Highlights From the 64th ASH Annual Meeting and Exposition
Updates on Three Ongoing Clinical Trials on Adults with Severe Hemophilia B
Presented by: Steven W. Pipe, MD, University of Michigan, Ann Arbor, Michigan, United States
Use the tabs below to review additional information about the abstracts discussed in this video.
Abstracts:
- Durability of Bleeding Protection and Factor IX Activity Levels Are Demonstrated in Individuals With and Without Adeno-associated Virus Serotype 5 Neutralizing Antibodies (Titers < 1:700) With Comparable Safety in the Phase 3 HOPE-B Clinical Trial of Etranacogene Dezaparvovec Gene Therapy for Hemophilia B
- Adults With Severe or Moderately Severe Hemophilia B Receiving Etranacogene Dezaparvovec in the HOPE-B Phase 3 Clinical Trial Continue to Experience a Stable Increase in Mean Factor IX Activity Levels and Durable Hemostatic Protection After 24 Months’ Follow-up
- Durability of Factor IX Activity and Bleeding Rate in People With Severe or Moderately Severe Hemophilia B After 5 Years of Follow-up in the Phase 1/2 Study of AMT-060, and After 3 Years of Follow-up in the Phase 2b and 2 Years of Follow-up in the Phase 3 Studies of Etranacogene Dezaparvovec (AMT-061)
RELATED CONTENT
Interactive Webinars
Long-term Outcomes: Durability and Safety
Presented by Prof. Margareth C. Ozelo, MD, PhD...
Obstacles and Opportunities
Presented by Frank W.G. Leebeek, MD, PhD...
Patient Support, Patient Counseling, and Monitoring
Presented by Lindsey A. George, MD...
Gene Therapy for FVIII
Presented by K. John Pasi, MB ChB, PhD, FRCP, FRCPath, FRCPC...
Update on Efficacy of Clinical Trials
Presented by Guy Young, MD...
Adeno-Associated Viral (AAV) Vector Gene Therapy: Application to Hemophilia
Presented by Barbara A. Konkle, MD...
Gene Therapy for Treatment of Hemophilia: An Introduction to Adeno-Associated Viral Vector Gene Transfer
Presented by Johnny Mahlangu, BSc, MBBCh, MMed, FCPath...
Gene Therapy for Treatment of Hemophilia: Common Concerns in Gene Therapy
Presented by Thierry VandenDriessche, PhD...
Gene Therapy for Treatment of Hemophilia: Other Strategies and Targets
Presented by Glenn F. Pierce, MD, PhD...
A History of Hemophilia Treatment: Non-replacement Therapy to Gene Therapy
Presented by Steven W. Pipe, MD...
Getting to Know Gene Therapy: Terminology and Concepts
Presented by David Lillicrap, MD...
Podcasts