[PART I – FINANCIAL INFORMATION](index=6&type=section&id=PART%20I%20%E2%80%93%20FINANCIAL%20INFORMATION) [Item 1. Financial Statements (unaudited)](index=6&type=section&id=Item%201.%20Financial%20Statements%20%28unaudited%29) This section presents the unaudited condensed consolidated financial statements for the quarter ended March 31, 2022, highlighting a net loss of **$35.0 million** and a strong cash position of **$337.0 million** sufficient for at least 12 months of operations [Condensed Consolidated Balance Sheets](index=6&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) Condensed Consolidated Balance Sheet Highlights (in thousands) | Account | March 31, 2022 | December 31, 2021 | | :--- | :--- | :--- | | Cash and cash equivalents | $336,978 | $375,145 | | Total assets | $444,026 | $476,771 | | Total liabilities | $91,111 | $95,025 | | Total stockholders' equity | $352,915 | $381,746 | [Condensed Consolidated Statements of Operations and Comprehensive Loss](index=7&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) Statement of Operations Summary (in thousands, except per share data) | Metric | Three Months Ended March 31, 2022 | Three Months Ended March 31, 2021 | | :--- | :--- | :--- | | Research and development | $25,554 | $18,753 | | General and administrative | $9,790 | $6,902 | | **Loss from operations** | **$(35,344)** | **$(25,655)** | | **Net loss** | **$(34,999)** | **$(25,562)** | | Net loss per share | $(0.61) | $(0.46) | [Condensed Consolidated Statements of Cash Flows](index=9&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) Cash Flow Summary (in thousands) | Activity | Three Months Ended March 31, 2022 | Three Months Ended March 31, 2021 | | :--- | :--- | :--- | | Net cash used in operating activities | $(35,435) | $(23,027) | | Net cash (used in) provided by investing activities | $(2,784) | $113,485 | | Net cash provided by financing activities | $52 | $212,957 | [Notes to Condensed Consolidated Financial Statements](index=10&type=section&id=Notes%20to%20Condensed%20Consolidated%20Financial%20Statements) - The company is an early-stage biotech focused on non-viral genetic medicines using closed-ended DNA (ceDNA) and a cell-targeted lipid nanoparticle (ctLNP) delivery system[24](index=24&type=chunk) - As of March 31, 2022, the company had an accumulated deficit of **$343.1 million**, and management expects existing cash and cash equivalents are sufficient to fund operations and capital expenditures for at least **12 months** from the report's issuance date[27](index=27&type=chunk) - In January 2021, the company raised net proceeds of **$211.3 million** from a follow-on public offering, and an "at-the-market" sales agreement for up to **$250.0 million** was established in August 2021, with no shares sold under it as of May 5, 2022[36](index=36&type=chunk) - Total unrecognized stock-based compensation cost as of March 31, 2022, was **$61.2 million**, expected to be recognized over a weighted-average period of **2.8 years** for stock options and **2.0 years** for restricted stock[48](index=48&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=19&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses the company's non-viral genetic medicine focus, increased operating expenses, and strong liquidity with **$337.0 million** cash sufficient into **2024** [Results of Operations](index=28&type=section&id=Results%20of%20Operations) Comparison of Operating Results (in thousands) | Metric | Q1 2022 | Q1 2021 | Change | | :--- | :--- | :--- | :--- | | Research and development | $25,554 | $18,753 | $6,801 | | General and administrative | $9,790 | $6,902 | $2,888 | | **Total operating expenses** | **$35,344** | **$25,655** | **$9,689** | | **Net loss** | **$(34,999)** | **$(25,562)** | **$(9,437)** | - The **$6.8 million** increase in R&D expenses was primarily driven by a **$3.1 million** increase in facilities costs related to the manufacturing facility lease, a **$2.4 