SVP Regulatory Affairs

uniQure•Lexington, KY

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. 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.

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.

Nice To Haves

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.

Responsibilities

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.
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.
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.
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.
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.