Quarterly Report for Quarter Ending September 30, 2025 (Form 10-Q)

Management's Discussion and Analysis of Financial Condition and Results of Operations.

You should read the following discussion and analysis of financial condition and operating results together with our financial statements and related notes appearing in this Quarterly Report on Form 10-Q ("Form 10-Q") and those included in our Annual Report on Form 10-K for the year ended December 31, 2024 (the "Form 10-K"). Some of the information contained in this discussion and analysis or set forth elsewhere in this Form 10-Q, including information with respect to our plans and strategy for our business and related financing, includes forward-looking statements that involve risks and uncertainties. As a result of many important factors, including those set forth in the "Risk Factors" section of this Form 10-Q, our actual results could differ materially from the results described in, or implied by, the forward-looking statements contained in the following discussion and analysis. For convenience of presentation some of the numbers have been rounded in the text below. Unless the context requires otherwise, references in this Management's Discussion and Analysis of Financial Condition and Results of Operations to the "Company," "we," "us" and "our" refer to MeiraGTx Holdings plc and its subsidiaries.

Overview

We are a vertically integrated, clinical-stage genetic medicines company with a broad pipeline of late-stage clinical programs, including Parkinson's disease, radiation-induced xerostomia and AIPL1-associated retinal dystrophy. Our clinical programs use targeted local delivery of small doses of genetic medicines to treat both inherited and more common conditions with severe unmet need. The successful development of the clinical pipeline is supported by our internal end-to-end manufacturing capabilities. We have two GMP viral vector production facilities, internal plasmid production for GMP, as well as an in-house Quality Control hub for stability and release, all fit for IND through commercial supply. In addition, we have developed a proprietary manufacturing platform with leading yield and quality aspects and commercial readiness. Our core capabilities in viral vector and capsid optimization allow increased potency, decreased dose and significantly reduced cost of goods for our genetic medicines. We have developed a transformative gene regulation platform using bespoke synthetic riboswitch technology invented in-house that allows for the precise, dose-responsive control of any transgene under the control of oral small molecules. We are focusing the riboswitch platform on in vivo delivery of biologic therapeutics such as the metabolic peptides GLP-1, GIP, glucagon, amylin, PYY and leptin via oral small molecules, as well as cell therapy for oncology and autoimmune diseases, and long-term intractable pain. We have developed unique comprehensive technology capabilities to apply genetic medicine to more common diseases, increasing efficacy, addressing novel targets, and expanding access in some of the largest disease areas where the unmet need remains high.

Our discussion of our financial condition and results of operations is based upon our financial statements, which have been prepared in accordance with generally accepted accounting principles in the United States ("GAAP"). Since our formation, we have devoted substantially all of our resources to developing our technology platform, establishing our viral vector manufacturing facilities and our GMP plasmid and DNA production facility and developing manufacturing processes, advancing the product candidates in our ophthalmology, salivary gland and neurodegenerative disease programs, building our intellectual property portfolio, organizing and staffing our Company, developing our business plan, raising capital, and providing general and administrative support for these operations. To date, we have financed our operations primarily with cash on hand, proceeds from the sales of our equity securities, debt financing, strategic collaborations and asset sales, including upfront and milestone payments in connection with the Collaboration, Option and License Agreement with Johnson & Johnson Innovative Medicine (formerly known as Janssen Pharmaceuticals, Inc.), dated as of January 30, 2019 (the "Collaboration Agreement"), which also provided us with research funding, and the Asset Purchase Agreement, dated as of December 20, 2023, we entered into with Johnson & Johnson Innovative Medicine (the "Asset Purchase Agreement") pursuant to which we sold and assigned to Johnson & Johnson Innovative Medicine, and Johnson & Johnson Innovative Medicine purchased and assumed, that certain License Agreement, dated February 5, 2019, by and between UCL Business Plc (now UCL Business Ltd.) ("UCLB"), on the one hand, and MeiraGTx UK II Limited and MeiraGTx Limited, on the other hand (the "UCLB RPGR License Agreement"), relating to the research, development, manufacture and exploitation of botaretigene sparoparvovec, or bota-vec (formerly referred to as AAV-RPGR), for the treatment of X-linked retinitis pigmentosa related to mutations in the retinitis pigmentosa GTPase regulator gene, or XLRP-RPGR (the "RPGR Product"), and other related assets as

described in the Asset Purchase Agreement. Through September 30, 2025, we received gross proceeds of approximately $632.2 million from sales of our equity securities, gross proceeds of approximately $75.0 million from issuance of debt, $130.0 million in upfront and milestone payments from the Collaboration Agreement with Johnson & Johnson Innovative Medicine, and $125.0 million from the Asset Purchase Agreement with Johnson & Johnson Innovative Medicine. As of September 30, 2025, we had cash, cash equivalents and restricted cash of $17.1 million, as well as $2.8 million in receivables due from Johnson & Johnson Innovative Medicine in connection with PPQ and transition services we provided to Johnson & Johnson Innovative Medicine.

