| Segment Reporting |
The Company is advancing a pipeline of innovative gene therapies seeking to deliver to patients suffering from rare and other devastating diseases single treatments with potentially curative results. The Company manages the operations related to these research and development activities within one operating segment because they rely on a common set of infrastructure, resources and technology of the products and production processes, types of customers, distribution methods and regulatory environment. The leadership team is identified as the Chief Operating Decision Maker (“CODM”). The CODM allocates resources to research projects and clinical candidates based on scientific data as well as quantitative and qualitative expected risk adjusted returns on investment. The CODM uses segment operating loss to monitor that cash operating losses remain within the approved budget. The accounting policies of the operating segment are the same as those described in the summary of significant accounting policies. | | | | | | | | | | | | | | | Three months ended June 30, | | Six months ended June 30, | | | 2025 | | 2024 | | 2025 | | 2024 | | | | (in thousands) | | (in thousands) | Revenue | | $ | 5,262 | | $ | 11,126 | | $ | 6,829 | | $ | 19,611 | Less: | | | | | | | | | | | | | Employee related expenses | | | (14,525) | | | (22,665) | | | (28,741) | | | (47,048) | Laboratory and development expenses | | | (15,593) | | | (9,404) | | | (31,560) | | | (18,480) | Professional fees | | | (3,124) | | | (2,696) | | | (5,181) | | | (4,959) | Facility expenses | | | (1,704) | | | (4,624) | | | (3,371) | | | (9,763) | Consumables | | | (697) | | | (3,472) | | | (1,633) | | | (6,684) | Other segment items(1) | | | (1,945) | | | (631) | | | (2,979) | | | (8,063) | Segment operating loss | | | (32,326) | | | (32,366) | | | (66,636) | | | (75,386) | Reconciliation | | | | | | | | | | | | | Depreciation and amortization expense | | | (3,630) | | | (2,670) | | | (8,157) | | | (5,299) | Share-based compensation expense | | | (5,084) | | | (6,880) | | | (9,494) | | | (14,071) | Fair value loss - contingent consideration | | | (2,254) | | | 1,786 | | | (3,470) | | | 1,621 | Foreign currency gains / (losses), net | | | 18,638 | | | (989) | | | 25,810 | | | (2,134) | Interest income | | | 3,524 | | | 5,805 | | | 7,651 | | | 12,313 | Interest expense - Royalty Financing Agreement | | | (13,770) | | | (12,438) | | | (27,079) | | | (24,839) | Interest expense - Hercules debt facility | | | (1,821) | | | (3,719) | | | (3,621) | | | (7,415) | Other reconciling items | | | (571) | | | (3,880) | | | 4,561 | | | (5,103) | Consolidated loss before income tax expense | | $ | (37,294) | | $ | (55,351) | | $ | (80,435) | | $ | (120,313) |
| (1) | Other segment items included in segment operating loss include costs related to intellectual property, information technology and insurance offset by income related to payments received from European authorities to subsidize the Company’s research and development in the Netherlands. |
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