Casebia Therapeutics

Job Title: Principal Scientist, Gene Therapy

Job Number: 22230

Location: Cambridge, MA

Job Description

Reporting directly to the VP of Ophthalmology and based in Cambridge, MA, the successful candidate will help guide a team of researchers in developing novel gene editing therapies, identifying new areas of research, prioritizing existing projects, and contributing to Casebia’s strategic research plans as it relates to ophthalmology. He/she will guide research projects through GLP preclinical studies, and transfer work to CROs and service providers as appropriate. A successful candidate will have deep experience in utilizing gene editing tools, preferably CRISPR/Cas9 but also TALEN or ZINC Finger Nucleases, plus experience with viral (e.g. AAV, Lentiviral) and/or non-viral (e.g. nanoparticle, liposome) delivery of these tools in vivo. Additionally, the candidate will be familiar with various inherited ophthalmic disorders, associated animal models, and have experience managing projects and research teams.


  • Lead the development and implementation of a preclinical research plan from discovery to IND/CTA
  • Identify and propose projects to senior management for approval
  • Serve as principal investigator or study director of non-clinical studies
  • Collaborate with and serve as scientific mentor to assigned Research Associates, directing the work of research associates at the laboratory bench
  • Assist VP of Ophthalmology in prioritizing and allocating resources
  • Leverage external research collaborators
  • Investigate, create, and/or develop new methods and technologies for project advancement
  • Assist in securing intellectual property, including detailed descriptions for patent filing and/or proof of concept work

Required Skills

  • PhD in a relevant scientific discipline with 12+ years’ industry experience, experience in genetic engineering is preferred
  • At least 5 years’ experience managing and leading a research team, preferably with experience getting to development candidate selection.
  • Preclinical research and development experience preferred
  • Direct experience with assays and technologies related to gene therapy, gene editing, viral and/or non-viral DNA/RNA delivery
  • Knowledge of the pathophysiology and treatment of ophthalmic disorders and related genetics
  • Prior success discovering and developing successful novel medicines
  • Expert knowledge of scientific principles and concepts
  • Significant knowledge of inherited ophthalmic disorders
  • Excellent oral and written communication skills
  • Ability to work in a team-oriented environment but also be a self-starter, internally motivated, with the ability to work independently to drive the research forward
  • Scientifically rigorous, highly organized, with significant attention to detail
  • Proven track record of leading research programs and establishing strong collaborations
  • High energy level, focus, a passion and sense of urgency for developing important new medicines for devastating diseases


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