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SEGMENT INFORMATION (Tables) | 3 Months Ended
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Mar. 31, 2025
SEGMENT INFORMATION |
Summary of operating segment expenses |
​ | ​ | ​ | ​ | ​ | ​ | ​
​ | ​ | Three months ended March 31,
​ | ​ | 2026 | ​ | 2025
Total revenue | ​ | $ | — | ​ | $ | —
​ | ​ | ​ | ​ | ​ | ​ | ​
Research and development expense: | ​ | ​ | ​ | ​ | ​ | ​
Compensation and benefits | ​ | ​ | 7,971,816 | ​ | ​ | 5,410,424
Duchenne muscular dystrophy program (Deramiocel) | ​ | ​ | 13,032,167 | ​ | ​ | 7,705,195
Exosomes platform research | ​ | ​ | 809,295 | ​ | ​ | 1,520,934
Other R&D segment expenses (1) | ​ | ​ | 2,156,138 | ​ | ​ | 1,325,471
Total research and development expense, excluding non-cash expense | ​ | ​ | 23,969,416 | ​ | ​ | 15,962,024
Stock-based compensation expense | ​ | ​ | 3,126,181 | ​ | ​ | 2,738,584
Depreciation and amortization | ​ | ​ | 281,315 | ​ | ​ | 214,964
Total research and development expense | ​ | ​ | 27,376,912 | ​ | ​ | 18,915,572
​ | ​ | ​ | ​ | ​ | ​ | ​
General and administrative expense: | ​ | ​ | ​ | ​ | ​ | ​
Compensation and benefits | ​ | ​ | 2,234,948 | ​ | ​ | 1,500,843
Other G&A segment expenses (2) | ​ | ​ | 4,115,764 | ​ | ​ | 1,375,233
Total general and administrative expense, excluding non-cash expense | ​ | ​ | 6,350,712 | ​ | ​ | 2,876,076
Stock-based compensation expense | ​ | ​ | 2,791,874 | ​ | ​ | 3,000,814
Depreciation and amortization | ​ | ​ | 253,355 | ​ | ​ | 190,486
Total general and administrative expense | ​ | ​ | 9,395,941 | ​ | ​ | 6,067,376
​ | ​ | ​ | ​ | ​ | ​ | ​
Operating loss | ​ | ​ | (36,772,853) | ​ | ​ | (24,982,948)
​ | ​ | ​ | ​ | ​ | ​ | ​
Investment income | ​ | ​ | 2,904,506 | ​ | ​ | 729,542
Other income (expense) | ​ | ​ | (72,998) | ​ | ​ | (138,188)
Total non-operating income, net | ​ | ​ | 2,831,508 | ​ | ​ | 591,354
​ | ​ | ​ | ​ | ​ | ​ | ​
Loss before income taxes | ​ | ​ | (33,941,345) | ​ | ​ | (24,391,594)
(Provision for) benefit from income taxes | ​ | ​ | — | ​ | ​ | —
​ | ​ | ​ | ​ | ​ | ​ | ​
Net loss | ​ | $ | (33,941,345) | ​ | $ | (24,391,594)
​
(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, business insurance, employee travel, and other facility and information technology costs.

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- Definition Tabular disclosure of the reconciliation of profit (loss) from reportable segments to the consolidated income (loss) before income tax expense (benefit) and discontinued operations. Includes, but is not limited to, reconciliation after income tax if income tax is allocated to the reportable segment. + References Reference 1: http://fasb.org/us-gaap/role/ref/legacyRef -Name Accounting Standards Codification -Topic 280 -SubTopic 10 -Section 50 -Paragraph 31 -Publisher FASB -URI https://asc.fasb.org/1943274/2147482810/280-10-50-31 Reference 2: http://fasb.org/us-gaap/role/ref/legacyRef -Name Accounting Standards Codification -Topic 280 -SubTopic 10 -Section 50 -Paragraph 30 -Subparagraph (b) -Publisher FASB -URI https://asc.fasb.org/1943274/2147482810/280-10-50-30 + Details
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