MeiraGTx Holdings plc

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 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 June 30, 2025, we received gross proceeds of approximately $632.2 million from sales of our equity securities, gross proceeds 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, 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

Previous wording

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 June 30, 2025, we had cash, cash equivalents and restricted cash of $34.4 million, as well as $2.3 million in receivables due from Johnson & Johnson Innovative Medicine in connection with PPQ and transition services we provided to Johnson & Johnson Innovative Medicine.

Current wording

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.

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 June 30, 2025 and 2024 were $38.8 million and $48.6 million, respectively. Our net losses for the six-month periods ended June 30, 2025 and 2024 were $78.8 million and $69.1 million, respectively. As of June 30, 2025, we had an accumulated deficit of $780.8 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.

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 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.

Previous wording

Our total operating expenses for the three-month periods ended June 30, 2025 and 2024 were $48.5 million and $46.2 million, respectively. For the six-month periods ended June 30, 2025 and 2024, our total operating expenses were $92.0 million and $93.7 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 six-month period ended June 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.

Current wording

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.

Previous wording

Based on our cash, cash equivalents, accounts receivable – related party and tax incentive receivable at June 30, 2025, together with the $17.0 million deposit received from Hologen Limited during the third quarter 2025 and the remaining 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 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.

Current wording

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

Previous wording

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 June 2025, Hologen made a $6.0 million payment as part of its commitment toward the Upfront Payment, and made an additional $17.0 million payment during the third quarter 2025.

Current wording

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.

Previous wording

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

Current wording

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.

Previous wording

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

Current wording

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.

Previous wording

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.

Current wording

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.

Previous wording

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:

Current wording

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

Previous 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.

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

Previous wording

General and administrative expenses were $12.3 million for the three months ended June 30, 2025, compared to $11.3 million for the three months ended June 30, 2024. The increase of $1.0 million was primarily due to an increase of $0.6 million in legal and accounting fees, an increase of $0.6 million in share-based compensation and an increase of $0.1 million in payroll and payroll related costs. These increases were partially offset by a decrease of $0.3 million in consulting fees.

Current wording

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.

Previous wording

Foreign currency gain was $8.6 million for the three months ended June 30, 2025 compared to a loss of $0.3 million for the three months ended June 30, 2024. The change of $8.9 million was primarily due to the weakeningof the U.S. dollar against the pound sterling and euro as it relates to the valuation of our intercompany payables and receivables.

Current wording

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 weakeningof the U.S. dollar against the pound sterling and euro as it relates to the valuation of our intercompany payables and receivables.

Previous wording

Interest income was $0.4 million for the three months ended June 30, 2025 compared to $0.8 million for the three months ended June 30, 2024. The decrease of $0.4 million was due to lower interest rates and cash balances held in interest bearing accounts during 2025.

Current wording

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.

Previous wording

Interest expense was $3.0 million for the three months ended June 30, 2025 compared to $3.3 million for the three months ended June 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.

Current wording

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.

Previous wording

General and administrative expenses were $21.7 million for the six months ended June 30, 2025, compared to $24.4 million for the six months ended June 30, 2024. The decrease of $2.7 million was primarily due to a $1.3 million gain on lease termination, a decrease of $0.7 million in share-based compensation, a decrease of $0.7 million in consulting fees, a decrease of $0.6 million in legal and accounting fees and a decrease of $0.2 million in other office related costs. These decreases were partially offset by an increase of $0.8 million in payroll and payroll-related costs.

Current wording

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.

Previous wording

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

Current wording

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

Previous wording

Research and development expenses for the six months ended June 30, 2025 were $66.3 million, compared to $69.3 million for the six months ended June 30, 2024. The decrease of $3.0 million was primarily due to a decrease of $10.8 million in manufacturing costs primarily due to a reclassification of batch costs to the AAV-GAD program and reclassification of cost of service revenue due to progress of PPQ services provided under the Asset Purchase Agreement and related agreements, a decrease of $1.1 million 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, a decrease of $0.1 million related to gene regulation research costs, and a decrease of $3.4 million in other research and development costs. These decreases were partially offset by an increase of $6.8 million in clinical trial expenses primarily due to an increase in costs associated with our AAV-GAD program, an increase of $1.3 million for the AAV-hAQP1 program, an increase of $1.1 million in other ocular diseases, an increase of $0.9 million in preclinical ocular diseases, a reduction in a prior period credit of $0.4 million for the AAV-CNBGB3 and AAV-CNBGA3 programs, and a reduction in reimbursements from Johnson & Johnson Innovative Medicine of $1.9 million.

