Focus Area Advanced Therapeutics

Gene Editing – Monogenetic Diseases

The Client

  • US-based biotech with clinical stage best-in-class gene editing platform in development for various oncology settings
  • Looking to leverage the broad applicability of proprietary gene editing technologies to a broad range of human diseases beyond oncology


  • Develop rational strategic plan for pursuing development of platform in areas outside of core oncology expertise
  • Prioritize therapeutic targets with genetic driver among the multitude of opportunities for gene editing within the rare disease space


  • Develop methodology for filtering rare genetic disease landscape based on selection criteria amenable to client platform technology
  • Map clinical stage gene therapy programs for genetic diseases with assessment of clinical value proposition

Valued Outcome

  • Pursuit of ex vivo cell-based development found to represent most attractive near-term opportunity in light of current regulatory and potential in-vivo delivery issues
  • Detailed profiles of 20 prioritized opportunities for client’s platform gene editing technologies within the rare disease space