Q32 Bio Inc. 10-Q

Item 1. Financial Statements. HOMOLOGY MEDICINES, INC. CONDENSED CONSOLIDATED BALANCE SHEETS (in thousands, except share and per share amounts) (UNAUDITED) As of September 30, 2020 December 31, 2019 Assets Current assets: Cash and cash equivalents $ 178,003 $ 53,774 Short-term investments — 208,614 Prepaid expenses and other current assets 3,252 4,189 Total current assets 181,255 266,577 Property and equipment, net 39,684 42,716 Right-of-use assets 6,149 — Restricted cash 1,274 1,274 Total assets $ 228,362 $ 310,567 Liabilities and stockholders' equity Current liabilities: Accounts payable $ 3,316 $ 2,608 Accrued expenses and other liabilities 8,426 7,644 Operating lease liabilities 2,497 — Deferred rent — 1,313 Deferred revenue 2,939 809 Total current liabilities 17,178 12,374 Non-current liabilities: Operating lease liabilities, net of current portion 13,540 — Deferred rent, net of current portion — 9,544 Deferred revenue, net of current portion 27,422 30,142 Total liabilities 58,140 52,060 Commitments and contingencies (Note 7) Stockholders’ equity: Preferred stock, $ 0.0001 par value, 10,000,000 shares authorized; no shares issued and outstanding — — Common stock, $ 0.0001 par value; 200,000,000 shares authorized; 45,263,849 and 45,138,408 shares issued as of September 30, 2020 and December 31, 2019, respectively; and 45,259,747 an d 45,116,742 shares outstanding as of September 30, 2020 and December 31, 2019, respectively 4 4 Additional paid-in capital 468,795 457,994 Accumulated other comprehensive gain — 183 Accumulated deficit ( 298,577 ) ( 199,674 ) Total stockholders’ equity 170,222 258,507 Total liabilities and stockholders' equity $ 228,362 $ 310,567 See notes to condensed consolidated financial statements. 5 HOMOLOGY MEDICINES, INC. CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (in thousands, except share and per share amounts) (UNAUDITED) Three months ended September 30, Nine months ended September 30, 2020 2019 2020 2019 Collaboration revenue $ 567 $ 441 $ 1,722 $ 1,103 Operating expenses: Research and development 20,417 25,691 77,197 69,056 General and administrative 8,423 6,038 24,986 16,431 Total operating expenses 28,840 31,729 102,183 85,487 Loss from operations ( 28,273 ) ( 31,288 ) ( 100,461 ) ( 84,384 ) Other income: Interest income 41 1,661 1,558 4,635 Total other income 41 1,661 1,558 4,635 Net loss $ ( 28,232 ) $ ( 29,627 ) $ ( 98,903 ) $ ( 79,749 ) Net loss per share-basic and diluted $ ( 0.62 ) $ ( 0.67 ) $ ( 2.19 ) $ ( 1.92 ) Weighted-average common shares outstanding-basic and diluted 45,227,231 43,904,812 45,196,459 41,503,545 See notes to condensed consolidated financial statements. 6 HOMOLOGY MEDICINES, INC. CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSS (in thousands) (UNAUDITED) Three months ended September 30, Nine months ended September 30, 2020 2019 2020 2019 Net loss $ ( 28,232 ) $ ( 29,627 ) $ ( 98,903 ) $ ( 79,749 ) Other comprehensive gain (loss): Change in unrealized gain (loss) on available for sale securities, net ( 11 ) 68 ( 183 ) 331 Total other comprehensive gain (loss) ( 11 ) 68 ( 183 ) 331 Comprehensive loss $ ( 28,243 ) $ ( 29,559 ) $ ( 99,086 ) $ ( 79,418 ) See notes to condensed consolidated financial statements. 7 HOMOLOGY MEDICINES, INC. CONDENSED CONSOLIDATED STATEMENTS OF STOCKHOLDERS’ EQUITY (in thousands, except share and per share amounts) (UNAUDITED) Common Stock $0.0001 Par Value Additional Paid-in Accumulated Other Comprehensive Accumulated Total Stockholders’ Shares Amount Capital Gain (Loss) Deficit Equity Balance at January 1, 2019 37,358,526 $ 3 $ 292,187 $ ( 77 ) $ ( 95,758 ) $ 196,355 Vesting of common stock from option exercises 31,584 — 18 — — 18 Issuance of common stock from option exercises 18,876 — 21 — — 21 Issuance of common stock pursuant to employee stock purchase plan 28,311 — 396 — — 396 Stock-based compensation — — 1,243 — — 1,243 Other comprehensive gain — — — 83 — 83 Net loss — — — — ( 23,852 ) ( 23,852 ) Balance at March 31, 2019 37,437,297 $ 3 $ 293,865 $ 6 $ ( 119,610 ) $ 174,264 Issuance of common stock in follow-on offering, net of discounts and issuance costs 6,388,889 1 134,524 — — 134,525 Vesting of common stock from option exercises 31,024 — 18 — — 18 Issuance of common stock from option exercises 12,404 — 55 — — 55 Stock-based compensation — — 1,970 — — 1,970 Other comprehensive gain — — — 180 — 180 Net loss — — — — ( 26,270 ) ( 26,270 ) Balance at June 30, 2019 43,869,614 $ 4 $ 430,432 $ 186 $ ( 145,880 ) $ 284,742 Vesting of common stock from option exercises 16,104 — 11 — — 11 Issuance of common stock from option exercises 43,429 — 209 — — 209 Issuance of common stock pursuant to employee stock purchase plan 26,923 — 433 — — 433 Stock-based compensation — — 1,986 — — 1,986 Other comprehensive gain — — — 68 — 68 Net loss — — — — ( 29,627 ) ( 29,627 ) Balance at September 30, 2019 43,956,070 $ 4 $ 433,071 $ 254 $ ( 175,507 ) $ 257,822 Common Stock $0.0001 Par Value Additional Paid-in Accumulated Other Comprehensive Accumulated Total Stockholders’ Shares Amount Capital Gain (Loss) Deficit Equity Balance at January 1, 2020 45,116,742 $ 4 $ 457,994 $ 183 $ ( 199,674 ) $ 258,507 Vesting of common stock from option exercises 15,542 — 12 — — 12 Issuance of common stock from option exercises 34,311 — 135 — — 135 Issuance of common stock pursuant to employee stock purchase plan 39,471 — 537 — — 537 Stock-based compensation — — 2,883 — — 2,883 Other comprehensive loss — — — ( 202 ) — ( 202 ) Net loss — — — — ( 35,331 ) ( 35,331 ) Balance at March 31, 2020 45,206,066 $ 4 $ 461,561 $ ( 19 ) $ ( 235,005 ) $ 226,541 Vesting of common stock from option exercises 1,011 — 4 — — 4 Issuance of common stock from option exercises 3,576 — 16 — — 16 Stock-based compensation — — 3,453 — — 3,453 Other comprehensive gain — — — 30 — 30 Net loss — — — — ( 35,340 ) ( 35,340 ) Balance at June 30, 2020 45,210,653 $ 4 $ 465,034 $ 11 $ ( 270,345 ) $ 194,704 Vesting of common stock from option exercises 1,011 — 5 — — 5 Issuance of common stock from option exercises 3,706 — 9 — — 9 Issuance of common stock pursuant to employee stock purchase plan 44,377 — 401 — — 401 Stock-based compensation — — 3,346 — — 3,346 Other comprehensive gain — — — ( 11 ) — ( 11 ) Net loss — — — — ( 28,232 ) ( 28,232 ) Balance at September 30, 2020 45,259,747 $ 4 $ 468,795 $ — $ ( 298,577 ) $ 170,222 See notes to condensed consolidated financial statements. 8 HOMOLOGY MEDICINES, INC. CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS (in thousands) (UNAUDITED) Nine months ended September 30, 2020 2019 Cash flows from operating activities: Net loss $ ( 98,903 ) $ ( 79,749 ) Adjustments to reconcile net loss to net cash used in operating activities: Depreciation 5,909 4,599 Noncash lease expense 686 — Stock-based compensation expense 9,682 5,199 Amortization of premium on short-term investments ( 197 ) ( 1,601 ) Changes in operating assets and liabilities: Prepaid expenses and other current assets 938 2,541 Accounts payable 486 1,511 Accrued expenses and other liabilities 924 2,121 Deferred revenue ( 590 ) ( 379 ) Deferred rent — 733 Operating lease liabilities ( 1,655 ) — Net cash used in operating activities ( 82,720 ) ( 65,025 ) Cash flows from investing activities: Purchases of short-term investments ( 19,991 ) ( 257,389 ) Maturities of short-term investments 228,620 243,725 Purchases of property and equipment ( 2,778 ) ( 18,108 ) Net cash provided by (used in) investing activities 205,851 ( 31,772 ) Cash flows from financing activities: Proceeds from issuance of common stock in follow-on public offering, net of discounts and issuance costs — 134,524 Proceeds from issuance of common stock pursuant to employee stock purchase plan 938 829 Proceeds from issuance of common stock from option exercises 160 285 Net cash provided by financing activities 1,098 135,638 Net change in cash, cash equivalents and restricted cash 124,229 38,841 Cash, cash equivalents and restricted cash, beginning of period 55,048 39,992 Cash, cash equivalents and restricted cash, end of period $ 179,277 $ 78,833 Supplemental disclosures of noncash investing and financing activities: Reclassification of liability for common stock vested $ 21 $ 47 Property and equipment additions included in accounts payable $ 677 $ 682 Property and equipment additions included in accrued expenses and other liabilities $ 126 $ 705 Unrealized (loss) gain on available for sale securities, net $ ( 183 ) $ 331 See notes to condensed consolidated financial statements. 