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CASE STUDY

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Gene therapy disease assessment

A gene therapy biotech with a novel approach to a fast-moving disease area needed an objective read on the opportunity and on whether it had a credible path to win there.

S351-108

Addressable population sized by mutation status

Situation

Novel approach, internal skepticism

  • Gene therapy biotech was considering a novel therapeutic approach that would be mutation agnostic, with a compelling research package supporting scientific hypothesis.
  • Company already with several assets in development, and CEO was unconvinced this program should be added to pipeline.
  • Client sought comprehensive evaluation and how it stacked against other programs we had evaluated previously for them.

Project

Competitive and commercial assessment

  • Conducted secondary research and KOL interviews to build:
    • Epi/market model
    • Competitive landscape
    • Robustness of current biomarker and clinical endpoints
    • Uptake scenarios based on efficacy thresholds for asset
  • Identified key product characteristics likely to drive success amidst competition.
  • Built financial model that included R&D program costs, revenue projections, NPV.

Competitor readouts mapped by target population

Revenue modeled across efficacy and competitive scenarios

Outcome

Conviction to advance IND

  • Recommended moving forward with key criteria to be evaluated during early-stage development that could inform future go/no-go decisions.
  • CEO and Board of Directors approved program, moved to IND stage.

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