We are a clinical stage company and have not generated any product revenues to date. We have ongoing clinical development programs and a broad pipeline of preclinical programs. Since inception, we have incurred significant operating losses. Our net losses for the three-month periods ended September 30, 2025 and 2024 were $50.5 million and $39.3 million, respectively. Our net losses for the nine-month periods ended September 30, 2025 and 2024 were $129.3 million and $108.4 million, respectively. As of September 30, 2025, we had an accumulated deficit of $831.3 million. We do not expect to generate revenue from sales of products unless and until we successfully initiate and complete clinical development and obtain regulatory approval for any product candidates, or satisfy our third party obligations.

Our total operating expenses for the three-month periods ended September 30, 2025 and 2024 were $46.5 million and $51.0 million, respectively. For the nine-month periods ended September 30, 2025 and 2024, our total operating expenses were $138.5 million and $144.6 million, respectively. We expect to continue incurring costs associated with our clinical activities for AAV-hAQP1 for the treatment of radiation-induced xerostomia and xerostomia associated with Sjogren's syndrome, AAV-GAD for the treatment of Parkinson's disease, as well as costs associated with the delivery of services under the Asset Purchase Agreement and related agreements. We also incurred expenses during the nine-month period ended September 30, 2025 and expect to continue to incur expenses related to research activities in additional therapeutic areas to expand our pipeline, developing our potentially transformative gene regulation technology, hiring additional personnel as needed in manufacturing, research, clinical operations, quality and other functional areas, and associated cash and share-based compensation expense, as well as the further development of internal manufacturing capabilities and capacity and other associated costs including the management of our intellectual property portfolio.

We will require additional capital in the future, which we may raise through equity offerings (including our "at-the-market" equity offering program), debt financings, marketing and distribution arrangements and other collaborations, strategic alliances and licensing arrangements or other sources to enable us to complete the development and potential commercialization of our product candidates. Furthermore, we expect to continue incurring costs associated with being a public company. Adequate additional financing may not be available to us on acceptable terms, or at all. Our failure to raise capital as and when needed would have a negative effect on our financial condition and our ability to pursue our business strategy. In addition, attempting to secure additional financing may divert the time and attention of our management from day-to-day activities and harm our product candidate development efforts. If we are unable to raise capital when needed or on acceptable terms, we would be forced to delay, reduce or eliminate certain of our research and development programs.

Based on our cash, cash equivalents, accounts receivable - related party and tax incentive receivable at September 30, 2025, together with the $75.0 million upfront payment from Eli Lilly and Company ("Lilly") as described below, and the $22.0 million deposit received from Hologen Limited to date during the fourth quarter 2025 and the remaining $150.0 million from the anticipated closing of the strategic collaboration with Hologen Limited as described below, we estimate that such funds will be sufficient to enable us to fund our operating expenses and capital expenditure requirements into the second half of 2027 and to repay our debt obligation of $75.0 million to Perceptive (due in August 2026). This estimate does not include the $135.0 million in potential near-term cash consideration from Lilly upon the achievement of certain development and regulatory approval milestones. This estimate also does not include the $285.0 million in milestones we are eligible to receive under the Asset Purchase Agreement upon first commercial sale of an RPGR Product in the United States and in at least one of the United Kingdom, France, Germany, Spain and Italy, for completion of the transfer of certain manufacturing technology to Johnson & Johnson Innovative Medicine and upon regulatory approval of a Johnson & Johnson Innovative Medicine-selected manufacturing facility in each of the United States and European Union, or EU, for commercial manufacture of the RPGR Product. We have based these estimates

on assumptions that may prove to be wrong, and we may use our available capital resources sooner than we currently expect. See "Liquidity and Capital Resources." Because of the numerous risks and uncertainties associated with the development of our product candidates, any future product candidates, our platform and technology and because the extent to which we may enter into collaborations with third parties for development of any of our product candidates is unknown, we are unable to estimate the amounts of increased capital outlays and operating expenses associated with completing the research and development of our product candidates.

Adequate additional funds may not be available to us on acceptable terms, or at all. To the extent that we raise additional capital through the sale of equity or convertible securities, your ownership interest will be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect your rights as a shareholder. Any future debt financing or preferred equity or other financing, if available, may involve agreements that include covenants limiting or restricting our ability to take specific actions, such as incurring additional debt, making capital expenditures or declaring dividends and may require the issuance of warrants, which could potentially dilute your ownership interests.

If we raise additional funds through collaborations, strategic alliances, or licensing arrangements with third parties, we may have to relinquish valuable rights to our technologies, future revenue streams, research programs or product candidates or grant licenses on terms that may not be favorable to us. If we are unable to raise additional funds through equity or debt financings when needed, we may be required to delay, limit, reduce, or terminate our product development programs or any future commercialization efforts or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves.

