CRISPR Therapeutics AG
For the period ended Mar 31, 2025. Filed May 6, 2025.
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Mar 31, 2025 · Filed May 6, 2025
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Notes to Condensed Consolidated Financial Statements
(unaudited)
- Basis of Presentation and Significant Accounting Policies
Basis of Presentation
The accompanying condensed consolidated financial statements are unaudited and have been prepared by the Company in accordance with accounting principles generally accepted in the United States of America, or U.S. GAAP.
The accompanying condensed consolidated financial statements include the accounts of the Company and its wholly-owned subsidiaries. All intercompany balances and transactions have been eliminated in consolidation. The Company views its operations and manages its business inone operating segment, which is the business of discovering, developing and commercializing therapies derived from or incorporating genome-editing technology. Certain information and footnote disclosures normally included in the Company’s annual financial statements have been condensed or omitted. These interim financial statements, in the opinion of management, reflect all normal recurring adjustments necessary for a fair presentation of the financial position and results of operations for the three-month interim periods ended March 31, 2025 and 2024.
The results of operations for the interim periods are not necessarily indicative of the results of operations to be expected for the full year. These interim financial statements should be read in conjunction with the audited financial statements for the year ended December 31, 2024, which are contained in the 2024 Annual Report on Form 10-K filed with the Securities and Exchange Commission, or the SEC, on February 11, 2025.
Use of Estimates
The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the amounts reported in the financial statements and accompanying notes. On an ongoing basis, the Company’s management evaluates its estimates, which include, but are not limited to, revenue recognition, equity-based compensation expense and reported amounts of expenses during the period. The Company bases its estimates on historical experience and other market-specific or other relevant assumptions that it believes to be reasonable under the circumstances. Actual results may differ from those estimates or assumptions. Changes in estimates are reflected in reported results in the period in which they become known.
Significant Accounting Policies
The significant accounting policies used in preparation of these condensed consolidated financial statements for the three months ended March 31, 2025 are consistent with those discussed in Note 2 to the consolidated financial statements in the Company’s 2024 Annual Report on Form 10-K filed with the SEC on February 11, 2025, with the exception of changes to the below policy.
Collaboration Arrangements
The Company records the elements of its collaboration agreements that represent joint operating activities in accordance with ASC, Accounting Standards Codification, or ASC, 808, Collaborative Agreements, or ASC 808. Accordingly, the elements of the collaboration agreements that represent activities in which both parties are active participants and to which both parties are exposed to the significant risks and rewards that are dependent on the commercial success of the activities, are recorded as collaborative arrangements.
The Company evaluates the proper presentation of the commercial activities and the profit and loss sharing associated with the collaboration agreements. ASC 808 states that when payments between parties in a collaborative arrangement are not within the scope of other authoritative accounting literature, the income statement classification should be based on the nature of the arrangement, the nature of its business operations and the contractual terms of the arrangement. To the extent that these payments are not within the scope of other authoritative accounting literature, the income statement classification for the payments shall be based on an analogy to authoritative accounting literature or if there is no appropriate analogy, a reasonable, rational and consistently applied accounting policy election.
Collaboration costs specific to the Vertex Hemoglobinopathy Agreements (as defined in Note 6) accounted for under ASC 808 are presented within “collaboration expense, net” in the condensed consolidated statements of operations and comprehensive loss. Refer to Note 6 to these condensed consolidated financial statements for further discussion on the Vertex Hemoglobinopathy Agreements.
Recent Accounting Pronouncements
In December 2023, the Financial Accounting Standards Board, or FASB, issued Accounting Standards Update, or ASU, 2023-09, Improvements to Income Tax Disclosures, which requires entities, on an annual basis, to disclose disaggregated information about their effective tax rate reconciliation and income taxes paid. The disclosure requirements will be applied on a prospective basis, with the option to apply them retrospectively. The standard is effective for fiscal years beginning after December 15, 2024, with early adoption permitted. The Company has adopted ASU 2023-09 and is currently evaluating the guidance, which is expected to only impact annual disclosures with no impact to the Company’s results of operations.
In November 2024, the FASB issued ASU 2024-03, Income Statement—Reporting Comprehensive Income—Expense Disaggregation Disclosures, which requires disclosure of additional information about specific expense categories in the notes to the financial statements on an interim and annual basis. The standard is effective for fiscal years beginning after December 15, 2026, and for interim periods beginning after December 15, 2027, with early adoption permitted. The Company is currently evaluating the disclosure requirements related to this new standard.
