Scientist II​/Senior Scientist Nonclinical Development

Position Overview

Our company is seeking an experienced Nonclinical Development Scientist II/Senior Scientist with a deep understanding of gene therapy and regulatory agency requirements. The ideal candidate will have preclinical experience in the field of gene therapy and be able to lead the development of products from the research stage through to clinical trials. The role will involve working closely with cross-functional teams to ensure the successful development of gene therapy products that meet regulatory standards and effectively translate scientific discoveries into clinical practice.

Reporting to the VP of Therapeutics, this role will deliver strategic and hands-on nonclinical development, working closely with Platform Development, Bioanalytical, and CMC teams.

• Design, oversee, perform and interpret nonclinical studies (non-GLP and GLP), including proof-of-concepts, pharmacology, biodistribution, and toxicology studies within established timelines.
• Manage CROs and external partners.
• Write, review, and edit preclinical study reports and nonclinical sections of regulatory submissions.
• Partner with platform development scientists to inform candidate selection strategy.
• Develop PD biomarker strategy across programs and work closely with bioanalytical scientists to ensure efficient hands-off.
• Maintain up-to-date knowledge of regulatory guidance.
• Communicate effectively with Key Opinion Leader (KOLs) to maintain up to date understanding of the literature.
• Develop strategies for preclinical development, including the identification and management of risks and opportunities.

• Ph.D. or equivalent in medicinal chemistry, pharmacology, or related discipline, with a minimum of 5 years of related experience in biotechnology or pharmaceutical industry.
• Demonstrated experience working with regulatory agencies, including leading interactions with FDA, EMA, and/or other relevant agencies.
• Strong preclinical experience in gene therapy.
• Demonstrated ability to work with CRO and to collaborate with cross-functional teams and external partners, including regulatory agencies, academic institutions, and biotech companies.
• Bioanalytical experience is preferred.

• Understanding of gene therapy and CRISPR based technologies.
• Strong communication skills, with the ability to effectively present scientific data and communicate complex scientific concepts to a wide range of stakeholders.
• Strong analytical and problem-solving skills, with the ability to effectively identify and address complex scientific and regulatory issues.
• Ability to work in a fast-paced environment, manage multiple projects, and meet deadlines.