Segment Information (Tables)

9 Months Ended

Sep. 30, 2025

Segment Reporting [Abstract]

Schedule of Results Of Operations by Reportable Segment

As of September 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 September 30,				Nine Months Ended September 30,
	2025		2024		2025		2024
Revenue under licensing agreements	$	—	$	—	$	—	$	925
Program specific expenses:
Rett syndrome	$	5,559	$	3,444	$	17,135	$	8,261
Batten disease	136		1,832		1,382		4,756
Early Discovery	614		1,329		2,621		4,500
Unallocated internal expenses:
Personnel-related	5,449		4,512		15,978		13,787
Stock-based compensation	1,669		1,272		4,815		3,211
Manufacturing	3,111		3,220		9,856		9,014
Other (a)	703		676		2,585		2,041
Total research and development expenses	$	17,241	$	16,285	$	54,372	$	45,570
General and administrative specific expenses:
Personnel-related	$	2,248	$	2,016	$	6,944	$	6,081
Stock-based compensation	1,697		1,201		6,007		2,620
Professional and consultant fees	1,088		969		3,335		3,469
Office-related	619		635		1,871		1,870
Other (b)	905		1,074		3,270		2,408
Total general and administrative expenses	$	6,557	$	5,895	$	21,427	$	16,448
Other income(c)	2,845		1,963		10,183		5,463
Net loss	$	(20,953)	$	(20,217)	$	(65,616)	$	(55,630)
(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.