9 HOMOLOGY MEDICINES, INC. NOTES TO CONDENSED CONSOLIDATED FINANCIAL STATEMENTS (in thousands, except share and per share amounts) (UNAUDITED) 1. NATURE OF BUSINESS AND BASIS OF PRESENTATION Nature of Business —Homology Medicines, Inc. (the “Company”) is a clinical stage biopharmaceutical company dedicated to translating proprietary gene editing and gene therapy technology into novel treatments for patients with rare genetic diseases with significant unmet medical needs by curing the underlying cause of the disease. The Company was founded in March 2015 as a Delaware corporation. Its principal offices are in Bedford, Massachusetts. Since its inception, the Company has devoted substantially all of its resources to recruiting personnel, developing its technology platform and advancing its pipeline of product candidates, developing and implementing manufacturing processes, building out manufacturing and research and development space, and maintaining and building its intellectual property portfolio. The Company is subject to a number of risks similar to those of other companies conducting high-risk, early-stage research and development of product candidates. Principal among these risks are dependency on key individuals and intellectual property, competition from other products and companies, and the technical risks associated with the successful research, development and manufacturing of its product candidates. The Company’s success is dependent upon its ability to continue to raise additional capital in order to fund ongoing research and development, obtain regulatory approval of its products, successfully commercialize its products, generate revenue, meet its obligations, and, ultimately, attain profitable operations. On April 2, 2018, the Company completed its initial public offering (“IPO”) with the sale of 10,350,000 shares of its common stock, including shares issued upon the exercise in full of the underwriters’ over-allotment option, at a public offering price of $ 16.00 per share, resulting in net proceeds of $ 150.8 million, after deducting underwriting discounts and commissions and offering expenses. Upon the closing of the IPO, all of the Company’s outstanding shares of convertible preferred stock automatically converted into 24,168,656 shares of common stock at the applicable conversion ratio then in effect. On April 12, 2019, the Company completed a follow-on public offering of its common stock. The Company sold 5,555,556 shares of its common stock at a public offering price of $ 22.50 per share and received net proceeds of $ 116.9 million, after deducting underwriting discounts and commissions and offering expenses. In addition, on April 26, 2019 and May 7, 2019, in connection with the exercise in full of the underwriters’ option to purchase additional shares, the Company issued an aggregate of 833,333 shares of its common stock at a public offering price of $ 22.50 per share and received net proceeds of $ 17.6 million, after deducting underwriting discounts and commissions. On March 12, 2020, the Company filed a Registration Statement on Form S-3 (File No. 333-237131) (the “Shelf”) with the Securities and Exchange Commission (“SEC”) in relation to the registration of common stock, preferred stock, debt securities, warrants and/or units of any combination thereof for a period up to three years from the date of the filing. The Shelf became effective on March 12, 2020. The Company also simultaneously entered into a sales agreement with Cowen and Company, LLC (“Cowen”), as sales agent, providing for the offering, issuance and sale by the Company of up to an aggregate $ 150.0 million of its common stock from time to time in “at-the-market” offerings under the Shelf (the “ATM”). The Company did no t sell any shares of common stock pursuant to the ATM during the nine months ended September 30, 2020. At September 30, 2020, there remained $ 150.0 million of common stock available for sale under the ATM. Simultaneous with the filing of the Shelf, the previous Registration Statement on Form S-3 (File No. 333-230664) filed with the SEC on April 1, 2019, and a prior sales agreement with Cowen providing for the offering, issuance and sale by the Company of up to an aggregate $ 100.0 million of its common stock from time to time in “at-the-market” offerings, with $ 76.8 million of remaining capacity, were terminated. To date, the Company has not generated any revenue from product sales and does not expect to generate any revenue from the sale of product in the foreseeable future. Through September 30, 2020, the Company has financed its operations primarily through public offerings of its common stock, the issuance of convertible preferred stock, and with proceeds from its collaboration and license agreement with Novartis Institutes of BioMedical Research, Inc. (“Novartis”) (see Note 10). During the nine months ended September 30, 2020, the Company incurred a net loss of $ 98.9 million and as of September 30, 2020, had $ 298.6 million in accumulated deficit. The Company expects to incur additional operating losses and negative operating cash flows for the foreseeable future. 10 Based on current projections, management now believes that existing cash and cash equivalents, together with the anticipated proceeds of $ 60.0 million from the Pfizer Inc. private placement (see Note 11), will enable the Company to continue its operations into the third quarter of 2022. In the absence of a significant source of recurring revenue, the continued viability of the Company beyond that point is dependent on its ability to continue to raise additional capital to finance its operations. There can be no assurance that the Company will be able to obtain sufficient capital to cover its costs on acceptable terms, if at all. Basis of Presentation — The accompanying unaudited condensed consolidated financial statements have been prepared by the Company in conformity with accounting principles generally accepted in the United States of America (“U.S. GAAP”) and pursuant to the rules and regulations of the SEC for interim financial statements. Certain information and footnote disclosures normally included in financial statements prepared in accordance with U.S. GAAP have been condensed or omitted pursuant to such rules and regulations. However, the Company believes that the disclosures are adequate to make the information presented not misleading. These condensed consolidated financial statements should be read in conjunction with the Company’s audited consolidated financial statements and the notes thereto for the year ended December 31, 2019, included in the Company's Annual Report on Form 10-K on file with the SEC. The unaudited interim condensed consolidated financial statements have been prepared on the same basis as the audited consolidated financial statements. In the opinion of management, the accompanying unaudited interim condensed consolidated financial statements contain all adjustments which are necessary for a fair statement of the Company’s financial position as of September 30, 2020, and consolidated results of operations for the three and nine months ended September 30, 2020 and 2019, and cash flows for the nine months ended September 30, 2020 and 2019. Such adjustments are of a normal and recurring nature. The results of operations for the three and nine months ended September 30, 2020 are not necessarily indicative of the results of operations that may be expected for the year ending December 31, 2020. 2. SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES Principles of Consolidation —The Company’s condensed consolidated financial statements include the accounts of the Company and its subsidiary, Homology Medicines Securities Corporation, a wholly owned Massachusetts corporation, for the sole purpose of buying, selling, and holding securities on the Company’s behalf. All intercompany balances and transactions have been eliminated in the condensed consolidated financial statements. Use of Estimates —The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets, liabilities, revenue, and expenses, and the disclosure of contingent assets and liabilities as of and during the reporting period. The Company bases its estimates and assumptions on historical experience when available and on various factors that it believes to be reasonable under the circumstances. Significant estimates and assumptions reflected in these condensed consolidated financial statements include, but are not limited to, revenue recognition, accrued research and development expenses and useful lives assigned to property and equipment. The Company assesses estimates on an ongoing basis; however, actual results could materially differ from those estimates. Comprehensive Income (Loss) —Comprehensive income (loss) is defined as the change in equity of a business enterprise during a period from transactions and other events and circumstances from non-owner sources. The Company’s only element of other comprehensive income (loss) is unrealized gains and losses on available-for-sale investments. Cash and Cash Equivalents and Restricted Cash —Cash and cash equivalents consist of standard checking accounts, money market accounts and certain investments. The Company considers all highly liquid investments with original or remaining maturities at the time of purchase of 90 days or less to be cash equivalents. Restricted cash consists of cash serving as collateral for letters of credit issued for security deposits for the Company’s facility leases in Bedford, Massachusetts. The following table provides a reconciliation of cash, cash equivalents and restricted cash to amounts shown in the condensed consolidated statements of cash flows: September 30, 2020 2019 (in thousands) Cash and cash equivalents $ 178,003 $ 77,559 Restricted cash 1,274 1,274 Total cash, cash equivalents and restricted cash $ 179,277 $ 78,833 11 Short-Term Investments —Short-term investments represent holdings of available-for-sale marketable securities in accordance with the Company’s investment policy and cash management strategy. Short-term investments mature within one-year from the balance sheet date. Investments in marketable securities are recorded at fair value, with any unrealized gains and losses reported within accumulated other comprehensive income as a separate component of stockholders’ equity until realized or until a determination is made that an other-than-temporary decline in market value has occurred. Any premium or discount arising at purchase is amortized and/or accreted to interest income and/or expense over the life of the of the underlying security. Such amortization and accretion, together with interest on securities, are included in interest income on the Company’s condensed consolidated statements of operations. The cost of marketable securities sold is determined based on the specific identification method and any realized gains or losses on the sale of investments are reflected as a component of other income. Deferred Offering Costs —The Company capitalizes incremental legal, professional accounting and other third-party fees that are directly associated with equity financings as other current assets until the transactions are completed. After equity financings are complete, these costs are recorded in stockholders’ equity as a reduction of additional paid-in capital generated as a result of the offering. Leases — In February 2016, the Financial Accounting Standards Board (“FASB”) issued Accounting Standards Update (“ASU”) No. 2016-02, Leases (Topic 842) (“ASU 2016-02”), which eliminated the tests for lease classification under prior U.S. GAAP and requires lessees to recognize right-of-use assets and related lease liabilities on the balance sheet. The FASB subsequently issued the following amendments to ASU 2016-02 that have the same effective date and transition date: ASU No. 2018-01 - Leases (Topic 842): Land Easement Practical Expedient for Transition to Topic 842 , ASU No. 2018-10 - Codification Improvements to Topic 842 , Leases , ASU No. 2018-11 – Leases (Topic 842): Targeted Improvements , ASU No. 2018-20 – Leases (Topic 842): Narrow-Scope Improvements for Lessors , and ASU No. 2019-01 – Leases (Topic 842): Codification Improvements . The Company adopted these amendments with ASU 2016-02 (collectively, the new leasing standards) effective January 1, 2020. The Company adopted the new leasing standards using the modified retrospective approach, as of January 1, 2020, with no restatement of prior periods or cumulative adjustments to retained earnings. The Company elected the package of practical expedients permitted under the transition guidance within the new standard, which, among other things, allows the Company to carry forward the historical lease classification. In addition, the Company elected the practical expedient not to apply the recognition requirements in the leasing standards to short-term leases (a lease that at commencement date has a lease term of 12 months or less and does not contain a purchase option that it is reasonably certain to exercise) and the practical expedient that permits lessees to make an accounting policy election (by class of underlying asset) to not separate lease components of a contract from non-lease components. The Company determines if an arrangement is a lease at contract inception. The Company’s contracts are determined to contain a lease when all of the following criteria based on the specific circumstances of the arrangement are met: (1) there is an identified asset for which there are no substantive substitution rights; (2) the Company has the right to obtain substantially all of the economic benefits from the identified asset; and (3) the Company has the right to direct the use of the identified asset. At the commencement date, operating lease liabilities and their corresponding right-of-use assets are recorded based on the present value of future lease payments over the expected lease term. The Company’s lease agreements do not provide an implicit rate. As a result, the Company utilizes an estimated incremental borrowing rate to discount lease payments, which is based on the rate of interest the Company would have to pay to borrow a similar amount on a collateralized basis over a similar term. Certain adjustments to the right-of-use asset may be required for items such as initial direct costs paid or lease incentives received. Operating lease cost is recognized over the expected term on a straight-line basis. Variable lease cost is recognized as incurred. The Company acts as sublessor related to a sublease of the Company's former headquarters. Fixed sublease payments received are recognized on a straight-line basis over the sublease term. Right-of-use assets are periodically evaluated for impairment. The expected lease term for those leases commencing prior to January 1, 2020 did not change with the adoption of the new leasing standards. The expected lease term for leases commencing after the adoption of the new leasing standards includes noncancelable lease periods and, when applicable, periods covered by an option to extend the lease if the Company is reasonably certain to exercise that option, as well as periods covered by an option to terminate the lease if the Company is reasonably certain not to exercise that option. 12 As a result of the adoption of the new leasing standards, on January 1, 2020, the Company recorded non-cash transactions to recognize a right-of-use asset of $ 6.8 million, operating lease liabilities of $ 17.7 million and the derecognition of deferred rent of $ 10.9 million originally accounted for under legacy guidance. The adoption did not have a material impact on the condensed consolidated statement of operations. For additional information on the adoption of the new leasing standards, refer to Note 7. January 1, 2020 (in thousands) Prior to adoption of new leasing standards Adjustment for adoption of new leasing standards As Adjusted Right-of-use assets (1)(2) $ — $ 6,835 $ 6,835 Deferred rent (2) $ 1,313 $ ( 1,313 ) $ — Deferred rent, net of current portion (2) $ 9,544 $ ( 9,544 ) $ — Operating lease liabilities (3) $ — $ 2,251 $ 2,251 Operating lease liabilities, net of current portion (3) $ — $ 15,441 $ 15,441 (1) Represents capitalization of operating right-of-use assets (2) Represents reclassification of deferred rent and incentives as a reduction to operating right-of-use assets (3) Represents recognition of operating lease liabilities Research and Development Costs —Research and development costs are charged to expense as incurred. Research and development expense consists of expenses incurred in performing research and development activities, including salaries and benefits, materials and supplies, preclinical and clinical expenses, stock-based compensation expense, depreciation of equipment, contract services, and other outside expenses. Costs for certain development activities are recognized based on an evaluation of the progress to completion of specific tasks using information provided to the Company by its vendors on their actual costs incurred. Payments for these activities are based on the terms of the individual arrangements, which may differ from the pattern of costs incurred, and are reflected in the consolidated financial statements as prepaid expense or accrued research and development