VP, Biometrics

uniQure•Lexington, KY

13h•Onsite

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. Reporting to the SVP, Head of Clinical Development, the VP, Biometrics will provide executive leadership for Biostatistics, Statistical Programming, and Data Management across the portfolio. This role will be responsible for the strategic direction, operational execution, and regulatory compliance of all clinical data activities across biostatistics, programming, and data management. They are responsible for building and guiding a multi-disciplinary team, providing strategic and operational leadership for all statistical and data functions within the Clinical Development organization. The VP, Biometrics, joins an established Biostats organization that includes a Senior Director, Biostatistics based in Lexington, MA and a capable team of internal and external experts with deep knowledge of uniQure’s programs. This role should build upon the existing team’s strengths, adding senior strategic leadership, combining data management with statistical programing and biostats, and elevating the function’s influence within the Clinical Development and the CMO Organization. The ideal candidate brings experience navigating complex environments for rare diseases and/or gene or cell therapies, has successfully been involved in Regulatory Agency interactions. This individual must have a demonstrated ability to partner across organizational lines, and the expertise breadth to contribute to development pathways for earlier-stage research assets as well as late-stage registration programs.

Requirements

PhD in Biostatistics or related quantitative field preferred.
15+ years of experience in biometrics/biostatistics within a biotech or pharma environment.
Proven track record in managing CROs and supporting regulatory submissions.

Nice To Haves

Experience with external control or real-world evidence–based regulatory strategies, including propensity score matching and natural history comparators.
Prior experience with neurology or CNS-targeted therapies.
Proven track record of building and sustaining productive partnerships across organizational boundaries, including with clinical development, and medical affairs

Responsibilities

Develop, implement, and advance the company’s statistics, programming, Real World Evidence (RWE), and Clinical Data Management capabilities.
Lead the statistical design and execution of clinical trials across the various stages of development, and registrational strategies.
Drive innovation in statistical methods, automation, and analytics to improve decision-making and execution speed.
Shape and evaluate clinical protocols including the preparation of statistical considerations sections, sample size calculations, interim looks, randomizations, and study design recommendations.
Establish and enforce governance for data standards, data quality, validation, and submission readiness.
Direct the biometrics strategy for regulatory submissions (INDs, NDAs, BLAs) and participate with health authority interactions (FDA, EMA, MHRA)
Navigate and resolve all statistical and data questions during regulatory review processes
Oversee Contract Research Organizations (CROs) and external vendors, establishing KPIs, ensuring quality deliverables and cost optimization aligning with internal standards and program needs.
Build, mentor, and manage a high-performing internal team, promoting a culture of excellence and continuous learning.
Serve as a core representative in cross-functional meetings, aligning biostatistics, programming, and data strategy with clinical development, regulatory affairs, and quality assurance.