Because of the numerous risks and uncertainties associated with drug development, we are unable to predict the timing or amount of increased expenses or when or if we will be able to achieve or maintain profitability. Even if we are able to generate revenue from product sales, we may not become profitable. If we fail to become profitable or are unable to sustain profitability on a continuing basis, then we may be unable to continue our operations at planned levels and be forced to reduce or terminate our operations.

Hologen Transactions

On March 9, 2025 (the "Hologen Signing Date"), we and our affiliates entered into a strategic collaboration with Hologen Limited, a non-cellular company limited by shares incorporated in Guernsey ("Hologen"), and its affiliates. Hologen is a leading developer of multi-modal generative AI foundation models of real-world clinical data for clinical medicine and pharmaceutical drug development. As part of the strategic collaboration, we and Hologen entered into the Framework Agreements (as defined below), pursuant to which we and our affiliates will receive from Hologen an upfront cash payment of $200 million (the "Upfront Payment") on the Hologen Closing Date (as defined below), and Hologen will provide additional funding of up to an additional $230 million as further described below. As part of the strategic collaboration, we also received 250,000 Class A shares of Hologen at a nominal price. Furthermore, we have the option to receive up to an additional 500,000 Class A shares of Hologen at a nominal price if certain funding obligations as described herein are not met by Hologen. During the nine months ended September 30, 2025, Hologen made $28.0 million in payments as part of its commitment toward the Upfront Payment, and made an additional $22.0 million in payments to date during the fourth quarter 2025.

The closing of the transactions contemplated by the Framework Agreements (the "Hologen Closing Date") is expected to occur in the fourth calendar quarter of 2025, subject to customary closing and funding conditions.

Neuro Framework Agreement

On the Hologen Signing Date, we, MeiraGTx Neuro UK Limited, a private company limited by shares incorporated in England and one of our wholly-owned subsidiaries ("MeiraGTx Neuro UK"), Hologen Neuro AI Limited, a non-cellular company limited by shares incorporated in Guernsey and an affiliate of Hologen ("Hologen Neuro"), and Hologen, entered into that certain Framework Agreement (the "Neuro Framework Agreement"), pursuant to which, on the Hologen Closing Date, we, MeiraGTx Neuro UK, MeiraGTx Neuro I, LLC, a Delaware limited liability company and one of our wholly-owned subsidiaries ("MeiraGTx Neuro US"), Hologen, Hologen Neuro and Hologen

Neuro AI UK Limited, a private company limited by shares incorporated in England and an affiliate of Hologen ("Hologen Neuro UK"), shall enter into a Collaboration and License Agreement (the "Hologen Collaboration Agreement") for the research, development, manufacture and commercialization of our (i) AAV-GAD investigational gene therapy for the treatment of Parkinson's disease, AAV-BDNF investigational gene therapy for the treatment of genetic obesity disorders and other potential locally delivered genetic medicines to the central nervous system (the "Clinical Programs") and (ii) proprietary device designed to effect the local delivery of a gene therapy product into the central nervous system or any topographic or subcutaneous tissue modification on the face and scalp, of humans or animals (the "Delivery Device"), in each case, in accordance with the terms and conditions of the Hologen Collaboration Agreement.

On the Hologen Closing Date, under the Hologen Collaboration Agreement, MeiraGTx Neuro US will receive the applicable portion of the Upfront Payment in consideration for granting to Hologen Neuro and Hologen Neuro UK, as of the Hologen Closing Date and subject to the license granted by Hologen Neuro and Hologen Neuro UK back to MeiraGTx Neuro UK, exclusive, worldwide, royalty-free, fully paid-up licenses to certain of our intellectual property rights for the research, development, manufacture and commercialization of the Clinical Programs and the Delivery Device. On the Hologen Closing Date, (a) MeiraGTx Neuro UK will receive Class A shares of Hologen Neuro representing a 30% ownership of the issued share capital of Hologen Neuro, in consideration for the provision of services to Hologen Neuro and Hologen Neuro UK as specified in the Hologen Collaboration Agreement, including services relating to the development of the Clinical Programs and the Delivery Device and certain other transition services, and (b) Hologen Guernsey will receive Class B shares of Hologen Neuro representing a 70% ownership of the issued share capital of Hologen Neuro, in consideration for paying the applicable portion of the Upfront Payment to MeiraGTx Neuro US, as well as a commitment to provide additional capital of up to $230 million to fund the development of the Clinical Programs and the Delivery Device. Additionally, Hologen will license to Hologen Neuro its proprietary multi-modal generative foundation models (LMMs), or large medicine models, pursuant to a license agreement mutually agreeable to the parties.

As of the Hologen Closing Date, Hologen Neuro shall be governed by a board of directors comprised of three representatives designated by Hologen and two representatives designated by MeiraGTx Neuro UK, and certain material business decisions (as further enumerated in the Neuro Framework Agreement) will require the approval of at least 70% of the directors then in office.