expense. Revenue Recognition— Revenue is recognized in accordance with FASB Accounting Standards Codification (“ASC”) Topic 606, Revenue from Contracts with Customers (“ASC 606”). On January 1, 2019, the Company adopted ASC 606 using the full retrospective transition method. Under ASC 606, the Company recognizes revenue when its customer obtains control of promised goods or services, in an amount that reflects the consideration which the entity expects to receive in exchange for those goods or services. To determine the appropriate amount of revenue to be recognized for arrangements determined to be within the scope of ASC 606, the Company performs the following five steps: (i) identification of the promised goods or services in the contract; (ii) determination of whether the promised goods or services are performance obligations including whether they are distinct in the context of the contract; (iii) measurement of the transaction price, including the constraint on variable consideration; (iv) allocation of the transaction price to the performance obligations; and (v) recognition of revenue when (or as) the Company satisfies each performance obligation. The Company only applies the five-step model to contracts when it is probable that the entity will collect consideration it is entitled to in exchange for the goods or services it transfers to the customer. The promised goods or services in the Company’s arrangements would likely consist of a license, rights to the Company’s intellectual property or research, development and manufacturing services. Performance obligations are promised goods or services in a contract to transfer a distinct good or service to the customer and are considered distinct when (i) the customer can benefit from the good or service on its own or together with other readily available resources and (ii) the promised good or service is separately identifiable from other promises in the contract. In assessing whether promised goods or services are distinct, the Company considers factors such as the stage of development of the underlying intellectual property, the capabilities of the customer to develop the intellectual property on its own or whether the required expertise is readily available and whether the goods or services are integral or dependent to other goods or services in the contract. 13 The Company estimates the transaction price based on the amount expected to be received for transferring the promised goods or services in the contract. The consideration may include fixed consideration and variable consideration. At the inception of each arrangement that includes variable consideration, the Company e valuates the amount of consideration to which the Company expects to be entitled to. The Company utilizes either the most likely amount method or expected value method to estimate the amount expected to be received based on which method best predicts the a mount expected to be received. The amount of variable consideration that is included in the transaction price may be constrained and is included in the transaction price only to the extent that it is probable that a significant reversal in the amount of th e cumulative revenue recognized will not occur in a future period. The Company’s contracts may include development and regulatory milestone payments that are assessed under the most likely amount method and constrained until it is probable that a significant revenue reversal would not occur. Milestone payments that are not within the Company’s control, such as regulatory approvals, are not considered probable of being achieved until those approvals are received. At the end of each reporting period, the Company re-evaluates the probability of achievement of such development and regulatory milestones and any related constraint, and if necessary, adjust its estimate of the overall transaction price. Any such adjustments are recorded on a cumulative catch-up basis, which would affect collaboration revenue in the period of adjustment. For arrangements that include sales-based royalties, including milestone payments based on the level of sales, and the license is deemed to be the predominant item to which the royalties relate, the Company recognizes revenue at the later of (i) when the related sales occur, or (ii) when the performance obligation to which some or all of the royalty has been allocated has been satisfied (or partially satisfied). To date, the Company has not recognized any royalty revenue resulting from the Company’s collaboration arrangement. The Company allocates the transaction price based on the estimated standalone selling price of each performance obligation. The Company must develop assumptions that require judgment to determine the stand-alone selling price for each performance obligation identified in the contract. The Company utilizes key assumptions to determine the stand-alone selling price, which may include other comparable transactions, pricing considered in negotiating the transaction and the estimated costs. Variable consideration is allocated specifically to one or more performance obligations in a contract when the terms of the variable consideration relate to the satisfaction of the performance obligation and the resulting amounts allocated are consistent with the amounts the Company would expect to receive for the satisfaction of each performance obligation. The consideration allocated to each performance obligation is recognized as revenue when control is transferred for the related goods or services. For performance obligations which consist of licenses and other promises, the Company utilizes judgment to assess the nature of the combined performance obligation to determine whether the combined performance obligation is satisfied over time or at a point in time and, if over time, the appropriate method of measuring progress. The Company evaluates the measure of progress for its over-time arrangements at each reporting period and, if necessary, updates the measure of progress and revenue recognized. Net Loss per Share— Basic net loss per share is computed by dividing net loss by the weighted-average number of common shares outstanding during the period. Diluted net loss per share is computed using the weighted-average number of common shares outstanding during the period and, if dilutive, the weighted-average number of potential shares of common stock. The weighted-average number of common shares included in the computation of diluted net loss gives effect to all potentially dilutive common equivalent shares, including outstanding stock options and unvested shares of common stock. Common stock equivalent shares are excluded from the computation of diluted net loss per share if their effect is antidilutive. In periods in which the Company reports a net loss attributable to common stockholders, diluted net loss per share attributable to common stockholders is generally the same as basic net loss per share attributable to common stockholders since dilutive common shares are not assumed to have been issued if their effect is anti-dilutive. Recent Accounting Pronouncements —The Jumpstart Our Business Startups Act of 2012 permits an emerging growth company to take advantage of an extended transition period to comply with new or revised accounting standards applicable to public companies until those standards would otherwise apply to private companies. As an emerging growth company, the Company has elected to take advantage of this extended transition period. In February 2016, the FASB issued new leasing standards to increase transparency and comparability among organization related to their leasing activities. For additional information on the adoption of the new leasing standards, please refer to section titled “Leases” above, and Note 7. 