v3.26.1
Segment Information (Tables)
 6 Months Ended
Jun. 30, 2026
Segment Reporting [Abstract]  
Schedule of Results of Operations by Reportable Segment
As of June 30, 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 June 30,Six Months Ended
June 30,
2026202520262025
Program specific expenses:
Rett syndrome$9,890 $7,052 $20,927 $11,576 
Batten disease60 401 238 1,246 
Early Discovery675 720 1,288 2,007 
Unallocated internal expenses:
Personnel-related5,990 5,125 12,446 10,529 
Stock-based compensation2,688 1,575 4,479 3,146 
Manufacturing4,876 3,635 9,073 6,742 
Other (a)
1,289 858 2,167 1,885 
Total research and development expenses$25,468 $19,366 $50,618 $37,131 
General and administrative specific expenses:
Personnel-related$2,785 $2,212 $5,411 $4,696 
Stock-based compensation5,172 1,833 7,010 4,310 
Professional and consultant fees1,233 970 2,635 2,247 
Office-related496 620 1,106 1,252 
Other (b)
1,516 1,080 3,239 2,364 
Total general and administrative expenses$11,202 $6,715 $19,401 $14,869 
Other income(c)
2,182 4,065 4,597 7,337 
Net loss$(34,488)$(22,016)$(65,422)$(44,663)
(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.