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