Director, Clinical Translation

Company: Uniqure NV
Location: Lexington
Posted on: April 8, 2021

Job Description:

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 Director, Clinical Translation will serve a key role of providing expert medical input to the company's ongoing portfolio of gene therapy-based rare diseases programs with a focus over pre-clinical gene therapy programs in neurologic and other disorders. This will include the ongoing assessment of clinical study design, administration procedures, clinical endpoints, and imaging/biofluid biomarkers applicable to disease modifying treatments across therapeutic areas and treatment targets where uniQure is developing gene therapy products (either in-licensed or internally developed). This assessment will form the basis for clinical feedback to the pre-clinical pipeline. The Director, Clinical Translation will report to the Vice President, Clinical Development, and collaborate closely with Research, Business Development/Commercial, and Medical Affairs to address future pipeline needs for uniQure moving forward. Responsibilities:

• Serve as the clinical translation expert in relation to assigned programs in the matrixed pre-clinical Project Teams (with Project Management, Research, Clinical Development, Regulatory Affairs, Medical Affairs, Business Development/Commercial)
• Develop and maintain assessments of clinical study design, administration procedures, clinical endpoints, and imaging/biofluid biomarkers applicable to disease modifying treatments across therapeutic areas and treatment targets
• Proactively identify potential clinical development program risks based upon landscape assessment and research findings and collaborate in development of mitigation strategies
• Collaborate with Early Development and handle day-to-day clinical translation questions from various internal stakeholders
• Collaborate with the Clinical Development Group and lead development of early Clinical Development Plans (CDPs) and Phase I/II protocol synopses for assigned pre-clinical programs
• Collaborate cross-functionally in achieving successful pre-IND regulatory engagements to address clinical translation considerations and driving preparation and submission of the IND
• Collaborate with Clinical Development and Clinical Operations in developing phase I/II protocols and with Research in development of initial Investigator Brochures
• Collaborate with the Business Development/Commercial part of the organization to provide clinical perspective on disease incidence and prevalence, identify unmet needs and therapeutic opportunities, and develop target product profiles aligned to the evolving treatment landscape to ensure development programs are aligned with commercial expectations
• Participate as a clinical translation expert in Company due-diligence exercises on potential in-licensing of pipeline gene therapy programs
• Serve as a liaison with external clinical experts, professional/patient organizations, and regulatory science consortium (eg. C-PATH) related to assigned pre-clinical programs
• Collaborate cross-functionally and provide clinical leadership for advisory board, virtual focus group and 1:1 engagement to generate insights on clinical translation The successful candidate we hire will be a sophisticated clinician/researcher who has the right degree of drive and independence with a very strong track record of accomplishments in the pharmaceutical industry. S/he will have the following mix of personal and professional characteristics:
  • Education/research (MD, MD/PhD, or PhD) and at least 5 years of industry experience required; Experience in gene therapy or neuroscience a plus.
  • Strong scientific background including both basic science research and clinical aspects of delivery of medical care; knowledge and experience should include clinical trial design, endpoints, and biomarkers and specific considerations associated with specialty biologics or disease modifying treatments and rare disorders
  • Experience collaborating cross-functionally in the assessment and optimization of product development, including generation of clinical insights on clinical translation of pre-clinical data, literature assessments, and direct engagement with healthcare professionals, societies/organizations, and patients/caregivers
  • Hands on experience and willingness to develop/update fully referenced white papers and slide libraries summarizing clinical translation issues
  • Hands on experience and willingness to engage with researchers, clinical experts, societies/organizations, and patients/caregivers to generate insights into clinical translation
  • Hands on experience leading discussions with clinical experts through live and virtual advisory boards and focus groups including developing agendas and presentations/materials
  • Proven ability to meet strict deadlines; manage competing priorities and changing demands
  • Sound organization and time management skills.
  • Good attention to detail and accuracy
  • Ability to follow instructions/guidelines, work independently and proactively on own initiative, and keep management/stakeholders informed.
  • 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
  • Outstanding communication skills; comfortable representing the company and vision to internal and external 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" contributor 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 15-25% time) to attend conferences, meet clinical experts, and co-lead/participate in advisory boards.

Keywords: Uniqure NV, Huntsville , Director, Clinical Translation, Healthcare , Lexington, Alabama