14 In June 2016, the FASB issued ASU No. 2016-13, Financial Instruments - Credit Losses (Topic 326): Measurement of Credit Losses on Financial Instruments (“ASU 2016-13”) to improve financial reporting by requir ing more timely recording of credit losses on loans and other financial instruments held by financial institutions and other organizations. ASU 2016-13 requires the measurement of all expected credit losses for financial assets held at the reporting date b ased on historical experience, current conditions and reasonable and supportable forecasts. ASU 2016-13 also requires enhanced disclosures to help investors and other financial statement users better understand significant estimates and judgments used in e stimating credit losses, as well as the credit quality and underwriting standards of an organization’s portfolio. ASU 2016-13 is effective for the Company beginning January 1, 2023, with early application permitted. The Company is currently evaluating the impact the adoption of this standard will have on its condensed consolidated financial statements. In June 2018, the FASB issued ASU No. 2018-07, Compensation—Stock Compensation (Topic 718): Improvements to Nonemployee Share-Based Payment Accounting (“ASU 2018-07”) , which changes certain aspects of the accounting for share-based payments granted to nonemployees. Under ASU 2018-07, most of the guidance on such payments to nonemployees would be aligned with the requirements for share-based payments granted to employees. The Company adopted ASU 2018-07 on January 1, 2020. The adoption of ASU 2018-07 did not have a material impact on the Company’s condensed consolidated financial statements and related disclosures. 3. SHORT-TERM INVESTMENTS The Company invests its excess cash in fixed income instruments denominated and payable in U.S. dollars including U.S. treasury securities, commercial paper, corporate debt securities and asset-backed securities in accordance with the Company’s investment policy that primarily seeks to maintain adequate liquidity and preserve capital. The Company has designated all investments as available-for-sale and therefore such investments are reported at fair value. Unrealized gains or losses on investments are recorded in accumulated other comprehensive income or loss, a component of stockholders’ equity, on the Company’s condensed consolidated balance sheets. The following table summarizes the Company’s investments, which are included in cash equivalents and short-term investments: As of September 30, 2020 Amortized Cost Unrealized Gains Unrealized Losses Fair Value (in thousands) Money market mutual funds $ 177,753 $ — $ — $ 177,753 Total $ 177,753 $ — $ — $ 177,753 As of December 31, 2019 Amortized Cost Unrealized Gains Unrealized Losses Fair Value (in thousands) Repurchase agreements $ 14,000 $ — $ — $ 14,000 Asset-backed securities 14,866 10 — 14,876 Money market mutual funds 39,524 — — 39,524 Commercial paper 41,259 — — 41,259 U.S. Treasury securities 59,926 45 — 59,971 Corporate debt securities 92,380 128 — 92,508 Total $ 261,955 $ 183 $ — $ 262,138 The Company utilizes the specific identification method in computing realized gains and losses. The Company had no realized gains and losses on its available-for-sale securities for the three and nine months ended September 30, 2020 and 2019. The contractual maturity dates of all of the Company’s investments are less than one year . 15 4. FAIR VALUE MEASUREMENTS The Company’s financial instruments consist of cash and cash equivalents, short-term investments, restricted cash and accounts payable. The carrying amount of cash, restricted cash and accounts payable are each considered a reasonable estimate of fair value due to the short-term maturity. Assets measured at fair value on a recurring basis were as follows: Description September 30, 2020 Quoted Prices (Unadjusted) in Active Markets for Identical Assets (Level 1) Significant Other Observable Inputs (Level 2) Significant Unobservable Inputs (Level 3) (in thousands) Cash equivalents: Money market mutual funds $ 177,753 $ 177,753 $ — $ — Total cash equivalents $ 177,753 $ 177,753 $ — $ — Description December 31, 2019 Quoted Prices (Unadjusted) in Active Markets for Identical Assets (Level 1) Significant Other Observable Inputs (Level 2) Significant Unobservable Inputs (Level 3) (in thousands) Cash equivalents: Money market mutual funds $ 39,524 $ 39,524 $ — $ — Repurchase agreements 14,000 — 14,000 — Total cash equivalents $ 53,524 $ 39,524 $ 14,000 $ — Short-term investments: Asset-backed securities $ 14,876 $ — $ 14,876 $ — Commercial paper 41,259 — 41,259 — U.S. Treasury securities 59,971 — 59,971 — Corporate debt securities 92,508 — 92,508 — Total short-term investments $ 208,614 $ — $ 208,614 $ — Total financial assets $ 262,138 $ 39,524 $ 222,614 $ — Short-term securities are valued using models or other valuation methodologies that use Level 2 inputs. These models are primarily industry-standard models that consider various assumptions, including time value, yield curve, volatility factors, default rates, current market and contractual prices for the underlying financial instruments, as well as other relevant economic measures. Substantially all of these assumptions are observable in the marketplace, can be derived from observable data or are supported by observable levels at which transactions are executed in the marketplace. There were no transfers between fair value measure levels during the three and nine months ended September 30, 2020 and 2019. 16 5. PROPERTY AND EQUIPMENT, NET Property and equipment, net consists of the following: As of September 30, 2020 December 31, 2019 (in thousands) Laboratory equipment $ 11,991 $ 10,837 Manufacturing equipment 7,754 4,911 Computers and purchased software 1,596 640 Furniture and fixtures 1,363 1,363 Leasehold improvements 29,960 30,862 Assets not yet in service 1,339 2,513 Property and equipment, at cost 54,003 51,126 Less: accumulated depreciation and amortization ( 14,319 ) ( 8,410 ) Property and equipment, net $ 39,684 $ 42,716 Depreciation expense for the three and nine months ended September 30, 2020 was approximately $ 2.0 million and $ 5.9 million, respectively, compared to $ 1.7 million and $ 4.6 million, respectively for the three and nine months ended September 30, 2019. There were no disposals of property and equipment during the three and nine months ended September 30, 2020 and 2019. Leasehold improvements consist primarily of costs associated with the buildout of the Company’s research and development, manufacturing and general office space in Bedford, Massachusetts. 6. ACCRUED EXPENSES AND OTHER LIABILITIES Accrued expenses and other liabilities consist of the following: As of September 30, 2020 December 31, 2019 (in thousands) Accrued compensation and benefits $ 5,641 $ 4,551 Accrued research and development expenses 1,997 2,258 Accrued professional fees 461 542 Accrued unvested common stock subject to repurchase 16 37 Accrued other 311 256 Total accrued expenses and other liabilities $ 8,426 $ 7,644 7. COMMITMENTS AND CONTINGENCIES Operating Leases —In September 2016, the Company entered into a noncancelable operating lease beginning in November 2016 for office, laboratory and manufacturing space in Bedford, Massachusetts, that expires in October 2021 , with an option for an additional three-year term. On August 13, 2018, the Company entered into a sublease agreement for the entire leased premises. The rent commencement date of the sublease was December 2018, and the sublease will terminate on the scheduled termination date of the original lease. Under the terms of the sublease, the subtenant is obligated to pay the Company aggregate base rent of approximately $ 2.7 million over the term of the sublease in addition to passthrough of operating expenses and real estate taxes charged by the landlord. 17 In December 2017, the Company entered into a noncancelable operating lease for approximately 67,000 square feet of research and development, manufacturing and general office space in Bedford, Massachusetts. The lease expires in February 2027 with an option for an additional five-year term. Rent became due under the lease in two phases; rent on the first 46,000 square feet started in September 2018 and rent on the remaining 21,000 square feet started in March 2019 . The initial annual base rent is $ 39.50 per square foot and will increase by three percent annually. The Company is obligated to pay, on a pro-rata basis, real estate taxes and operating costs related to the premises. The lease agreement entered into in December 2017 allowed for a tenant improvement allowance not to exceed $ 10.9 million, which the Company received in full, to be applied to the total cost of tenant improvements to the leased premises. The unamortized balance of the tenant improvement allowance was included in deferred rent and has been recorded as a reduction to the operating right-of-use asset upon the adoption of the new leasing standards. The Company maintains letters of credit, secured by restricted cash, for security deposits totaling $ 1.3 million as of September 30, 2020 and December 31, 2019, respectively, in conjunction with its current leases. The following table summarizes operating lease costs as well as sublease income for the nine months ended September 30, 2020: Nine months ended September 30, 2020 Amount (in thousands) Operating lease costs $ 1,869 Sublease income ( 685 ) Net lease