Scientist II/Senior Scientist, Nonclinical Development (Gene Therapy)

South San Francisco, CA

Our client is an early-stage biotech in the San Francisco Bay Area developing a next-generation gene regulation platform to enable safe and durable control of targeted gene expression across multiple therapeutic areas. They are seeking a Senior Scientist (or Sci II) with deep gene therapy experience to lead programs from research through IND-enabling studies and into early clinical development. This role owns the design and execution of in vivo nonclinical studies, works closely with internal platform, bioanalytical, and CMC teams, and interfaces with external partners and regulators. Reporting to the VP of Therapeutics, you will drive both strategic planning and hands-on execution of nonclinical activities for a growing pipeline.

Responsibilities

• Design, oversee, and interpret in vivo nonclinical studies (non-GLP and GLP), including proof‑of‑concept, pharmacology, biodistribution, and toxicology studies, within agreed timelines.
• Lead and manage CROs and other external partners, including protocol development, study monitoring, data review, and issue resolution.
• Write, review, and edit preclinical study protocols, reports, and nonclinical sections of regulatory submissions (e.g., IND/CTA briefing packages, meeting materials).
• Partner with platform scientists to inform candidate selection, dose and schedule, and translational strategy for new programs.
• Develop and implement pharmacodynamic biomarker strategies across programs and work closely with bioanalytical teams to ensure robust assay support and sample handling.
• Maintain current knowledge of relevant regulatory guidance and industry best practices in nonclinical development of gene therapies and advanced modalities.
• Engage with external experts and Key Opinion Leaders (KOLs) to stay aligned with evolving science, safety considerations, and translational approaches in the field.
• Identify key nonclinical risks and opportunities for each program and propose clear mitigation and development strategies.

Qualifications

• Ph.D. or equivalent in pharmacology, toxicology, medicinal chemistry, molecular biology, or a related discipline.
• Minimum 5 years of relevant biotech or pharmaceutical industry experience in nonclinical / preclinical development, with a strong focus on gene therapy or genetic medicines.
• Demonstrated experience leading nonclinical studies (in vivo pharmacology, biodistribution, toxicology) and working effectively with CROs.
• Proven track record of direct interactions with health authorities (e.g., FDA, EMA), including preparation of nonclinical content for regulatory submissions and participation in meetings.
• Strong understanding of gene therapy concepts and CRISPR or other gene regulation/editing technologies.
• Excellent written and verbal communication skills, with the ability to present complex data and recommendations to technical and non‑technical stakeholders.
• Ability to operate in a fast‑paced, resource‑lean environment, manage multiple projects, and drive execution to timelines.

Preferred Skills

• Bioanalytical experience supporting gene or cell therapy programs (e.g., assay strategy for biodistribution, immunogenicity, PD biomarkers).
• Experience contributing to IND/CTA filings and early‑phase clinical trial start-up for gene therapy or nucleic acid-based therapeutics.
• Familiarity with GLP, ICH, and other nonclinical regulatory guidelines relevant to advanced therapies.
• Proven success working in small or emerging biotech environments with cross‑functional teams (e.g., CMC, clinical, regulatory, discovery).