- Marketable Securities
The following table summarizes cash equivalents and marketable securities held at March 31, 2025 and December 31, 2024 (in thousands), which are recorded at fair value. The table below excludes $137.7 million and $193.9 million of cash at March 31, 2025 and December 31, 2024, respectively.
| AmortizedCost | GrossUnrealizedGains | GrossUnrealizedLosses | Fair Value | |
|---|---|---|---|---|
| Cash equivalents: | ||||
| Money market funds | $66,748 | $— | $— | $66,748 |
| Commercial paper | 23,475 | — | (2) | 23,473 |
| Corporate debt securities | 914 | — | — | 914 |
| U.S. Treasury securities | 6,339 | — | — | 6,339 |
| Total cash equivalents | 97,476 | — | (2) | 97,474 |
| Marketable securities: | ||||
| Corporate debt securities | 1,141,075 | 4,263 | (614) | 1,144,724 |
| Certificates of deposit | 107,242 | — | — | 107,242 |
| Government-sponsored enterprise securities | 269,836 | 596 | (176) | 270,256 |
| Commercial paper | 91,587 | 21 | (4) | 91,604 |
| Total marketable debt securities | 1,609,740 | 4,880 | (794) | 1,613,826 |
| Corporate equity securities | 10,387 | — | (4,112) | 6,275 |
| Total marketable securities | 1,620,127 | 4,880 | (4,906) | 1,620,101 |
| Total cash equivalents and marketable securities | $1,717,603 | $4,880 | $(4,908) | $1,717,575 |
| Cash equivalents: | ||||
| Money market funds | $74,155 | $— | $— | $74,155 |
| Corporate debt securities | 882 | — | — | 882 |
| Commercial paper | 29,271 | — | (9) | 29,262 |
| Total cash equivalents | 104,308 | — | (9) | 104,299 |
| Marketable securities: | ||||
| U.S. Treasury securities | 5,936 | 2 | — | 5,938 |
| Corporate debt securities | 1,136,255 | 3,442 | (1,592) | 1,138,105 |
| Certificates of deposit | 52,372 | — | — | 52,372 |
| Government-sponsored enterprise securities | 266,877 | 482 | (497) | 266,862 |
| Commercial paper | 127,805 | 34 | (39) | 127,800 |
| Total marketable debt securities | 1,589,245 | 3,960 | (2,128) | 1,591,077 |
| Corporate equity securities | 10,387 | 4,600 | (495) | 14,492 |
| Total marketable securities | 1,599,632 | 8,560 | (2,623) | 1,605,569 |
| Total cash equivalents and marketable securities | $1,703,940 | $8,560 | $(2,632) | $1,709,868 |
As of March 31, 2025, marketable debt securities were in a net unrealized gain position of $4.1 million. As of December 31, 2024, marketable debt securities were in a net unrealized gain position of $1.8 million. The Company has recorded a net unrealized gain of $2.3 million and a net unrealized loss of $3.5 million during thethree months ended March 31, 2025 and 2024, respectively, related to its debt securities, which is included in comprehensive loss on the condensed consolidated statements of operations and comprehensive loss.
As of March 31, 2025 and December 31, 2024, the aggregate fair value of marketable securities that were in an unrealized loss position for less than twelve months was $288.8 million and $451.9 million, respectively. As of March 31, 2025 and December 31, 2024, the aggregate fair value of marketable securities that were in an unrealized loss position for more than twelve months was $3.5 million and $31.8 million, respectively. As of March 31, 2025, no securities in an unrealized loss position for more than twelve months will mature beyond one year. As of December 31, 2024, no securities in an unrealized loss position for more than twelve months had maturities beyond one year.
As of March 31, 2025, marketable equity securities were in a net unrealized loss position of $4.1 million. As of December 31, 2024, marketable equity securities were in a net unrealized gain position of $4.1 million. The Company has recorded a net unrealized loss of $8.2 million during thethree months ended March 31, 2025, related to its equity securities. The Company didnot hold marketable equity securities as of March 31, 2024. These amounts are included in comprehensive loss on the condensed consolidated statements of operations and comprehensive loss.
The Company determined that there is no material credit risk associated with the above investments as of March 31, 2025. The Company has the intent and ability to hold such securities until recovery. As a result, the Company did not record any charges for credit-related impairments for its marketable securities for the three months ended March 31, 2025 and 2024. No available-for-sale debt securities held as of March 31, 2025 had remaining maturities greater than thirty months.
