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LB/CO01.1 : A Novel Adeno Associated Virus (AAV) Gene Therapy (FLT180a) Achieves Normal FIX Activity Levels in Severe Hemophilia B (HB) Patients (B-AMAZE Study)

Researchers

Presenter

  • P. Chowdary

Principal Investigators

  • S. Shapiro

  • M. Makris

  • G. Evans

  • S. Boyce

  • K. Talks

  • G. Dolan

  • U. Reiss

  • M. Phillips

  • A. Riddell

  • M.R. Peralta

  • M. Quaye

  • E. Tuddenham

  • J. Krop

  • G. Short

  • S. Kar

  • A. Smith

  • A. Nathwani

Medical Centers

  • Katharine Dormandy Haemophilia and Thrombosis Centre, Royal Free London NHS Foundation Trust, London, United Kingdom

  • Kings College London, London, United Kingdom

  • Churchill Hospital, Oxford Haemophilia & Thrombosis Centre and Oxford NIHR BRC, Oxford, United Kingdom

  • University of Sheffield, Sheffield, United Kingdom

  • Kent & Canterbury Hospital, Kent, United Kingdom

  • University of Southampton, Southampten, United Kingdom

  • Newcastle Haemophilia Comprehensive Care Centre, Newcastle

  • Guy's And St Thomas NHS Foundation Trust, London, United Kingdom

  • St Jude Childrens Research Hospital, Memphis, TN, United States

  • Freeline, Boston, United States

  • Freeline, Stevenage, England, United Kingdom

Locations

  • United Kingdom

  • United States

Companies

  • N/A

Study Components

Therapeutic Area

  • Blood/haematological Diseases

Disease

  • Hemophilia

Biomarkers

  • N/A

Drug/Treatment

  • N/A

Outcome

  • N/A

Study Design

  • Adaptive

  • Open Label

  • Multicenteric

Phase

  • I/II

Study Id's

  • N/A

Sponsors

  • N/A

Result

  • N/A

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