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Evaluation

Pharmaceuticals

Regulatory Considerations for Gene Therapy in Pediatric Patients

Wednesday, May 14, 2025
08:30 - 09:30 CEST
Location: The Arc (level 3)
Learning Level: Foundational
Description: Despite ongoing advancements in gene therapy research, pediatric patients remain underrepresented in this area. A solutions circle would offer key stakeholders an opportunity to address and discuss the knowledge gaps surrounding gene therapy in pediatric populations. The primary goal of the group would be to examine the current regulatory landscape and explore potential pathways to address this unmet need in the medical field.

Learning Objectives:

  • Participants will be able to identify and articulate the key challenges and gaps in current gene therapy research for pediatric patients, including issues related to long-term safety, immune response, and dosing strategies.
  • Participants will be able to analyze the current regulatory framework for pediatric gene therapy and propose actionable strategies for developing study designs and clinical trials that address the unmet medical needs of this population.