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Education

Gene Therapy for FVIII

Interactive Webinars

Presented by K. John Pasi, MB ChB, PhD, FRCP, FRCPath, FRCPC

Learning from Clinical Trials and Preparing for Clinical Practice

Virtual Learning Sessions

Presented by Steven W. Pipe, MD and Guy Young, MD

HTC Readiness: A Global Perspective

Podcasts

Presented by Steven W. Pipe, MD and Debra Pollard, RN

HTC Readiness: A Multidisciplinary Perspective

Podcasts

Presented by Steven W. Pipe, MD and Debra Pollard, RN

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

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)

Conference Coverage

Pratima Chowdary, MRCP, FRCPath

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

Presented by Lindsey A. George, MD

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

Conference Coverage

Paul Batty, MBBS, PhD

Update on Efficacy of Clinical Trials

Interactive Webinars

Presented by Guy Young, MD

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

Conference Coverage

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

Conference Coverage

John Pasi, MB ChB, PhD, FRCP, FRCPath, FRCPCH

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

Conference Coverage

Steven W. Pipe, MD

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

Conference Coverage

Glenn F. Pierce, MD, PhD

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

Conference Coverage

Alok Srivastava, MD, FRACP, FRCPA, FRCP

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

Conference Coverage

H. Trent Spencer, PhD

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

Conference Coverage

Glenn F. Pierce, MD, PhD

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

Interactive Webinars

Presented by Barbara A. Konkle, MD

What Do Hemophilia Treatment Centers Need to Consider Now in Preparation for the Anticipated Availability of Gene Therapy?

Podcasts

Presented by Steven W. Pipe, MD and Nigel Key, MB, ChB. FRCP

Interpreting Vector Doses in Hemophilia Gene Therapy Clinical Trials

Podcasts

Presented by Steven W. Pipe, MD and Nigel Key, MB, ChB. FRCP

Hemophilia Gene Therapy in Patients With History of Inhibitors

Podcasts

Presented by Steven W. Pipe, MD and Nigel Key, MB, ChB. FRCP

Laboratory Measurement of Hemophilia Gene Therapy Products/Factor Activity

Podcasts

Presented by Steven W. Pipe, MD and Nigel Key, MB, ChB. FRCP

Adeno-Associated Virus (AAV) Mediated Gene Transfer: Why is This the Predominant Strategy for Hemophilia Gene Therapy?

Podcasts

Presented by Steven W. Pipe, MD and Nigel Key, MB, ChB. FRCP

Part 1: Pre-existing Immunity Against AAV Serotypes and What This Means for Hemophilia Gene Therapy Clinical Trials

Podcasts

Presented by Steven W. Pipe, MD and Nigel Key, MB, ChB. FRCP

Part 2: Pre-existing Immunity Against AAV Serotypes and What This Means for Hemophilia Gene Therapy Clinical Trials

Podcasts

Presented by Steven W. Pipe, MD and Nigel Key, MB, ChB. FRCP

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Interactive Webinars

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Supported by educational grants from Bayer, BioMarin, CSL Behring, Freeline Therapeutics Limited, Pfizer Inc., Spark Therapeutics, and uniQure, Inc.

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