Why Join Us
Ultragenyx is a clinical stage biotechnology company committed to bringing to market novel products for the treatment of rare and ultra-rare diseases with an initial focus on serious debilitating metabolic genetic diseases. We are launching five Phase 2 clinical trials and with another preclinical trial in the pipeline. Where possible, our strategy is to acquire and retain global commercialization rights to our products. Come join our team during this exciting time of growth. We are located in Novato, CA, 28 miles north of San Francisco.
We are looking for an experienced Pharmacologist and Toxicologist scientist with the passion and creativity to develop great medicines for rare and ultra-rare diseases. Working closely with the CEO, VP Research and VP Regulatory, the Director, Pharmacology and Toxicology will be responsible for the preparation of pharmacology and toxicology components of regulatory submissions for all clinical study programs in support of IND and NDA packages and respond to inquiries from regulatory authorities. The Director will have experience in evaluating and addressing global pharmacology and toxicology issues. The Director, Pharmacology and Toxicology will also direct and lead the Non-Clinical Research team and activities to deliver drug activity, safety and disposition analyses.
Responsibilities, including but not limited to:
• Responsible for draft, edit and finalization of nonclinical study and technical reports and nonclinical sections for IND and BLA submissions, as well as required reports for maintenance of applications to ensure regulatory compliance
• Participate in discussions with FDA to obtain feedback and respond to inquiries related to nonclinical development.
• Critically review and edit CRO toxicology study reports, analyze and interpret data and coordinate report finalization
• Ensure appropriate quality of documentation for internal department studies compatible with global regulatory submission requirements.
• Design and manage nonclinical studies (GLP and non-GLP) for inclusion in regulatory submissions, including pharmacology, pharmacokinetics and toxicology studies
• Provide analysis and interpretation of pharmacology and toxicology issues in preparation for final reports.
• Ensure timely and accurate communication of study results and interpretation to appropriate internal teams.
• Maintain a current understanding of toxicology literature and methodology, as well as the scientific literature related to the specific drug discovery projects
• Provide direction and mentorship of Nonclinical Research team.
• The individual will be a self-starter with excellent management capabilities and who is seeking to be part of an innovative team that to support the company’s drug development process.
• Ph.D. in Biochemistry, Pharmacology /Toxicology or Biological Sciences required with more than 10 years of industry experience in conducting drug discovery research and development.
• Required experience in preparing regulatory submissions, including full BLA/NDAs or significant sNDAs, as well as multiple INDs
• Experience responding to regulatory questions related to pharmacology, pharmacokinetics and toxicology
Understanding of CMC, QA, regulatory and clinical functions to guide nonclinical activities setting up products for success at all stages of development.
• Experience with both small molecule, protein-based and protein-drug conjugates for metabolic and genetic disease targets.
• Experience in establishing and managing external CROs with timely delivery of clear and accurate well written study findings.
• Ability to synthesize and interpret diverse, multidisciplinary data sets
• Experience in designing and interpreting nonclinical safety studies
• Strong knowledge of pre-clinical drug development and FDA and ICH guidance documents including GLP regulations for nonclinical studies with small molecules and biologics is essential
• Demonstrated experience in evaluating PK/PD correlations, PK modeling and parameter estimation. Experience evaluating the pharmacokinetic properties of therapeutic antibodies is a plus.