Phase 1–2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia B

2022147 citationsJournal Articlebronze Open Access

Authors

Pratima Chowdary · The Royal Free Hospital

Susan Shapiro · Oxford BioMedica (United Kingdom)

Michael Makris · University of Sheffield

Gillian Evans · University Foundation

Sara Boyce · University Hospital Southampton NHS Foundation Trust

Kate Talks · Newcastle upon Tyne Hospital

Gerard Dolan

Abstract

Sustained factor IX levels in the normal range were observed with low doses of FLT180a but necessitated immunosuppression with glucocorticoids with or without tacrolimus. (Funded by Freeline Therapeutics; ClinicalTrials.gov numbers, NCT03369444 and NCT03641703; EudraCT numbers, 2017-000852-24 and 2017-005080-40.).

Topics & Keywords

Virus-based gene therapy research Hemophilia Treatment and Research CAR-T cell therapy research

UN Sustainable Development Goals

Good health and well-being

Publication Details

Published in: New England Journal of Medicine

Volume 387, Issue 3, pp. 237-247

DOI: 10.1056/nejmoa2119913

Field-Weighted Citation Impact: 26.54