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.
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.
Authors
Sahana Kunanayagam
Michelle C. Wang
Catrina M. Loucks
Facundo Garcia-Bournissen
Ruud H. J Verstegen
Tamorah Lewis
Page last reviewed: 27-Aug-2026
Key takeaway
- Cell and gene therapies are changing treatment for children with rare diseases, including conditions with few or no effective treatment options
- These therapies behave differently from traditional drugs, so researchers need new ways to understand how they move through and affect the body
- Children are not simply “small adults”. Age, organ development, and the immune system can affect how these therapies work and what dose is appropriate
- Preclinical research is especially important because pediatric rare disease trials often involve very small numbers of patients, making it harder to determine the right dose from clinical data alone
- Longer-term monitoring is critical, particularly for cell therapies that can remain active in the body for months or even years
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