MeiraGTx Holdings plc

Previous wording

We are a vertically integrated, clinical-stage genetic medicines company with a broad pipeline of late-stage clinical programs supported by end-to-end manufacturing capabilities. We have internal plasmid production for good manufacturing practices, or GMP, two GMP viral vector production facilities 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, core capabilities in viral vector design and optimization and a potentially transformative riboswitch gene regulation platform technology that allows for the precise, dose-responsive control of gene expression by oral small molecules. We are focusing the riboswitch platform on the delivery of metabolic peptides including GLP-1, GIP, glucagon and PYY using oral small molecules, as well as cell therapy for oncology and autoimmune diseases. We have developed the technology 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. We are an exempted company incorporated under the laws of the Cayman Islands in 2018, and prior to that, we commenced operations as MeiraGTx Limited, a private limited company incorporated under the laws of England and Wales in 2015.

Current wording

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 onin 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.

Previous wording

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 Series A ordinary shares, convertible preferred C shares and ordinary shares, debt financing and 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, 2024, we received gross proceeds of approximately $618.1 million from sales of our ordinary shares, Series A ordinary shares and convertible preferred C shares, gross proceeds of approximately $75.0 million from issuance of debt, $130.0 million from the Collaboration Agreement, and $115.0 million from the Asset Purchase Agreement. As of September 30, 2024, we had cash, cash equivalents and restricted cash of

Current wording

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 and 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 March 31, 2025, we received gross proceeds of approximately $626.7 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 &

Previous wording

$125.0 million, as well as $3.3 million in receivables due from Johnson & Johnson Innovative Medicine in connection with transition services we provided to Johnson & Johnson Innovative Medicine.

Current wording

Johnson Innovative Medicine, and $125.0 million from the Asset Purchase Agreement with Johnson & Johnson Innovative Medicine. As of March 31, 2025, we had cash, cash equivalents and restricted cash of $68.6 million, as well as $0.7 million in receivables due from Johnson & Johnson Innovative Medicine in connection with PPQ and transition services we provided to Johnson & Johnson Innovative Medicine.

Previous wording

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, 2024 and 2023 were $39.3 million and $44.3 million, respectively. For the nine-month periods ended September 30, 2024 and 2023, our net losses were $108.4 million and $104.2 million, respectively. As of September 30, 2024, we had an accumulated deficit of $662.6 million. We do not expect to generate revenue from sales of products for several years, if at all. Under the Collaboration Agreement, we received an upfront payment in the amount of $100.0 million in March 2019 and a milestone payment in the amount of $30.0 million in December 2021. Additionally, pursuant to the Collaboration Agreement, we received research and development funding for certain research, manufacturing and clinical development costs. On December 20, 2023, we entered into an Asset Purchase Agreement with Johnson & Johnson Innovative Medicine pursuant to which the Company sold and assigned to Johnson & Johnson Innovative Medicine a License Agreement between the Company and UCLB relating to the research, development, manufacture and exploitation of the RPGR Product, and other related assets as described in the Asset Purchase Agreement. In connection with entering into the Asset Purchase Agreement, we entered into a Termination Agreement with Johnson & Johnson Innovative Medicine terminating the Collaboration Agreement. The Company and Johnson & Johnson Innovative Medicine also entered into a Supply Agreement on December 20, 2023 (the “Supply Agreement”) pursuant to which the Company agreed to manufacture and supply the RPGR Product for Johnson & Johnson Innovative Medicine. In December 2023, we received a non-refundable upfront payment of $65.0 million in connection with the Asset Purchase Agreement, and in the first quarter of 2024 we received a milestone payment of $50.0 million in connection with the achievement of the initiation of the extension study for the Phase 3 LUMEOS clinical trial for the RPGR Product.

Current wording

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 March 31, 2025 and 2024 were $40.0 million and $20.4 million, respectively. As of March 31, 2025, we had an accumulated deficit of $742.0 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.

