Strategic Development of Gene-Editing Platform Technology in Rare Disease Space

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

Challenge

• 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

Approach

• 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