Segment Information

12 Months Ended

Sep. 30, 2025

Segment Reporting [Abstract]

Segment Information

SEGMENT INFORMATION

We operate in a single segment dedicated to the discovery, development, manufacturing and commercialization of RNAi therapeutics. The Company's RNAi therapeutics are comprised of siRNAs that function upstream of conventional medicines by potently silencing messenger RNA (“mRNA”) that encode for proteins implicated in the cause or pathway of disease, thus preventing them from being made. Consistent with our operational structure, our Chief Executive Officer (“CEO”), as the CODM, manages and allocates resources on a consolidated basis at the global corporate level. Our global research and development and technical operations and quality organizations are responsible for the discovery, development, and supply of products. Commercial efforts that coordinate the marketing, sales and distribution of these products are organized by geographic region and therapeutic area. All of these activities are supported by corporate staff functions. Managing and allocating resources at the corporate level enables our CEO to assess the overall level of resources available and how to best deploy these resources in line with our overarching long-term, corporate-wide strategic goals. The determination of a single segment is consistent with the consolidated financial information regularly reviewed by the CODM for the purposes of evaluating performance, forecasting future period financial results, allocating resources and setting incentive targets.

Consistent with our management reporting, results of our operations are reported on a consolidated basis for purposes of segment reporting. The CEO evaluates performance and decides how to allocate resources based on consolidated net loss that is reported on the consolidated statements of operations and comprehensive income (loss). The measure of segment assets is reported on the consolidated balance sheets as total assets. The CEO uses consolidated net loss to evaluate income generated from the Company’s business activities in deciding how to allocate company resources (such as pursuing clinical development or entering a strategic collaboration), monitoring budget versus actual results, and establishing management’s compensation. Please refer to the consolidated financial statements for further information related to these measures of segment performance. In addition, research and development and selling, general and administrative expenses are significant segment expenses regularly provided to the CEO with the following categories:

Research and Development

	Year Ended September 30,
	2025		2024		2023
	(in thousands)
Candidate costs	$	347,571	$	259,280	$	162,459
R&D discovery costs	66,788		74,150		55,586
Salaries	109,085		96,418		73,668
Facilities related	29,233		25,782		16,267
Total research and development expense, excluding non-cash expense	$	552,677	$	455,630	$	307,980
Stock compensation	32,582		33,586		34,332
Depreciation and amortization	21,900		16,654		10,876
Total research and development expense	$	607,159	$	505,870	$	353,188

General & Administrative

	Year Ended September 30,
	2025		2024		2023
	(in thousands)
Salaries	$	31,916	$	27,589	$	22,999
Professional, outside services, and other	53,589		24,733		20,720
Facilities related	5,625		4,116		3,415
Total general and administrative expense, excluding non-cash expense	$	91,130	$	56,438	$	47,134
Stock compensation	30,785		40,382		43,798
Depreciation/amortization	2,028		1,941		1,617
Total general and administrative expense	$	123,943	$	98,761	$	92,549