ADDED
As of March 25, 2026, a total of 414,274,017 shares of common stock, $0.01 par value per share were outstanding.
Forward-looking statements may include, but are not limited to, statements about: our estimates regarding the sufficiency of our cash resources and our expenses, capital requirements and need for substantial additional financing, and our ability to obtain the substantial additional financing that we need to support our operations and to continue to operate as a going concern, including the possibility that at any time we may elect or may be required to cease operations entirely, liquidate all or a portion of our assets, and/or seek protection under the U.S.
Bankruptcy Code in the very near term; our expectations concerning the availability and adequacy of data and a clear regulatory pathway to support a potential Biologics License Application, or BLA, submission for isaralgagene civaparvovec and the anticipated timing and acceptance of such submission, and the potential for the BLA to be approved by the U.S.
These statements reflect our current views with respect to future events, are based on assumptions and involve known and unknown risks, uncertainties and other factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements.
We will need substantial additional funding in the very near term to execute our operating plan and to continue to operate as a going concern.
Bankruptcy Code in the very near term, and you may lose all or part of your investment.
If we are not able to find collaborators, or if our collaborators do not diligently pursue product development efforts, we will not be able to secure sufficient capital to continue to operate as a going concern.
In particular, we are engaged in early stage business development discussions with potential counterparties concerning a commercialization agreement for our Fabry disease program, but have been unsuccessful in consummating any such transaction to date.
There can be no assurance that we will be able to secure a commercialization partner for our Fabry disease program or partner or sell any other programs in a timely manner, on acceptable terms, or at all, and if we are unable to execute such an agreement providing us with significant upfront funding in the very near term, we will not be able to secure sufficient capital to continue to operate as a going concern.
Disruptions at the FDA, including due to a reduction in workforce and/or inadequate funding, could prevent the FDA from performing normal functions on which our business relies, which could negatively impact our business.
REMOVED
As of March 13, 2025, a total of 224,710,019 shares of common stock, $0.01 par value per share were outstanding.
These statements reflect our current views with respect to future events, are based on assumptions and are subject to risks and uncertainties.
4 Table of Conte nts SUMMARY OF RISK FACTORS Our business involves significant risks.
We need substantial additional funding to execute our operating plan and to continue to operate as a going concern.
If adequate funds are not available to us on a timely basis, or at all, we will be required to take additional actions to address our liquidity needs, including additional cost reduction measures such as further reducing operating expenses and delaying, reducing the scope of, discontinuing or altering our research and development activities, which would have a material adverse effect on our business and prospects, or we may be required to cease operations entirely, liquidate all or a portion of our assets, and/or seek protection under the U.S.
Bankruptcy Code, and you may lose all or part of your investment.
If we are not able to find collaborators, or if our collaborators do not diligently pursue product development efforts, we may not be able to secure sufficient capital to continue to operate as a going concern or to develop our technologies or product candidates.
Even if our product development efforts are successful and even if the requisite regulatory approvals are obtained, our products may not gain market acceptance among physicians, patients, healthcare payors and the medical community.
5 Table of Conte nts We have experienced and may continue to experience difficulties in hiring, integrating and retaining qualified skilled employees.
We have in the past, and may in the future, be unable to comply with the listing standards of the Nasdaq Stock Market LLC.