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

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

Highlights from the NHF 15th Workshop on Novel Technologies and Gene Transfer for Hemophilia

Conference Coverage

A History of Hemophilia Treatment: Non-replacement Therapy to Gene Therapy

Interactive Webinars

Gene Therapy for Treatment of Hemophilia: Other Strategies and Targets

Interactive Webinars

Getting to Know Gene Therapy: Terminology and Concepts

Interactive Webinars

A History of Hemophilia Treatment: Factor Replacement to Gene Therapy

Interactive Webinars

Subcategories

Monthly Roundup

Interactive Webinars

Podcasts

Conference Coverage

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

Gene Therapy Illustrated Clinical Trials

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

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