- Fair Value Measurements
The following tables present information about the Company’s financial assets measured at fair value on a recurring basis and indicate the fair value hierarchy classification of such fair values as of March 31, 2025 and December 31, 2024 (in thousands):
| Fair Value Measurements at | ||||
|---|---|---|---|---|
| Total | Level 1 | Level 2 | Level 3 | |
| Cash and cash equivalents: | ||||
| Cash | $137,710 | $137,710 | $— | $— |
| Money market funds | 66,748 | 66,748 | — | — |
| Commercial paper | 23,473 | — | 23,473 | — |
| Corporate debt securities | 914 | — | 914 | — |
| U.S. Treasury securities | 6,339 | — | 6,339 | — |
| Marketable securities: | ||||
| Corporate debt securities | 1,144,724 | — | 1,144,724 | — |
| Certificates and term deposits | 107,242 | — | 107,242 | — |
| Government-sponsored enterprise securities | 270,256 | — | 270,256 | — |
| Commercial paper | 91,604 | — | 91,604 | — |
| Corporate equity securities | 6,275 | 875 | 5,400 | — |
| Total | $1,855,285 | $205,333 | $1,649,952 | $— |
| Fair Value Measurements at | ||||
| Total | Level 1 | Level 2 | Level 3 | |
| Cash and cash equivalents: | ||||
| Cash | $193,958 | $193,958 | $— | $— |
| Money market funds | 74,155 | 74,155 | — | — |
| Corporate debt securities | 882 | — | 882 | — |
| Commercial paper | 29,262 | — | 29,262 | — |
| Marketable securities: | ||||
| U.S. Treasury securities | 5,938 | — | 5,938 | — |
| Corporate debt securities | 1,138,105 | — | 1,138,105 | — |
| Certificates of deposit | 52,372 | — | 52,372 | — |
| Government-sponsored enterprise securities | 266,862 | — | 266,862 | — |
| Commercial paper | 127,800 | — | 127,800 | — |
| Corporate equity securities | 14,492 | 2,391 | 12,101 | — |
| Total | $1,903,826 | $270,504 | $1,633,322 | $— |
Marketable securities classified as Level 1 within the valuation hierarchy consist of corporate equity securities with quoted prices in active markets. Marketable securities classified as Level 2 within the valuation hierarchy generally consist of U.S. treasury securities and government agency securities, certificates of deposit, corporate bonds, commercial paper and warrants to purchase common shares of publicly traded companies. The Company estimates the fair value of these marketable securities by taking into consideration valuations obtained from third-party pricing sources.
- Property and Equipment, net
Property and equipment, net, consists of the following (in thousands):
| As of | ||
|---|---|---|
| March 31, | December 31, | |
| 2025 | 2024 | |
| Computer equipment | $4,248 | $3,833 |
| Furniture, fixtures and other | 8,554 | 8,554 |
| Laboratory equipment | 42,204 | 42,008 |
| Leasehold improvements | 145,852 | 145,852 |
| Construction work in process | 5,536 | 6,118 |
| Total property and equipment, gross | 206,394 | 206,365 |
| Accumulated depreciation | (76,968) | (72,272) |
| Total property and equipment, net | $129,426 | $134,093 |
Depreciation expense for the three months ended March 31, 2025 and 2024 $4.7 million and $4.8 million, respectively.
- Accrued Expenses
Accrued expenses consist of the following (in thousands):
| As of | ||
|---|---|---|
| March 31, | December 31, | |
| 2025 | 2024 | |
| Payroll and employee-related costs | $6,086 | $18,443 |
| Research and development costs | 17,508 | 15,549 |
| Collaboration costs | 55,297 | — |
| Licensing fees | 806 | 1,850 |
| Professional fees | 3,426 | 3,086 |
| Intellectual property costs | 1,031 | 1,513 |
| Other | 461 | 631 |
| Total | $84,615 | $41,072 |
- Significant Contracts
Agreements with Vertex
For purposes of this Note 6 and Note 7, CASGEVY (exagamglogene autotemcel [exa-cel]) is referred to as “CASGEVY”.
2015 collaboration
In 2015, the Company entered into a strategic collaboration, option and license agreement, or the 2015 Collaboration Agreement, with Vertex Pharmaceuticals Incorporated, or Vertex. The 2015 Collaboration Agreement is focused on the use of the Company’s CRISPR/Cas9 gene editing technology to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. The Company and Vertex amended the 2015 Collaboration Agreement in 2017 and 2019 with Amendment No. 1 and Amendment No. 2, respectively, namely to clarify Vertex’s option rights under the 2015 Collaboration Agreement and to modify certain definitions and provisions of the 2015 Collaboration Agreement to make them consistent with the JDA (as defined below) and the 2019 Collaboration Agreement (as defined below). In 2017, Vertex exercised an option granted to it under the 2015 Collaboration Agreement to obtain a co-exclusive license to develop and commercialize hemoglobinopathy and beta-globin targets, and in 2019, Vertex exercised the remaining options granted to it under the 2015 Collaboration Agreement to exclusively license certain collaboration targets developed under the 2015 Collaboration Agreement.
