UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 ___________________________________________________________________ FORM 10-Q ___________________________________________________________________ (Mark One) QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES ☒ EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2023 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 Numbe ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 _______________________________________________________________________________ FORM 10-K _______________________________________________________________________________ ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2022 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission Fi ...

[PART I—FINANCIAL INFORMATION](index=5&type=section&id=PART%20I%E2%80%94FINANCIAL%20INFORMATION) [Financial Statements](index=5&type=section&id=Item%201.%20Financial%20Statements) The company reported increased net losses for Q3 and the nine months ended September 30, 2021, with an accumulated deficit of $613.7 million, while total assets increased to $490.6 million and cash decreased to $332.7 million [Condensed Consolidated Balance Sheets](index=5&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) As of September 30, 2021, total assets increased to $490.6 million, driven by operating lease assets, while cash decreased to $332.7 million, and total liabilities rose sharply to $144.6 million Condensed Consolidated Balance Sheet Highlights (in thousands) | Account | Sep 30, 2021 | Dec 31, 2020 | | :--- | :--- | :--- | | **Assets** | | | | Cash, cash equivalents & short-term investments | $332,705 | $429,729 | | Operating lease right-of-use assets | $100,174 | $19,376 | | Total assets | $490,581 | $482,567 | | **Liabilities & Equity** | | | | Lease liability (current & non-current) | $122,365 | $30,708 | | Total liabilities | $144,581 | $48,220 | | Accumulated deficit | $(613,667) | $(502,536) | | Total stockholders' equity | $346,000 | $434,347 | [Condensed Consolidated Statements of Operations and Comprehensive Loss](index=6&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) Net loss increased to $38.4 million for Q3 2021 and $111.1 million for the nine-month period, primarily due to higher R&D and G&A expenses, partially offset by $7.5 million in license revenue Statement of Operations Summary (in thousands) | Metric | Q3 2021 | Q3 2020 | Nine Months 2021 | Nine Months 2020 | | :--- | :--- | :--- | :--- | :--- | | License Revenue | $0 | $0 | $7,500 | $0 | | Research and Development | $24,069 | $16,653 | $66,657 | $50,581 | | General and Administrative | $14,453 | $11,351 | $52,546 | $30,989 | | Operating Loss | $(38,522) | $(28,004) | $(111,703) | $(81,570) | | Net Loss | $(38,362) | $(27,769) | $(111,131) | $(79,875) | | Net Loss per Share | $(0.39) | $(0.31) | $(1.13) | $(0.99) | [Condensed Consolidated Statements of Cash Flows](index=9&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) Net cash used in operating activities increased to $82.0 million for the nine months ended September 30, 2021, while investing activities provided $54.8 million, and financing activities provided $2.0 million Cash Flow Summary (Nine Months Ended Sep 30, in thousands) | Activity | 2021 | 2020 | | :--- | :--- | :--- | | Net cash used in operating activities | $(82,016) | $(57,067) | | Net cash provided by (used in) investing activities | $54,826 | $(291,743) | | Net cash provided by financing activities | $2,022 | $355,609 | | **Net (decrease) increase in cash** | **$(25,168)** | **$6,799** | [Notes to Condensed Consolidated Financial Statements](index=10&type=section&id=Notes%20to%20Condensed%20Consolidated%20Financial%20Statements) The notes highlight the company's clinical-stage status, accumulated deficit of $613.7 million, sufficient cash runway, and significant events including license revenue and new lease agreements with related impairment charges - The company is a clinical-stage gene therapy company with an accumulated deficit of **$613.7 million** as of September 30, 2021[27](index=27&type=chunk)[28](index=28&type=chunk) - Management believes its cash, cash equivalents, and short-term investments of **$332.7 million** are sufficient to fund operations for at least twelve months from the financial statement issuance date[28](index=28&type=chunk) - In January 2021, the company recognized **$7.5 million** in non-refundable upfront license revenue from an agreement with Lexeo Therapeutics[40](index=40&type=chunk)[41](index=41&type=chunk) - The company recorded a **$1.1 million** impairment charge related to right-of-use assets and leasehold improvements for a terminated lease in Redwood City[44](index=44&type=chunk) - The company entered into a new lease for a facility in North Carolina and subsequently subleased it, determining there was no impairment as the undiscounted sublease income exceeds the asset group's carrying amount[48](index=48&type=chunk)[50](index=50&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=17&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses the strategic shift to focus ADVM-022 on wet AMD after discontinuing the DME program due to toxicity, noting increased operating losses from higher R&D and G&A expenses, and confirming sufficient cash to fund operations