Following the Hologen Closing Date and in accordance with the terms of the Hologen Collaboration Agreement, the parties shall negotiate in good faith and enter into clinical and commercial supply agreements, pursuant to which MeiraGTx Neuro UK (directly, or through affiliates or subcontractors) shall manufacture and supply AAV-GAD, AAV-BDNF and other potential locally delivered genetic medicines to the central nervous system.

Manufacturing Framework Agreement

On the Hologen Signing Date, MeiraGTx Manufacturing Limited, a private company limited by shares incorporated in England and one of our wholly-owned subsidiaries ("MeiraGTx Manufacturing"), MeiraGTx Limited, a private company limited by shares incorporated in England and one of our wholly-owned subsidiaries ("MeiraGTx Limited"), and Hologen, entered into that certain Framework Agreement (the "Manufacturing Framework Agreement" and, together with the Neuro Framework Agreement, the "Framework Agreements"), pursuant to which, on the Hologen Closing Date and in exchange for the applicable portion of the Upfront Payment, Hologen will acquire a minority interest in MeiraGTx Manufacturing, an entity that will comprise our flexible and scalable end-to-end genetic medicines manufacturing business. Hologen will also contribute a portion of the annual funding to MeiraGTx Manufacturing.

As of the Hologen Closing Date, MeiraGTx Manufacturing shall be governed by a board of directors comprised of three representatives designated by MeiraGTx Limited and two representatives designated by Hologen, and certain material business decisions (as further enumerated in the Manufacturing Framework Agreement) will require the approval of at least 70% of the directors then in office.

For a period of twelve months following the Hologen Closing Date, Hologen has an exclusive, irrevocable option to purchase additional shares in MeiraGTx Manufacturing at a specified price, such that following exercise of such option, Hologen shall own 40% of the issued share capital of MeiraGTx Manufacturing in the aggregate. In the

event that Hologen does not exercise its option, we have an exclusive, irrevocable option to purchase all of the shares of MeiraGTx Manufacturing held by Hologen for the same price that Hologen paid for such shares. Such option shall be exercisable anytime beginning on the third anniversary of the Hologen Closing Date and ending three years thereafter.

Acquisition of Smart Immune Assets

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In July 2025, our new, wholly-owned French subsidiary MeiraGTx Cell Therapies acquired through a French insolvency proceeding certain assets and operations of Smart Immune, a French clinical-stage biotechnology company that developed ProTcell, a T-cell progenitor-based cell therapy platform that harnesses the patient's own thymus to rapidly re-arm the immune system against a wide range of potential conditions, including cancer and autoimmune conditions. Following a public tender process, the Paris court of economic activities chose our offer to acquire a majority of Smart Immune's assets and operations, including twenty of its employees. We paid a purchase price of €250,000 plus a €100,000 transfer fee under a license agreement. As a result of the acquisition, we intend to advance the development of, among other things, off-the-shelf, allogenic ProTcell-derived CAR-T therapies that incorporate our riboswitch technology platform.

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Lilly Transaction

On November 7, 2025 (the "Effective Date"), our affiliates MeiraGTx Ocular UK Limited ("MeiraGTx Ocular"), MeiraGTx Limited and MeiraGTx UK II Limited, entered into a strategic collaboration and license agreement with Lilly (the "Lilly Collaboration Agreement")for the research, development and commercialization of genetic medicines in and related to the area of ophthalmology.Under the Lilly Collaboration Agreement, we havegranted Lilly exclusive, worldwide rights to research, develop and commercializeour product candidate AAV-AIPL1, which treatsLeber congenital amaurosis 4, or LCA4, caused by mutations in the AIPL1gene, as well as two other preclinical product candidates which are intended to treat otherinherited retinal dystrophies.As of the Effective Date, Lilly has (i) an exclusive license to proprietary intravitreal capsids for use with up to five targets, relating to or useful in the field of ophthalmology, to be selected by Lilly, (ii) an exclusive license to proprietary pan-retinal or rod-specific promoters for use with up to five targets, relating to or useful in the field of ophthalmology, to be selected by Lillyand (iii) a right of first designation with respect to certain target-specific transactions that MeiraGTx Ocular or its affiliates may seek to pursue in the field of ophthalmology. Lilly also has a right of first negotiation for use of our proprietary riboswitch technology in the field of ophthalmological gene editing.

Under the terms of the Lilly Collaboration Agreement, we will receive an upfront payment of $75.0 million after signing the Lilly Collaboration Agreement and will be eligible to receive up to over $400.0 million in total milestone payments, including up to $135.0 million in other potential near-term cash consideration upon the achievement of certain development and regulatory approval milestones. Lilly will have the right to research, develop and commercialize products under the Lilly Collaboration Agreement, at its cost. The Lilly Collaboration Agreement also provides for tiered royalties to be paid to MeiraGTx Ocular.

