Preclinical Director, Biologics Engineering

About the CompanyThe mission of Neurogene is to treat devastating neurological diseases to improve the lives of patients and families impacted by these rare diseases. Neurogene is developing novel approaches and treatments to address the limitations of conventional gene therapy in central nervous system disorders. This includes selecting a delivery approach to maximize distribution to target tissues and designing products to maximize potency and purity for an optimized efficacy and safety profile. The Company's novel and proprietary EXACT transgene regulation platform technology allows for the delivery of therapeutic levels while limiting transgene toxicity associated with conventional gene therapy. Neurogene has constructed a state-of-the-art gene therapy manufacturing facility in Houston, Texas. CGMP production of NGN-401 was conducted in this facility and will support pivotal clinical development activities. This transformative work is driven by our highly collaborative people who come together each day to bring us closer to achieving our mission. As an employee of Neurogene, you will be part of building a culture that honors patient and caregiver mindsets and nurtures innovation, creative problem solving and a strong sense of purpose.About the RoleThis candidate will need to be a team player in a small company environment in this cross-functional role, leading the integration of modified rAAV into the Neurogene pipeline. Reporting to the Executive Director, Preclinical Development, this individual will be responsible for shaping and executing Neurogene's internal strategy for modifying AAV capsids to improve neuronal tropism. The ideal candidate will be a highly motivated and creative thinker, who is able to review and interpret scientific literature and use it to craft hypotheses that shape the research and development strategy at Neurogene. The ideal candidate will have direct experience designing rAAV capsid and vector genome variants and a knowledge of rAAV manufacturing processes. An understanding of receptor targeting, monoclonal antibody characterization, protein engineering and analytical assay development is highly valued. Ideally the candidate will have strong experience in immunogenicity profiling.ResponsibilitiesDesign the biological strategy and testing approach for novel BBB-penetrant rAAV capsidsDevelop methods to assess capsid properties including safety (immune response)Use knowledge gained through prior hands-on experience and/or review of relevant literature to develop robust scientific strategiesMaintain an up-to-date understanding of the novel AAV capsid and novel gene therapy delivery landscape through literature review and conference attendanceManage projects at contract research organizations and work collaboratively across internal preclinical, CMC and translational functionsQualificationsEducation PhD in biomedical sciences Experience 7-10 years of relevant R&D experience in biotech or pharmaRequired SkillsBackground in molecular biology, vectorology and protein binding assaysStrong knowledge of biologics manufacturingExperience in analytical assay developmentBackground in immunology is a plusExcellent interpersonal written and verbal communication skills for presentations, report writing, and day-to-day project team communicationsContinuous strong focus on meeting or exceeding expectations in executing deliverables, performance and results.Ability to track and measure performance against defined metrics.Self-starter with high degree of energy, independence, initiative, and self-motivation, as illustrated by the ability to instill a sense of pace and urgency into the team.Someone who moves fast and decisively in a balanced manner, with a passion for the growing company.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.

Created: 2025-01-20