into 2024 - The company is focusing on developing ADVM-022 for wet age-related macular degeneration (wet AMD) and has discontinued development for diabetic macular edema (DME)[63](index=63&type=chunk)[66](index=66&type=chunk) - The decision to halt the DME program followed a dose-limiting toxicity observed at the high dose (6 x 10^11 vg/eye) in the Phase 2 INFINITY trial, with plans for a new Phase 2 trial in wet AMD to evaluate lower doses[66](index=66&type=chunk)[67](index=67&type=chunk) - Reflecting revised timelines and capital priorities, the company has subleased its new GMP manufacturing facility in Durham, North Carolina, and will rely on existing contract manufacturing partners[68](index=68&type=chunk) Comparison of Operating Expenses (in thousands) | Expense Category | Nine Months 2021 | Nine Months 2020 | Change | | :--- | :--- | :--- | :--- | | Research and development | $66,657 | $50,581 | $16,076 | | General and administrative | $52,546 | $30,989 | $21,557 | - The increase in G&A expenses for the nine months ended Sep 30, 2021 was primarily due to a **$9.3 million** increase in personnel costs, **$6.6 million** in professional services (mainly for a proxy contest), and costs related to new leases and asset impairment[100](index=100&type=chunk) - As of September 30, 2021, the company had **$332.7 million** in cash, cash equivalents, and short-term investments, which is expected to fund operations into 2024[103](index=103&type=chunk) [Quantitative and Qualitative Disclosures About Market Risk](index=24&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company reported no material changes in foreign currency exchange and interest rate risks for the nine months ended September 30, 2021 - No material changes in foreign currency exchange and interest rate risks were reported for the nine months ended September 30, 2021[118](index=118&type=chunk) [Controls and Procedures](index=24&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded that disclosure controls and procedures were effective as of September 30, 2021, with no material changes to internal control over financial reporting during the quarter - The CEO and Interim CFO concluded that as of September 30, 2021, the company's disclosure controls and procedures were effective[120](index=120&type=chunk) - No material changes to internal control over financial reporting occurred during the third quarter of 2021[121](index=121&type=chunk) [PART II—OTHER INFORMATION](index=25&type=section&id=PART%20II%E2%80%94OTHER%20INFORMATION) [Legal Proceedings](index=25&type=section&id=Item%201.%20Legal%20Proceedings) The company reported no material legal proceedings during the period - Not applicable[125](index=125&type=chunk) [Risk Factors](index=25&type=section&id=Item%201A.%20Risk%20Factors) This section outlines significant financial, clinical development, manufacturing, intellectual property, commercialization, and operational risks, including dependence on ADVM-022 and challenges inherent in gene therapy [Risks Related to Financial Position and Need for Capital](index=26&type=section&id=Risks%20Related%20to%20Our%20Financial%20Position%20and%20Need%20for%20Capital) The company faces ongoing significant operating losses and requires substantial additional capital for clinical development and commercialization, with no guarantee of availability or favorable terms - The company has incurred significant operating losses since inception and expects them to continue for the foreseeable future, with no guarantee of ever achieving profitability[127](index=127&type=chunk) - The company will need to raise substantial additional capital to fund operations, and failure to do so could force it to delay, reduce, or eliminate research and development programs[130](index=130&type=chunk) [Risks Related to Discovery and Development](index=28&type=section&id=Risks%20Related%20to%20the%20Discovery%20and%20Development%20of%20Our%20Product%20Candidates) The business is highly dependent on ADVM-022, facing long, expensive, and uncertain gene therapy development, with risks of serious side effects, program discontinuation, and unpredictable timelines due to novel technology - The business heavily depends on the success of one or more product candidates, primarily ADVM-022, and failure to develop, get approval for, or commercialize it would materially harm the business[137](index=137&type=chunk) - Serious complications or side effects, such as the dose-limiting toxicity observed in the INFINITY trial, could lead to discontinuation of clinical programs or refusal of regulatory approval[143](index=143&type=chunk) - The company's gene therapy platform is a novel technology, making it difficult to predict the time, cost, and regulatory pathway for development and approval[151](index=151&type=chunk)[152](index=152&type=chunk) [Risks Related to Manufacturing](index=34&type=section&id=Risks%20Related%20to%20Manufacturing) The company faces significant manufacturing risks, including challenges in developing robust processes, reliance on limited third-party contractors, and the inherent complexity of gene therapy production at commercial scale - Inability to develop and maintain robust and reliable manufacturing processes could delay clinical trials and force program termination[168](index=168&type=chunk) - The