uniQure(QURE) - 2022 Q4 - Annual Report

Financial Milestones - The company reported a significant milestone payment of $75.0 million from CSL Behring upon the first sale of HEMGENIXÔ in the European Union prior to July 2, 2023[14]. - The company may need to raise additional funding, which could be affected by a$75.0 million milestone payment related to HEMGENIXÔ sales in the EU[14]. Clinical Trials and Product Development - The company anticipates continued reliance on third parties for conducting clinical trials, which may impact timelines and regulatory compliance[14]. - The company has encountered delays in clinical trials and may continue to face challenges in demonstrating the safety and efficacy of its product candidates[14]. - The company may not successfully build a pipeline of additional product candidates using its gene therapy technology platform[14]. - The company is focused on building a pipeline of additional product candidates using its gene therapy technology platform[14]. - The company is reliant on third parties for conducting clinical trials, which may not perform satisfactorily or meet regulatory requirements[14]. Regulatory and Compliance Risks - The company is subject to significant government regulations regarding its manufacturing facility, and failure to comply could harm its business[14]. - The company must comply with complex data protection laws, and failure to do so may result in penalties[14]. - The company is exposed to risks associated with data protection laws across different jurisdictions, with potential penalties for non-compliance[14]. Operational Challenges - The ongoing Covid pandemic has materially affected the company's operations and supply chain[15]. - The ongoing Covid pandemic has materially affected the company's business, operations, and supply chain[15]. - The company faces risks related to competition, pricing, and reimbursement for its product candidates, which could adversely affect revenue[14]. - The company faces competition and challenges in employee recruitment and retention, which could adversely affect its business[14]. Internal Controls and Security - The company emphasizes the importance of maintaining effective internal controls to ensure accurate reporting and prevent fraud[15]. - The company’s internal computer systems may suffer security breaches or disruptions, impacting product development programs[14]. Funding and Intellectual Property - The company may need to raise additional funding, which may not be available on acceptable terms, potentially impacting product development efforts[14]. - The company relies on licenses of intellectual property from third parties, which may not be available in the future on commercially reasonable terms[14]. - The company relies on licenses of intellectual property from third parties, which may not be available on commercially reasonable terms[14].