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v3.25.3

SEGMENT INFORMATION (Details) | 3 Months Ended | 9 Months Ended
--- | --- | ---
Sep. 30, 2025 USD ($) | Jun. 30, 2025 USD ($) | Mar. 31, 2025 USD ($) | Sep. 30, 2024 USD ($) | Jun. 30, 2024 USD ($) | Mar. 31, 2024 USD ($) | Sep. 30, 2025 USD ($) segment | Sep. 30, 2024 USD ($)
SEGMENT INFORMATION |  |  |  |  |  |  |  |
Number of reportable segment | segment |  |  |  |  |  |  | 1 |
Revenues |  |  |  | $ 2,261,642 |  |  |  | $ 11,139,956
Research and development: |  |  |  |  |  |  |  |
Total research and development | $ 20,359,098 |  |  | 11,807,867 |  |  | $ 61,321,924 | 35,413,649
General and administrative: |  |  |  |  |  |  |  |
General and Administrative Expense, Total | 5,924,942 |  |  | 3,463,655 |  |  | 17,664,198 | 10,593,308
LOSS FROM OPERATIONS | (26,284,040) |  |  | (13,009,880) |  |  | (78,986,122) | (34,867,001)
Total non-operating income, net | 1,713,393 |  |  | 453,152 |  |  | 4,113,090 | 1,516,418
NET LOSS | (24,570,647) | $ (25,910,791) | $ (24,391,594) | (12,556,728) | $ (10,999,782) | $ (9,794,073) | (74,873,032) | (33,350,583)
Single reportable segment |  |  |  |  |  |  |  |
SEGMENT INFORMATION |  |  |  |  |  |  |  |
Revenues |  |  |  | 2,261,642 |  |  |  | 11,139,956
Research and development: |  |  |  |  |  |  |  |
Total research and development | 20,359,098 |  |  | 11,807,867 |  |  | 61,321,924 | 35,413,649
General and administrative: |  |  |  |  |  |  |  |
Compensation and benefits | 3,314,529 |  |  | 2,323,384 |  |  | 11,672,488 | 7,516,797
Other G&A segment expenses | 2,610,413 |  |  | 1,140,271 |  |  | 5,991,710 | 3,076,511
General and Administrative Expense, Total | 5,924,942 |  |  | 3,463,655 |  |  | 17,664,198 | 10,593,308
LOSS FROM OPERATIONS | (26,284,040) |  |  | (13,009,880) |  |  | (78,986,122) | (34,867,001)
Total non-operating income, net | 1,713,393 |  |  | 453,152 |  |  | 4,113,090 | 1,516,418
NET LOSS | (24,570,647) |  |  | (12,556,728) |  |  | (74,873,032) | (33,350,583)
Single reportable segment | Compensation and benefits |  |  |  |  |  |  |  |
Research and development: |  |  |  |  |  |  |  |
Total research and development | 9,586,659 |  |  | 4,461,233 |  |  | 27,176,680 | 13,314,847
Single reportable segment | Duchenne muscular dystrophy program (Deramiocel) |  |  |  |  |  |  |  |
Research and development: |  |  |  |  |  |  |  |
Total research and development | 7,310,101 |  |  | 5,430,428 |  |  | 24,854,222 | 16,674,567
Single reportable segment | Exosomes platform research |  |  |  |  |  |  |  |
Research and development: |  |  |  |  |  |  |  |
Total research and development | 1,474,937 |  |  | 842,952 |  |  | 4,160,004 | 2,518,052
Single reportable segment | Other R&D segment expenses |  |  |  |  |  |  |  |
Research and development: |  |  |  |  |  |  |  |
Total research and development | $ 1,987,401 |  |  | $ 1,073,254 |  |  | $ 5,131,018 | $ 2,906,183

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