Delivering strong patient impact is critical to Syncona’s strategy of building companies that can develop transformational treatments for patients in areas of high unmet need. The positive impact a therapy can have on patients is integrated into our investment process and the ongoing management of our portfolio.
Patient impact is a core part of our investment and portfolio management processes
Syncona’s strategy is to create, build and scale companies around exceptional science, to deliver transformational treatments for patients in areas of high unmet need. We focus on developing treatments for patients by working in close partnership with world- class academic founders and management teams. Our patient impact framework outlines the key patient impact factors we assess when we are considering an investment opportunity.
We believe that this framework provides a tool which can be used by our stakeholders to understand the overall impact our portfolio can have on patients suffering from devastating diseases, whilst also providing a clear structure which can be used for monitoring progress across the Syncona portfolio.
1. The number of existing cases of a disease.
Applying our framework
Our patient impact framework has been developed to apply to companies across our investment strategy. We expect there will be some variation in the application of the framework across investment opportunities and existing companies, where there is broad diversification across therapeutic area and development stage.
Our framework provides clear criteria against which the investment team can assess potential opportunities. This will be implemented through the integration of the framework into investment memos, as well as due diligence for later-stage opportunities.
Our framework also supports our approach to integrating patient impact into our ongoing management of the portfolio. As our portfolio companies continue on their developmental pathways, we work alongside management teams to refine clinical and pipeline strategies. This means that through time the application of the framework to an individual company may change. This will be integrated throughout our standard cycle of monitoring progress at companies. The framework will also be integrated into our standard cycle of reporting to the Syncona Board.
Through time, we intend to use our framework to provide reporting to our stakeholders so that they can quantify the overall patient impact of the Syncona portfolio. The full integration of the framework across our investment and portfolio management processes is a key priority for Syncona in FY2024/5.
| INVESTMENT CONSIDERATION |
DEFINITION | GRADE 1 | GRADE 2 | GRADE 3 |
| Impact on patient quality of life |
Measures the improvement in quality of life conferred to the patient, as inferred by measures including Quality of Life (QoL) score | Low | Medium | High |
| Addressable patient numbers (prevalence) |
Measures the total patient population in a disease area being targeted by a therapy | <20,000 | 20,000-500,000 | >500,000 |
| Proximity to regulatory filing |
The clinical development stage of a therapy, measured by years to Biologics License Application (BLA) or equivalent | >6 years | 3-6 years | <3 years |
| Life threatening | Extent to which the disease can threaten the life of a patient | Modest impact | High impact | Life threatening |
| Curative potential | Extent to which a therapy has the potential to cure a patient | Significant symptom relief |
Disease stabilisation or significant change in progression |
Disease cure |
| Availability of existing treatments |
Measures the availability of alternative treatment options for the specific disease area | Available and effective treatment options |
Available but poor treatment options | No existing treatments |
| Category leading platform |
Extent to which the company has developed a world-class technology platform which cannot be replicated |
Low platform potential |
Strong platform potential |
Leading platform potential |
| Maturity |
Assessment of the maturity of a company, measured through several factors with an increased weighting for companies at a later stage. Inputs include, but are not limited to: – Scientific provenance |
Nascent (0-1 out of 5) |
Substantial (2-4 out of 5) |
Complete (5 out of 5) |