Description
Position Overview
- Work Location: Remote with ~25-30% travel
- Reports To (Position): VP, Medical Affairs
Neurogene is seeking a highly collaborative Executive Director, Evidence Generation and Scientific Communications to lead the generation and execution of the comprehensive evidence that will support NGN-401 and our broader pipeline. This is a critical and highly visible role at a pivotal time for the company. This leader will identify the evidence gaps that matter most to clinicians, multidisciplinary care teams, patients, caregivers, and payers, and turn them into a clear, prioritized plan. Working closely with Clinical Development, Biostatistics, Regulatory, HEOR/Market Access, Medical Affairs and leading external experts, this individual will drive that plan from concept through execution. Beyond evidence generation, this leader will oversee execution of our publication and congress plan, and manuscripts, and will serve as a scientific subject matter expert in the Medical, Legal, and Regulatory (MLR) review process, and support broader scientific communication priorities across Medical Affairs.
This role offers the opportunity to shape how the data for a potentially transformative gene therapy are generated, interpreted, and shared with the scientific community. We are looking for someone who brings scientific rigor, sound judgment, and the ability to influence across functions.
Experience in rare disease, pediatric neurology, genetic medicine, and/or gene therapy is highly valued. We are equally interested in exceptional candidates who have led evidence generation and publication programs in complex specialty or rare disease settings.
In rare disease, every data point matters. The quality of the evidence we generate, and how clearly and credibly we communicate it, will directly shape our ability to serve patients and families.
Requirements
Accountabilities and Responsibilities
Evidence Generation Strategy
• Lead development of the integrated Medical Affairs evidence generation plan for NGN-401 and pipeline programs, aligned with medical, clinical development, and corporate priorities.
• Collaborate with external experts, investigators, and cross-functional partners (including Clinical Development, Biostatistics, Regulatory, HEOR/Market Access, and Field Medical) to understand the evidence needs of the scientific and clinical community.
• Identify and prioritize key evidence gaps across the needs of clinicians, multidisciplinary care teams, patients, caregivers, and payers.
• Translate evidence gaps into a clear, prioritized roadmap based on scientific impact, strategic relevance, feasibility, and resources.
• Partner with Medical Affairs leadership to ensure the evidence plan is integrated with medical strategy, HCP engagement, and communication priorities.
• Provide medical input into endpoints, patient populations, and exploratory analyses that are meaningful to clinical practice. Continuously evaluate evidence priorities as the clinical, scientific, and treatment landscape evolves.
Evidence Generation Execution
• Drive delivery of the integrated evidence generation plan from concept through completion, in partnership with Clinical Development, Biostatistics, and Clinical Operations, who lead study design and operational execution. Initiatives may include post-hoc and exploratory analyses, natural history and registry studies, real-world evidence, caregiver-reported outcomes and burden-of-care research, and investigator-sponsored research.
• Shape study concepts and research questions and provide medical and scientific input into protocols and statistical analysis plans so that studies address the priority evidence gaps.
• Track progress against the evidence plan, milestones, budget, and report status, risks, and decisions to Medical Affairs leadership and cross-functional governance. Manage the medical communications agency and medical writers, including scopes of work, timelines, and budgets, and oversee development of abstracts, posters, oral presentations, and manuscripts to ensure timely, high-quality submissions.
Cross-Functional and External Expert Partnership
• Serve as a trusted Medical Affairs partner to Clinical Development, Biostatistics, Regulatory, HEOR/Market Access, Clinical Operations, Patient Advocacy, and Commercial.
• Build and maintain strong scientific relationships with investigators, academic collaborators, and key external experts.
• Lead or support scientific advisory boards, steering committees, and expert panels that inform evidence priorities and scientific communication needs.
• Partner with Field Medical to translate field and KOL insights into evidence generation priorities.
• Monitor the scientific and competitive landscape and assess implications for evidence priorities.
Publications and Congress Execution
• Oversee execution of the publication and congress plan, ensuring abstracts, posters, oral presentations, encores, and manuscripts are scientifically accurate and delivered on time.