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Recent Development Highlights and Anticipated Milestones

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Strategic Collaboration with Eli Lilly and Company to Develop and Commercialize Genetic Medicines in Ophthalmology:

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AAV2-hAQP1 for the Treatment of Radiation-Induced Xerostomia:

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Additional indications for AAV2-hAQP1:

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AAV-GAD for the Treatment of Parkinson's Disease:

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Strategic Collaboration with Hologen AI:

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Botaretigene Sparoparvovec for the Treatment of X-linked Retinitis Pigmentosa (XLRP):

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Riboswitch Gene Regulation Technology Platform for in vivo Delivery:

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Components of Our Results of Operations

Service Revenue - Related Party

Our service revenue consisted of the process performance qualification ("PPQ") services performed in connection with the Asset Purchase Agreement and related agreements.

Operating Expenses

Our operating expenses since inception have consisted primarily of general and administrative costs and research and development costs. Since 2024, we have incurred expenses classified as cost of service revenue - related party performed in connection with the Asset Purchase Agreement and related agreements.

Cost of Service Revenue - Related Party

Our cost of service revenue consisted of the PPQ services performed in connection with the Asset Purchase Agreement and related agreements.

General and Administrative Expenses

General and administrative expenses consist primarily of salaries and other related costs, including share-based compensation, for personnel in our executive, finance, legal, business development and administrative functions. General and administrative expenses also include legal fees relating to intellectual property and corporate matters; professional fees for accounting, auditing, tax and consulting services; insurance costs; travel expenses; and office facility-related expenses, which include direct depreciation costs.

We have incurred, and expect to continue to incur, increased expenses associated with being a public company, including costs of accounting, audit, legal, regulatory and tax-related services associated with maintaining compliance with Nasdaq and SEC requirements; director and officer insurance costs; and investor and public relations costs.

Research and Development Expenses

Research and development expenses consist primarily of costs incurred for our research activities, including our discovery efforts, and the development of our product candidates, and include:

We expense research and development costs as incurred.

Research and development activities are central to our business model. We expect to continue incurring increasing research and development costs associated with our clinical activities for AAV-hAQP1 for the treatment of radiation-induced xerostomia and xerostomia associated with Sjogren's syndrome, as well as for AAV-GAD for the treatment of Parkinson's disease, although certain of these increases relating to AAV-GAD are expected to be offset by the funding provided by Hologen after the anticipated closing of the strategic collaboration we entered into with them. In addition, we expect to continue to incur expenses related to research activities in additional therapeutic areas to expand our pipeline and develop our potentially transformative gene regulation technology.

We cannot determine with certainty the duration and costs of future clinical trials of our product candidates or any other product candidate we may develop or if, when, or to what extent we will generate revenue from the commercialization and sale of any product candidate for which we obtain marketing approval. We may never succeed in obtaining marketing approval for any product candidate. The duration, costs and timing of clinical trials and

development of our existing product candidates or any other product candidate we may develop will depend on a variety of factors, including:

A change in the outcome of any of these variables with respect to the development of a product candidate could mean a significant change in the costs and timing associated with the development of that product candidate. For example, if the U.S. Food and Drug Administration (the "FDA") or another U.S. or foreign regulatory authority were to require us to conduct clinical trials beyond those that we anticipate will be required for the completion of clinical development of a product candidate, or if we experience significant delays in our clinical trials due to patient enrollment or other reasons, we would be required to expend significant additional financial resources and time on the completion of clinical development.

Other Non-Operating Income (Expense)

Other non-operating income (expense) includes the following:

Foreign Currency (Loss) Gain

Our condensed consolidated financial statements are presented in U.S. dollars, which is our reporting currency. The financial position and results of operations of our subsidiaries are measured using the foreign subsidiaries' local currency as the functional currency, either the pound sterling or the euro. These entities' cash accounts holding U.S. dollars and intercompany payables and receivables are remeasured based upon the exchange rate at the date of remeasurement with the resulting gain or loss included in the condensed consolidated statements of operations and comprehensive loss.

Interest Income

Interest income is comprised on interest earned on our interest-bearing bank accounts.

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Interest Expense

Interest expense consists of interest expense and amortization of the debt discount in connection with the debt financing described in Note 10 to our condensed consolidated financial statements.

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Gain on Sale of Nonfinancial Assets

The gain on sale of nonfinancial assets represents the value allocated to the nonfinancial assets sold and assigned to Johnson & Johnson Innovative Medicine including the UCLB RPGR License Agreement relating to the

research, development, manufacture and exploitation of the RPGR Product, and other related assets pursuant to the Asset Purchase Agreement, net of carrying value.

Other Comprehensive Loss

Other comprehensive loss includes the following:

Foreign Currency Translation Gain (Loss)

Revenue and expenses of subsidiaries have been translated into U.S. dollars at average exchange rates prevailing during the period. Assets and liabilities have been translated at the rates of exchange on the condensed consolidated balance sheet date. The resulting translation gain and loss adjustments are recorded directly as a separate component of shareholders' equity and as other comprehensive loss on the condensed consolidated statements of operations and comprehensive loss.

