SVP Regulatory Affairs

Place in the Organization

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.

Job Description

The SVP, Regulatory Affairs reports to the Chief Medical Officer (CMO) and joins an established regulatory organization that includes a VP of Regulatory Affairs based in Amsterdam and a capable team with deep institutional knowledge of uniQure’s programs. This role seeks to build upon the existing team’s strengths, adding senior strategic leadership, broadening global regulatory reach, and elevating the function’s influence at the executive and Board level. Within the CMO’s organization, the SVP operates as a strategic peer to the SVP, Clinical Development, jointly driving Clinical-Regulatory strategy, endpoint design, and development planning. Equally, the SVP builds deep enterprise partnerships with Medical Affairs, Market Access, and Commercial—functions within the Chief Customer and Strategy Officer’s (CCBO) organization—ensuring regulatory strategy, labeling, and evidence generation are aligned with commercial positioning and patient access goals. The SVP also partners with the Chief Scientific and Business Officer, CEO, and the broader executive team on uniQure’s global regulatory agenda.
The ideal candidate brings deep experience navigating complex regulatory environments for rare diseases and/or gene therapies, has successfully led at least one BLA or MAA through to marketing authorization, and brings credibility with the FDA’s CBER as well as international agencies. This individual must have the temperament to lead through regulatory ambiguity, a demonstrated ability to partner across organizational lines, and the breadth to weigh into regulatory and development pathways for earlier-stage research assets—not solely late-stage registration programs.

Key result areas (major duties, accountabilities and responsibilities)
Global Regulatory Strategy

• Develop, own, and execute the global regulatory strategy for AMT-130 and all pipeline programs—from research-stage assets through commercial products—including filings with the FDA, MHRA, EMA, and other international authorities.
• With CMO, lead all FDA/CBER interactions, including Type A, Type B, and pre-BLA meetings. Serve as the company’s senior regulatory voice in health authority engagements.
• Architect parallel international regulatory pathways, with immediate priority on the UK MHRA and EU/EMA strategies.
• Weigh into regulatory, research, and development pathways for earlier-stage pipeline assets (TLE, Fabry, future indications), shaping IND/CTA strategies and target product profiles from the outset.
• Advise the CEO, CMO, Board, and executive team on regulatory risk, strategy, and health authority feedback. Translate regulatory dynamics into clear strategic options for executive decision-making.

Label Strategy, Indication Expansion, and Cross-Organizational Partnership

• Partner with the SVP, Clinical Development on protocol design, endpoint selection, and evidence strategies that optimize both the probability of regulatory success and the breadth of the resulting product label.
• Build strong cross-organizational partnerships with Medical Affairs, Market Access, and Commercial within the CCSO’s organization, ensuring regulatory strategy, labeling, and health authority engagement are aligned with positioning and patient access goals.
• Provide regulatory input across the full portfolio lifecycle—from research-stage target validation through commercial lifecycle management—ensuring regulatory considerations inform go/no-go decisions and portfolio prioritization.
• Participate as a core member of the CMO’s leadership team and as a key cross-functional partner to the CCSO’s and CSBO’s organizations, ensuring regulatory strategy bridges research, development and commercial functions.

BLA/MAA Filing and Submissions

• Lead the preparation and submission of BLA, MAA, and other marketing applications for uniQure’s lead program, overseeing regulatory components of the eCTD/CTD in coordination with Clinical, CMC, Nonclinical, and Biostatistics.
• Manage regulatory aspects of external control methodology and other novel statistical approaches in collaboration with Biostatistics and external advisors.
• Ensure regulatory readiness for the anticipated Phase III program(s), including protocol design input, endpoint strategy, and adaptive design considerations.
• Manage post-submission interactions with health authorities, including information requests, advisory committee preparation, and approval negotiations.

International Regulatory and Commercial Regulatory Infrastructure

• Build a multi-jurisdiction filing strategy leveraging MHRA, EMA, and other approvals to enable market access in Saudi Arabia (SFDA), Gulf states, and other markets with formal reliance pathways.
• Partner with the CCSO’s organization to develop commercial regulatory infrastructure for product launch, including labeling strategy, post-marketing commitments, and lifecycle management.

Regulatory Policy and Intelligence

• Monitor and advise on the evolving global regulatory landscape for gene therapies and ATMPs, including FDA/CBER policy shifts and international harmonization developments.
Engage with industry consortia, regulatory policy groups, and advocacy organizations to shape the external regulatory environment for gene therapies in rare disease.

Qualifications & Skills
Essential Requirements

• Advanced degree (PhD, PharmD, MD, or equivalent) in a relevant scientific discipline.
• 15 years of progressive regulatory affairs experience in biopharmaceuticals, with at least 10 years in rare disease or gene therapy product development.
• Demonstrated experience leading at least one successful BLA or MAA submission through to marketing authorization for a biologic or gene therapy product.
• Direct experience interacting with FDA/CBER, including leading formal health authority meetings (Type A, Type B, pre-BLA/pre-MAA). Global filing experience with MHRA, EMA, or other major international agencies required.
• Experience providing regulatory strategy across the full development lifecycle, including earlier-stage research and preclinical assets, IND/CTA-enabling programs, and late-stage registration.
• Strong leadership capabilities with experience managing regulatory teams and elevating inherited teams. Exceptional communication skills with demonstrated ability to present to regulators, boards, and executive leadership.

Strongly Preferred

• Experience with external control or real-world evidence–based regulatory strategies.
• Prior experience with neurology or CNS-targeted therapies.
• Proven track record of building and sustaining productive partnerships across organizational boundaries, including with CMC, clinical, commercial, medical affairs, and market access functions outside the regulatory leader’s direct reporting line.
• Understanding of CMC regulatory requirements for gene therapy products, including vector manufacturing, potency assays, and comparability.
• Experience with regulatory aspects of surgical or procedure-based therapies (e.g., intracranial delivery, intrathecal administration).
• Prior experience in a publicly traded, rare disease–focused biotechnology company.
• Experience partnering with research and early development teams to shape regulatory strategy for preclinical and IND-stage programs across multiple modalities or indications.

Personal Attributes

• Resilient and composed under pressure: comfortable in regulatory ambiguity, with the ability to maintain strategic clarity and team morale through setbacks.
• Patient-centered: deeply motivated by the urgency of bringing disease-modifying therapies to patients with no treatment options.
• Strategically assertive: advocates firmly and constructively with regulatory agencies while maintaining productive relationships.
• Organizationally fluent: naturally builds trust across reporting lines, skilled at driving alignment with leaders in commercial, medical affairs, and market access functions outside their direct organization.
• Entrepreneurial: energized by the pace and impact of a mid-cap biotechnology company advancing multiple first-in-class programs.

Salary Range: $400,000 - $450,000

The estimated salary range reflects an anticipated range for this position. The actual base salary offered may depend on a variety of factors, including the qualifications of the individual applicant, relevant years of experience, specific and unique skills, level of education attained, certifications or other professional licenses held, and the location in which the applicant lives and/or from where they will be performing the job. The actual base salary offered will be in accordance with state or local minimum wage requirements for the job location.