• Partner with external authors and the medical communications agency/medical writers to manage drafts, data verification, author review, and approvals.
• Provide direct scientific input and review on publication and congress materials.
• Ensure compliance with Good Publication Practice (GPP), ICMJE recommendations, and Neurogene publication policies.
• Represent Neurogene at scientific congresses and external meetings as appropriate.
Scientific Communications and Medical Review
• Serve as a scientific subject matter expert in the MLR review process, ensuring materials are scientifically accurate, fair balanced, and consistent with the evidence.
• Contribute to the scientific platform, core narrative, and lexicon, and ensure new data are reflected consistently across scientific communications.
• Support other scientific communication priorities, including medical information, field medical resources, congress materials, and educational content.
• Ensure consistency between emerging evidence, publications, and external scientific exchange.
Operational Excellence and Compliance
• Help build the processes, tools, and governance needed to support evidence generation and publications as Neurogene grows.
• Manage evidence generation and publication resources and budgets responsibly.
• Establish appropriate metrics to track delivery against milestones, timelines, and budgets.
• Ensure all activities are conducted in accordance with applicable laws, regulations, industry standards, and Neurogene policies.
• Contribute broadly as a leader within Medical Affairs, supporting priorities where responsibilities may extend beyond traditional functional boundaries.
Minimum Requirements
Education
• Advanced scientific or clinical degree (PharmD, PhD, MD/DO) required
Experience
• 10+ years of biopharmaceutical industry, including significant experience in Medical Affairs, evidence generation, and/or scientific communications
• Demonstrated experience identifying evidence gaps and translating them into executable evidence generation plans
• Hands-on experience shaping and delivering a broad range of evidence generation activities, such as clinical trial and post-hoc analyses, natural history, registry, and realworld evidence studies, patient- and caregiver-reported outcomes research, and investigator-sponsored or collaborative research
• Proven track record managing publication and congress plans, including abstracts,
posters, oral presentations, and manuscripts, with direct agency and author management
• Working knowledge of GPP, ICMJE, and compliance requirements for scientific
exchange and publications
• Experience in rare disease, neurology, and/or pediatric disease, ideally within gene
therapy, genetic medicine, or another advanced therapeutic modality
• Pre-launch and/or launch experience required
• Experience in a small or emerging biotech, with comfort operating as both a strategic
leader and a hands-on contributor
• Willingness to travel to support scientific congresses, advisory boards, and other
external engagements
Knowledge, Skills and Attributes
• Ability to critically interpret clinical and statistical data and translate findings into clear,
clinically meaningful insights.
• Ability to understand and articulate complex medical and scientific information to
internal and external stakeholders and tailor information to specific audiences.
• Outstanding scientific writing, presentation, and oral communication skills.
• Exceptional attention to detail and project management skills, with a track record of
delivering high-quality work against firm deadlines.
• Ability to identify, cultivate, and maintain key external relationships with investigators
and external experts.
• Demonstrated ability to influence without authority and work effectively across
functions and levels of the organization.
• Ability to manage competing priorities and projects.
• Thrive in a fast-paced, innovative environment while remaining agile and adaptive.
• Flexibility to learn new skills, work without having the complete picture, and handle
uncertainty.
• Ability to maintain the highest degree of confidentiality and integrity; uphold the
company's high ethics and professionalism.
• Adhere to corporate compliance policies in all activities, including those related to
clinical trials, publications, scientific interactions with internal and external groups, and
unsolicited requests for medical/scientific information.
• Self-starter with a high degree of energy, independence, initiative, and self-motivation.
• Passionate, collaborative, creative, willing to take prudent risks, and motivated by the
creation of a company focused on transformative treatments for patients and their
families.
This job description has been developed based upon the expected and current duties, responsibilities and requirements for the position. Because job requirements evolve with the changing needs of Neurogene's business, this description is subject to change and may be modified at any time, whether formally or informally.
