Welcome to Gene Therapy Patient Engagement

Gene Therapy Patient Engagement is dedicated to providing detailed case studies and actionable insights on how to incorporate patient input more effectively into gene therapy development. Whilst everyone knows the patient is important, it’s evident that there are several unique nuances within gene therapy where a lot can be learned from those who have been through the experience, including:

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Discovering the Tools Being Used to Create and Distribute Educational Materials on Gene Therapy
Understand the strategies behind developing virtual resources and reaching patients in the context of the current pandemic, with insights from Amicus Therapeutics & uniQure

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Integrating Patient Input More Effectively in Clinical Trial Design
Hear how Lysogene and AGTC are incorporating patient input into clinical trial design, through designing of bespoke clinical endpoints that are meaningful to patients and regulators, and developing patient advisory boards and councils to support this process

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Navigating the Challenges Around the Realities of Committing to a Gene Therapy Trial
Explore the ways that remote-based clinical assessments can be used to support long-term patient commitment required for gene therapy trials, and explore how to manage and align expectations aross stakeholders, with insights shared from Nationwide Children’s Hospital, GenSight Biologics & bluebird bio

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Forging Mutually Beneficial, Long-Lasting Partnerships with Patient Advocacy Organizations
Discover the ways in which patient advocacy groups and industry have collaborated on gene therapy development, with insights from CureDuchenne & Pfizer

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Investigating Early Stage Patient Engagement and its Importance for Gene Therapy
Hear how FARA are preparing their patient communities early in light of potential gene therapies entering the pipeline, and understand how Takeda are engaging patients in R&D to truly understand their disease and set up strong foundations for later stage development