Chris Williams

Prototyping DeSci to Accelerate Cures for Rare Genetic Diseases

The understanding of biology and genetics have opened up a new frontier of gene therapies that could provide cures to 1000s of debilitating diseases impacting millions of patients around the world. While big Pharma companies focus on more prevalent (profitable) indications, 100s underfunded public and private research institutions are working in silos to develop these cures. Helping researchers to overcome cost and risk barriers can accelerate the development of bespoke therapies for smaller patient groups. We hypothesize that DeSci concepts and tools could provide platforms for supporting this research on a broad scale. However, the complete absence of DeSci from this space may require a soft introduction followed by rapid adaptation to meet the most pressing needs of the industry. The goal of this session is to identify a Minimum Viable Product that can be prototyped within this space to solve relevant problems and provide immediate value. Format Short presentation on the biotechnology ecosystem and types of players involved (Big Pharma, Small Biotechs, Public Research, Patient Advocates, etc.) followed by a whiteboard assisted design thinking session for web2/web3 tools to prototype in this space. Proposed Duration 45 minutes My Role as Facilitator ● Set context for the discussion ● Answer clarifying questions ● Challenge ideas from the perspective of the user Participant Roles ● Ask Questions! ● Design Thinking ● Ideate on web2 and web3 tools that could be prototyped in translational medicine