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SEGMENT INFORMATION

9 Months Ended

Sep. 30, 2025

SEGMENT INFORMATION

SEGMENT INFORMATION

9.            SEGMENT INFORMATION

The Company operates as a single operating segment. The Company’s Chief Executive Officer, who serves as the Chief Operating Decision Maker (“CODM”), is responsible for allocating resources and assessing performance. The CODM reviews the Company’s operating results on an aggregate basis to make decisions about resource allocation, evaluate financial performance, and manage the overall business. Accordingly, the Company’s operations are managed as one reportable segment focused on the development and commercialization of its therapeutic candidates.

The following table represents consolidated net loss summarized by the significant segment expenses regularly reviewed by the CODM for the three and nine months ended September 30, 2025 and 2024:

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Three months ended September 30,

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Nine months ended September 30,

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2025

2024

2025

2024

Total revenues

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$

—

​

$

2,261,642

​

$

—

​

$

11,139,956

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​

​

​

​

​

​

​

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​

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Research and development:

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​

​

​

​

​

​

​

​

​

​

​

Compensation and benefits

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​

9,586,659

​

​

4,461,233

​

​

27,176,680

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​

13,314,847

Duchenne muscular dystrophy program (Deramiocel)

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​

7,310,101

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​

5,430,428

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​

24,854,222

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​

16,674,567

Exosomes platform research

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1,474,937

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​

842,952

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​

4,160,004

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​

2,518,052

Other R&D segment expenses (1)

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​

1,987,401

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​

1,073,254

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5,131,018

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​

2,906,183

Total research and development

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20,359,098

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11,807,867

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61,321,924

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​

35,413,649

General and administrative:

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​

​

​

​

​

​

​

​

​

​

​

Compensation and benefits

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​

3,314,529

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​

2,323,384

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11,672,488

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7,516,797

Other G&A segment expenses (2)

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2,610,413

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1,140,271

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5,991,710

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3,076,511

Total general and administrative

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5,924,942

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3,463,655

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17,664,198

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​

10,593,308

Operating loss

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​

(26,284,040)

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(13,009,880)

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(78,986,122)

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​

(34,867,001)

Total non-operating income, net

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​

1,713,393

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​

453,152

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4,113,090

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​

1,516,418

Net loss

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$

(24,570,647)

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$

(12,556,728)

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$

(74,873,032)

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$

(33,350,583)

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(1) Other R&D segment expenses primarily include other pipeline development costs, depreciation, amortization, 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

The entire disclosure for reporting segments including data and tables. Reportable segments include those that meet any of the following quantitative thresholds a) it's reported revenue, including sales to external customers and intersegment sales or transfers is 10 percent or more of the combined revenue, internal and external, of all operating segments b) the absolute amount of its reported profit or loss is 10 percent or more of the greater, in absolute amount of 1) the combined reported profit of all operating segments that did not report a loss or 2) the combined reported loss of all operating segments that did report a loss c) its assets are 10 percent or more of the combined assets of all operating segments.

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