Associate Director, Pharmacology & Toxicology

About the role

The Associate Director, Pharmacology & Toxicology will lead the design and execution of non‑clinical development programs for in‑vivo gene‑editing therapeutics. This senior position is responsible for shaping strategy, overseeing CRO‑managed studies, and ensuring regulatory compliance across NMPA, FDA, and EMA.

Key responsibilities

• Develop and lead non‑clinical pharmacology, pharmacokinetics, and toxicology study strategies for gene‑editing pipelines (liver, ophthalmology, CNS, etc.).
• Oversee end‑to‑end execution of GLP and non‑GLP outsourced programs, including protocol review, timeline tracking, budget control, and final report delivery.
• Apply innovative models (humanized mouse, non‑human primate) to assess distribution, persistence, and editing efficiency.
• Lead off‑target risk assessment by integrating bioinformatics predictions with high‑depth NGS validation.
• Design and interpret toxicology studies addressing CRISPR/Cas immunogenicity, chromosomal translocations, and insertional mutagenesis.
• Establish biomarker and gene‑editing efficiency assessment systems (Indel rates, base conversion rates).
• Draft and review non‑clinical modules for IND/BLA submissions and serve as the primary liaison with regulatory agencies.
• Guide and develop the pharmacology & toxicology team, fostering cross‑functional collaboration with Discovery, CMC, Clinical, and Regulatory Affairs.

Required profile

• Master’s or PhD in Pharmacology, Toxicology, Molecular Biology, Veterinary Medicine or related field.
• 8–12 years of non‑clinical R&D experience, including at least 3 years in a management role.
• Successful full IND submission experience.
• Prior experience with in‑vivo gene‑editing, mRNA‑LNP, AAV or other cell & gene therapy platforms is highly preferred.
• Deep knowledge of NMPA, FDA (CBER/OTP), EMA, and ICH non‑clinical regulations.

Required skills

• In‑vivo gene‑editing (CRISPR/Cas) techniques
• Pharmacokinetics (PK) and toxicology study design
• GLP and non‑GLP study oversight
• Regulatory submission preparation (IND, BLA)
• Bioinformatics for off‑target analysis
• Next‑generation sequencing (NGS) data analysis
• Animal model development (humanized mouse, non‑human primate)