Segment Information (Tables)

6 Months Ended

Jun. 30, 2025

Segment Reporting [Abstract]

Schedule of Results Of Operations by Reportable Segment

As of June 30, 2025 and 2024, the Company did not have any significant long-lived assets located outside of the United States. Information on the segment and reconciliation to net loss is as follows (in thousands):

	Three Months Ended June 30,				Six Months Ended June 30,
	2025		2024		2025		2024
Revenue under licensing agreements	$	—	$	925	$	—	$	925
Program specific expenses:
Rett syndrome	$	7,052	$	2,798	$	11,576	$	4,817
Batten disease	401		1,734		1,246		2,924
Early Discovery	720		1,705		2,007		3,171
Unallocated internal expenses:
Personnel-related	5,125		4,557		10,529		9,275
Stock-based compensation	1,575		1,375		3,146		1,939
Manufacturing	3,635		2,815		6,742		5,794
Other (a)	858		760		1,885		1,365
Total research and development expenses	$	19,366	$	15,744	$	37,131	$	29,285
General and administrative specific expenses:
Personnel-related	$	2,212	$	1,964	$	4,696	$	4,065
Stock-based compensation	1,833		939		4,310		1,419
Professional and consultant fees	970		998		2,247		2,500
Office-related	620		631		1,252		1,235
Other (b)	1,080		783		2,364		1,334
Total general and administrative expenses	$	6,715	$	5,315	$	14,869	$	10,553
Other income(c)	4,065		1,642		7,337		3,500
Net loss	$	(22,016)	$	(18,492)	$	(44,663)	$	(35,413)
(a) The Other expense segment items category within research and development expense is mainly comprised of: Consultant fees for programs not specified above, IT Software and network support and rent expenses. (b) The Other expense segment items category within general and administrative expense is mainly comprised of: Insurance, IT software and network support, market research, and tax expenses. (c) Other income included in net loss is mainly comprised of: Interest income, interest expense, other income and other expense.