decreases in prepaid expenses and other current assets of $1.8 million, contract assets of $0.6 million, and other receivables of $0.5 million, and an increase of $1.5 million in accounts payable.

Current wording

Cash used in operating activities for the six months ended June 30, 2023, was primarily due to our net loss of $57.6 million, adjusted by non-cash charges of $4.7 million and net changes in our operating assets and liabilities of $9.7 million. Our non-cash charges were primarily comprised of (i) $6.7 million of stock-based compensation, (ii) $1.2 million of depreciation and amortization expense, and (iii) non-cash lease expense of $1.0 million; which were partially offset by (i) amortization of investment premiums of $3.6 million, and (ii) change in the fair value of the MSKCC success payments liability of $0.5 million. The changes in our operating assets and liabilities were due to (i) decreases in prepaid expenses and other current assets of $2.4 million, contract assets of $0.8 million, and other receivables of $0.7 million, and (ii) increases of $5.2 million in deferred revenue (including $7.5 million related to the Pfizer Investment) and $2.0 million in accounts payable; partially offset by (i) an increase of $0.6 million in accounts receivable, and (ii) decreases of $0.5 million in operating lease liabilities and $0.4 million in accrued expenses and other current liabilities.

Previous wording

During the three months ended March 31, 2024, and 2023, cash provided by investing activities was $23.0 million, and $20.7 million, respectively.

Current wording

During the six months ended June 30, 2024, and 2023, cash provided by investing activities was $44.2 million, and $31.8 million, respectively.

Previous wording

Cash provided by investing activities for the three months ended March 31, 2024, was primarily due to proceeds from the sales and maturities of marketable securities of $97.1 million, partially offset by purchases of marketable securities of $71.2 million, in-process research and development of $1.5 million, and property and equipment of $1.4 million.

Current wording

Cash provided by investing activities for the six months ended June 30, 2024, was primarily due to proceeds from the sales and maturities of marketable securities of $204.9 million, partially offset by purchases of marketable securities of $155.4 million, in-process research and development of $1.6 million, and property and equipment of $3.7 million.

Previous wording

Cash provided by investing activities for the three months ended March 31, 2023, was primarily due to proceeds from the sales and maturities of marketable securities of $98.7 million, partially offset by sales of marketable securities of $75.9 million and property and equipment of $2.0 million.

Current wording

Cash provided by investing activities for the six months ended June 30, 2023, was primarily due to proceeds from the sales and maturities of marketable securities of $171.0 million, partially offset by purchases of marketable securities of $134.7 million and property and equipment of $4.6 million.

Previous wording

During the three months ended March 31, 2024, and 2023, cash provided by financing activities was $12.5 million and $1.6 million, respectively.

Current wording

During the six months ended June 30, 2024, and 2023, cash provided by financing activities was $12.6 million and $19.2 million, respectively.