Segment Information (Tables)

3 Months Ended

Mar. 31, 2025

Segment Reporting [Abstract]

Schedule of Results Of Operations by Reportable Segment

As of March 31, 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 March 31,
	2025		2024
Program specific expenses:
Rett syndrome	$	4,524	$	2,019
Batten disease	845		1,190
Early Discovery	1,287		1,466
Unallocated internal expenses:
Personnel-related	5,404		4,718
Stock-based compensation	1,570		564
Manufacturing	3,107		2,979
Other (a)	1,023		605
Total research and development expenses	$	17,760	$	13,541
General and administrative specific expenses:
Personnel-related	$	2,484	$	2,101
Stock-based compensation	2,478		480
Professional and consultant fees	1,277		1,502
Office-related	632		604
Other (b)	1,288		551
Total general and administrative expenses	$	8,159	$	5,238
Other income (expense)(c)	3,272		1,858
Net loss	$	(22,647)	$	(16,921)
(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 (expense) included in net loss includes interest income, interest expense, other income and other expense.