Ethical challenges for a new generation of early-phase pediatric gene therapy trials

Alexander A. Iyer, Dimah Saade, Diana Bharucha-Goebel, A. Reghan Foley, Gilberto ‘Mike’ Averion, Eduardo Paredes, Steven Gray, Carsten G. Bönnemann, Christine Grady, Saskia Hendriks, Annette Rid

Research output: Contribution to journalReview articlepeer-review

6 Scopus citations


After decades of setbacks, gene therapy (GT) is experiencing major breakthroughs. Five GTs have received US regulatory approval since 2017, and over 900 others are currently in development. Many of these GTs target rare pediatric diseases that are severely life-limiting, given a lack of effective treatments. As these GTs enter early-phase clinical trials, specific ethical challenges remain unresolved in three domains: evaluating risks and potential benefits, selecting participants fairly, and engaging with patient communities. Drawing on our experience as clinical investigators, basic scientists, and bioethicists involved in a first-in-human GT trial for an ultrarare pediatric disease, we analyze these ethical challenges and offer points to consider for future GT trials.

Original languageEnglish (US)
Pages (from-to)2057-2066
Number of pages10
JournalGenetics in Medicine
Issue number11
StatePublished - Nov 2021

ASJC Scopus subject areas

  • Genetics(clinical)


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