Hemoglobinopathies collaboration
In 2017, following Vertex's exercise of its option to obtain a co-exclusive license to develop and commercialize hemoglobinopathy and beta-globin targets, the Company and Vertex entered into a joint development and commercialization agreement, or the JDA, and agreed for potential hemoglobinopathy treatments, including CASGEVY, the Company and Vertex would share equally all research and development costs and worldwide revenues. In 2021, the Company and Vertex amended and restated the JDA, or the A&R Vertex JDCA (as amended and in effect, from time to time), pursuant to which the parties agreed to, among other things, (a) adjust the governance structure for the collaboration and adjust the responsibilities of each party thereunder, whereby Vertex leads and has all decision making (i.e., control) in relation to the CASGEVY program prospectively; (b) adjust the allocation ofnet profits and net losses between the parties with respect to CASGEVY only, which will be allocated 40% to the Company and 60% to Vertex, prospectively; and (c) exclusively license (subject to the Company’s reserved rights to conduct certain activities) certain intellectual property rights to Vertex relating to the specified product candidates and products (including CASGEVY) that may be researched, developed, manufactured and commercialized on a worldwide basis under the A&R Vertex JDCA. Additionally, for 2022, 2023, and 2024, the Company had an option to defer a portion of its share of costs under the A&R Vertex JDCA if spending on the CASGEVY program exceeded specified amounts. Any deferred amounts under the A&R Vertex JDCA, as amended, are only payable to Vertex as an offset against future profitability of the CASGEVY program and the amounts payable are capped at a specified maximum amount per year.
In December 2023, the Company entered into an amendment to the A&R Vertex JDCA, or Amendment No. 1 to the A&R Vertex JDCA, with Vertex related to the global development, manufacturing, and commercialization of CASGEVY. Pursuant to Amendment No. 1 to the A&R Vertex JDCA, among other things, the Company and Vertex agreed to (a) allocate certain costs arising from a license agreement with a third party, resulting in a current payment due to Vertex by the Company of $20.0 million upon an event specified in Amendment No. 1 to the A&R Vertex JDCA, and (b) adjust, under certain specified circumstances, the timing of and portion of the Company’s share of costs it is permitted to defer under the agreement.
Letter Agreement
In May 2024, Vertex and the Company entered into a letter agreement, or the Letter Agreement, with respect to the priority review voucher issued by the FDA to Vertex as the sponsor of the rare pediatric disease product application for CASGEVY. Vertex and the Company agreed that if Vertex utilizes or transfers the priority review voucher prior to the first calendar year in which the CASGEVY program generates a net profit, Vertex will pay the Company $43.0 million or an amount equal to 42% of the net proceeds from such transfer, as applicable. If the CASGEVY program begins generating calendar-year net profits prior to such utilization or transfer, Vertex will instead pay the Company up to $43.0 million set-off by deductions Vertex would otherwise be eligible to take against the CASGEVY program’s net profits due to the Company related to amounts deferred previously by the Company.
Collaboration in the field of diabetes
In 2021, the Company and ViaCyte, Inc., or ViaCyte, entered into a joint development and commercialization agreement, or the ViaCyte JDCA, to jointly develop and commercialize product candidates and shared products for the diagnosis, treatment or prevention of diabetes type 1, diabetes type 2 or insulin dependent / requiring diabetes throughout the world. In the third quarter of 2022, Vertex acquired ViaCyte, and ViaCyte became a wholly-owned subsidiary of Vertex. In March 2023, (1) the Company and ViaCyte entered into an amendment to the ViaCyte JDCA, or the ViaCyte JDCA Amendment, and adjusted certain rights and obligations of the Company and ViaCyte under the ViaCyte JDCA, and (2) the Company and Vertex entered into a non-exclusive license agreement, or the Non-Ex License Agreement, pursuant to which the Company agreed to license to Vertex, on a non-exclusive basis, certain of its gene editing intellectual property to exploit certain products for the diagnosis, treatment or prevention of diabetes type 1, diabetes type 2 or insulin dependent / requiring diabetes throughout the world. Subsequently, ViaCyte elected to opt-out of the ViaCyte JDCA. Per the opt-out terms, the on-going collaboration assets are now wholly owned by the Company, subject to a royalty on future sales owed to ViaCyte. The opt-out became effective in early February 2024.
In 2024, the Company received a $10.0 million research milestone achieved in the fourth quarter of 2024 and recorded a receivable of $25.0 million as of December 31, 2024 related to an additional research milestone achieved under the Non-Ex License Agreement in the fourth quarter of 2024. The Company is eligible to receive additional milestone payments under the Non-Ex License Agreement of up to $125.0 million in aggregate, which are dependent on the achievement of pre-determined research, development and commercial milestones for certain products utilizing the licensed intellectual property. Additionally, the Company is eligible to receive tiered royalties on the sales of certain products in the low to mid-single digits.