Previous wording

Our total operating expenses for the three-month periods ended September 30, 2024 and 2023 were $51.0 million and $37.9 million, respectively. For the nine-month periods ended September 30, 2024 and 2023, our total operating expenses were $144.6 million and $105.3 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, as well as for AAV-GAD for the treatment of Parkinson’s disease. We also incurred expenses during the nine-month period ended September 30, 2024 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.

Current wording

Our total operating expenses for the three-month periods ended March 31, 2025 and 2024 were $43.5 million and $47.5 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 three-month period ended March 31, 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.

Previous wording

Based on our current cash, cash equivalents, tax incentive receivable and accounts receivable – related party at September 30, 2024 and the near-term milestone payments we expect to receive under the Asset Purchase Agreement, we estimate that we will be able to fund our operating expenses and capital expenditure requirements into the second quarter of 2026. This estimate 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 Janssen and upon regulatory approval of a Janssen-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.

Current wording

Based on our cash, cash equivalents, accounts receivable – related party and tax incentive receivable at March 31, 2025, together with the proceeds 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 2027 and to repay our debt obligation of $75.0 million to Perceptive (due in August 2026). This estimate 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 Medicne 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.

Previous wording

Botaretigene Sparoparvovec for the Treatment of XLRP:

Current wording

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

Previous wording

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

Current wording

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.

Previous wording

Research and development activities are central to our business model. We expect to continue incurring 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. In addition, we expect to continue to incur expenses related to research activities in additional therapeutic areas to expand our pipeline and developing our potentially transformative gene regulation technology.

Current wording

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.

Previous wording

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 MeiraGTx UK II Limited, MeiraGTx Ireland DAC, MeiraGTx Netherlands B.V., MeiraGTx Belgium and MeiraGTx B.V. are measured using the foreign subsidiaries’ local currency as the functional currency. 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.

Current wording

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.

Previous wording

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

Current wording

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.

Previous wording

The (loss) 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.

Current wording

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.

Previous wording

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.

Current wording

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.

Previous wording

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 license and collaboration 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.

Current wording

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.

Previous wording

There was no license revenue for the three months ended September 30, 2024, compared to $5.1 million for the three months ended September 30, 2023. The decrease is due to the termination of the Collaboration Agreement concurrent with the execution of the Asset Purchase Agreement.

Current wording

Cost of service revenue was $1.4 million for the three months ended March 31, 2025, due to progress of PPQ services under the Asset Purchase Agreement and related agreements. There was no cost of service revenue for the three months ended March 31, 2024.

Previous wording

General and administrative expenses were $37.1 million for the nine months ended September 30, 2024, compared to $35.2 million for the nine months ended September 30, 2023. The increase of $2.0 million was primarily due to an increase of $1.3 million in legal and accounting fees, $0.6 million in payroll and payroll-related costs, $0.8 million in other office related costs and $0.5 million in consulting fees. These increases were partially offset by a decrease of $0.6 million in share-based compensation and $0.6 million in insurance costs.

Current wording

General and administrative expenses were $9.4 million for the three months ended March 31, 2025, compared to $13.2 million for the three months ended March 31, 2024. The decrease of $3.8 million was primarily due to a decrease of $1.3 million in share-based compensation, a decrease of $1.3 million in legal and accounting fees, a $1.3 million gain due to the early termination of a lease agreement, a decrease of $0.4 million in consulting fees and a decrease of $0.2 million in other office related costs. These decreases were partially offset by an increase of $0.7 million in payroll and payroll related costs.

Previous wording

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.

Current wording

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.

Previous wording

Research and development expenses for the nine months ended September 30, 2024 were $95.5 million, compared to $70.1 million for the nine months ended September 30, 2023. The increase of $25.4 million was primarily due to a decrease of $60.6 million in reimbursements from Johnson & Johnson Innovative Medicine as the reimbursement for the nine months ended September 30, 2023 was in connection with research funding provided under the Collaboration Agreement, which was terminated on December 20, 2023. Expenses related to our preclinical programs increased $0.3 million primarily related to the development of our gene regulation technology. These increases were partially offset by a decrease in clinical trial expenses of $11.5 million primarily related to bota-vec as Johnson & Johnson Innovative Medicine is now primarily funding the expenses related to this program as a result of the Asset Purchase Agreement, a decrease in manufacturing costs of $22.2 million due to a decrease in manufacturing material purchases, lower batch costs of clinical material during the nine months ended September 30, 2024 compared to the nine months ended September 30, 2023, which costs were charged to the clinical programs as well as a reclassification of cost of service revenue based on progress of PPQ services provided under the Asset Purchase Agreement and related agreements. Additionally, other research and development costs decreased by $1.8 million.

