A US-based biotech with clinical stage, best-in-class gene editing platform with development-stage programs for various oncology indications. The client was looking to leverage the broad applicability of their own and additional proprietary gene editing technologies to a broad range of human diseases beyond oncology
To develop a rational strategic plan for pursuing the development of platform technologies in areas outside of core oncology expertise and to prioritise these therapeutic targets with genetic drivers amongst the multitude of opportunities for gene editing within the rare disease space
We developed a methodology for filtering the rare genetic disease landscape based on selection criteria amenable to the client platform technology. We mapped clinical-stage gene therapy programs for genetic diseases with an assessment of the clinical value proposition
Client pursued ex vivo cell-based platform found to represent most attractive near-term opportunity in light of current regulatory and potential in-vivo delivery issues. Delivered detailed profiles of 20 prioritised opportunities for the client’s platform gene editing technologies within the rare disease space
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