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

@article{Chowdary2022Phase1T,
  title={Phase 1-2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia B.},
  author={Pratima Chowdary and Susan Shapiro and Michael Makris and Gillian Evans and Sara Boyce and Kate L. Talks and Gerard Dolan and Ulrike M Reiss and Mark Phillips and Anne Riddell and Maria Rita Peralta and Michelle Quaye and David W. Patch and Edward G. D. Tuddenham and Allison P. Dane and Marie Watiss{\'e}e and Alison Long and Amit Nathwani},
  journal={The New England journal of medicine},
  year={2022},
  volume={387 3},
  pages={
          237-247
        }
}
BACKGROUND FLT180a (verbrinacogene setparvovec) is a liver-directed adeno-associated virus (AAV) gene therapy that uses a synthetic capsid and a gain-of-function protein to normalize factor IX levels in patients with hemophilia B. METHODS In this multicenter, open-label, phase 1-2 trial, we assessed the safety and efficacy of varying doses of FLT180a in patients with severe or moderately severe hemophilia B (factor IX level, ≤2% of normal value). All the patients received glucocorticoids with… 
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