ReviewPediatric research2026
Clinical pharmacology insights from recent cell and gene therapy approvals relevant to pediatrics.
Review in Pediatric research, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
1 citing paper in PubMed.
- Regulatory Convergence in Cell and Gene Therapy: Harmonizing Quality, CMC, and Approval Pathways Across the FDA, EMA, PMDA, and Emerging Markets.Biotech (Basel (Switzerland)) · 2026Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors.
Funding
Abstract
The United States Food and Drug Administration has approved 44 cell and gene therapies as of May 2025, including 14 for pediatric use (excluding cord blood). This signifies an advancement in the treatment of diseases and conditions that, as of yet, cannot be effectively managed with available small-molecule drugs and therapeutic proteins. This review explores the pharmacokinetic and first-in-human dosing considerations unique to cell and gene therapies. For in vivo gene therapies, development considerations include vector shedding and biodistribution. Traditional pharmacokinetic principles do not apply to ex vivo gene therapies, given the modification of cells outside the body before re-infusion. Cellular kinetics such as expansion and persistence are relevant in cell therapies like Chimeric Antigen Receptor T cells and Tumor Infiltrating Lymphocytes. A common trend of cell and gene therapy development is the reliance on preclinical models to inform safety, efficacy, and dose selection, with first-in-human dosing further guided by data from related therapies. Rare disease and pediatric drug development present additional challenges due to small patient populations, emphasizing the importance of preclinical evidence and safety considerations. The article outlines the key considerations for cell and gene therapies that are vital for expanding their use. IMPACT: This article provides a synthesis of pediatric rare disease clinical pharmacology considerations for recently approved cell and gene therapies, highlighting how advanced therapeutic products compare with traditional pharmacokinetic and pharmacodynamic frameworks used for small-molecule drugs. The development of advanced therapeutics depends on preclinical modeling, first-in-human dosing strategies, clinical trial design, and regulatory guidance for pediatric use, emphasizing the need for a practical primer on emerging advanced therapeutic products for researchers.
Identifiers
42236924What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.