million** increase in personnel costs, and a **$1.3 million** increase in stock-based compensation, partly offset by a **$1.2 million** decrease in external manufacturing costs due to the transition to an in-house process[86](index=86&type=chunk) - The **$2.9 million** increase in G&A expenses was mainly due to a **$1.3 million** rise in stock-based compensation and a **$1.0 million** increase in personnel-related costs, both resulting from higher headcount[88](index=88&type=chunk) [Liquidity and Capital Resources](index=30&type=section&id=Liquidity%20and%20Capital%20Resources) - As of March 31, 2022, the company had cash and cash equivalents of **$337.0 million**[90](index=90&type=chunk) - Management believes that existing cash and cash equivalents will be sufficient to fund operating expenses and capital expenditures into **2024**[67](index=67&type=chunk)[102](index=102&type=chunk) - Net cash used in operating activities increased to **$35.4 million** in Q1 2022 from **$23.0 million** in Q1 2021, primarily due to the higher net loss[91](index=91&type=chunk)[92](index=92&type=chunk)[94](index=94&type=chunk) - Net cash provided by financing activities was only **$0.1 million** in Q1 2022, compared to **$213.0 million** in Q1 2021, which included **$211.9 million** in net proceeds from a follow-on public offering[98](index=98&type=chunk) [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=36&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company's primary market risk is interest rate sensitivity on its **$337.0 million** cash and cash equivalents, with no marketable securities or hedging instruments used - The company's primary market risk is interest rate sensitivity on its cash and cash equivalents, which totaled **$337.0 million** as of March 31, 2022[108](index=108&type=chunk) - The company did not hold any marketable securities as of March 31, 2022, and does not currently use hedging instruments to manage interest rate risk[108](index=108&type=chunk) [Item 4. Controls and Procedures](index=36&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded that the company's disclosure controls and procedures were effective as of March 31, 2022, with no material changes to internal controls during the quarter - Based on an evaluation as of March 31, 2022, the CEO and CFO concluded that the company's disclosure controls and procedures were effective at the reasonable assurance level[110](index=110&type=chunk) - There were no changes in internal control over financial reporting during Q1 2022 that have materially affected, or are reasonably likely to materially affect, these controls[111](index=111&type=chunk) [PART II – OTHER INFORMATION](index=38&type=section&id=PART%20II%20%E2%80%93%20OTHER%20INFORMATION) [Item 1A. Risk Factors](index=38&type=section&id=Item%201A.%20Risk%20Factors) This section directs readers to the detailed discussion of risk factors in the company's Annual Report on Form 10-K, which could materially affect future results - The report directs investors to carefully consider the risk factors discussed in Part I, Item 1A of the company's Annual Report on Form 10-K[112](index=112&type=chunk) [Item 6. Exhibits](index=39&type=section&id=Item%206.%20Exhibits) This section lists all exhibits filed with the quarterly report, including a lease amendment and certifications from the Principal Executive and Financial Officers - Exhibits filed with the report include a Third Amendment to the Lease with BMR-Rogers Street LLC, and certifications from the Principal Executive Officer and Principal Financial Officer pursuant to the Sarbanes-Oxley Act of 2002[114](index=114&type=chunk) [Signatures](index=40&type=section&id=Signatures) The report is formally signed and authorized by the company's principal executive and financial officers - The report was signed on May 5, 2022, by Geoff McDonough, President and Chief Executive Officer, and Matthew Norkunas, Chief Financial Officer[119](index=119&type=chunk)

☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2021 Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-K (Mark One) OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission file number: 001-39319 GENERATION BIO CO. (Exact name of registrant as specified in its charter) Delaware 81-4301284 (State or othe ...

generation bio | --- | --- | --- | --- | --- | --- | --- | --- | --- | |-------|-------|-------|-------|-------|-------|-------|------------------------------------------------------------------------------------------------|----------| | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | Leading durable | | | | | | | | | | redosable | scalable | | | | | | | | | non-viral genetic medicines FOR MILLIONS OF PATIENTS LIVING WITH RARE AND PREVALENT DISEASES | | | | | | ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 10-Q ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2021 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission File Number: 001-39319 GENERATION BIO CO. (Exact name of registrant as specified in its charter) Delaware 81-4301284 (State or other ...

Financial Performance - The company reported net losses of $56.4 million for the six months ended June 30, 2021, compared to $35.4 million for the same period in 2020, with an accumulated deficit of $245.3 million as of June 30, 2021[86]. - The net loss for the three months ended June 30, 2021, was $30.8 million, compared to a net loss of $17.7 million for the same period in 2020, reflecting an increase in losses of 73.7%[108]. - Total operating expenses for the six months ended June 30, 2021, were $56.5 million, up from $35.8 million in 2020, indicating a 57.9% increase[113]. - Cash used in operating activities for the six months ended June 30, 2021, was $48.5 million, compared to $33.3 million in 2020, representing a 45.6% increase[118]. - Interest income for the six months ended June 30, 2021, was $0.1 million, down from $0.4 million in 2020, a decrease of 71.4%[116]. - The company expects to incur significant expenses and operating losses for the foreseeable future as it continues its research and development activities[117]. - Existing cash, cash equivalents, and marketable securities are projected to fund operations into 2024, but additional financing may be required sooner than expected[125]. - The company does not have any committed external sources of funds and will need to seek further funding through various means, which may lead to dilution for stockholders[125]. - If sufficient capital is not raised, the company may need to significantly curtail or discontinue research, development programs, or commercialization efforts[126]. Research and Development - The company plans to submit an IND application for its hemophilia A program in 2023 and expects to report Factor VIII expression data in non-human primates by year-end[81]. - The company is advancing a broad portfolio of programs targeting rare and prevalent diseases, initially focusing on liver and retina diseases[79]. - The company aims to enable patients to produce therapeutic antibodies from their own cells for years from a single dose, with in vivo studies showing a peak antibody concentration of 8µg/ml[80]. - The increase in research and development expenses was primarily due to heightened preclinical activity and increased headcount in the R&D function[114]. - Research and development expenses increased to $22.7 million for the three months ended June 30, 2021, up from $13.5 million in the same period of 2020, representing a 68.3% increase[109]. - The company expects substantial increases in expenses related to ongoing activities, particularly in preclinical and clinical trials for product candidates[125]. Manufacturing and Operations - The company has developed a rapid enzymatic synthesis process (RES) that shortens the ceDNA production cycle time from 28 days to one day, aiming to manufacture drug candidates cost-effectively[83]. - The company plans to invest up to $45 million in a new cGMP-compliant manufacturing facility in Waltham, Massachusetts, expected to be operational in 2023[84]. - The company plans to transition all portfolio programs to the RES manufacturing process to enhance scalability and reduce variability[83]. General and Administrative Expenses - General and administrative expenses rose to $8.2 million for the three months ended June 30, 2021, compared to $4.3 million in 2020, marking an increase of 92.5%[111]. COVID-19 Impact - The company is closely monitoring the impact of the COVID-19 pandemic on its operations and has implemented safety measures for its employees[93]. Accounting Policies - Critical accounting policies related to accrued research and development expenses and stock-based compensation are deemed most critical for financial statement preparation[129]. - There have been no material changes to critical accounting policies and estimates from those disclosed in the Annual Report[129]. - The company currently has no off-balance sheet arrangements[130]. - Recently adopted accounting pronouncements that may impact financial position and results of operations are disclosed in the Quarterly Report[131]. - The company is classified as a smaller reporting company and is not required to provide certain market risk disclosures[132].

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 10-Q ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2021 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to GENERATION BIO CO. (Exact name of registrant as specified in its charter) Delaware 81-4301284 (State or other jurisdiction of incorporation or organ ...

generation bio | --- | --- | --- | --- | --- | --- | --- | --- | --- | |-------|-------|-------|-------|-------|-------|-------|-------|-------| | | | | | | | | | | | | | | | | | | | | NASDAQ: GBIO APRIL 2021 Forward-looking statements 2 Any statements in this presentation about future expectations, plans and prospects for the Company, including statements about the Company's strategic plans or objectives, technology platforms, research and clinical development plans, manufacturing plans and goals, and othe ...

