Sr. / Director, In vivo Pharmacology

Posted on May 25,

Orna Therapeutics, Inc. is a Cambridge-based biotechnology startup on a mission to create therapies for diseases inaccessible to existing approaches. Built on novel circular RNA technology developed at MIT, Orna is leading the next wave of RNA therapeutics with our best-in-class expression and delivery platform. We are seeking an exceptional Sr / Director, In vivo Pharmacologyto identify novel therapeutic opportunities and advancing our platform for enhanced tissue delivery, efficacy and safety. The successful candidate will build and lead a highly integrated scientific team working seamlessly with research teams to drive projects to completion, bringing new and exciting drugs to IND. A deep understanding of gene therapy and the appropriate application of in vivo models to assess target engagement, disease interruption, and potential liabilities is expected. Emerging problems will be met with evidence-based solutions.

Extensive experience working with rodent and non-rodent animal models internally and with CROs is an essential requirement. In addition to bench assays, the leader will understand and correctly interpret data from in vivo studies including gross findings, clinical pathology, histologic changes, and in situ molecular profiling by immunohistochemistry, in situ hybridization, spatial-seq, etc. The position requires an ability to see beyond the ordinary and identify novel opportunities revealed by disparate data streams.

The ideal candidate holds a PhD in a biomedical discipline, and / or board certification in veterinary or and is recognized leader in both investigative research. The position requires a deep understanding of gene therapy and the appropriate application of in vivo models to assess target engagement, disease interruption, and potential liabilities. The successful candidate will have strong communication skills, team-first collegiality, and an ability to manage multiple parallel activities in a fast-paced environment.


  • Work with leadership team and scientists to create and execute research strategies for target discovery, platform and safety to enable clear go/no-go decisions
  • Responsibility for managing internal and outsourced studies around target identification and validation, on- and off-target in vivo pharmacology, phenotypic outcomes, and IND-enabling studies
  • Participate in drug discovery collaborations across expertise areas
  • Select, design, and validate appropriate models and outcome assessments for internal and outsourced programs
  • Plan and execute early- and late-stage preclinical studies using appropriate models
  • Outline and communicate research goals, design key studies and analyze data
  • Provide guidance on circulating and tissue biomarkers for clinical candidates
  • Effectively communicate study outcomes and significance to diverse stakeholders
  • Leverage a network of scientific advisors and consultants to execute research activities for preclinical and translational programs


  • PhD in the life sciences and/or DVM/MD
  • 12+ years’ experience as an independent in vivo biomedical researcher
  • 4+ years managing PhD-level scientists in biopharma
  • Solid record of publications and inventions
  • Experience in gene therapy including RNAi, mRNA and/or CRISPR/Cas9
  • Strong background in disease research with a thorough understanding of signaling pathways and mechanism of action for targeted therapies
  • Experience analyzing a broad array of molecular, cell biology and in vivo outcomes
  • Track record of novel target discovery and safety assessment
  • Demonstrated ability to manage and work with teams of highly skilled scientists and research associates in a fast-paced, entrepreneurial environment
  • Excellent interpersonal skills, leadership abilities, and oral and written communication
  • Comfortable delivering against challenging commitments
  • Experience working with external partners to accomplish drug discovery and developmental objectives
  • Strong track-record of working on cross-functional project teams as a project leader
  • Demonstrated ability to deliver on multiple projects in parallel


Orna Therapeutics, Inc. is an equal opportunity employer.