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v3.26.1

SEGMENT INFORMATION (Details) | 3 Months Ended
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Mar. 31, 2026 USD ($) segment | Mar. 31, 2025 USD ($)
SEGMENT INFORMATION |  |
Number of operating segment | segment | 1 |
Number of reportable segment | segment | 1 |
Research and development expense: |  |
Total research and development expense | $ 27,376,912 | $ 18,915,572
General and administrative expense: |  |
Total general and administrative expense | 9,395,941 | 6,067,376
LOSS FROM OPERATIONS | (36,772,853) | (24,982,948)
Investment income | 2,904,506 | 729,542
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 | 0 | 0
NET LOSS | (33,941,345) | (24,391,594)
Single reportable segment |  |
Research and development expense: |  |
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 | 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
LOSS FROM OPERATIONS | (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)
NET LOSS | (33,941,345) | (24,391,594)
Single reportable segment | Compensation and benefits |  |
Research and development expense: |  |
Total research and development expense, excluding non-cash expense | 7,971,816 | 5,410,424
Single reportable segment | Duchenne muscular dystrophy program (Deramiocel) |  |
Research and development expense: |  |
Total research and development expense, excluding non-cash expense | 13,032,167 | 7,705,195
Single reportable segment | Exosomes platform research |  |
Research and development expense: |  |
Total research and development expense, excluding non-cash expense | 809,295 | 1,520,934
Single reportable segment | Other R&D segment expenses |  |
Research and development expense: |  |
Total research and development expense, excluding non-cash expense | $ 2,156,138 | $ 1,325,471

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