Скачать или смотреть C-Path Global Impact 2025: Development of Gene-based Therapies in Pediatrics

The panel discussion from Critical Path Institute’s (C-Path) Global Impact Conference (CGIC) 2025 is led by C-Path Vice President of Rare/Orphan and Pediatric Diseases Collin Hovinga, who is joined by FDA Associate Director for Pediatrics Najat Bouchkouj, Novartis Pediatric Center of Excellence’s Ki Lee Milligan, Director for Clinical Research and Development at NCCHD Hidefumi Nakamura, Eli Lilly Senior Research Advisor Mary Short, Solid Bioscience Head of Clinical Science Patrick Gonzalez. The conversation centers on the development, regulatory considerations, and clinical trial design of pediatric gene therapies, with a focus on cell and gene therapy approval processes, patient inclusion, and long-term follow-up.

The session highlights the complexities and challenges in bringing gene therapies to pediatric populations, emphasizing the importance of early patient inclusion, collaboration across stakeholders, and innovative trial designs.

Key Insights

Early and continuous patient involvement is essential to design meaningful pediatric gene therapy trials.

Regulatory frameworks (PREA, BPCA) and guidances guide pediatric trials but require flexible, case-by-case application.

Real-world data and natural history studies are increasingly vital to supplement or replace traditional control arms, especially in rare pediatric diseases.

Long-term follow-up (5-15 years) is critical to monitor safety (e.g., mutagenesis risk) and durability, balanced with minimizing patient burden through decentralized approaches.

Multidisciplinary site infrastructure and consistent evaluator training are necessary for complex gene therapy trials.

Industry motivation is often driven by patient community connections and transformational company missions, not solely commercial incentives.

International collaboration and harmonization efforts are expanding to streamline global pediatric gene therapy development

Ethical transparency and informed consent are paramount, particularly given the one-time nature and risks of gene therapies SUBSCRIBE to C-Path's YouTube Channel Now ►►    / criticalpathinstitute  

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About Critical Path Institute C-Path is an independent, nonprofit established in 2005 as a public-private partnership, in response to the FDA’s Critical Path Initiative. C-Path’s mission is to lead collaborations that advance better treatments for people worldwide. Globally recognized as a pioneer in accelerating drug development, C-Path has established numerous international consortia, programs and initiatives that currently include more than 1,600 scientists and representatives from government and regulatory agencies, academia, patient organizations, disease foundations and pharmaceutical and biotech companies. With dedicated team members located throughout the world, C-Path’s global headquarters is located in Tucson, Arizona and C-Path’s Europe subsidiary is headquartered in Amsterdam, Netherlands. For more information, visit c-path.org. #CPath #drugdiscovery #researchgrants #globalhealth #drugdevelopment #datasharing #researchfunding #raredisease #academicresearch #regulatoryscience #globalhealth #consortia #collaboration #FDA #EMA #T1D #Parkinsons #Alzheimers #PKD #Duchenne #ALS #biomarkers