Our patient impact framework

Sustainability pillar
A focus on delivering a strong patient impact

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.

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
patient numbers
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
treatment options
Available but poor treatment options No existing
Category leading
Extent to which the company has developed a world-class
technology platform which cannot be replicated
Low platform
Strong platform
Leading platform

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
– Emerging data from lead asset
– Syndicate of investors
– Build out of an experienced executive team
– Operations capable of delivering registrational studies

(0-1 out of 5)
(2-4 out of 5)
(5 out of 5)