Highlights From the 30th Congress of ISTH
Elucidating the Mechanism Behind the AAV-Derived Factor VIII Assay Discrepancy
Relationship Between Transgene-Produced FVIII and Bleeding Rates 2 Years After Gene Transfer With Valoctocogene Roxaparvovec: Results From Gener8-1
Results from B-LIEVE, a Phase 1/2 Dose-Confirmation Study of FLT180a AAV Gene Therapy in Patients With Hemophilia B
Highlights from the ASGCT 25th Annual Meeting
Stable Hemostatic Correction and Improved Hemophilia-Related Quality of Life: Final Analysis from the Pivotal Phase 3 HOPE-B Trial of Etranacogene Dezaparvovec
Delivery of CRISPR/Cas9 mRNA LNPs to Repair a Small Deletion in FVIII Gene in Hemophilia A Mice
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
Stabilin-2 Promoter Modulates the Immune Response to FVIII After Lentiviral Vector Delivery in Hemophilic Mice
Highlights From The WFH 2022 World Congress
Using the World Federation of Haemophilia Gene Therapy Registry for the Long-term Follow-up of Haemophilia Patients Treated with Gene Therapy
Available for CME Credit!
Topic: Gene Therapy in Hemophilia
Accreditation Type: AMA PRA Category 1 Credit(s)™
Release Date: June 6, 2022
Expiration Date: June 5, 2023
Estimated Time to Complete Activity: 15 minutes
Highlights From the 15th Annual Congress of EAHAD
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
Efficacy and Safety of Valoctocogene Roxaparvovec Gene Transfer for Severe Haemophilia A: Results From the GENEr8-1 Two-Year Analysis
Steven W. Pipe, MD
Impact of Valoctocogene Roxaparvovec Gene Transfer for Severe Haemophilia A on Health-related Quality of Life
Johnny Mahlangu, BSc, MBBCh, MMed, FCPath
Highlights From NHF's 16th Workshop on Novel Technologies and Gene Transfer for Hemophilia
Highlights From the 63rd ASH Annual Meeting
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
Bleeding Data Across Baseline FIX Expression Levels in People With Hemophilia B: An Analysis Using the ‘Factor Expression Study
Follow-up of More Than 5 Years in a Cohort of Patients With Hemophilia B Treated With Fidanacogene Elaparvovec Adeno-Associated Virus Gene Therapy
Ben J. Samelson-Jones, MD, PhD
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
Pratima Chowdary, MRCP, FRCPath
Efficacy and Safety of Valoctocogene Roxaparvovec Adeno-associated Virus Gene Transfer for Severe Hemophilia A: Results From the Phase 3 GENEr8-1 Trial
Margareth C. Ozelo, MD, PhD
Liver Safety Case Report From the Phase 3 HOPE-B Gene Therapy Trial in Adults With Hemophilia B
Steven W. Pipe, MD
52 Week Efficacy and Safety of Etranacogene Dezaparvovec in Adults With Severe or Moderate-severe Hemophilia B: Data From the Phase 3 HOPE-B Gene Therapy Trial
Steven W. Pipe, MD
Investigation of Early Outcomes Following Adeno-associated Viral Gene Therapy in a Canine Hemophilia Model
David Lillicrap, MD, FRCPC
Clinical Outcomes in Adults With Hemophilia B With and Without Pre-existing Neutralizing Antibodies to AAV5: 6 Month Data From the Phase 3 Etranacogene Dezaparvovec HOPE-B Gene Therapy Trial
Wolfgang A. Miesbach, MD, PhD
Evolution of AAV Vector Gene Therapy Is Ongoing in Hemophilia. Will the Unique Features of BAY 2599023 Address the Outstanding Needs?
