WFH Conference Briefings

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

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

Steven W. Pipe, MD

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

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

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ASGCT Conference Briefings

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

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

Steven W. Pipe, MD

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

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

Glen F. Pierce, MD, PhD

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

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

Alok Srivastava, FRACP, FRCPA, FRCP

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

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

Glen F. Pierce, MD, PhD

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Conference Coverage from Orlando

Conference Coverage from Orlando, featuring expert interviews on the latest advances in Gene Therapy.

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Conference Coverage – Live from Orlando 2019

Recent Updates on Gene Therapy Clinical Trials in Hemophilia B
Long term Safety and Efficacy Data from Gene Therapy Trials in Hemophilia Provide Optimism
Long term Expression of AAV mediated FVIII Gene Transfer and Vector Integration Events in Hemophilia
Novel Approaches to Hemophilia Gene Therapy in Preclinical Development
Preexisting Antibodies to AAV and Consequences for Hemophilia Gene Therapy
The Future of Hemophilia Gene Therapy
Preparing for the Approval of Gene Therapy for Hemophilia