Conference Coverage from ISTH 2019 in Melbourne
Education for the Global
Hemophilia Community
Interactive Webinar
Getting to Know Gene Therapy: Terminology and Concepts
Presented by David Lillicrap, MD
Latest News
Clinical Advances in Gene Therapy Updates on Clinical Trials of Gene Therapy in Haemophilia
Science Translational Medicine (05/22/19) Vol. 11, No. 493 Milani, Michela; Annoni, Andrea; Moalli, Federica; et al.
Therapeutic hFIX Activity Achieved After Single AAV5-hFIX Treatment in Hemophilia B Patients and NHPs with Pre-existing Anti-AAV5 NABs
Science Translational Medicine (05/22/19) Vol. 11, No. 493 Milani, Michela; Annoni, Andrea; Moalli, Federica; et al.
The Impact of Pre-Existing Immunity on the Non-Clinical Pharmacodynamics of AAV5-Based Gene Therapy
Science Translational Medicine (05/22/19) Vol. 11, No. 493 Milani, Michela; Annoni, Andrea; Moalli, Federica; et al.
Hyperactivity of Factor IX Padua (R338L) Depends on Factor VIIIa Cofactor Activity
Science Translational Medicine (05/22/19) Vol. 11, No. 493 Milani, Michela; Annoni, Andrea; Moalli, Federica; et al.
ISTH Announces Launch of New Global Education Initiative in Gene Therapy For Hemophilia
Science Translational Medicine (05/22/19) Vol. 11, No. 493 Milani, Michela; Annoni, Andrea; Moalli, Federica; et al.
Conference Coverage – ISTH 2019 in Melbourne
The ALTA Study: SB-525 Gene Therapy in Adults with Hemophilia A
Prevalence of Preexisting AAV Antibodies and Implications for Gene Therapy in Hemophilia
Results of the AMT-060 and AMT-061 Gene Therapy Trials in Patients with Hemophilia B
Initial Results of the Phase 1/2 Alta Study: SB-525 Gene Therapy in Adults with Hemophilia A
One Year Follow-up of Patients with Hemophilia B Following SPK-9001 Gene Transfer
AMT-061 Gene Transfer in Adults with Hemophilia B: Early Results of a Phase 2b Trial
Current Status of Gene Therapy for Hemophilia
FLT180a: Next Generation AAV Vector for Hemophilia B
Stable Expression of FIX: Updated Results of the AMT-060 Gene Therapy Trial in Patients with Hemophilia B.
Lentiviral Vector-based Factor VIII and Factor IX Gene Therapy in Non-human Primates
Valoctocogene Roxaparvovec AAV Gene Therapy for Patients with Hemophilia A: New Results
Looking Ahead at Gene Therapy for Hemophilia
