The Importance of Prioritizing Indications for Cell and Gene Therapy-Focused Companies

The cell and gene therapy (CGT) market is vibrant, advancing rapidly, and has tremendous promise. Analyst reports show the market size reaching $21.33 billion in 2026 at a compound annual growth rate of 25.6%. 16 out of the 20 largest biopharmaceutical companies by revenue have portfolios with cell and gene therapy assets, demonstrating the legitimacy and promise of this field.

Often, biotech companies have great science but need in-depth industry insights to make strategic decisions and reflect value on their product portfolios. These companies require extensive data and advanced bioinformatics as well as development feasibility and commercial metrics to determine their potential target. Prioritizing an indication for a CGT asset is a multistep process but a necessary part of a biotech’s evolution and ultimate success.

At Kineticos, our approach combines years of life science experience and market research with insightful analysis to produce actionable and success-driven recommendations. Our approach to indication prioritization employs a strategic analytic process that provides a risk-mitigated path to market for any CGT approach.

Understanding your Company’s Approach and Likely Areas of Success

The first step in the process is to carefully consider the development or commercial implications of developing your platform for the initially chosen indication or indication space. A deep understanding of the unique perspective of your company can greatly drive the initial indication list, which could include several hundred indications. Once the first list is established, the following steps should be taken to prioritize the right indication.

Initial Triage of Indications

The first step of triage requires a thorough disease overview: considering the epidemiology standard of care,  unmet needs, and approved drugs in the space.

The work done in this step will help guide the decisions of which indications to prioritize or omit.  Average yearly cases, 5-year survival rates, average yearly deaths,  and mortality rates are all important factors to consider. Standard of care is particularly important. An indication where there are already several efficacious options on the market will be a difficult area for a CGT to make a meaningful impact. Furthermore, disease progression plays a unique role in CGT approaches where cost and increased manufacturing times can make some approaches less suitable to indications that progress rapidly. The mortality rate of the selected indication can dictate when your treatment will be administered. As an example of these considerations, all approved CAR-T cell therapies are 3^rd line treatments for relapsed/refractory cancers. If the goal is to move a potential therapy to 2^nd or first-line treatment, consider choosing indications with high mortality rates and few effective options for the standard of care.

The key takeaway here is that the indication and target landscapes are complex and contain multiple factors that will influence clinical studies and commercialization. Due diligence here will go a long way to ensure a higher probability of selecting an indication that will have scientific, clinical, and commercial success.

Mapping out the clinical and regulatory landscape for cell and gene therapies targeting a range of potential applications will help to determine what is the appropriate lead indication. Start by reviewing the recent clinical trial paths for the identified targets. It helps paint a clearer picture of these trials by stage, type, geography, and endpoints. Other key points to consider are average trial cost and cost per patient, average trial enrollment, and average duration.

The next step of triaging is to identify and review the top companies and potential competitors in space. The evaluation should include key attributes, capabilities, strengths and weaknesses, and a pipeline assessment for any company exploring similar indications. The goal is to understand the competition’s clinical strategy and learning opportunities.

Specific Triage with Primary Research and Modeling

Finally, for these highly prioritized indications, perform a risk-adjusted net present value (rNPV) estimation to better understand potential returns for investors in the company’s technology. A positive rNPV may show that a relatively inexpensive, short clinical development of the targeted therapy makes all considered indications in the space attractive to pursue. The goal is to provide the company with enough data to determine which indication to select for its lead clinical program.

Conclusion

Investigating potential lead indications for therapeutic assets is critical for commercial success. A CGT biotech needs to understand the clinical and regulatory landscape for a range of potential therapeutic areas, to determine what is the appropriate lead indication. Important steps can be made to make sure the right disease is selected:

• Indication assessment
• Clinical trial assessment
• Competitive landscape
• KOL interviews
• Risk-adjusted net present value

By following these steps, a biotech company can confidently prioritize a series of indications for a risk-adjusted, value-inflecting portfolio.