Steven W. Pipe, MD
Efficacy and Safety of Etranacogene Dezaparvovec in Adults With Severe or Moderate-severe Hemophilia B: First Data From the Phase 3 Hope-B Gene Therapy Trial
Steven W. Pipe, MD
Characterisation of Adeno-Associated Virus Vector Persistence After Long-Term Follow-up in the Haemophilia a Dog Model
Paul Batty, MBBS, PhD
Etranacogene Dezaparvovec (AAV5-Padua hFIX variant), an Enhanced Vector for Gene Transfer in Adults With Severe or Moderate-Severe Hemophilia B: Two Year Data From a Phase 2b Trial
Annette von Drygalski, MD, PharmD
AMT-060 Gene Therapy in Adults With Severe or Moderate-Severe Hemophilia B Confirm Stable FIX Expression and Durable Reductions in Bleeding and Factor IX Consumption for Up to 5 Years
Frank W.G. Leebeek, MD, PhD
Follow-up on a Novel Adeno-Associated Virus (AAV) Gene Therapy (FLT180a) Achieving Normal FIX Activity Levels in Severe Hemophilia B (HB) Patients (B-AMAZE Study)
Pratima Chowdary, MRCP, FRCPath
AMT-060 Gene Therapy in Adults With Severe or Moderate-Severe Hemophilia B Confirm Stable FIX Expression and Durable Reductions in Bleeding and Factor IX Consumption for Up to 5 Years
Wolfgang A. Miesbach, MD, PhD
First Data From the Phase 3 HOPE-B Gene Therapy Trial: Efficacy and Safety of Etranacogene Dezaparvovec (AAV5-Padua hFIX variant; AMT-061) in Adults With Severe or Moderate-Severe Hemophilia B Treated Irrespective of Pre-Existing Anti-Capsid Neutralizing
Steven W. Pipe, MD
Etranacogene Dezaparvovec (AAV5-Padua hFIX variant), an Enhanced Vector for Gene Transfer in Adults With Severe or Moderate-Severe Hemophilia B: Two Year Data From a Phase 2b Trial
Steven W. Pipe, MD
Updated Follow-up of the Alta Study, a Phase 1/2 Study of Giroctocogene Fitelparvovec (SB-525) Gene Therapy in Adults With Severe Hemophilia
Barbara A. Konkle, MD
ISTH Conference Briefings
Phase I/II Trial of SPK-8011: Stable and Durable FVIII Expression for > 2 Years With Significant ABR Improvements in Initial Dose Cohorts Following AAV-Mediated FVIII Gene Transfer for Hemophilia A
Lindsey A. George, MD
Long-term Vector Genome Outcomes and Immunogenicity of AAV FVIII Gene Transfer in the Hemophilia A Dog Model
Paul Batty, MBBS, PhD
A Novel Adeno Associated Virus (AAV) Gene Therapy (FLT180a) Achieves Normal FIX Activity Levels in Severe Hemophilia B (HB) Patients (B-AMAZE Study)
Pratima Chowdary, MRCP, FRCPath
Frequency, Location and Nature of AAV Vector Insertions After Long-term Follow-up of FVIII Transgene Delivery in a Hemophilia A Dog Model
Paul Batty, MBBS, PhD
WFH Conference Briefings
Recent Progress in the Development of AMT-061 (Etranacogene Dezaparvovec) for Persons With Severe or Moderately Severe Hemophilia B
Steven W. Pipe, MD
First-in-human Four-year Follow-up Study of Durable Therapeutic Efficacy and Safety: AAV Gene Therapy With Valoctocogene Roxaparvovec for Severe Haemophilia A
John Pasi, MB ChB, PhD, FRCP, FRCPath, FRCPCH
ASGCT Conference Briefings
First-in-human Gene Therapy Study of AAVhu37 Capsid Vector Technology in Severe Hemophilia A: Safety and FVIII Activity Results
Steven W. Pipe, MD
Utilising Apheresis to Remove Neutralizing AAV Antibodies in Patients Previous Excluded From Gene Therapy
Glenn F. Pierce, MD, PhD
Development of a Clinical Candidate AAV3 Vector for Gene Therapy of Hemophilia B
Alok Srivastava, FRACP, FRCPA, FRCP
AAV Integration Analysis After Long Term Follow-up in Hemophilia A Dogs Reveals the Genetic Consequences of AAV-Mediated Gene Correction
Glenn F. Pierce, MD, PhD
Conference Coverage from Orlando
Conference Coverage from Orlando, featuring expert interviews on the latest advances in Gene Therapy.
Conference Coverage – Live from Orlando 2019






