The Place and Potential for Gene Therapy

Session details:

Gene therapy has proven its value in rare diseases, in approved therapies demonstrating the ability to address these at their biological source with potentially long-lasting effects from a single treatment. The next phase of gene therapy is to tackle more common serious chronic diseases, such as Parkinson’s disease and heart failure, which represent great global burdens. Achieving this ambition will require innovation across multiple areas, including targets and biology, treatment delivery, manufacturing scale, and patient access systems. Cross-ecosystem collaboration will be essential to translating research and development of gene therapy innovations into real outcomes for patients.

Key takeaways:

• How gene therapy delivers or modifies genetic instructions to address disease at its biological root, enabling the potential for long-lasting impact.
• While some success has initially been seen in rare diseases, larger unmet medical need lies in more prevalent serious chronic conditions, such as Parkinson’s disease and heart failure.
• The innovations needed across delivery, manufacturing, scale, and access to support broader patient populations.
• The role of cross-sector collaboration in translating research and development of gene therapy innovation into real-world patient impact.