Current wording

Research and development expenses for the three months ended March 31, 2025 were $32.8 million, compared to $34.3 million for the three months ended March 31, 2024. The decrease of $1.5 million was primarily due to a decrease of $1.0 million in manufacturing costs primarily due to a reclassification of cost of service revenue due to progress of PPQ services provided under the Asset Purchase Agreement and related agreements and a decrease of $2.2 million in other research and development expenses. These decreases were partially offset by a reduction in reimbursements from Johnson & Johnson Innovative Medicine of $0.7 million, an increase of $0.9 million in clinical trial expenses primarily due to an increase in costs associated with our AAV-hAQP1 program and our other ocular disease programs. The increase in clinical trial expenses was partially offset by a decrease in costs related to bota-vec as Johnson & Johnson Innovative Medicine is now primarily funding the expenses related to this program as a result of the Asset Purchase Agreement. Additionally, expenses related to our preclinical programs increased $0.1 million.

Previous wording

Interest income was $1.2 million for the three months ended September 30, 2024 compared to $0.5 million for the three months ended September 30, 2023. The increase of $0.7 million was due to higher interest rates and cash balances during 2024.

Current wording

Interest income was $1.0 million for the three months ended March 31, 2025 compared to $1.1 million for the three months ended March 31, 2024. The decrease of $0.1 million was due to lower interest rates and cash balances held in interest bearing accounts during 2025.

Previous wording

Interest expense was $9.9 million for the nine months ended September 30, 2024 compared to $9.8 million for the nine months ended September 30, 2023. The increase of $0.1 million was primarily due to a higher 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

Current wording

Interest expense was $3.0 million for the three months ended March 31, 2025 compared to $3.2 million for the three months ended March 31, 2024. The decrease of $0.2 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.

Previous wording

(Loss) Gain on Sale of Nonfinancial Assets

Current wording

Gain on Sale of Nonfinancial Assets

Previous wording

The loss on sale of nonfinancial assets was $0.6 million for the three months ended September 30, 2024, which was a result of an adjustment to the allocation of the transaction price and $50.0 million milestone payment to the performance obligations identified under the Asset Purchase Agreement. The nonfinancial assets were sold and assigned to Johnson & Johnson Innovative Medicine which include the UCLB RPGR License Agreement relating to the research, development, manufacture and exploitation of the RPGR Product, and other related assets described in the Asset Purchase Agreement.

Current wording

The gain on sale of nonfinancial assets was $0 for the three months ended March 31, 2025 compared to $29.0 million for the three months ended March 31, 2024. This decrease was a result of the recognition of the $50.0 million milestone during the three months ended March 31, 2024, which was 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 described in the Asset Purchase Agreement.

Previous wording

Since our inception, we have incurred significant operating losses. For the nine months ended September 30, 2024, we used $81.2 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.

Current wording

Since our inception, we have incurred significant operating losses. For the three months ended March 31, 2025, we used $36.8 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.

