Long-term safety and efficacy outcomes of valoctocogene roxaparvovec gene transfer up to 6 years post-treatment.
acinar cell carcinoma
adeno-associated virus
clinical trial
gene therapy
haemophilia
phase 2
Journal
Haemophilia : the official journal of the World Federation of Hemophilia
ISSN: 1365-2516
Titre abrégé: Haemophilia
Pays: England
ID NLM: 9442916
Informations de publication
Date de publication:
05 Feb 2024
05 Feb 2024
Historique:
revised:
07
12
2023
received:
21
08
2023
accepted:
30
12
2023
medline:
6
2
2024
pubmed:
6
2
2024
entrez:
6
2
2024
Statut:
aheadofprint
Résumé
Valoctocogene roxaparvovec uses an adeno-associated virus serotype 5 (AAV5) vector to transfer a factor VIII (FVIII) coding sequence to individuals with severe haemophilia A, providing bleeding protection. To assess safety and efficacy of valoctocogene roxaparvovec 5-6 years post-treatment. In a phase 1/2 trial, adult male participants with severe haemophilia A (FVIII ≤1 IU/dL) without FVIII inhibitors or anti-AAV5 antibodies received valoctocogene roxaparvovec and were followed for 6 (6 × 10 No new treatment-related safety signals emerged. During year 6, a participant in the 6 × 10 Valoctocogene roxaparvovec safety and efficacy profiles remain largely unchanged; genomic investigations showed no association with a parotid tumour.
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Subventions
Organisme : BioMarin Pharmaceutical
Informations de copyright
© 2024 The Authors. Haemophilia published by John Wiley & Sons Ltd.
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