Segment Information (Tables)

6 Months Ended

Jun. 30, 2025

Segment Reporting [Abstract]

Schedule of Reconciliation to Consolidated Operating Expenses

A reconciliation to consolidated operating expenses as our single segment operating loss for the three and six months ended June 30, 2025 and 2024 is included in the table below:


		Three Months Ended June 30,								Six Months Ended June 30,
		2025				2024				2025				2024
		(in thousands)								(in thousands)
AATD program		$	1,846			$	3,172			$	3,292			$	6,365
INHBE program			3,515				1,646				6,054				2,381
DMD program			2,681				4,051				5,719				7,101
HD program			789				3,636				1,956				6,015
Other research and development expenses(1), including RNA editing, PRISM, others			34,638				27,888				67,070				51,978
Total research and development expenses			43,469				40,393				84,091				73,840
General and administrative expenses			17,989				14,296				36,346				27,845
Total operating expenses		$	61,458			$	54,689			$	120,437			$	101,685

(1) Includes expenses related to other research and development programs, identification of potential drug discovery candidates, compensation-related expenses, internal manufacturing expenses, equipment repairs and maintenance expense, facility-related expenses, and other operating expenses, which are not allocated to specific programs.