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Education

Stable Hemostatic Correction and Improved Hemophilia-Related Quality of Life: Final Analysis from the Pivotal Phase 3 HOPE-B Trial of Etranacogene Dezaparvovec

Conference Coverage

Steven W. Pipe, MD

Delivery of CRISPR/Cas9 mRNA LNPs to Repair a Small Deletion in FVIII Gene in Hemophilia A Mice

Conference Coverage

Chun-Yu Chen, PhD

Stabilin-2 Promoter Modulates the Immune Response to FVIII After Lentiviral Vector Delivery in Hemophilic Mice

Conference Coverage

Antonia Follenzi, MD, PhD

A Factor VIII Variant With Elimination of the N-glycosylation Site at 2118 Reduced Anti-FVIII Immune Responses in Gene Therapy Treated Hemophilia A Mice

Conference Coverage

Carol H. Miao, MD

Using the World Federation of Haemophilia Gene Therapy Registry for the Long-term Follow-up of Haemophilia Patients Treated with Gene Therapy

Conference Coverage

Barbara A. Konkle, MD

Impact of Valoctocogene Roxaparvovec Gene Transfer for Severe Haemophilia A on Health-related Quality of Life

Conference Coverage

Johnny Mahlangu, BSc, MBBCh, MMed, FCPath

Dose Selection and Study Design for B-LIEVE, A Phase 1/2 Dose Confirmation Clinical Trial of FLT180A Gene Therapy for Patients with Hemophilia B

Conference Coverage

Pratima Chowdary, MRCP, FRCPath

Final Analysis From the Pivotal Phase 3 HOPE-B Gene Therapy Trial: Stable Steady-state Efficacy and Safety of Etranacogene Dezaparvovec in Adults With Severe or Moderately Severe Hemophilia B

Conference Coverage

Wolfgang Miesbach, MD

Efficacy and Safety of Valoctocogene Roxaparvovec Gene Transfer for Severe Haemophilia A: Results From the GENEr8-1 Two-Year Analysis

Conference Coverage

Steven W. Pipe, MD

Breaking Transgene Tolerance via Immunosuppression

Conference Coverage

Valder R. Arruda, MD, PhD

AAV Integration Summary and Implications

Conference Coverage

Frederic D. Bushman, PhD

Relationship Between Endogenous, Transgene FVIII Expression and Bleeding Events Following Valoctocogene Roxaparvovec Gene Transfer for Severe Hemophilia A: A Post-Hoc Analysis of the GENEr8-1 Phase 3 Trial

Conference Coverage

Steven W. Pipe, MD

Bleeding Data Across Baseline FIX Expression Levels in People With Hemophilia B: An Analysis Using the ‘Factor Expression Study

Conference Coverage

Steven W. Pipe, MD

Targeting Sinusoial Endothelium

Conference Coverage

Valder R. Arruda, MD, PhD

Follow-up of More Than 5 Years in a Cohort of Patients with Hemophilia B Treated With Fidanacogene Elaparvovec Adeno-Associated Virus Gene Therapy

Conference Coverage

Steven W. Pipe, MD

Factor IX Expression Within the Normal Range Prevents Spontaneous Bleeds Requiring Treatment Following FLT180a Gene Therapy in Patients With Severe Hemophilia B: Long-term Follow-up Study of the B-Amaze Program

Conference Coverage

Pratima Chowdary, MRCP, FRCPath

Gene Therapy as a Global Solution

Podcasts

Presented by Daniel Hart, BSc, MBChB, MRCP, FRCPath, PhD and Johnny Mahlangu, BSc, MBBCh, MMed, FCPath

 

Preparing Treatment Centers for Administration

Podcasts

Presented by Daniel Hart, BSc, MBChB, MRCP, FRCPath, PhD and Wolfgang A. Miesbach, MD, PhD

Patient Perspectives for Hemophilia in Gene Therapy

Podcasts

Presented by Daniel Hart, BSc, MBChB, MRCP, FRCPath, PhD and Declan Noone

Gene Therapy for Hemophilia A and B Compared to Available Treatments

Podcasts

Presented by Daniel Hart, BSc, MBChB, MRCP, FRCPath, PhD and Maria Elisa Mancuso, MD, PhD

Gene Therapy in Hemophilia Ongoing and Future Research and What to Expect

Podcasts

Presented by Daniel Hart, BSc, MBChB, MRCP, FRCPath, PhD and Dr. Steven W. Pipe, MD

Importance of Monitoring Individuals With Hemophilia for Safety and Efficacy in the Short Term and Long Term

Podcasts

Presented by Daniel Hart, BSc, MBChB, MRCP, FRCPath, PhD and Pratima Chowdary, MRCP, FRCPath

Monitoring of Vector Integration Sites in In Vivo Gene Therapy Approaches by Liquid-Biopsy-Integration-Site Sequencing

Conference Coverage

Daniela Cesana, PhD

Overcoming anti-AAV pre-existing immunity to achieve safe and efficient gene transfer in clinical settings

Conference Coverage

Giuseppe Ronzitti, PhD

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Supported by educational grants from Bayer, BioMarin, Freeline Therapeutics Limited, Pfizer Inc., Shire, Spark Therapeutics, and uniQure, Inc.

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