Commercial assessment of early-stage gene editing technology for sickle cell disease and β-thalassemia

Our client asked us to identify and prioritize opportunities for a technologically novel approach to gene editing for two indications in which it may be a late entry.

CRA approach

  • Performed secondary research and literature review
    • Developed disease overview, current treatment dynamics, unmet needs, and addressable patient populations
    • Assessed competitor pipelines for gene therapy, gene editing, and other technologies
  • Conducted qualitative market research with leading KOLs in sickle cell disease, β-thalassemia, stem cell transplants, and gene therapy

Client impact

  • Our rare disease experience, pipeline review, and market research helped target opportunities for the future product to play an essential role in a rapidly evolving treatment landscape
  • Provided specific recommendations for target gene and delivery approach for client’s editing technology that could provide maximal clinical value and competitive differentiation
  • Identified key patient segments based on genetics, intensity of current care, new treatment risks, and other factors to identify the patients most addressable with the client’s future product
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