cost $ 1,184 Rent expense, net of amortization of the deferred rent incentives was $ 0.4 million and $ 1.0 million, respectively, for the three and nine months ended September 30, 2019. The maturities of our operating lease liabilities and minimum lease payments as of September 30, 2020 were as follows: For the Years Ending December 31, Amount (in thousands) 2020 $ 969 2021 3,834 2022 2,987 2023 3,077 2024 3,169 Thereafter 7,197 Total undiscounted lease payments $ 21,233 Less: imputed interest ( 5,196 ) Present value of operating lease liabilities $ 16,037 The following table summarizes the lease term and discount rate as of September 30, 2020: As of September 30, 2020 Weighted-average remaining lease term (years) Operating leases 6.0 Weighted-average discount rate Operating leases 9.9 % The following table summarizes cash paid for amounts included in the measurement of the Company’s operating lease liabilities for the nine months ended September 30, 2020. Nine months ended September 30, 2020 (in thousands) Cash paid for amounts included in the measurement of lease liabilities Operating cash flows from operating leases $ 2,838 18 8. STOCK INCENTIVE PLANS 2015 Stock Incentive Plan In December 2015, the Company’s Board of Directors adopted the 2015 Stock Incentive Plan (the “2015 Plan”), which provided for the grant of qualified incentive and nonqualified stock options or restricted stock awards to the Company’s employees, officers, directors, advisors, and outside consultants. Stock options generally vest over a four-year period and expire ten years from the date of grant. Certain options provide for accelerated vesting if there is a change in control, as defined in the 2015 Plan. At September 30, 2020, there were no additional shares available for future grant under the 2015 Plan. 2018 Incentive Award Plan In March 2018, the Company’s Board of Directors adopted and the Company’s stockholders approved the Homology Medicines, Inc. 2018 Incentive Award Plan (the “2018 Plan” and, together with the 2015 Plan, the “Plans”), which became effective on the day prior to the first public trading date of the Company’s common stock. Upon effectiveness of the 2018 Plan, the Company ceased granting new awards under the 2015 Plan. The 2018 Plan provides for the grant of incentive stock options, nonqualified stock options, restricted stock awards, restricted stock units, stock appreciation rights and other stock or cash-based awards to employees and consultants of the Company and directors of the Company. The number of shares of common stock initially available for issuance under the 2018 Plan was 3,186,205 shares of common stock plus the number of shares subject to awards outstanding under the 2015 Plan that expire, terminate or are otherwise surrendered, cancelled, forfeited or repurchased by the Company on or after the effective date of the 2018 Plan. In addition, the number of shares of common stock available for issuance under the 2018 Plan is subject to an annual increase on the first day of each calendar year beginning on January 1, 2019 and ending on and including January 1, 2028 equal to the lesser of (i) 4 % of the Company’s outstanding shares of common stock on the final day of the immediately preceding calendar year and (ii) such smaller number of shares of common stock as determined by the Company’s Board of Directors, provided that not more than 20,887,347 shares of common stock may be issued under the 2018 Plan upon the exercise of incentive stock options. Therefore, on January 1, 2020, an additional 1,804,670 shares were added to the 2018 Plan, representing 4 % of total common shares outstanding at December 31, 2019. At September 30, 2020, there were 2,430,357 shares available for future grant under the 2018 Plan. 2018 Employee Stock Purchase Plan In March 2018, the Company’s Board of Directors adopted and the Company’s stockholders approved the Homology Medicines, Inc. 2018 Employee Stock Purchase Plan (the “2018 ESPP”). The 2018 ESPP allows employees to buy Company stock through after-tax payroll deductions at a discount from market value. The 2018 ESPP is intended to qualify as an “employee stock purchase plan” under Section 423 of the Internal Revenue Code. The number of shares of common stock initially available for issuance under the 2018 ESPP was 353,980 shares of common stock plus an annual increase on the first day of each calendar year beginning on January 1, 2019 and ending on and including January 1, 2028 equal to the lesser of (i) 1 % of the Company’s outstanding shares of common stock on the final day of the immediately preceding calendar year and (ii) such smaller number of shares of common stock as determined by the Company’s Board of Directors, provided that not more than 4,778,738 shares of common stock may be issued under the 2018 ESPP. Therefore, on January 1, 2020, an additional 451,167 shares were added to the 2018 ESPP, representing 1 % of total common shares outstanding at December 31, 2019. At September 30, 2020, there were 1,039,651 shares available for future issuance under the 2018 ESPP. Under the 2018 ESPP, employees may purchase common stock through after-tax payroll deductions at a price equal to 85 % of the lower of the fair market value on the first trading day of an offering period or the last trading day of an offering period. The 2018 ESPP generally provides for offering periods of six months in duration that end on the final trading day of each February and August. In accordance with the Internal Revenue Code, no employee will be permitted to accrue the right to purchase stock under the 2018 ESPP at a rate in excess of $25,000 worth of shares during any calendar year during which such a purchase right is outstanding (based on the fair market value per share of the Company’s common stock as of the first day of the offering period). During the nine months ended September 30, 2020, 83,848 shares were issued under the 2018 ESPP for aggregate proceeds to the Company of $ 0.9 million. During the nine months ended September 30, 2019, 55,234 shares were issued under the 2018 ESPP for aggregate proceeds to the Company of $ 0.8 million. Pursuant to the 2018 ESPP, the Company recorded stock-based compensation of $ 0.1 million during the three and nine months ended September 30, 2020, and $ 0.1 million during the three and nine months ended September 30, 2019. 19 Stock-based compensation expense The Company recognizes compensation expense for awards to employees based on the grant date fair value of stock-based awards on a straight-line basis over the period during which an award holder provides service in exchange for the award, which is generally the vesting period. The fair value of each option award is estimated on the date of grant using the Black-Scholes option- pricing model, with the assumptions noted in the table below. Expected volatility for the Company’s common stock was determined based on an average of the historical volatility of a peer group of publicly traded companies that are similar to the Company. The expected term of options granted to employees was calculated using the simplified method, which represents the average of the contractual term of the option and the weighted-average vesting period of the option. The Company uses the simplified method because it does not have sufficient historical option exercise data to provide a reasonable basis upon which to estimate expected term. The contractual life of the options was used for the expected term of options granted to non-employees. The assumed dividend yield is based upon the Company’s expectation of not paying dividends in the foreseeable future. The risk-free rate is based on the U.S. Treasury yield curve in effect at the time of grant for periods commensurate with the expected term of the award. The Company recognizes forfeitures as they occur. The assumptions used in the Black-Scholes option pricing model are as follows: Three months ended September 30, Nine months ended September 30, 2020 2019 2020 2019 Expected volatility 64.6% — 65.2% 62.4% — 63.2% 63.2% — 65.4% 62.4% — 64.7% Weighted-average risk-free interest rate 0.31% — 0.40% 1.36% — 1.94% 0.31% — 1.73% 1.36% — 2.60% Expected dividend yield — % — % — % — % Expected term (in years) 6.0-6.25 6.25 5.5-6.25 6.25 Underlying common stock fair value $10.10 — $16.48 $15.92 — $21.45 $10.10 — $21.75 $15.92 — $30.34 The Company recorded stock-based compensation expense related to stock options and shares purchased under the 2018 ESPP as follows: Three months ended September 30, Nine months ended September 30, 2020 2019 2020 2019 (in thousands) Research and development $ 1,572 $ 1,124 $ 4,640 $ 2,786 General and administrative 1,774 863 5,042 2,413 $ 3,346 $ 1,987 $ 9,682 $ 5,199 As of September 30, 2020, there was $ 32.6 million of unrecognized compensation expense related to unvested employee and non-employee share-based compensation arrangements granted under the Plans. The unrecognized compensation expense is estimated to be recognized over a period of 2.6 years at September 30, 2020. A summary of option activity under the Plans during the nine months ended September 30, 2020 is as follows: Number of Options Weighted- Average Exercise Price per Share Weighted- Average Remaining Contractual Term (in Years) Aggregate Intrinsic Value (in thousands) Outstanding at January 1, 2020 4,843,018 $ 14.78 8.5 $ 33,809 Granted 1,200,650 $ 18.43 Exercised ( 41,593 ) $ 3.84 Cancelled/Forfeited ( 189,243 ) $ 23.31 Outstanding at September 30, 2020 5,812,832 $ 15.33 8.1 $ 11,299 Vested and expected to vest at September 30, 2020 5,812,832 $ 15.33 8.1 $ 11,299 Exercisable at September 30, 2020 2,574,662 $ 11.70 7.3 $ 9,618 20 The total intrinsic value of options exercised during the nine months ended September 30, 2020 and 2019 was $ 0.5 million and $ 1.4 million, respectively. The weighted-average grant date fair value per share of options granted during the nine months ended September 30, 2020 and 2019 was $ 10.73 and $ 14.70 , respectively. Stock options granted pursuant to the 2015 Plan permit option holders to elect to exercise unvested options in exchange for unvested common stock. Options granted under the 2015 Plan that are exercised prior to vesting will continue to vest according to the respective option agreement, and such unvested shares are subject to repurchase by the Company at the optionee’s original exercise price in the event the optionee’s service with the Company voluntarily or involuntarily terminates. A summary of the Company’s unvested common stock from early exercises that is subject to repurchase by the Company is as follows: Shares Unvested shares - January 1, 2020 21,666 Vested ( 17,564 ) Issued — Repurchased — Unvested shares - September 30, 2020 4,102 As of September 30, 2020 and December 31, 2019, 4,102 shares and 21,666 shares, respectively, remained subject to a repurchase right by the Company, with a related liability included in accrued expenses and other liabilities in the condensed consolidated balance sheets of less than $ 0.1 million as of September 30, 2020 and December 31, 2019. 9. NET LOSS PER SHARE The Company’s potential dilutive securities, which include unvested common stock from the early-exercise of stock options and outstanding common stock options, have been excluded from the computation of diluted net loss per share as the effect would be to reduce the net loss per share. Therefore, the weighted average number of common shares outstanding used to calculate both basic and diluted net loss per share attributable to common stockholders is the same. The Company excluded the following potential common shares, presented based on amounts outstanding at September 30, 2020 and 2019, from the computation of diluted net loss per share attributable to common stockholders because including them would have had an anti-dilutive effect: As of September 30, 2020 2019 Unvested common stock from early exercise of options 4,102 72,577 Stock options to purchase common stock 5,812,832 3,876,428 Total 5,816,934 3,949,005 10. COLLABORATION AND LICENSE AGREEMENT Summary of Agreement In November 2017, the Company entered into a collaboration and license agreement with Novartis (as amended, the “Collaboration Agreement”) for the research, development, manufacturing and commercialization of products using the Company’s gene-editing technology for the treatment of certain ophthalmic targets and sickle cell disease. In February 2019, Novartis elected to discontinue the sickle cell disease program under the Collaboration Agreement effective in August 2019. In May 2020, Novartis confirmed an ophthalmic target for a therapeutic editing program. Together, the companies are focusing their resources on advancing this program to development candidate selection. Additionally, in accordance with the terms of the Collaboration Agreement, Novartis’ right to substitute other ophthalmic targets expired. The Company also continues to work with Novartis on identifying new targets for the collaboration based on the existing exploratory research component of the agreement, which was extended for an additional six months through an amendment to the Collaboration Agreement executed in October 2020. 21 Under the terms of the Collaboration Agreement, the Company grante d Novartis a research license, a development and commercialization license, and a manufacturing license, under certain of its intellectual property rights to research, develop, manufacture and commercialize ophthalmic targets. Upon entering into the Collab oration Agreement, the Company received an upfront, nonrefundable payment of $ 35.0 million and issued additional shares of its Series B preferred stock to Novartis for consideration of $ 10.0 million. The Company recorded the Series B preferred stock at its estimated fair value of $ 11.7 million, including $ 1.7 million of the upfront payment, and allocated the remaining $ 33.3 million of the upfront payment to the Collaboration Agreement. The Collaboration Agreement consists of a research term, where the Company and Novartis are collaborating to perform research and conduct preclinical development to identify candidates that modulate ophthalmic targets. The Collaboration Agreement also includes exploratory work on the applicability of the gene-editing technology with respect to other gene targets. The Company’s obligation to perform such exploratory research has been extended pursuant to the October 2020 amendment to the Collaboration Agreement and now concludes in May 2021. Taking into account the expiration of Novartis’ rights to substitute other ophthalmic targets, the Company and Novartis will focus resources on the confirmed ophthalmic target. The Company is responsible for the manufacturing of proprietary research grade human hematopoietic stem cell derived adeno-associated virus vectors (“AAVHSCs”) during the research term. Research activities performed by the Company are being reimbursed at a full-time equivalent rate (“FTE”) and manufacturing activities will be reimbursed at cost, up to a maximum of $ 17.0 million during the research term, as specified and defined in the Collaboration Agreement. Novartis is required to pay the Company a target fee of $ 5.0 million if the target meets certain success criteria during the research term (the “target fee trigger date”). The research term will continue for five years from the effective date of the Collaboration Agreement. Pursuant to the Collaboration Agreement, the Company will also participate on a joint steering committee and a joint manufacturing subcommittee, with equal representation from both the Company and Novartis. Novartis has the exclusive right to develop and commercialize one ophthalmic candidate or product arising from the research activities. Subject to certain limitations pursuant to the terms of the Collaboration Agreement, Novartis will fund all development and commercialization costs. The Company will be responsible for manufacturing candidates and products for Novartis during the development and commercialization terms. The Company’s manufacturing activities will be reimbursed at cost during the development term and at cost plus a margin during the commercialization term, as defined in the Collaboration Agreement. If the Company is not able to manufacture candidates or products that meet the quality or quantity requirements of Novartis, then Novartis shall have the right to designate a third-party contract manufacturer or manufacture such candidates or products itself. In accordance with the Collaboration Agreement, taking into consideration Novartis’ election to discontinue the sickle cell disease program and the expiration of Novartis’ rights to make substitutions of an ophthalmic target, the Company is also eligible to receive up to a total of $ 265.0 million in milestone payments, including up to $ 90.0 million in development milestone payments, up to $ 85.0 million in regulatory milestone payments and up to $ 90.0 million in commercial milestone payments, with respect to the licensed products. The Company is also eligible to earn tiered royalties on net sales of licensed products by Novartis, its affiliates or sublicensees, ranging from mid single-digit, to sub-teen double-digit percentages, and