Critical Accounting Policies and Use of Estimates

Management's discussion and analysis of our financial condition and results of operations is based on our condensed consolidated financial statements, which have been prepared in accordance with GAAP. The preparation of these condensed consolidated financial statements requires us to make estimates and judgements that affect the reporting amounts of assets, liabilities, revenues and expenses and the disclosure of contingent assets and liabilities in our condensed consolidated financial statements. On an ongoing basis, we evaluate our estimates and judgements, including those related to service revenue, share-based compensation and accrued expenses. We base our estimates on historical experience, known trends and events and various other factors that we believe to be reasonable under the circumstances, the results of which form the basis for making judgements about the carrying value of assets and liabilities that are not readily apparent from our sources. Actual results may differ from these estimates under different assumptions.

The Company's critical accounting policies, significant judgements and estimates are included in the Company's Form 10-K for the year ended December 31, 2024 and Note 2 to our unaudited condensed consolidated financial statements included elsewhere in this Form 10-Q.

Results of Operations

Comparison of Three Months Ended September 30, 2025 and 2024

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Service Revenue - Related Party

Service revenue was $0.4 million for the three months ended September 30, 2025, compared to $10.9 million for the three months ended September 30, 2024. The decrease of $10.5 million was due to decreased activity of PPQ services under the Asset Purchase Agreement and related agreements as the work was substantially completed as of September 30, 2025.

Cost of Service Revenue - Related Party

Cost of service revenue was $0.3 million for the three months ended September 30, 2025 compared to $12.0 million for the three months ended September 30, 2024. The decrease of $11.7 million was due to decreased activity of PPQ services under the Asset Purchase Agreement and related agreements as the work was substantially completed as of September 30, 2025.

General and Administrative Expenses

General and administrative expenses were $13.6 million for the three months ended September 30, 2025, compared to $12.7 million for the three months ended September 30, 2024. The increase of $0.9 million was primarily due to an increase of $0.5 million in rent and facilities costs, an increase of $0.3 million due to a loss on disposal of equipment, furniture and fixtures, an increase of $0.3 million in consulting fees, an increase of $0.2 million in share-based compensation and a $0.4 million increase in other office related costs. These increases were partially offset by a decrease of $0.8 million in legal and accounting fees.

Research and Development Expenses

Research and development expenses for the three months ended September 30, 2025 and 2024 were as follows (in thousands):

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Clinical program expenses represent the direct costs for each clinical trial plus the cost of the clinical trial material charged from the manufacturing costs.

Manufacturing expenses represent the costs to manufacture clinical trial material, including payroll, facilities, manufacturing supplies, raw materials, quality control and quality assurance. Upon completion of the manufacture of a batch of clinical trial material, the standard cost of manufacturing the batch of clinical trial material is charged to the clinical programs.

Preclinical program expenses represent the direct costs for each group of preclinical programs.

Other research and development expenses represent costs that are not allocated to a specific clinical or preclinical program, such as payroll and payroll related costs, share-based compensation, travel, rent and facilities costs, depreciation and other non-program specific expenses.

Research and development expenses for the three months ended September 30, 2025 were $32.5 million, compared to $26.2 million for the three months ended September 30, 2024. The increase of $6.3 million was primarily due to an increase in manufacturing costs due to both a lower allocation of clinical trial material batch costs to our clinical programs and a lower allocation of costs to cost of service revenue reflecting PPQ services provided under the Asset Purchase Agreement and related agreements being substantially complete by September 30, 2025. Other cost increases arose in our clinical programs for other ocular diseases, primarily due to an increase in manufactured clinical trial material batches related to these programs, and our preclinical programs for gene regulation reflecting preclinical studies initiated during the three months ended September 30, 2025. These increases were partially offset by a decrease in our AAV-hAQP1 program due to the required clinical trial material being substantially manufactured in the prior year.

Foreign Currency (Loss) Gain

Foreign currency loss was $1.6 million for the three months ended September 30, 2025 compared to a gain of $3.5 million for the three months ended September 30, 2024. The change of $5.1 million was primarily due to the weakening of the U.S. dollar against the pound sterling and euro as it relates to the valuation of our intercompany payables and receivables.

Interest Income

Interest income was $0.2 million for the three months ended September 30, 2025 compared to $1.2 million for the three months ended September 30, 2024. The decrease of $1.0 million was due to lower interest rates and cash balances held in interest bearing accounts during 2025.

Interest Expense

Interest expense was $3.1 million for the three months ended September 30, 2025 compared to $3.4 million for the three months ended September 30, 2024. The decrease of $0.3 million was primarily due to a lower interest rate in connection with the debt financing described in Note 10 to our unaudited condensed consolidated financial statements included elsewhere in this Form 10-Q.

