Senior Director, Clinical Development

uniQure•Lexington, KY

18h•$246,000 - $318,000

About The Position

At uniQure, we are delivering on the promise of gene therapy – and delivering hope for patients facing urgent unmet medical needs. Every role in our organization carries profound purpose; whether you're in research, operations, access, or support, your contributions impact patients. We're seeking passionate professionals who thrive in high-stakes environments, uphold rigorous quality standards, and share our relentless commitment to transforming the lives of patients. Join us in making the impossible possible, where your expertise becomes a catalyst for life-changing treatments. The Senior Director, Clinical Development, is responsible for providing clinical and medical oversight and guidance on gene therapy-based development programs at the clinical development stages. In collaboration with the VP and Clinical Development Leader, they cover a wide spectrum of responsibilities from formulation of clinical development strategies and study concept generation to design, implementation, execution of clinical studies, and submission to/approval from Health Authorities and Health technology assessments bodies in collaboration with medical Affairs and market access to ensure commercial success. He/she supports also the research organization in the Therapeutic Area of expertise. The Senior Director, Clinical Development will work closely with the Clinical Development leadership to support efforts for identifying current and future pipeline needs moving forward. They will also act as both the internal and external subject matter expert for Huntington’s Disease as well as other undisclosed programs. This position reports into the SVP of Clinical Development.

Requirements

MD with at least 7 years post qualification experience in clinical research area, in Pharmaceutical or Biotech company. Experience as Investigator in clinical trials is a plus
Proven record of successfully leading and managing teams
Deep knowledge of drug development and clinical trial process based on previous deliverables
Excellent knowledge of regulatory requirements/ICH guidelines
Demonstrated experience in working with several project teams into late-stage development and submission process
Experience in interactions with Health Authorities (FDA, CHMP/EMA, PMDA Japan)
Clinical research experience in at least one of the indications within the assigned Therapeutic Area is preferred
Sound knowledge of regulatory requirements/ICH guidelines
Good organizational, problem solving, interpersonal and communication skills
Hands-on and results driven attitude with a good sense of urgency
Experience negotiating with key stakeholders to influence, convince, and gain their buy-in and agreement
Thinks globally and understands the big picture (e.g., competitor activities, potential implications and consequences of own activities and decisions)
Excellent written and spoken communication skills in English
Proven ability to meet strict deadlines; manage competing priorities and changing demands
Sound organization and time management skills
Good attention to detail and accuracy whilst maintaining high quality GCP standards
Ability to be flexible and receptive to changing process demands
Willingness and aptitude to learn new skills, and support activities across service lines
Ability to establish and maintain effective working relationships with coworkers, managers and clients – many with diverse cultural backgrounds
Skill required to create buy-in from functional groups and departments
Great communication skills; comfortable representing the company and vision to internal and investor communities.
Confident, independent and decisive; a self-starter, proactive and results-oriented with high performance standards. Passionate, persistent and tenacious. Strong ability to prioritize and operate with a sense of urgency.
“Hands-on”, “roll-up-the-sleeves” leader who understands the need for involvement at all levels of activity within a small company environment. Extremely bright with a highly strategic nature but also balanced with a biotech mindset and an attitude that gets things done.
Understanding of and willingness to meet applicable regulatory, quality and compliance standards. Trustworthy with highest integrity, committed to ethics and scientific standards.
Willingness to travel (domestic/international, approximately 30% time) to oversee ongoing trials, meet investigators, attend conferences, and participate in advisory boards.

Responsibilities

Represents and defends the assigned programs in the matrixed Core Project Teams (with Project Management, Clinical Operations, Regulatory Affairs, Database Management & Programming, Biostatistics, Commercial, and Pre-Clinical)
In collaboration with the Clinical Development Leader, contribute to the design, implementation, execution, medical monitoring and reporting of requisite clinical studies at all phases of pipeline development (following applicable regulations SOPs and internal guidelines under guidance and support of senior operation team members)
Proactively identify program risks, and create and implement mitigation strategies
Provide medical expertise internally for all responsible project activities assigned, through a deep understanding of the science, medicine, product environment and competitive landscape relating to the disease area
Provide medical expertise externally based on thorough scientific review of relevant product data and consultation with internal and external experts
Oversee the execution of the clinical studies within the assigned project area
Organize and lead medical aspects of clinical advisory boards and study specific committees (steering committees, data monitoring committees, safety event committees) together with the clinical team
Collaborate cross-functionally in the preparation of the CDP, clinical protocols, investigator brochures, target product profiles, annual IND reports, severe adverse advent reports, periodic safety update reports, clinical study reports
Participate in the ongoing interpretation and analysis of clinical trial results including human pharmacokinetics, pharmacodynamics, laboratory, neuroimaging, and antibody data, review and approve clinical trial coding, safety monitoring in collaboration with pharmacovigilance expert
Participate in various scientific advice meetings with the EMA and FDA, or other Regulatory bodies
Continual review and analysis of the pertinent medical (disease-specific) literature
Supervise the preparation and/or delivery of abstracts, posters, and slide presentation at external Conference Symposia, and manuscripts for peer-reviewed journals
Collaboratively interface with the company’s Research department to bring a medical disease perspective and analysis of unmet need to new gene therapy product candidates (either in-licensed or internally developed) to optimally differentiate new gene therapy products, and to identify new opportunities beyond initial indication of AAV products
Focused medical review of emerging pre-clinical product candidates at Stage Gate #1, with design of an early high-level clinical development plan (CDP)
Collaborate with the commercial part of the organization as needed for perspective on disease incidence and prevalence of orphan indications and to ensure development programs are aligned with commercial expectations
With Business Development, participate as expert in Company due-diligence exercises on potential in-licensing of pipeline gene therapy programs