Education

Hemophilia is a congenital hemorrhagic condition caused by mutations in coagulation factors VIII or IX. The current standard therapy for bleeding prevention begins in childhood with frequent intravenous injections of coagulation factor concentrates. Although this procedure helps with bleeding management, it is also time-consuming and requires a lifetime of painful needle sticks.

The World Federation of Hemophilia Gene Therapy Registry

Conference Coverage

Updated Follow-up of the Alta Study, a Phase 1/2 Study of Giroctocogene Fitelparvovec (SB-525) Gene Therapy in Adults With Severe Hemophilia

Conference Coverage

Latest Advances in Gene Therapy for Hemophilia in Portuguese

Podcasts

Obstacles and Opportunities

Interactive Webinars

Key Considerations: Advances in Gene Therapy for Hemophilia

Interactive Webinars

Stepping Into the Future: Hemophilia Gene Therapy Clinical Trial Updates

Interactive Webinars

Patient Support, Patient Counseling, and Monitoring

Interactive Webinars

Gene Therapy for FVIII

Interactive Webinars

Learning from Clinical Trials and Preparing for Clinical Practice

Interactive Webinars

Frequency, Location and Nature of AAV Vector Insertions After Long-term Follow-up of FVIII Transgene Delivery in a Hemophilia A Dog Model

Conference Coverage

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

Conference Coverage

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

Conference Coverage

Long-term Vector Genome Outcomes and Immunogenicity of AAV FVIII Gene Transfer in the Hemophilia A Dog Model

Conference Coverage

Update on Efficacy of Clinical Trials

Interactive Webinars

Recent Progress in the Development of AMT-061 (Etranacogene Dezaparvovec) for Persons With Severe or Moderately Severe Hemophilia B

Conference Coverage

First-in-human Four-year Follow-up Study of Durable Therapeutic Efficacy and Safety: AAV Gene Therapy With Valoctocogene Roxaparvovec for Severe Haemophilia A

Conference Coverage

First-in-human Gene Therapy Study of AAVhu37 Capsid Vector Technology in Severe Hemophilia A: Safety and FVIII Activity Results

Conference Coverage

Utilising Apheresis to Remove Neutralizing AAV Antibodies in Patients Previous Excluded From Gene Therapy

Conference Coverage

Development of a Clinical Candidate AAV3 Vector for Gene Therapy of Hemophilia B

Conference Coverage

Development of Coagulation Factor VIII Transgenes That Confer Greater Potency to Gene Therapies for Hemophilia A Through Ancestral Sequence Reconstruction

Conference Coverage

AAV Integration Analysis After Long Term Follow-up in Hemophilia A Dogs Reveals the Genetic Consequences of AAV-Mediated Gene Correction

Conference Coverage

Adeno-Associated Viral (AAV) Vector Gene Therapy: Application to Hemophilia

Interactive Webinars

Gene Therapy for Treatment of Hemophilia: An Introduction to Adeno-Associated Viral Vector Gene Transfer

Interactive Webinars

Gene Therapy for Treatment of Hemophilia: Common Concerns in Gene Therapy

Interactive Webinars
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Supported through educational grants from BioMarin, CSL Behring, Pfizer Inc., Roche, and Spark Therapeutics

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