Accounting Analysis
For purposes of this Note 6, the 2015 Collaboration Agreement, Amendment No. 1, Amendment No. 2, A&R Vertex JDCA, Amendment No. 1 to the A&R Vertex JDCA are collectively referred to as the “Vertex Hemoglobinopathy Agreements” and the Non-Ex License Agreement and ViaCyte JDCA Amendment are collectively referred to as the “March 2023 Diabetes Agreements.”
The Vertex Hemoglobinopathy Agreements and the March 2023 Diabetes Agreements include components of a customer-vendor relationship as defined under ASC 606, Revenue from Contracts with Customers, or ASC 606, collaborative arrangements as defined under ASC 808, and research and development costs as defined under ASC 730, Research and Development, or ASC 730. Specifically, with regards to the March 2023 Diabetes Agreements, the Company concluded that the non-exclusive license is a performance obligation under ASC 606 and the ongoing research and development services under the ViaCyte JDCA Amendment are a unit of account under ASC 808.
The Company has determined that recognition criteria for the Letter Agreement has not been met and will not be met until the priority review voucher is (i) utilized or (ii) there is sufficient profitability such that Vertex is obligated to pay the Company under the Letter Agreement.
Accounting Analysis Under ASC 606
Accounting for the March 2023 Diabetes Agreements
Recognition of Revenue
No revenue was recognized under the March 2023 Diabetes Agreements for thethree months ended March 31, 2025 and 2024, respectively.
Milestones under the Non-Ex License Agreement
As of March 31, 2025, the Company is eligible to receive potential future milestone payments from Vertex of up to $125.0 million in the aggregate under the Non-Ex License Agreement depending on the achievement of pre-determined research, development and commercial milestones for certain products utilizing the licensed intellectual property. Additionally, the Company is eligible to receive tiered royalties on the sales of certain products in the low to mid-single digits.
Each of the remaining milestones under the Non-Ex License Agreement are fully constrained as of March 31, 2025. There is uncertainty as to whether the events to obtain the research and developmental milestones will be achieved given the nature of clinical development and the stage of the CRISPR/Cas9 technology. The remaining research, development and regulatory milestones will be constrained until it is probable that a significant revenue reversal will not occur. Commercial milestones and royalties relate predominantly to a license of intellectual property and are determined by sales or usage-based thresholds. The commercial milestones and royalties are accounted for under the royalty recognition constraint and will be accounted for as constrained variable consideration. The Company applies the royalty recognition constraint for each commercial milestone and will not recognize revenue for each until the subsequent sale of a licensed product (achievement of each) occurs.
Accounting for the Vertex Hemoglobinopathy Agreements
Recognition of Revenue
No revenue was recognized under the Vertex Hemoglobinopathy Agreements for thethree months ended March 31, 2025 and 2024.
Deferred revenue
As of March 31, 2025 and December 31, 2024, there wasno current deferred revenue related to the Vertex Hemoglobinopathy Agreements. As of March 31, 2025, there was $12.3 million of non-current deferred revenue related to the Vertex Hemoglobinopathy Agreements, which is unchanged from December 31, 2024. The transaction price allocated to the remaining performance obligations was $12.3 million.
Milestones under the Vertex Hemoglobinopathy Agreements
The Company has evaluated the milestones that may be received in connection with the Vertex Hemoglobinopathy Agreements.
Under the 2015 Collaboration Agreement and subsequent amendments, the Company is eligible to receive up to $410.0 million in additional development, regulatory and commercial milestones and royalties on net product sales for each of the three collaboration targets that Vertex licensed in 2019. Each milestone is payable only once per collaboration target, regardless of the number of products directed to such collaboration target that achieve the relevant milestone event.
The Company has the option to conduct research at its own cost in certain defined areas. If such research is beneficial to a program under the Vertex Hemoglobinopathy Agreements and the applicable product ultimately achieves regulatory approval in such areas, the Company could be entitled to receive from Vertex certain milestone payments aggregating to high eight digits.
In addition to the milestones described under the Vertex Hemoglobinopathy Agreements, the Company is eligible to receive potential future payments of up to $775.0 million upon the successful achievement of specified development, regulatory and commercial milestones under a strategic collaboration and license agreement with Vertex from 2019 for the development and commercialization of products for the treatment of Duchenne muscular dystrophy and myotonic dystrophy Type 1, or DM1. The Company is also eligible to receive tiered royalties on future net sales on any products that may result from this collaboration; however, the Company has the option to forego the DM1 milestones and royalties to co-develop and co-commercialize all DM1 products globally.
