Refinement and pressure testing of the launch strategy for a gene replacement therapy asset in a congenital rare disease therapy area, alongside a scenario planning exercise that identified key issues to address based on the characterization of future competitor launch scenarios.


The client’s key objective was to refine and pressure-test the launch strategy for their new gene replacement therapy, using a competitor scenario planning process. The strategy needed to consider:

  • The rapidly evolving competitor landscape, with multiple new entrants anticipated
  • The potential for a range of different competitor launch label indications, the uncertainty around approval timings, and the impact of these on the launch plan
  • The overarching need for a gene therapy market entrant to assess the target patient opportunity segments at launch


We employed a matrix scenario framework to map likely competitor launch scenarios and their probabilities, evaluating the implications of eachscenario.

We developed mitigation strategies to cover positioning, patient segment priorities and adeno-associated virus (AAV) gene therapy eligibility screening.

We were able to identify key market and competitor trigger points to support ongoing tracking and evaluation of risk.


We created a set of clear plans associated with each competitor scenario, which the client could activate immediately when a set of ‘scenario indicators’ relating to the market were satisfied.