GENERAL SUMMARY4D Molecular Therapeutics (4DMT), located in Emeryville, CA, is a clinical-stage biotechnology company developing targeted genetic medicines using its proprietary AAV vector platform to treat serious unmet medical conditions. The Vice President, Regulatory Affairs Strategy, reporting to the Chief Regulatory and Quality Officer, is the company's senior global regulatory strategist, accountable for designing and executing integrated US, EU, and international regulatory strategies that take 4DMT's programs from IND through BLA/MAA and approval in major markets. The role sets regulatory direction on program and executive teams, leads the company's most consequential interactions with FDA, EMA, and other health authorities, and leads the Regulatory Strategy team.
MAJOR DUTIES & RESPONSIBILITIES:- Develops and leads integrated global regulatory strategies (US, EU, UK, Japan, and other key markets) for 4DMT's AAV gene therapy programs from IND through BLA/MAA and approval, aligned with corporate objectives and supported by risk assessments, probability-of-success analyses, and contingency plans.
- Designs global development and submission sequencing, including CTA timing, BLA and MAA filing strategy, and use of FDA-EMA parallel scientific advice and other collaborative review mechanisms.
- Builds regulatory evidence strategies for novel endpoints and trial designs, including surrogate or intermediate endpoints, external controls, and real-world evidence where appropriate, consistent with FDA and EMA expectations for gene therapy.
- Serves as the regulatory lead on program and governance teams, translating regulatory requirements and precedent into development plans, target product profiles, and evidence-generation strategies.
- Identifies and pursues expedited and special regulatory pathways in each region, including Fast Track, RMAT, Breakthrough Therapy, Priority Review, Accelerated Approval, the Commissioner's National Priority Voucher program, PRIME, ILAP, SAKIGAKE, and orphan designations.
- Plans and leads health authority interactions, including pre-IND, INTERACT, Type A/B/C, End-of-Phase 2, and pre-BLA meetings, EMA scientific advice, and PMDA consultations; directs briefing document strategy and preparation, prepares project teams, and serves as a primary company contact with FDA (CBER/OTP), EMA, and other agencies.
- Leads regulatory strategy for, and oversees preparation of, major submissions, including INDs/CTAs and amendments, annual reports, DSURs, orphan and expedited program designation requests, pediatric plans (iPSP/PIP), and BLA/MAA, ensuring filings are accurate, high quality, and submitted on time.
- Partners with clinical, nonclinical, CMC, biostatistics, safety/pharmacovigilance, and medical affairs to define submission content and to ensure development plans are acceptable to regulators.
- In partnership with Regulatory CMC and Technical Operations, ensures regulatory strategy accounts for AAV-specific CMC topics, including potency assays, comparability, and manufacturing process changes.
- Shapes target product profiles and target labeling strategy across regions in partnership with Medical Affairs and Commercial.
- Provides regulatory strategy input to life cycle plans, including indication expansion, new populations, and post-approval commitments, in partnership with the Life Cycle Management team.
- Partners with Regulatory Operations on submission planning and timelines for global filings.
- Leads ex-US regulatory strategy and execution, adapting global plans to regional requirements (EMA/CAT, MHRA, PMDA, Health Canada, and others) and directing regional partners, CROs, and consultants.
- Partners with Quality to support Pre-Approval Inspection and BIMO readiness, ensuring submitted content is consistent with actual processes and data.
- Builds, leads, and develops the Regulatory Strategy team, including global regulatory leads, with responsibility for goal setting, budget, resourcing, and talent development.
- Educates and trains company employees on regulatory requirements, processes, and health authority expectations.
- Communicates regulatory strategies, risks, and probability of success to executive leadership, the Board as needed, cross-functional teams, and partners/collaborators; supports business development due diligence.
- Maintains current knowledge of the global regulatory environment for gene therapy (including FDA CBER/OTP and EMA CAT/ATMP frameworks), regulatory precedent, and the competitive landscape, and communicates implications to program teams and senior management.
- Shapes the external regulatory environment through engagement with industry groups, health authority workshops, and comments on draft guidance relevant to gene therapy.
- Proactively identifies regulatory risks and proposes solutions that keep development plans on time while maintaining the highest standards of quality and compliance.
QUALIFICATIONS: Education:
- S. degree in the life sciences or a related discipline required
- Advanced degree (Ph.D., J.D., PharmD, MBA, and/or M.S.) in the life sciences preferred
Experience:
- S. with 18+ years of experience in the biopharmaceutical or biotechnology industries, or 14+ years with an M.S. degree, or 10+ years with a PhD or PharmD, with the majority in Regulatory Affairs.
- 8+ years of people management experience leading regulatory strategy teams. An equivalent mix of education and experience will be considered.
- Experience within the viral vector gene therapy field required; AAV highly desirable. Direct experience with FDA CBER/OTP required.
- Senior responsibility for at least one successful BLA or MAA (gene therapy, cell therapy, or biologic preferred), including direct ownership of global (US and EU) regulatory strategy through approval.
- Demonstrated success negotiating novel development plans, endpoints, or expedited pathways with FDA CBER/OTP and EMA.
- Track record leading EU regulatory strategy, including EMA scientific advice, PRIME or CAT interactions, and centralized MAA procedures; Japan or other international experience preferred.
- Experience obtaining expedited program designations (e.g., RMAT, Breakthrough Therapy, Fast Track, PRIME, orphan).
- Ophthalmology or pulmonology experience a plus.
Qualifications/Skills:
- Expert knowledge of global regulations and guidance (FDA, EMA, ICH, MHRA, PMDA) and how they shape an integrated global regulatory strategy.
- Proven ability to develop and execute global regulatory strategies that deliver approvals.
- Strong track record of collaborative working relationships with FDA, EMA, and other health authorities that have led to successful outcomes for the business.
- Ability to work within a team environment, report on projects and drive them to conclusions.
- Self-starter who works with a sense of urgency and acts as a good team player working with other disciplines.
- Adaptability, flexibility, independence and resourcefulness to roll-up-sleeves and multi-task to thrive in small company environment.
- Ability to proactively identify challenges, possess strategic foresight and implement appropriate course of action
- Must have thorough knowledge of drug development and regulatory submission processes.
- Excellent scientific and regulatory writing skills, including briefing documents and responses to health authority questions.
- Demonstrated experience preparing and filing FDA and EMA submissions, including a BLA or MAA, required.
- Excellent organizational, communication and time management skills are required
- Must be able to innovate, analyze and solve problems with minimal supervision and strong attention to detail.
Travel: 15-25%, including international travel. Hybrid or on-site role, expected to be on site in Emeryville, CA at least three times a month.
Physical Requirements and Working Conditions:
- Physical Activity - Repetitive motions: Making substantial movements (motions) of the wrists, hands, and/or fingers. Fingering: Picking, pinching, typing or otherwise working, primarily with fingers rather than with whole hand or arm as in handling. Talking: Expressing or exchanging ideas by means of the spoken word.
- Physical Requirements - Sedentary work: Exerting up to 10 pounds of force occasionally and/or a negligible amount of force frequently or constantly to lift, carry, push, pull or otherwise move objects, including the human body. Sedentary work involves sitting most of the time. Jobs are sedentary if walking and standing are required only occasionally, and all other sedentary criteria are met.
- Working Conditions - The worker is not substantially exposed to adverse environmental conditions (as in typical office or administrative work).
Base salary compensation range: $326,000 - $381,000
Please note, the base salary compensation range and actual salary offered to the final candidate depends on various factors: candidate's geographical location, relevant work experience, skills, and years of experience.