Each of the remaining milestones described above are fully constrained as of March 31, 2025. There is uncertainty that the events to obtain the research and developmental milestones will be achieved given the nature of clinical development and the stage of the CRISPR/Cas9 technology. The remaining research, development and regulatory milestones will be constrained until it is probable that a significant revenue reversal will not occur. Commercial milestones and royalties relate predominantly to a license of intellectual property and are determined by sales or usage-based thresholds. The commercial milestones and royalties are accounted for under the royalty recognition constraint and will be accounted for as constrained variable consideration. The Company applies the royalty recognition constraint for each commercial milestone and will not recognize revenue for each until the subsequent sale of a licensed product (achievement of each) occurs.
Accounting Analysis under ASC 808
Vertex Hemoglobinopathy Agreements
In connection with the Vertex Hemoglobinopathy Agreements, the Company identified the following collaborative elements, which are accounted for under ASC 808: (i) development and commercialization services for shared products, including any transition services related to CASGEVY under the A&R Vertex JDCA; (ii) R&D Services for follow-on products; and (iii) committee participation. The related impact of the cost sharing under the Vertex Hemoglobinopathy Agreements is included within collaboration expense, net, in the condensed consolidated statements of operations and comprehensive loss. During the three months ended March 31, 2025 and 2024, the Company recognized $57.5 million and $47.0 million of collaboration expense, net, respectively. Collaboration expense, net, during thethree months ended March 31, 2025 and 2024 was net of $0.1 million and $0.8 million of reimbursements from Vertex, respectively.
- Commitments and Contingencies
Leases
Refer to Note 7 to the consolidated financial statements in the Company’s 2024 Annual Report on Form 10-K filed with the SEC on February 11, 2025 for discussion of the Company’s lease arrangements.
Litigation
In the ordinary course of business, the Company is or has been from time to time involved in lawsuits, investigations, proceedings and threats of litigation related to, among other things, the Company’s intellectual property estate (including certain in-licensed intellectual property), commercial arrangements and other matters. Such proceedings may include quasi-litigation, inter partes administrative proceedings in the U.S. Patent and Trademark Office and the European Patent Office or patent offices in other countries, involving the Company’s intellectual property estate including certain in-licensed intellectual property. The outcome of any of the foregoing, regardless of the merits, is inherently uncertain. In addition, litigation and related matters are costly and may divert the attention of Company’s management and other resources that would otherwise be engaged in other activities. If the Company is unable to prevail in any such proceedings, the Company’s business, results of operations, liquidity and financial condition could be adversely affected.
Letters of Credit
As of March 31, 2025, the Company had restricted cash of $11.5 million, representing letters of credit securing the Company’s obligations under certain leased facilities. The letters of credit are secured by cash held in a restricted depository account, with $3.5 million included in “Prepaid expenses and other current assets” and $8.0 million included in “Restricted cash” on the Company's condensed consolidated balance sheets as of March 31, 2025.
Research, Manufacturing, License and Intellectual Property Agreements
The Company has engaged several research institutions and companies to identify new delivery strategies and applications of the Company’s gene editing technology. The Company is also a party to a number of license agreements which require significant upfront payments and may be required to make future royalty payments and potential milestone payments from time to time. In addition, the Company is also a party to intellectual property agreements, which require maintenance and milestone payments from time to time. Further, the Company is a party to a number of manufacturing agreements that require upfront payments for the future performance of services.
In connection with these agreements, on a product-by-product basis, the counterparties are eligible to receive up to low eight-digit potential payments upon specified research, development and regulatory milestones. In addition, on a product-by-product basis, the counterparties are eligible to receive potential commercial milestone payments based on specified annual sales thresholds. The potential payments are low-single digit percentages of the specified annual sales thresholds. The counterparties are also eligible to receive low single-digit royalties on future net sales.
Under certain circumstances and if certain contingent future events occur, Vertex is eligible to receive up to $395.0 million in potential specified research, development, regulatory and commercial milestones and tiered single-digit percentage royalties on future net sales related to a specified target under an amendment to the 2015 Collaboration Agreement (as such term is defined in Note 6 above). In addition, Vertex has the option to conduct research at its own cost in certain defined areas that, if beneficial to a program under the Vertex Hemoglobinopathy Agreements and the applicable product ultimately achieves regulatory approval, could result in the Company owing Vertex certain milestone payments aggregating to high eight digits, subject to certain limitations on the profitability of the program.
