The Company has not generated revenue from product sales to date. KRESLADI™ received FDA accelerated approval in March 2026. The Company is currently advancing commercial launch activities, including Qualified Treatment Center onboarding, manufacturing readiness, and patient access initiatives. Commercial product revenue will depend on the timing of treatment center activation and patient treatment. KRESLADI™ was approved under the FDA's accelerated approval pathway based on an increase in neutrophil CD18 and CD11a surface expression. Continued approval may be contingent upon verification and description of clinical benefit through ongoing clinical follow-up and additional post-marketing data collection.

Operations of the Company are subject to certain risks and uncertainties, including, among others, uncertainty of drug candidate development, technological uncertainty, uncertainty regarding patents and proprietary rights, having limited commercial manufacturing, marketing and sales experience, dependency on key personnel, compliance with government regulations, and the need to obtain additional financing. Drug candidates currently under development will require significant additional research and development efforts, including extensive preclinical and clinical testing and regulatory approval, prior to commercialization. These efforts require significant amounts of additional capital, adequate personnel infrastructure, and extensive compliance-reporting capabilities. The commercialization of KRESLADI™ is subject to significant operational, logistical and reimbursement-related risks. The delivery of autologous gene therapies requires complex coordination, including treatment center onboarding, manufacturing, and vein-to-vein logistics, which may impact the timing and pace of commercial adoption.

The Company's product candidates are in the development and clinical stage. There can be no assurance that the Company's research and development efforts will be successfully completed, that adequate protection for the Company's intellectual property will be obtained, that any additional products developed will obtain necessary government approval, or that any approved products will be commercially successful. Even if the Company's product development efforts are successful, it is uncertain when, if ever, the Company will generate significant revenue from product sales. The Company operates in an environment of rapid technological change and substantial competition from pharmaceutical and biotechnology companies.

In July 2025, the Company implemented a strategic corporate reorganization designed to align its resources with its highest-priority programs, namely, the AAV-based genetic medicines platform focused on cardiovascular diseases. As part of this effort, the Company reduced its workforce by approximately 30%.

The Company's consolidated financial statements have been prepared on the basis of continuity of operations, realization of assets and the satisfaction of liabilities in the ordinary course of business. The Company has incurred recurring losses and negative cash flows from operations and had an accumulated deficit of $1.37 billion as of June 30, 2026. As of June 30, 2026, the Company had $283.7 million of cash, cash equivalents, and investments. In April 2026, the Company entered into an agreement to sell its PRV for $180 million, and the transaction closed in June 2026. The proceeds from the sale provided substantial non-dilutive capital, strengthened the Company's balance sheet, enhanced liquidity, and increased the Company's financial flexibility to execute on its focused cardiovascular genetic medicines strategy and other strategic priorities. The additional capital provides the Company with greater flexibility to advance its clinical, regulatory, manufacturing, and commercial priorities in support of its long-term strategic objectives. Based on the Company's current operating plan and its cash, cash equivalents, investments, including proceeds received from the PRV sale, the Company believes it has sufficient capital to fund operations into the second quarter of 2028.

In the longer term, the future viability of the Company is dependent on its ability to generate cash from operating activities or to raise additional capital to finance its operations. The Company expects to continue to generate operating losses for the foreseeable future and to finance its future cash needs through, but not limited to, one or a combination of equity offerings, debt financings, collaborations, strategic partnerships and alliances or licensing arrangements. If the Company is unable to obtain funding, the Company would be forced to delay, reduce or eliminate some or all of its R&D programs, preclinical and clinical testing or commercialization efforts, which could adversely affect its business prospects. The Company’s failure to raise capital as and when needed could have a negative impact on its financial condition and ability to pursue its business strategies.

The Company may also face challenges retaining key personnel and maintaining continuity across teams, which could impair its ability to advance clinical programs, meet regulatory milestones, or pursue long-term strategic objectives. Potential litigation or other employee-related claims arising from the workforce reduction could divert management attention and further increase costs. Any of these factors could have a material adverse effect on our business, operating results, and financial condition.