New Paper Provides Practical Guide to Cell and Gene Therapies in Pediatric Rare Disease Research
Cell and gene therapies are rapidly transforming the pediatric rare disease treatment landscape and are offering new possibilities for conditions that have historically had few or no effective treatment options.
RareKids-CAN Pharmacology Sub-Platform Lead Dr. Tamorah Lewis (left) and Pharmacology Sub-Platform Trainee and lead author, Michelle Wang (right).
To help clinicians, researchers and trainees navigate this rapidly evolving field, members of the RareKids-CAN Pharmacology Sub-Platform have published a new review article in Pediatric Research exploring the clinical pharmacology principles behind recently approved cell and gene therapies relevant to pediatric populations.
The paper, Clinical Phamacology Insights from Recent Cell and Gene Therapy Approvals Relevant to Pediatrics, examines lessons learned from the growing number of approved advanced therapies and highlights key considerations for their development, evaluation and clinical use.
As of May 2025, the U.S. Food and Drug Administration has approved 44 cell and gene therapies, including 14 therapies with pediatric use. Many of the therapies target rare disease that begin in childhood and have potential to provide long-term benefits for patients and their families.
"By working in pediatric rare disease research, I have seen how much excitement surrounds the potential of ATMPs and genetic medicines.” - Michelle Wang
Approximately 80 percent of rare diseases have a genetic basis. Cell and gene therapies have, therefore, become increasingly important in pediatric rare disease research. However, these therapies differ significantly from traditional medications. Rather than relying on conventional pharmacology concepts alone, researchers must consider factors such as viral vector biodistribution, vector shedding, cellular expansion, persistence, and long-term safety monitoring. The white paper explains these concepts and outlines how they influence clinical trial design, first-in-human dosing strategies, and regulatory decision-making.
The publication also highlights the unique challenges of conducting pediatric rare disease clinical trials. Small patient populations, ethical considerations, developmental difference between children and adults, and the need for specialized safety monitoring all require careful planning.
The authors emphasize the critical role of preclinical models in information dose selection and evaluating safety before therapies reach children in clinical trials.
Dr. Tamorah Lewis, Lead of the RareKids-CAN Pharmacology Sub-Platform and senior author of the paper, said the team was motivated by a desire to make this emerging area of research more accessible.
"My goal in mentoring the team during the writing of this paper was to make ATMPs understandable for the every-day clinician and researcher. There are many teams across Canada that have an interest in learning more about cell and gene therapies, but the current pharmacology papers written about these topics are not accessible or understandable without a lot of prior expertise. I hope that our White Paper can demystify these therapies and get teams excited about the future of research in rare disease."
"My goal in mentoring the team during the writing of this paper was to make ATMPs understandable for the every-day clinician and researcher.” - Dr. Tamorah Lewis
For lead author and RareKids-CAN Pharmacology Sub-Platform Trainee Michelle Wang, the paper was an opportunity to connect traditional pharmacology concepts with emerging therapeutic approaches.
"By working in pediatric rare disease research, I have seen how much excitement surrounds the potential of ATMPs and genetic medicines. Our team's aim was to bridge the gap between traditional pharmacology principles and these emerging ATMPs by creating a practical and accessible resource that highlights the key concepts researchers, clinicians, and trainees, need to understand as the field continues to grow."
The authors note that as advanced therapies continue to emerge as promising treatments for are diseases, it is increasingly important for clinicians, researchers, and trainees to understand their unique pharmacological characteristics. By providing a practical overview of key concepts, the review helps build knowledge and capacity across Canada’s pediatric rare disease research community and supports the continued advancement of innovative therapies for children and families.