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Associate Director/Director, Toxicology

Orna Therapeutics
Cambridge, MA Full Time
POSTED ON 2/22/2025
AVAILABLE BEFORE 5/22/2025

Position Overview :

We are seeking a highly experienced and motivated Associate Director / Director of Toxicology to join our team, with a focus on the safety evaluation of RNA-based therapies. The successful candidate will report to the Head of Preclinical and lead preclinical toxicology programs, ensuring the safe advancement of RNA therapeutics from discovery through clinical development. This individual will be instrumental in designing, managing, and interpreting toxicology studies, supporting regulatory submissions, and working closely with cross-functional teams to drive program success.

Key Responsibilities :

  • Lead the design and execution of nonclinical toxicology studies (GLP and non-GLP) to evaluate the safety profile of RNA-based therapeutics, including circRNA and mRNA.
  • Serve as the subject matter expert (SME) on toxicology, providing strategic input on safety assessments specific to lipid nanoparticles (LNPs) and RNA-based therapies.
  • Manage relationships with external contract research organizations (CROs) conducting toxicology studies, ensuring study integrity and alignment with program goals.
  • Analyze and interpret toxicology data to assess the safety and risk profiles of LNP-RNA therapies, providing insights to guide project decisions and regulatory strategies.
  • Collaborate closely with internal teams in pharmacology, discovery, clinical development, and regulatory affairs to align toxicology plans with overall program objectives.
  • Contribute to the preparation and review of regulatory submissions, including INDs, CTAs, and IBs, with a specific focus on RNA therapeutic safety data.
  • Stay current with evolving regulatory guidelines and industry trends related to RNA therapeutics and toxicology, ensuring compliance with global regulatory requirements (FDA, EMA, ICH).
  • Mentor and guide junior scientists within the preclinical department, promoting a culture of scientific excellence and teamwork.
  • Present toxicology findings at internal meetings and represent the company in regulatory interactions regarding toxicology data.

Qualifications :

  • PhD in Toxicology, Pharmacology, or a related life sciences field with at least [6-8 ] years of experience in toxicology within the biotechnology or pharmaceutical industry.
  • Strong background in the design and interpretation of toxicology studies for LNP and RNA-based modalities (e.g., mRNA,circRNA).
  • Experience working with GLP-compliant CROs and managing outsourced toxicology studies.
  • Demonstrated expertise in contributing to successful regulatory submissions (INDs, CTAs) for RNA or nucleic acid-based therapies.
  • Familiarity with the unique safety considerations and regulatory requirements for RNA therapeutics, including biodistribution, immunogenicity, and off-target effects.
  • Excellent leadership, communication, and organizational skills, with the ability to thrive in a dynamic, fast-paced biotech environment.
  • Strong analytical skills and the ability to make data-driven decisions in a cross-functional setting.
  • Preferred Qualifications :

  • Board certification in toxicology (DABT).
  • Experience with next-generation RNA platforms or novel delivery technologies (e.g., lipid nanoparticles, viral vectors).
  • Previous experience in a biotechnology startup or early-stage company, particularly in the RNA therapeutics space.
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