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Understanding patients’ unmet needs to inform the R&D strategy for gene therapies

The Challenge:

Our partner, AAVantgarde Bio, a gene therapies company developing two proprietary novel platforms to deliver large genes addressing indications with high unmet need not addressable by conventional AAV gene therapies, were looking to expand their disease portfolio and were  looking at prioritising research into areas where they will have the greatest impact.


New indications decision making  involves engagement with Key Opinion Leaders (KOLs) and other healthcare experts, but it is difficult to engage with enough rare disease patients, through traditional research methods, to understand the lived experience of these rare conditions.


AAVantgarde Bio asked White Swan to provide insight into the unmet needs, priorities and impact on life for patients with two rare genetic conditions.


What We Did:

Using Black Swan’s proprietary technology and White Swan’s proprietary taxonomy, combined with clustering and insights expertise, we analysed over 7k social posts from Reddit, Forums, Blogs, News & review sites that related to the two conditions of interest. We assessed a global data set covering a two-year period with a focus on UK & US insights. We adopted a flexible and iterative approach working closely with AAVantgarde Bio and deepening our analysis into key insights as required.


The Results:

Our analysis highlighted patients' everyday experiences of living with the condition. Amongst other factors, we uncovered the importance of early diagnosis and interventions to help patients prepare for a life with the condition. We also surfaced insights into patients’ experiences of publicly available genetic testing and how this informed their life choices and the perceived barriers to existing treatment options.

From these insights, AAVantgarde Bio was able to gain a better understanding of the lived experience of these conditions and what really matters to patients. These have helped inform prioritisation and investment decisions for the gene therapy pipeline.



Understanding patients’ unmet needs to inform the R&D strategy for gene therapies

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