v3.26.1
SEGMENT INFORMATION (Details)
3 Months Ended 6 Months Ended
Jun. 30, 2026
USD ($)
Mar. 31, 2026
USD ($)
Jun. 30, 2025
USD ($)
Mar. 31, 2025
USD ($)
Jun. 30, 2026
USD ($)
segment
Jun. 30, 2025
USD ($)
SEGMENT INFORMATION            
Number of operating segment | segment         1  
Number of reportable segment | segment         1  
Research and development expense:            
Total research and development expense $ 28,866,292   $ 22,047,254   $ 56,243,204 $ 40,962,826
General and administrative expense:            
Total general and administrative expense 14,079,328   5,670,280   23,475,269 11,737,656
LOSS FROM OPERATIONS (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
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) $ (33,941,345) (25,910,791) $ (24,391,594) (74,674,999) (50,302,385)
Single reportable segment            
Research and development expense:            
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 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
LOSS FROM OPERATIONS (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
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)
Single reportable segment | Compensation and benefits            
Research and development expense:            
Total research and development expense, excluding non-cash expense 9,577,971   7,510,545   17,549,787 12,920,970
Single reportable segment | Duchenne muscular dystrophy program (Deramiocel)            
Research and development expense:            
Total research and development expense, excluding non-cash expense 13,053,071   9,838,927   26,085,238 17,544,121
Single reportable segment | Exosomes platform research            
Research and development expense:            
Total research and development expense, excluding non-cash expense 523,529   1,164,133   1,332,824 2,685,067
Single reportable segment | Other R&D segment expenses            
Research and development expense:            
Total research and development expense, excluding non-cash expense $ 2,155,161   $ 1,369,691   $ 4,311,299 $ 2,695,162