Background
Neurogene exists to provide medicines to 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!

Role Purpose: The applicant will be a team player in a small company environment in this highly cross-functional role. Reporting to the Executive Director of Preclinical Development, this person will assist in developing and executing our non-clinical strategy. This role will collaborate across the company and support 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

• Assist with the development and execution of experiments for inclusion in regulatory submissions, including safety pharmacology and toxicology (including genotoxicity) and bridging efficacy studies
• Contribute specific in vivo experimental designs and test conditions based on knowledge gained through prior hands-on experience and/or review of relevant technical literature
• 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 with cross-functional teams to ensure all work is completed to high quality in a timely and scientifically rigorous manner

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 may include 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 supportive role within the company and will provide responsibility for preclinical development. The role will involve working closely with the Executive Director of Preclinical Development as well as operational teams across the company.
• Leadership Responsibility The role is to provide support in preclinical development and safety studies in a highly cross functional manner. As well as working with or managing vendors, the role will involve coordinating projects with the internal cross-functional team and assistance with regulatory documents.

  Minimum Required Qualifications/Skills

• Education: PhD in Neuroscience, molecular genetics or a related field
• Experience: 2-5 years of non-clinical R&D experience in biotech or pharma
• Knowledge
  • Strong understanding of biomolecular assay development and validation requirements
  • Knowledge of AAV gene therapy technologies as applied to neurological diseases
• Skills
  • Experience in working with preclinical animal models of disease and designing studies to evaluate safety and efficacy of novel drug products
  • 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 assay development and validation to support FDA filings is a benefit
  • Experience in gene therapy preferred
  • Knowledge of biostatistics to evaluate data from preclinical model efficacy studies and assays is a benefit
  • Excellent interpersonal communication and written skills for presentations, report writing, and day-to-day project team communications