such royalties are potentially subject to various reductions and offsets. If any of the exploratory research efforts are advanced into formal research and development programs, the parties will negotiate the economics including potential milestone payments for such programs at that time. Unless earlier terminated, the Collaboration Agreement will continue on a target-by-target basis until the expiration of all applicable royalty terms with respect to all products that modulate such target on a country-by-country-basis. Either party may terminate the agreement on a target-by-target basis for the other party’s material breach with respect to such target, or in the event of the other party’s bankruptcy. Novartis may terminate the agreement for convenience on a target-by-target basis. The Company may terminate the agreement if Novartis files, or joins a third party in filing or maintaining, a patent challenge against certain of the patent rights licensed to Novartis under the terms of the agreement. There are no performance, cancellation, termination or refund provisions in the arrangement that contain material financial consequences to the Company. Revenue Recognition The Company determined that the (1) research, development and commercialization and manufacturing licenses, (2) the research activities performed by the Company (3) service on the joint committees and (4) manufacturing during the research and development terms represented a single performance obligation under the Collaboration Agreement. Since the option for Novartis to obtain manufacturing during the commercialization term from the Company is at a price that would reflect the standalone selling price, the option does not provide Novartis with a material right and is therefore not a performance obligation under the contract. The Company has concluded that the research, development and commercialization and manufacturing licenses are not distinct from the research activities and manufacturing during the research and development term as Novartis cannot benefit from the licenses without the Company performing the research and manufacturing services. These services are so specialized and rely on the Company’s expertise in gene editing technologies not available in the marketplace. As a result, these licenses have been combined with the research activities, service on the joint committees and the manufacturing during the research and development terms as a single performance obligation. 22 The transaction price consists of the $ 33.3 million non-refundable upfront payment, net of amounts classified as equity, and projected reimbursable research and manufacturing activities, which have been estimated using the expected value method. None of the target fees or development and regulatory milestone payments have been included in the transaction price, as all are fully constrained. As part of the evaluation of the constraint, the Company considered numerous factors, including the fact that achievement of the milestones is outside of the Company’s control and is contingent upon the success of the Company’s preclinical studies, clinical trials, Novartis’ efforts and the receipt of regulatory approval. In addition, the Company has determined that the commercial milestones and sales-based royalties will be recognized when the related sales occur as they were determined to relate predominately to the licenses transferred to Novartis, and therefore have also been excluded from the transaction price. The Company will re-evaluate the transaction price, including estimated variable consideration included in the transaction price and all constrained amounts, in each reporting period and as uncertain events are resolved or other changes in circumstances occur. The Company identified only one performance obligation in the Collaboration Agreement. Therefore, the Company will allocate the transaction price at the onset of the contract to the single performance obligation. The Company recognizes revenue over time using a cost-to-cost method, which it believes best depicts the transfer of control to the customer. Under the cost-to-cost method, the extent of progress towards completion is measured based on the ratio of actual costs incurred to the total estimated costs expected upon satisfying the identified performance obligation. Under this method, revenue is recorded over the performance period as a percentage of the estimated transaction price based on the extent of progress towards completion. As of September 30, 2020, the aggregate amount of the transaction price related to the unsatisfied portion of the performance obligation is $ 30.4 million, and the Company will recognize this revenue as the research and manufacturing activities are performed, which is expected to occur over a period of time that is estimated will conclude in 2027. The Company does not expect collaboration revenue to be recognized evenly over this period as it will be recognized on a percentage of completion basis as the Company performs the research, development and manufacturing services, which will likely vary from period to period. In estimating the total costs to satisfy its single performance obligation pursuant to the Novartis agreement, the Company is required to make significant estimates including the expected time it will take to fulfill the performance obligation, which currently is expected to be approximately ten years from the date the Collaboration Agreement was entered into, and the expected costs associated with manufacturing during the research and development terms for a novel manufacturing process as well as the probability of success of a target to move into the development phase. The Company has made estimates of such costs utilizing its experience with similar product candidates, however not identical to those in the Collaboration Agreement. In making such estimates, significant judgment is required to evaluate those key assumptions related to cost estimates. The cumulative effect of revisions to the total estimated costs to complete the Company’s single performance obligation will be recorded in the period in which the changes are identified and amounts can be reasonably estimated. While such revisions will have no impact on the Company’s reported cash flows, a significant change in these assumptions and estimates could have a material impact on the timing and amount of revenue recognized in future periods. During the three and nine months ended September 30, 2020, the Company recognized revenue under the Collaboration Agreement of $ 0.6 million and $ 1.7 million, respectively, of which $ 0.2 million and $ 0.6 million was included in deferred revenue at the beginning of each period. During the three and nine months ended September 30, 2019, the Company recognized revenue under the Collaboration Agreement of $ 0.4 million and $ 1.1 million, respectively, of which $ 0.2 million and $ 0.4 million, respectively, was included in deferred revenue at the beginning of each period. As of September 30, 2020 and December 31, 2019, there was approximately $ 30.4 million and $ 31.0 million of deferred revenue related to the Collaboration Agreement, respectively. In addition, as of September 30, 2020 and December 31, 2019, the Company has recorded accounts receivable of $ 0.4 million related to reimbursable research and development costs under the Collaboration Agreement, which are included in prepaid expenses and other current assets on the condensed consolidated balance sheets as of each such date. 11. SUBSEQUENT EVENTS On November 9, 2020, the Company entered into a stock purchase agreement (the “Stock Purchase Agreement”) with Pfizer Inc. (“Pfizer”), pursuant to which Pfizer has agreed to purchase 5,000,000 shares of the Company’s common stock through a private placement transaction (the “Private Placement”) at a purchase price of $ 12.00 per share, for an aggregate purchase price of $ 60.0 million. Under the Stock Purchase Agreement, Pfizer was granted an exclusive right of first refusal (“ROFR”) for a 30-month period (the “ROFR Period”) beginning on the date of the closing of the Private Placement (collectively, the “ROFR Provision”), to negotiate a potential collaboration on the development and commercialization of HMI-102 and HMI-103. Pfizer may exercise its right of first refusal under the ROFR Provision one time for each of HMI-102 and HMI-103 during the ROFR period. Additionally during the ROFR period, Pfizer has the right to designate a member to join the Company’s Scientific Advisory Board to participate in matters related to the development of these programs. * * * * * * 23