Comparison of Nine Months Ended September 30, 2025 and 2024

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Service Revenue - Related Party

Service revenue was $6.0 million for the nine months ended September 30, 2025 compared to $11.9 million for the nine months ended September 30, 2024. The $5.9 million decrease was due to decreased activity of PPQ services under the Asset Purchase Agreement and related agreements as the work was substantially completed as of September 30, 2025.

Cost of Service Revenue - Related Party

Cost of service revenue was $4.4 million for the nine months ended September 30, 2025 compared to $12.0 million for the nine months ended September 30, 2024. The decrease of $7.6 million was due to decreased activity of PPQ services under the Asset Purchase Agreement and related agreements as the work was substantially completed as of September 30, 2025.

General and Administrative Expenses

General and administrative expenses were $35.3 million for the nine months ended September 30, 2025, compared to $37.1 million for the nine months ended September 30, 2024. The decrease of $1.8 million was primarily due to a $1.4 million gain on lease termination, a decrease of $1.5 million in legal and accounting fees, a decrease of $0.5 million in share-based compensation, a decrease of $0.1 million in insurance costs and a decrease of $0.1 million in depreciation. These decreases were partially offset by an increase of $0.9 million in payroll and payroll-related costs, an increase of $0.4 million due to a loss on disposal of equipment, furniture and fixtures and $0.5 million in other office related costs.

Research and Development Expenses

Research and development expenses for the nine months ended September 30, 2025 and 2024 were as follows (in thousands):

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Clinical program expenses represent the direct costs for each clinical trial plus the cost of the clinical trial material charged from the manufacturing costs.

Manufacturing expenses represent the costs to manufacture clinical trial material, including payroll, facilities, manufacturing supplies, raw materials, quality control and quality assurance. Upon completion of the manufacture of a batch of clinical trial material, the cost of manufacturing the batch of clinical trial material is charged to the clinical programs.

Preclinical program expenses represent the direct costs for each group of preclinical programs.

Other research and development expenses represent costs that are not allocated to a specific clinical or preclinical program, such as payroll and payroll related costs, share-based compensation, travel, rent and facilities costs, depreciation and other non-program specific expenses.

Research and development expenses for the nine months ended September 30, 2025 were $98.8 million, compared to $95.5 million for the nine months ended September 30, 2024. The increase of $3.3 million was primarily due to an increase in costs associated with our AAV-GAD program and other ocular diseases, both reflecting increased clinical trial material manufactured in the period. Other cost increases also arose in our preclinical programs for ocular diseases and gene regulation reflecting preclinical studies initiated in the period. These increases were partially offset by a decrease in costs related to our AAV-hAQP1 program due to the required clinical trial material being substantially manufactured in the prior year, a decrease in other research and development expenses due to a decrease in share-based compensation expenses and a decrease in costs related to the bota-vec program as Johnson & Johnson Innovative Medicine is now primarily funding the expenses related to this program as a result of the Asset Purchase Agreement.

Foreign Currency Gain

Foreign currency gain was $10.8 million for the nine months ended September 30, 2025 compared to a gain of $2.6 million for the nine months ended September 30, 2024. The change of $8.1 million was primarily due to the weakening of the U.S. dollar against the pound sterling and euro as it relates to the valuation of our intercompany payables and receivables.

Interest Income

Interest income was $1.5 million for the nine months ended September 30, 2025 compared to $3.1 million for the nine months ended September 30, 2024. The decrease of $1.6 million was due to lower interest rates and cash balances during 2025.

Interest Expense

Interest expense was $9.1 million for the nine months ended September 30, 2025 compared to $9.9 million for the nine months ended September 30, 2024. The decrease of $0.7 million was primarily due to a lower interest rate in connection with the debt financing described in Note 10 to our unaudited condensed consolidated financial statements included elsewhere in this Form 10-Q

Gain on Sale of Nonfinancial Assets

There was no gain on sale of nonfinancial assets during the nine months ended September 30, 2025 compared to $28.4 million for the nine months ended September 30, 2024. This decrease was a result of the recognition of the $50.0 million milestone allocated to the nonfinancial assets sold and assigned to Johnson & Johnson Innovative Medicine being fully recognized during 2023 and 2024.

Liquidity and Capital Resources

Since our inception, we have incurred significant operating losses. For the nine months ended September 30, 2025, we used $93.0 million in cash flows from operations. We did not generate positive cash flows from operations during the period and there are no assurances that we will generate positive cash flows in the future. Additionally, there are no assurances that we will be successful in obtaining an adequate level of financing for the development and commercialization of our product candidates. We expect to incur significant expenses and operating losses for the foreseeable future as we advance the preclinical and clinical development of our product candidates. We expect that our research and development and general and administrative costs will increase in connection with conducting preclinical studies and clinical trials for our product candidates, building out internal capacity to have products manufactured to support preclinical studies and clinical trials as well as to manufacture commercial products, expanding our intellectual property portfolio, and providing general and administrative support for our operations. As a result of these incurred and expected expenses we will need additional capital to fund our operations, which we may obtain from additional equity or debt financings, collaborations, licensing arrangements, or other sources.

