Climate Change Data

Environmental Metrics

Climate Goals & Targets

Environmental Challenges

• Uncertain and costly research and development process.
• Risks and uncertainties related to macroeconomic factors, including ongoing overseas conflicts, disruptions in access to bank deposits and lending commitments due to bank failure, on the global business environment, healthcare systems and business and operations of us and our collaborators, including the initiation and operation of clinical trials; the research and development process; the uncertain timing and unpredictable results of clinical trials, including whether preliminary or initial clinical trial data will be representative of final clinical trial data and whether final clinical trial data will validate the safety, efficacy and durability of product candidates; the impacts of clinical trial delays, pauses and holds on clinical trial timelines and commercialization of product candidates; the unpredictable regulatory approval process for product candidates across multiple regulatory authorities; the manufacturing of products, product candidates and capsids; the commercialization of approved products; the potential for technological developments that obviate technologies used by us and our collaborators; the potential for us or our collaborators to breach or terminate collaboration agreements; the potential for us to fail to realize our expected benefits of our collaborations; the uncertainty of our future capital requirements, financial performance and results, our lack of capital resources to fully develop, obtain regulatory approval for and commercialize our product candidates, including our ability to secure collaboration for some of our programs, our ability to secure the funding required to advance our preclinical programs and/or initiate a potential registrational trial of isaralgagene civaparvovec in a timely manner or at all; and our need for substantial additional funding to execute our operating plan and to operate as a going concern, including the risk we will be unable to obtain the funding necessary to advance our preclinical and clinical programs and to otherwise operate as a going concern in which case we may be required to cease operations entirely, liquidate all or portion of our assets and/or seek protection under applicable bankruptcy laws middle all or a portion of out.

Mitigation Strategies

• Focused resources and reduced OpEx by ~50% year-on-year.
• Expect to reduce Non-GAAP OpEx to under $105M in 2025 as we transition our legacy programs.

Supply Chain Management

Climate-Related Risks & Opportunities

Environmental Metrics

Climate Goals & Targets

Supply Chain Management

Climate-Related Risks & Opportunities