Neurogene exists to provide medicines to cure or substantially improve the lives of families and people living with neurologic impairment. The day to day difficulty of caring for someone with a devastating neurologic disorder is a heavy burden for families, and we imagine a world where families no longer have to hear the message that their rare disease has no treatment. As an employee of Neurogene, you will be helping to make this dream a reality, and part of a culture of people in a start-up environment on a mission to make the world a better place!
The Senior Director, Preclinical Development will be a leader and team player in a small company environment. A highly cross-functional and highly visible role, this person will play the lead role in developing and executing our non-clinical strategy critical to translate promising preclinical results supporting our clinical plans. This role will lead the filing of multiple investigational new drug applications and be part of the transformative shift the company is making as we move from the pre-clinical stage to IND enabling studies and to accelerate clinical development.
Primary Accountabilities, Duties, and Responsibilities
Independently plan and execute experiments for inclusion in regulatory submissions, including safety pharmacology and toxicology (including genotoxicity) and bridging studies.
Manage relationships and provide oversight of research conducted by CROs and other third parties
Provide oversight of sponsored research activities at academic partner laboratories
Analyze and evaluate experimental data and summarize findings and relevance to both project and company objectives
Ensure quality and rigor across nonclinical development programs both internally and at third parties
Drafting pre-clinical and safety sections for regulatory documents
Work closely within cross-functional teams to ensure all work is completed to high quality in a timely and scientifically rigorous manner
Support biomarker strategy for Neurogene’s portfolio, linking preclinical with clinical to enable robust translational science
Participate in business development activities including the assessment of novel technologies and programs
Essential Job Functions
Work Schedule The role will mostly involve standard working hours but flexibility is required to attend meetings and calls.
Travel The role will necessitate travel to meet vendors and attend conferences and meetings
Quality Responsibility A key aspect of the post is to ensure scientific rigor across preclinical activities and that regulatory standards are met for all IND enabling studies.
Job Scope This is a key role within the company and will provide primary responsibility for preclinical development. The role will involve working closely with the CSO as well as operational teams across the company.
Leadership Responsibility The role is to provide leadership in preclinical development and safety studies. The role is highly cross-functional and is also outward-facing. As well as managing vendors, the role will involve managing scientific staff internally as the development team grows.
Minimum Required Qualifications/Skills
Education PhD in Neuroscience, molecular genetics or a related field
Experience 5+ years of non-clinical R&D experience in biotech or pharma
Strong understanding of FDA and European nonclinical regulatory requirements with hands-on experience generating study reports and regulatory documentation supporting submissions
Knowledge of AAV gene therapy technologies as applied to neurological diseases
Experience in molecular biology including, designing and executing studies employing AAV vectors and the analysis of samples using PCR, qPCR, ELISA, western blot, immunolabeling, histological techniques, and microscopy
Experience in gene therapy preferred
Experience in modeling neurological diseases to assess therapeutic efficacy in gene therapy
Experience in designing safety and toxicology studies to support IND filings