Under the A&R Vertex JDCA, for 2022, 2023 and 2024, the Company had an option to defer a portion of its share of costs if spending on the CASGEVY program exceeded $110.3 million, which the Company exercised in each such year, resulting in deferred costs of $221.8 million, in the aggregate. Any deferred amounts under the A&R Vertex JDCA, as amended, are only payable to Vertex as an offset against future profitability of the CASGEVY program and the amounts payable are capped at a specified maximum amountper year. These deferred costs on the CASGEVY program will be recognized by us when recoverability of such deferred amounts by Vertex is probable and the amount can be reasonably estimated. As of March 31, 2025, no contingent payments have been accrued to date.
- Share Capital
All of the Company’s common shares are authorized under Swiss corporate law with a nominal value of 0.03 CHF per share. Though the nominal value of common shares is stated in Swiss francs, the Company continues to use U.S. dollars as its reporting currency for preparing the condensed consolidated financial statements.
As of March 31, 2025, the Company's share capital consists of 89,905,892 registered common shares with a nominal value of CHF 0.03 per share,8,202,832 registered common shares reserved for potential issuance of bonds or similar instruments and 19,537,850 registered common shares reserved for the Company’s employee equity incentive plans. In addition, the Board of Directors is authorized to conduct one or more increases of the share capital at any time until June 8, 2028, or the expiration of the capital band if earlier, up to an upper limit of CHF 3,142,094.52 by issuing a corresponding number of registered shares with a nominal value of CHF 0.03 each to be fully paid in. As of March 31, 2025, the number of shares that may be issued under the capital band is 14,830,592 registered common shares.
Common Share Issuances
At-the-Market Offerings
In August 2019, the Company entered into an Open Market Sale Agreement SM with Jefferies LLC, or Jefferies, under which the Company was able to offer and sell, from time to time at its sole discretion through Jefferies, as its sales agent, its common shares, par value of CHF 0.03 per share, or the August 2019 Sales Agreement. In January 2021, in connection with the August 2019 Sales Agreement, the Company filed a prospectus supplement with the SEC to offer and sell, from time to time, common shares having aggregate gross proceeds of up to $600.0 million. In August 2024, the Company filed a new prospectus supplement with the SEC, which replaced the previous prospectus supplements filed in January 2021 and July 2021, respectively, to offer and sell, from time to time, the common shares remaining under the original prospectus supplement and July 2021 prospectus supplement having aggregate gross proceeds of up to $378.6 million, or, together with the January 2021 prospectus supplement and July 2021 prospectus supplement, the 2021 ATM.
For the three months ended March 31, 2025, the Company issued and sold an aggregate of 0.2 million common shares under the 2021 ATM at an average price of $54.33 per share for aggregate proceeds of $8.7 million. Activity under the 2021 ATM was not material for thethree months ended March 31, 2024. Equity issuance costs, excluding stamp taxes, were not material for the three months ended March 31, 2025 and 2024.
As of March 31, 2025, the Company has issued and sold an aggregate of 2.1 million common shares under the 2021 ATM at an average price of $117.41 per share for aggregate proceeds of $241.9 million, which were net of equity issuance costs of $3.3 million, excluding stamp taxes. As of March 31, 2025, common shares having aggregate gross proceeds up to $354.8 million remain available under the 2021 ATM.
Additional Financings
In February 2024, the Company entered into an investment agreement for the sale of approximately $280.0 million of its common shares to a group of institutional investors in a registered direct offering, at a price per share of $71.50. The Company received net proceeds of $279.0 million, excluding stamp taxes due of $2.8 million.
- Stock-based Compensation
During thethree months ended March 31, 2025 and 2024, the Company recognized the following stock-based compensation expense (in thousands):
| Three Months Ended March 31, | ||
|---|---|---|
| 2025 | 2024 | |
| Research and development | $9,732 | $11,182 |
| General and administrative | 10,480 | 8,223 |
| Total | $20,212 | $19,405 |
Stock option activity
The following table summarizes stock option activity for thethree months ended March 31, 2025:
| Shares | Weighted-averageexercise priceper share | |
|---|---|---|
| Outstanding at December 31, 2024 | 7,288,883 | $58.07 |
| Granted | 894,655 | 42.10 |
| Exercised | (39,996) | 36.65 |
| Cancelled or forfeited | (257,062) | 58.84 |
| Outstanding at March 31, 2025 | 7,886,480 | $56.34 |
| Exercisable at March 31, 2025 | 5,039,236 | $58.98 |
| Vested and expected to vest at March 31, 2025 | 7,886,480 | $56.34 |
As of March 31, 2025, total unrecognized compensation expense related to stock options was $85.2 million, which the Company expects to recognize over a remaining weighted-average period of 2.8 years.