Compliance and Regulatory Costs - The company has incurred increased costs due to operating as a newly public entity, with significant legal, accounting, and compliance expenses expected to rise as it transitions from an Emerging Growth Company (EGC) status [716]. - The Sarbanes-Oxley Act and other regulations impose requirements that will increase legal and financial compliance costs, necessitating the hiring of additional financial and accounting personnel [716]. - The company is required to furnish a report on internal control over financial reporting starting with the Annual Report for the year ended December 31, 2021, which may involve significant costs and challenges [718]. - There is a risk that the company may not conclude that its internal control over financial reporting is effective, which could lead to adverse reactions in the financial markets [718]. Financial Performance and Position - Generation Bio Co. reported total assets of $294.155 million as of December 31, 2020, a significant increase from $42.140 million in 2019, representing a growth of 596% [818]. - The company incurred a net loss of $80.523 million for the year ended December 31, 2020, compared to a net loss of $61.317 million in 2019, reflecting a 31% increase in losses [820]. - Generation Bio Co. reported comprehensive loss of $80.514 million for 2020, compared to a comprehensive loss of $61.308 million in 2019, an increase of 31.2% [820]. - The company’s total operating expenses for 2020 were $81.114 million, up from $62.302 million in 2019, representing a 30.3% increase [820]. - The Company reported a net loss of $80.5 million for the year ended December 31, 2020, with an accumulated deficit of $189.0 million as of the same date [832]. Research and Development - Research and development expenses rose to $58.532 million in 2020, up from $50.134 million in 2019, indicating a 16.7% increase in investment in R&D [820]. - Research and development costs are expensed as incurred, including salaries, stock-based compensation, and external vendor costs [850]. - The Company recorded research and development expenses of less than $0.1 million under its license agreements with NIH and UMass for both 2020 and 2019 [910][916]. Capital and Funding - The company raised $213.900 million from its initial public offering in 2020, contributing to a net cash increase of $47.757 million for the year [826]. - The Company may need to obtain additional funding through public or private equity offerings, debt financings, or collaborations, which may not be on acceptable terms [833]. - As of March 18, 2021, the Company expects its cash, cash equivalents, and marketable securities to be sufficient to fund operating expenses and capital expenditures for at least 12 months [832]. Tax Matters - Changes in tax laws, including a reduction of the corporate tax rate from 35% to 21%, may adversely affect the company's financial condition [724]. - The CARES Act allows for the carryback of Net Operating Losses (NOLs) for up to five years, which could impact the company's tax liabilities [726]. - As of December 31, 2020, the Company had federal net operating loss carryforwards of $177.3 million, which may offset future taxable income [899]. - The Company recorded no income tax benefits for net operating losses incurred for the years ended December 31, 2020 and 2019 due to uncertainty in realizing benefits [897]. - The effective income tax rate for the years ended December 31, 2020 and 2019 was 0.0% [899]. Stockholder and Corporate Governance - The company is subject to various provisions in its corporate charter that may make acquisitions more difficult, potentially affecting stockholder interests [728]. - The company is governed by Delaware law, which includes provisions that could limit stockholder actions and affect the ability to replace management [729]. - The number of shares authorized for issuance under the 2017 Stock Incentive Plan increased from 8,407,405 to 10,275,717 shares in January 2020 [884]. - As of December 31, 2020, 2,500,761 shares remained available for future issuance under the 2020 Stock Incentive Plan, with an increase of 1,878,800 shares effective January 1, 2021 [887]. Operational Risks - The company maintains disaster recovery plans, but natural disasters or catastrophic events could significantly disrupt operations and financial performance [734]. - The Company relies on third-party suppliers for drug substance and product, which poses a risk of supply interruptions affecting its programs [838]. - The Company has incurred recurring losses and expects to continue generating operating losses in the foreseeable future [832]. Lease and Asset Management - The Company expects to recognize a lease liability of approximately $49.7 million and a related right-of-use asset of approximately $33.4 million upon adopting new lease accounting standards [869]. - Future minimum lease payments as of December 31, 2020, total $64,875,000, with the largest payment of $27,570,000 due thereafter [921]. - The total assets measured at fair value on a recurring basis amounted to approximately $263.3 million as of December 31, 2020 [874]. Employee Compensation and Stock Options - The Company recorded stock-based compensation expense of $8.4 million for the year ended December 31, 2020, up from $4.2 million in 2019 [896]. - The total intrinsic value of stock options exercised during the year ended December 31, 2020 was $20.0 million, compared to $0.1 million in 2019 [892]. - The total fair value of restricted common stock vested during the year ended December 31, 2020 was approximately $15.8 million, compared to $7.0 million in 2019 [895]. - The Company has unrecognized compensation cost related to unvested stock-based awards of $27.1 million, expected to be recognized over a weighted average period of 3.2 years [896].

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 10-Q ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2020 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission File Number: 001-39319 GENERATION BIO CO. (Exact name of registrant as specified in its charter) Delaware 81-4301284 (State or other ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 10-Q ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2020 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission File Number: 001-39319 GENERATION BIO CO. (Exact name of registrant as specified in its charter) Delaware 81-4301284 (State or other jurisdiction of incorpo ...