v3.26.1
Segment Information (Tables)
 3 Months Ended
Mar. 31, 2026
Segment Reporting [Abstract]  
Schedule of Results of Operations by Reportable Segment
As of March 31, 2026 and 2025, 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,
20262025
Program specific expenses:
Rett syndrome$11,037 $4,524 
Batten disease178 845 
Early Discovery613 1,287 
Unallocated internal expenses:
Personnel-related6,456 5,404 
Stock-based compensation1,791 1,570 
Manufacturing4,197 3,107 
Other (a)
878 1,023 
Total research and development expenses$25,150 $17,760 
General and administrative specific expenses:
Personnel-related$2,626 $2,484 
Stock-based compensation1,838 2,478 
Professional and consultant fees1,402 1,277 
Office-related610 632 
Other (b)
1,723 1,288 
Total general and administrative expenses$8,199 $8,159 
Other income(c)
2,415 3,272 
Net loss$(30,934)$(22,647)
(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.