Restricted stock activity
The following table summarizes restricted stock activity for thethree months ended March 31, 2025:
| Shares | Weighted-AverageGrant DateFair Value | |
|---|---|---|
| Unvested balance at December 31, 2024 | 2,397,643 | $59.21 |
| Granted | 656,721 | 42.11 |
| Vested | (396,943) | 67.98 |
| Cancelled or forfeited | (129,989) | 61.44 |
| Unvested balance at March 31, 2025 | 2,527,432 | $53.28 |
As of March 31, 2025, total unrecognized compensation expense related to unvested restricted common shares was $103.6 million, which the Company expects to recognize over a remaining weighted-average vesting period of 3.0 years.
- Net Loss Per Share Attributable to Common Shareholders
Basic net loss per share is calculated by dividing net loss attributable to common shareholders by the weighted-average number of common shares outstanding during the period. Diluted net loss per share is calculated by adjusting weighted-average shares outstanding for the dilutive effect of common stock equivalents outstanding for the period using the treasury stock method. For purposes of the diluted net loss per share calculation, stock options, unvested restricted common shares and ESPP shares are considered to be common stock equivalents but are excluded from the calculation of diluted net loss per share, as their effect would be anti-dilutive; therefore, basic and diluted net loss per share were the same for all periods presented as a results of the Company’s net loss. The Company’s net loss is net loss attributable to common shareholders for all periods presented.
The Company did not include the securities in the following table in the computation of the net loss per share calculations because the effect would have been anti-dilutive during each period:
| Three Months Ended March 31, | ||
|---|---|---|
| 2025 | 2024 | |
| Outstanding options | 7,886,480 | 7,164,349 |
| Unvested restricted common shares | 2,527,432 | 2,082,032 |
| ESPP | 13,330 | 8,630 |
| Total | 10,427,242 | 9,255,011 |
- Income Taxes
During the three months ended March 31, 2025 and 2024, the Company recorded an income tax provision of $1.1 million and $0.7 million, respectively, representing an effective tax rate of (0.8%) and (0.6%), respectively. The income tax provision for thethree months ended March 31, 2025 is primarily attributable to the income generated by the Company's U.S. subsidiaries. The difference in the statutory tax rate and effective tax rate is primarily a result of the jurisdictional mix of earnings, research credits generated, and the valuation allowance recorded against certain deferred tax assets. The Company maintains a valuation allowance against certain deferred tax assets that are not more-likely-than-not realizable.
- Segment Information
The Company operates asone operating segment, which is the business of discovering, developing and commercializing therapies derived from or incorporating genome-editing technology. The determination of a single business segment is consistent with the consolidated financial information regularly provided to the Company’s chief operating decision maker, or CODM. The Company’schief executive officer, as the CODM, uses consolidated, single-segment financial information for purposes of evaluating performance, making operating decisions, allocating resources and planning and forecasting for future periods.
The CODM assesses performance and decides how to allocate resources based on consolidated net loss. The measure is used to monitor budget verses actual results to evaluate the performance of the segment.
The measure of segment assets is reported on the condensed consolidated balance sheets as total consolidated assets. All material long-lived assets are located in the United States. Long-lived assets consist of property and equipment, net, and operating lease right-of-use assets.
The following table illustrates information about segment revenue, significant segment expenses and segment operating loss for the periods presented (in thousands):
| Three Months Ended March 31, | ||
|---|---|---|
| 2025 | 2024 | |
| Revenue: | $865 | $504 |
| Less1: | ||
| Research and development expense2 | 60,333 | 62,350 |
| General and administrative expense3 | 6,517 | 7,551 |
| Collaboration expense, net | 57,509 | 46,966 |
| Stock-based compensation expense | 20,212 | 19,405 |
| Depreciation expense | 4,718 | 4,819 |
| Other segment items4 | (12,428) | (23,996) |
| Segment net loss | (135,996) | (116,591) |
| Reconciliation of profit or loss: | ||
| Adjustments or reconciling items | — | — |
| Consolidated net loss | (135,996) | (116,591) |
(1) The significant expense categories and amounts align with the segment-level information that is regularly provided to the CODM.
(2) Research and development expense for the three months ended March 31, 2025 and 2024 excludes $9.7 million and $11.2 million of stock-based compensation expense, respectively, and $2.4 million and $2.6 million of depreciation expense, respectively.
(3) General and administrative expense for the three months ended March 31, 2025 and 2024 excludes $10.5 million and $8.2 million of stock-based compensation expense, respectively, and $2.3 million and $2.2 million of depreciation expense, respectively.
(4) Other segment items include interest income, net, the change in fair value of corporate equity securities and income tax expense.
The Company operates in the United States and Switzerland. Collaboration revenue is attributed to the CRISPR Therapeutics AG entity, which is domiciled in Switzerland.
The text of this filing, as the company filed it with the SEC, without its images or exhibits. This is not investment advice.