Medical Director, Clinical Trial Lead

About the role

Place in the Organization uniQure is dedicated to bring innovative therapies or unmet medical needs to patients. In doing so each function in uniQure is directly or indirectly responsible for the lives of patients. This responsibility should be part of our day-to-day working life by (i) living up to high quality standards and (ii) by recognizing the sense of urgency in areas of high medical need. The Medical Director will lead and oversee clinical trials based in the US. The successful candidate will be responsible for leading clinical trials, engaging with Key Opinion Leaders (KOLs) and stakeholders, and ensuring the successful execution and monitoring of ongoing trial activities, including safety and efficacy assessments. This role requires a deep understanding of trial execution, with a strong background in clinical research and drug development, preferably in neurology or rare disease.

Responsibilities

• Lead and oversee clinical trials focused, ensuring adherence to regulatory and ethical standards.
• Collaborate with the clinical development team to support study design, implement, and monitor clinical trial protocols, with a focus on safety, efficacy, and regulatory compliance.
• Provide expert guidance on patient selection criteria, dosing regimens, and endpoints.
• Develop and maintain strong relationships with Key Opinion Leaders (KOLs), clinical investigators, study coordinators, and other relevant stakeholders.
• Serve as a key point of contact for external partners, including clinical sites and contract research organizations (CROs) in terms of medical questions related to study readouts and assessments.
• Represent the company at scientific conferences, patient events and advisory boards.
• Oversee the monitoring of trial activities, including patient safety, adverse event reporting, and efficacy assessments.
• Support the study team in the review and interpretation of clinical trial data, providing insights and recommendations