company relies on a limited number of vendors, including single-source vendors, for manufacturing, who may not meet regulatory requirements or have sufficient capacity[175](index=175&type=chunk) - Gene therapy manufacturing is novel and complex, with few specialized contract manufacturers, increasing the risk of supply shortages and delays[174](index=174&type=chunk) [Risks Relating to Intellectual Property](index=40&type=section&id=Risks%20Relating%20to%20Our%20Intellectual%20Property) The company's success hinges on obtaining and maintaining intellectual property protection, facing risks of insufficient patent coverage, reliance on potentially terminable third-party licenses, and potential infringement claims - Commercial success depends on protecting intellectual property, but there is no assurance that patents will be issued or afford sufficient protection against competitors[200](index=200&type=chunk) - The company relies on licenses from third parties (e.g., University of California, Cornell, Virovek) for key technology, and the loss of these licenses could materially harm the business[214](index=214&type=chunk)[216](index=216&type=chunk) - Some in-licensed intellectual property was developed with U.S. government funding and is subject to federal regulations, including "march-in" rights and a preference for U.S.-based manufacturing[233](index=233&type=chunk) [Risks Related to Commercialization](index=48&type=section&id=Risks%20Related%20to%20Commercialization%20of%20Our%20Product%20Candidates) Commercialization risks include uncertain market acceptance, intense competition, challenges in securing adequate reimbursement for novel single-administration therapies, and the company's lack of sales and marketing capabilities - Even with regulatory approval, product candidates may not gain market acceptance among physicians, patients, and payers, which is critical for commercial success[240](index=240&type=chunk) - The company faces intense competition from well-established therapies like EYLEA and LUCENTIS, as well as other gene therapy companies like REGENXBIO[243](index=243&type=chunk)[245](index=245&type=chunk) - The company faces uncertainty related to pricing and reimbursement, as its product candidates are designed for single administration, and obtaining adequate coverage is crucial but not guaranteed[271](index=271&type=chunk) [Risks Related to Business Operations](index=55&type=section&id=Risks%20Related%20to%20Our%20Business%20Operations) Operational risks include negative public perception of gene therapy, dependence on key personnel, growth management challenges, COVID-19 impacts, cybersecurity threats, and compliance with complex healthcare and privacy laws - Negative public opinion and increased regulatory scrutiny of gene therapy, potentially fueled by adverse events in the field, could damage perception of the company's products and delay approvals[277](index=277&type=chunk)[279](index=279&type=chunk) - The COVID-19 pandemic has impacted business practices and may continue to affect clinical trials, supply chains, and regulatory processes, with the full extent of future impact remaining uncertain[284](index=284&type=chunk)[290](index=290&type=chunk) - The company is subject to evolving and stringent privacy and data security laws (HIPAA, CCPA, GDPR), and failure to comply could result in significant fines, litigation, and reputational damage[303](index=303&type=chunk)[306](index=306&type=chunk)[308](index=308&type=chunk) [Unregistered Sales of Equity Securities and Use of Proceeds](index=65&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) The company reported no unregistered sales of equity securities during the period - None[338](index=338&type=chunk) [Defaults Upon Senior Securities](index=66&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities) The company reported no defaults upon senior securities - None[340](index=340&type=chunk) [Mine Safety Disclosures](index=66&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) This item is not applicable - Not applicable[341](index=341&type=chunk) [Other Information](index=66&type=section&id=Item%205.%20Other%20Information) On November 1, 2021, the company terminated a building lease for a $0.4 million fee and concurrently subleased a portion of the premises to Revolution Medicines, Inc - On November 1, 2021, the company amended a lease to terminate one of its buildings, paying a **$0.4 million** fee[343](index=343&type=chunk) - Concurrently, the company subleased a portion of the terminated premises to Revolution Medicines, Inc. for a monthly base rent of **$0.1 million**[343](index=343&type=chunk) [Exhibits](index=67&type=section&id=Item%206.%20Exhibits) This section lists exhibits filed with the Form 10-Q, including separation and consulting agreements and required officer certifications - Exhibits filed include separation and consulting agreements with Angela Thedinga, and CEO/CFO certifications as required by Sarbanes-Oxley[346](index=346&type=chunk)

(Mark One) ___________________________________________________________________ FORM 10-Q ___________________________________________________________________ Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 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 Adve ...