Previous wording

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 ordinary shares, series A ordinary shares and convertible preferred C shares and the issuance of debt. In March 2019 and December 2021, we received a $100.0 million upfront payment and a $30.0 million milestone payment, respectively, in connection with the Collaboration Agreement, which also provided us with research funding. On December 20, 2023, we entered into an Asset Purchase Agreement with Johnson & Johnson Innovative Medicine pursuant to which the Company sold and assigned to Johnson & Johnson Innovative Medicine the UCLB RPGR License Agreement relating to the research, development, manufacture and exploitation of the RPGR Product, and other related assets as described in the Asset Purchase Agreement. In connection with entering into the Asset Purchase Agreement, we entered into a Termination Agreement with Johnson & Johnson Innovative Medicine terminating the Collaboration Agreement. The Company and Johnson & Johnson Innovative Medicine also entered into a Supply Agreement on December 20, 2023 pursuant to which we agreed to manufacture and supply the RPGR Product for Johnson & Johnson Innovative Medicine. In December 2023, we received a non-refundable upfront payment of $65.0 million in connection with the Asset Purchase Agreement, and in the first quarter of 2024 we received a milestone payment of $50.0 million in connection with the achievement of the initiation of the extension study for the Phase 3 LUMEOS clinical trial for the RPGR Product.

Current wording

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.

Previous wording

As of September 30, 2024, we had $125.0 million of cash, cash equivalents and restricted cash.

Current wording

As of March 31, 2025, we had $68.6 million of cash, cash equivalents and restricted cash.

Previous wording

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.

Current wording

During the three months ended March 31, 2025, our cash used in operating activities of $36.8 million was primarily due to a net loss of $40.0 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 $3.2 million, which consisted primarily of $4.9 million of share-based compensation, $3.1 million of depreciation and amortization, $0.3 million of non-cash interest and $0.1 million of net change in right-of-use-assets and liabilities offset by $3.7 million of a foreign currency gain, $1.4 million of a gain on termination of lease liabilities and a $0.1 million gain on disposal of equipment, furniture and fixtures. Additionally, operating assets, consisting of accounts receivable – related party, contract assets – related party, inventory, prepaid expenses, tax incentive receivable and other current assets, decreased by $6.9 million and operating liabilities, consisting of accounts payable, accrued expenses, other current liabilities and deferred revenue – related party, decreased by $7.0 million.

Previous wording

During the nine months ended September 30, 2023, our cash used in operating activities of $90.8 million was primarily due to a net loss of $104.2 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 charges of $34.3 million, which consisted primarily of $20.8 million of share-based compensation, $2.9 million of a foreign currency loss, $9.8 million of depreciation and amortization and $0.8 million of amortization of debt discount. Additionally, operating assets, consisting of accounts receivable-related party, prepaid expenses, tax incentive receivable and other current assets, increased by $0.4 million and operating liabilities, consisting of accounts payable, accrued expenses and deferred revenue – related party, decreased by $20.5 million.

Current wording

During the three months ended March 31, 2024, our cash used in operating activities of $36.7 million was primarily due to a net loss of $20.4 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 of $18.0 million, which consisted primarily of $29.0 million of a gain on sale of nonfinancial assets, $7.0 million of share-based compensation, $3.3 million of depreciation and amortization, $0.5 million of a foreign currency loss and $0.3 million of amortization of debt discount. Additionally, operating assets, consisting of accounts receivable – related party, prepaid expenses, other assets and other current assets, decreased by $0.2 million and operating liabilities, consisting of accounts payable, accrued expenses, other current liabilities and deferred revenue – related party, increased by $1.5 million.

Previous wording

Net cash used in investing activities for the nine months ended September 30, 2023 of $17.1 million consisted of purchases of property and equipment for our manufacturing, laboratory and process development facilities and buildout costs of our new facilities.

Current wording

Net cash used in investing activities for the three months ended March 31, 2025 of $1.7 million consisted of purchases of property and equipment for our manufacturing, laboratory and process development facilities.

Previous wording

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.

Current wording

Net cash provided by investing activities for the three months ended March 31, 2024 of $27.3 million consisted of $29.0 million from proceeds from the sale of nonfinancial assets offset by $1.7 million of purchases of property and equipment for our manufacturing, laboratory and process development facilities.

Previous wording

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.

Current wording

Net cash provided by financing activities was $1.7 million for the three months ended March 31, 2025, which consisted of $4.5 million net proceeds from an “at-the-market” offering of our ordinary shares, offset by the payment of $2.8 million to cover tax withholding obligations upon the vesting of restricted share unit awards.