Conference Coverage

Gene Therapy Implementation - A Conversation with Dr. Lishel Horn

Histopathological characterization of immune-mediated hepatocellular injury following gene therapy in hemophilia

Durable efficacy and safety in responders: 5-Year post hoc end-of-study analysis of etranacogene dezaparvovec in the phase 3 HOPE-B trial

Improved hFIX and hFVIII expression with ITR-modified AAV vectors in hemophilic mice

Evolution Directed Gene Engineered (EDGE) Gain-of-Function Factor VIII Variants Enhance Gene Therapy for Hemophilia A

Enhancing Global Collaboration: The Role of Data Governance in the WFH Gene Therapy Registry

Natural History of AAV5 Neutralizing Antibodies in Adults with Haemophilia B

Developing Evidence-Based Guidelines for AAV Gene Therapy in Hemophilia

Factor VIII Expression From a Novel F8 Transgene Through a Lentiviral Vector Transduced CD34+ Autologous Hematopoietic Stem Cells for Gene Therapy of Severe Hemophilia Α

Use of Fidanacogene Elaparvovec, a Gene Therapy Vector, to Deliver a Stable, Fully Functional Human Factor IX Transgene for the Treatment of Hemophilia B: A Combined Analysis of Safety

Efficacy and Safety of Giroctocogene Fitelparvovec in Adults With Moderately Severe to Severe Hemophilia Α

BeCoMe–9: A Phase 1/2 Dose Escalation and Expansion Study of be-101 for the Treatment of Adults With Moderately Severe or Severe Hemophilia B

Subcategories

Highlights From the 17th Annual Congress of EAHAD

Highlights From the 65th ASH Annual Meeting and Exposition

Highlights From the ESGCT 30th Annual Congress

Highlights From the 31st Congress of ISTH

Highlights From the 32nd Congress of ISTH

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

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