Prenatal Somatic Cell Gene Therapies: Charting a Path Toward Clinical Applications (Proceedings of the CERSI-FDA Meeting).
drug development
fetal medicine
immunopharmacology
neurology
pediatrics
perinatology
pharmacogenetics/pharmacogenomics
rare diseases
regulatory/scientific affairs
women's health
Journal
Journal of clinical pharmacology
ISSN: 1552-4604
Titre abrégé: J Clin Pharmacol
Pays: England
ID NLM: 0366372
Informations de publication
Date de publication:
09 2022
09 2022
Historique:
received:
20
05
2022
accepted:
24
07
2022
entrez:
15
9
2022
pubmed:
16
9
2022
medline:
17
9
2022
Statut:
ppublish
Résumé
We are living in a golden age of medicine in which the availability of prenatal diagnosis, fetal therapy, and gene therapy/editing make it theoretically possible to repair almost any defect in the genetic code. Furthermore, the ability to diagnose genetic disorders before birth and the presence of established surgical techniques enable these therapies to be delivered safely to the fetus. Prenatal therapies are generally used in the second or early third trimester for severe, life-threatening disorders for which there is a clear rationale for intervening before birth. While there has been promising work for prenatal gene therapy in preclinical models, the path to a clinical prenatal gene therapy approach is complex. We recently held a conference with the University of California, San Francisco-Stanford Center of Excellence in Regulatory Science and Innovation, researchers, patient advocates, regulatory (members of the Food and Drug Administration), and other stakeholders to review the scientific background and rationale for prenatal somatic cell gene therapy for severe monogenic diseases and initiate a dialogue toward a safe regulatory path for phase 1 clinical trials. This review represents a summary of the considerations and discussions from these conversations.
Identifiants
pubmed: 36106778
doi: 10.1002/jcph.2127
pmc: PMC9547535
mid: NIHMS1838001
doi:
Types de publication
Journal Article
Review
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
S36-S52Subventions
Organisme : NICHD NIH HHS
ID : K12 HD001262
Pays : United States
Organisme : NICHD NIH HHS
ID : R01 HD107190
Pays : United States
Organisme : NINDS NIH HHS
ID : R35 NS122306
Pays : United States
Informations de copyright
© 2022, The American College of Clinical Pharmacology.
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