Current wording

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.

Previous wording

Foreign currency gain was $12.3 million for the six months ended June 30, 2025 compared to a loss of $0.8 million for the six months ended June 30, 2024. The change of $13.1 million was primarily due to the weakeningof the U.S. dollar against the pound sterling and euro as it relates to the valuation of our intercompany payables and receivables.

Current wording

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 weakeningof the U.S. dollar against the pound sterling and euro as it relates to the valuation of our intercompany payables and receivables.

Previous wording

Interest income was $1.4 million for the six months ended June 30, 2025 compared to $1.9 million for the six months ended June 30, 2024. The decrease of $0.5 million was due to lower interest rates and cash balances during 2025.

Current wording

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.

Previous wording

Interest expense was $6.1 million for the six months ended June 30, 2025 compared to $6.5 million for the six months ended June 30, 2024. The decrease of $0.4 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

Current wording

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

Previous wording

There was no gain on sale of nonfinancial assets during the six months ended June 30, 2025 compared to $29.0 for the six months ended June 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 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 in 2024.

Current wording

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.

Previous wording

Since our inception, we have incurred significant operating losses. For the six months ended June 30, 2025, we used $80.8 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

Current wording

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.

Previous wording

Based on our current cash, cash equivalents, accounts receivable – related party, and tax incentive receivable at June 30, 2025, together with the $17.0 million deposit received from Hologen during the third quarter 2025 and the remaining 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 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 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.

Current wording

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.

Previous wording

As of June 30, 2025, we had $34.4 million of cash, cash equivalents and restricted cash.

Current wording

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

Previous wording

During the six months ended June 30, 2025, our cash used in operating activities of $80.8 million was primarily due to a net loss of $78.8 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 $3.5 million, which consisted primarily of $12.3 million of a foreign exchange gain, $10.6 million of share-based compensation, $6.2 million of depreciation and amortization, $1.4 million of gain on termination of lease liability, $0.1 million gain on disposal of equipment, furniture and fixtures, and $0.6 million of amortization of debt discount. Additionally, operating assets, consisting of accounts receivable – related party, prepaid expenses, tax incentive receivable, other assets and other current assets, decreased by $5.8 million and operating liabilities, consisting of accounts payable, accrued expenses and deferred revenue – related party, decreased by $11.4 million.

Current wording

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.

Previous wording

net loss included non-cash income and expense of $8.0 million, which consisted primarily of $29.0 million of a gain on sale of nonfinancial assets, $12.8 million of share-based compensation, $6.5 million of depreciation and amortization, $0.8 million of a foreign currency loss, $0.5 million of amortization of debt discount and a $0.4 million loss on disposal of equipment, furniture and fixtures. Additionally, operating assets, consisting of accounts receivable – related party, prepaid expenses, tax incentive receivable, other assets and other current assets, decreased by $19.5 million and operating liabilities, consisting of accounts payable, accrued expenses and deferred revenue – related party, increased by $1.3 million.

Current 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.Investing Activities

Previous wording

Net cash used in investing activities for the six months ended June 30, 2025 of $2.5 million consisted of purchases and disposals of property and equipment for our manufacturing, laboratory and process development facilities.

Current wording

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.

Previous wording

Net cash provided by investing activities for the six months ended June 30, 2024 of $26.0 million consisted of $29.0 million from proceeds from the sale of nonfinancial assets offset by $3.0 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 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.

Previous wording

Net cash provided by financing activities was $7.2 million for the six months ended June 30, 2025, which consisted of $9.9 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.

Current wording

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.

Previous wording

Net cash provided by financing activities was $0.3 million for the six months ended June 30, 2024, which consisted of $2.5 million of proceeds from an “at-the-market” offering of our ordinary shares, net of one-time costs relating to the preparation and filing of the shelf registration statement, accounting and legal fees, and negotiation of the sales agreement offset by the payment of $2.2 million to cover tax withholding obligations upon the vesting of restricted share unit awards.

Current 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.