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Summary of Significant Accounting Policies and Recent Accounting Pronouncements (Tables)

3 Months Ended

Mar. 31, 2025

Summary of Significant Accounting Policies and Recent Accounting Pronouncements

Schedule of Restricted Cash

​

​	​	​	​	​	​	​
​	​	March 31,		​	December 31,
​	​	2025		​	2024
Cash and cash equivalents		$	66,523		$	103,659
Restricted cash	​		2,087	​		2,009
Total cash, cash equivalents and restricted cash in the condensed consolidated statement of cash flows	​	$	68,610	​	$	105,668

Schedule of Financial Assets and Liabilities Measured at Fair Value

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​

​

​

​

​

​

​

​

​

​

​

​

​

​

​

Fair Value Measurement Using:

​

​

​

Significant

Significant Other

Significant

​

​

March 31,

​

Observable Inputs

​

Observable Inputs

​

Unobservable

Description

​

2025

​

(Level 1)

​

(Level 2)

​

(Level 3)

Cash equivalents

​

$

57,020

​

$

57,020

​

$

—

​

$

—

Restricted cash

​

$

2,087

​

$

2,087

​

$

—

​

$

—

​

​

​

​

​

​

​

​

​

​

​

​

​

​

​

​

Fair Value Measurement Using:

​

​

​

Significant

Significant Other

Significant

​

​

December 31,

​

Observable Inputs

​

Observable Inputs

​

Unobservable

Description

​

2024

​

(Level 1)

​

(Level 2)

​

(Level 3)

Cash equivalents

​

$

80,930

​

$

80,930

​

$

—

​

$

—

Restricted cash

​

$

2,009

​

$

2,009

​

$

—

​

$

—

Schedule of Change in Asset Retirement Obligations

​

​	​	​	​	​	​	​
​	​	For the Three-Month Periods Ended March 31,
​	​	2025		​	2024
Balance at beginning of period		$	2,821		$	2,401
Change in estimate	​	​	(1,576)	​	​	—
Amortization of interest	​		63	​		48
Effects of exchange rate changes	​		36	​		(9)
Balance at end of period	​	$	1,344	​	$	2,440

Schedule of Securities Excluded from Computation of Diluted Net Loss per Share

​

​	​	​	​	​
​		March 31,		March 31,
​		2025		2024
Share options		8,022,989		8,484,644
Restricted share units	​	3,389,750	​	4,094,750
Deferred share units	​	—	​	185,000
Warrants	​	700,000	​	700,000
​		12,112,739		13,464,394

Schedule of net loss for the segment

​

​	​	​	​	​	​	​
​	​	For the Three-Month Periods Ended March 31,
​	​	2025		​	2024
Service revenue - related party		$	1,926		$	697
Operating expenses:	​	​	​	​	​	​
Cost of service revenue - related party	​	​	1,378	​	​	—
General and administrative	​		6,348	​		8,864
Clinical programs:	​	​	​	​	​	​
Botaretigene sparoparvovec	​	​	—	​	​	3,957
AAV-hAQP1	​	​	4,852	​	​	1,663
AAV-CNGB3 / AAV-CNGA3	​	​	—	​	​	(1,179)
AAV-GAD	​	​	1,459	​	​	2,170
Other ocular diseases	​	​	1,445	​	​	293
Manufacturing	​	​	17,359	​	​	18,360
Preclinical programs:	​	​	​	​	​	​
Gene regulation	​	​	2,286	​	​	2,499
Neurodegenerative diseases	​	​	426	​	​	391
Preclinical ocular diseases	​	​	625	​	​	327
Other research and development1	​	​	(624)	​	​	541
Johnson & Johnson Innovative Medicine reimbursement	​	​	—	​	​	(652)
Share-based compensation	​	​	4,887	​	​	6,960
Depreciation and amortization	​	​	3,081	​	​	3,275
Total operating expenses	​	​	43,522	​	​	47,469
Other segment items2	​	​	1,615	​	​	26,330
Segment net loss	​	$	(39,981)	​	​	(20,442)
Adjustments or reconciling items	​	​	—	​	​	—
Net loss	​	$	(39,981)	​	$	(20,442)

​

¹ Other research and development is comprised of all other costs including payroll and payroll related costs, travel, rent and facilities costs and other non-program specific expenses.

² Other segment items is comprised of foreign currency gain (loss), interest income, interest expense and gain on sale of nonfinancial assets.

Schedule of Long-lived Assets by Geographical Area

​

​	​	​	​	​	​	​
​		March 31,			December 31,
​	​	2025		​	2024
United States	​	$	2,515	​	$	7,209
United Kingdom	​		28,255	​		28,419
European Union	​	​	103,487	​	​	100,845
​	​	$	134,257	​	$	136,473

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