| SEGMENT INFORMATION |
16. SEGMENT INFORMATION The Company operates as a single operating segment. The Company’s Chief Executive Officer, who serves as the Chief Operating Decision Maker (“CODM”), is responsible for allocating resources and assessing performance. The CODM reviews the Company’s operating results on an aggregate basis to make decisions about resource allocation, evaluate financial performance, and manage the overall business. Accordingly, the Company’s operations are managed as one reportable segment focused on the development and commercialization of its therapeutic candidates. The following table represents consolidated net loss summarized by the significant segment expenses regularly reviewed by the CODM for the three and six months ended June 30, 2026 and 2025. | | | | | | | | | | | | | | | Three months ended June 30, | | Six months ended June 30, | | | 2026 | | 2025 | | 2026 | | 2025 | Total revenue | | $ | — | | $ | — | | $ | — | | $ | — | | | | | | | | | | | | | | Research and development expense: | | | | | | | | | | | | | Compensation and benefits | | | 9,577,971 | | | 7,510,545 | | | 17,549,787 | | | 12,920,970 | Duchenne muscular dystrophy program (Deramiocel) | | | 13,053,071 | | | 9,838,927 | | | 26,085,238 | | | 17,544,121 | Exosomes platform research | | | 523,529 | | | 1,164,133 | | | 1,332,824 | | | 2,685,067 | Other R&D segment expenses (1) | | | 2,155,161 | | | 1,369,691 | | | 4,311,299 | | | 2,695,162 | Total research and development expense, excluding non-cash expense | | | 25,309,732 | | | 19,883,296 | | | 49,279,148 | | | 35,845,320 | Stock-based compensation expense | | | 3,260,674 | | | 1,930,467 | | | 6,386,855 | | | 4,669,051 | Depreciation and amortization | | | 295,886 | | | 233,491 | | | 577,201 | | | 448,455 | Total research and development expense | | | 28,866,292 | | | 22,047,254 | | | 56,243,204 | | | 40,962,826 | | | | | | | | | | | | | | General and administrative expense: | | | | | | | | | | | | | Compensation and benefits | | | 3,140,431 | | | 2,139,615 | | | 5,375,379 | | | 3,640,458 | Other G&A segment expenses (2) | | | 7,870,353 | | | 1,561,190 | | | 11,986,117 | | | 2,936,424 | Total general and administrative expense, excluding non-cash expense | | | 11,010,784 | | | 3,700,805 | | | 17,361,496 | | | 6,576,882 | Stock-based compensation expense | | | 2,802,251 | | | 1,716,687 | | | 5,594,125 | | | 4,717,501 | Depreciation and amortization | | | 266,293 | | | 252,788 | | | 519,648 | | | 443,273 | Total general and administrative expense | | | 14,079,328 | | | 5,670,280 | | | 23,475,269 | | | 11,737,656 | | | | | | | | | | | | | | Operating loss | | | (42,945,620) | | | (27,717,534) | | | (79,718,473) | | | (52,700,482) | | | | | | | | | | | | | | Investment income | | | 2,211,295 | | | 1,793,352 | | | 5,115,801 | | | 2,522,894 | Interest expense | | | — | | | — | | | — | | | — | Other income (expense) | | | 2,271 | | | 14,991 | | | (70,727) | | | (123,197) | Total non-operating income, net | | | 2,213,566 | | | 1,808,343 | | | 5,045,074 | | | 2,399,697 | | | | | | | | | | | | | | Loss before income taxes | | | (40,732,054) | | | (25,909,191) | | | (74,673,399) | | | (50,300,785) | (Provision for) benefit from income taxes | | | (1,600) | | | (1,600) | | | (1,600) | | | (1,600) | | | | | | | | | | | | | | Net loss | | $ | (40,733,654) | | $ | (25,910,791) | | $ | (74,674,999) | | $ | (50,302,385) |
| (1) | Other R&D segment expenses primarily include other pipeline development costs, and other facility costs. |
| (2) | Other G&A segment expenses primarily include accounting, legal and other professional fees, consulting expenses, pre-commercial initiatives, business insurance, employee travel, and other facility and information technology costs. |
The asset information provided to the CODM for the single operating segment is consistent with the amounts reported in the consolidated balance sheets.
|