We do not currently have any approved products and have never generated any revenue from product sales. We have historically financed our operations primarily through cash on hand and proceeds from the sale of our equity securities, the issuance of debt and upfront and milestone payments from the Collaboration Agreement and Asset Purchase Agreement.

Based on our current cash, cash equivalents, accounts receivable - related party, and tax incentive receivable at September 30, 2025, together with the $75.0 million upfront payment from Lilly, and the $22.0 million deposit received from Hologen to date during the fourth quarter 2025 and the remaining $150.0 million proceeds from the anticipated closing of the strategic collaboration with Hologen, we estimate that we will be able to fund our operating expenses and capital expenditure requirements into the second half of 2027 and to repay our debt obligation of $75.0 million to Perceptive (due in August 2026). This estimate does not include the $135.0 million in potential near-term cash consideration from Lilly upon the achievement of certain development and regulatory approval milestones. This estimate also does not include the $285.0 million in milestones we are eligible to receive under the Asset Purchase Agreement upon first commercial sale of an RPGR Product in the United States and in at least one of the United Kingdom, France, Germany, Spain and Italy, for completion of the transfer of certain manufacturing technology to Johnson & Johnson Innovative Medicine and upon regulatory approval of a Johnson & Johnson Innovative Medicine-selected manufacturing facility in each of the United States and European Union for commercial manufacture of the RPGR Product. We have based these estimates on assumptions that may prove to be wrong, and we could utilize our available capital resources sooner than we expect.

Cash Flows

As of September 30, 2025, we had $17.1 million of cash, cash equivalents and restricted cash.

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The following table summarizes our sources and uses of cash, cash equivalents and restricted cash for the period presented:

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Operating Activities

During the nine months ended September 30, 2025, our cash used in operating activities of $93.0 million was primarily due to a net loss of $129.3 million as we incurred expenses associated with research activities on our clinical programs, manufacturing of our clinical trial materials, preclinical research programs and general and administrative expenses. The net loss included non-cash income and expense of $14.9 million, which consisted primarily of $16.5 million of share-based compensation, $9.4 million of depreciation and amortization, $0.8 million of amortization of debt discount, $0.3 million of loss on disposal of equipment, furniture and fixtures, $0.1 million of amortization of interest on asset retirement obligations, $10.8 million of a foreign exchange gain, and $1.5 million of gain on termination of lease liabilities. Additionally, operating assets, consisting of accounts receivable - related party, contract assets - related party, inventory, prepaid expenses, tax incentive receivable, other assets and other current assets, decreased by $1.2 million and operating liabilities, consisting of accounts payable, accrued expenses, other current liabilities and deferred revenue - related party, increased by $20.2 million.

During the nine months ended September 30, 2024, our cash used in operating activities of $81.2 million was primarily due to a net loss of $108.4 million as we incurred expenses associated with research activities on our clinical programs, manufacturing of our clinical trial materials, preclinical research programs and general and administrative expenses. The net loss included non-cash income and expense of $1.0 million, which consisted primarily of $28.4 million of a gain on sale of nonfinancial assets, $19.1 million of share-based compensation, $9.8 million of depreciation and amortization, $2.6 million of a foreign currency gain, $0.9 million of non-cash interest, a $0.4 million loss on disposal of equipment, furniture and fixtures and $0.2 million of net change in right-of-use-assets and liabilities. Additionally, operating assets, consisting of accounts receivable - related party, prepaid expenses, tax incentive receivable, other assets and other current assets, decreased by $13.5 million and operating liabilities, consisting of accounts payable, accrued expenses, other current liabilities and deferred revenue - related party, increased by $14.7 million.Investing Activities

Net cash used in investing activities for the nine months ended September 30, 2025 of $3.0 million consisted of purchases of property and equipment for our manufacturing, laboratory and process development facilities of $3.5 million offset by proceeds from the sale of equipment, furniture and fixtures of $0.5 million.

Net cash provided by investing activities for the nine months ended September 30, 2024 of $24.7 million consisted of $28.4 million from proceeds from the sale of nonfinancial assets offset by $3.7 million of purchases of property and equipment for our manufacturing, laboratory and process development facilities.

Financing Activities

Net cash provided by financing activities was $6.9 million for the nine months ended September 30, 2025, which consisted of $9.9 million net proceeds from an "at-the-market" offering of our ordinary shares which was offset by the payment of $3.0 million to cover tax withholding obligations upon the vesting of restricted share unit awards.

Net cash provided by financing activities was $50.3 million for the nine months ended September 30, 2024, which consisted of $52.6 million net proceeds from the issuance of our ordinary shares, offset by the payment of $2.3 million to cover tax withholding obligations upon the vesting of restricted share unit awards.

Off-Balance Sheet Arrangements

We have not entered into any off-balance sheet arrangements under applicable SEC rules and do not have any holdings in variable interest entities.