SEGMENT INFORMATION (Tables)

6 Months Ended

Jun. 30, 2026

SEGMENT INFORMATION

Summary of operating segment expenses


		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.