Highlights From the 30th Congress of ISTH

CME INFORMATION

Elucidating the Mechanism Behind the AAV-Derived Factor VIII Assay Discrepancy

Elucidating the Mechanism Behind the AAV-Derived Factor VIII Assay Discrepancy

Anna Sternberg, PhD

Relationship Between Transgene-Produced FVIII and Bleeding Rates 2 Years After Gene Transfer With Valoctocogene Roxaparvovec: Results From Gener8-1

Relationship Between Transgene-Produced FVIII and Bleeding Rates 2 Years After Gene Transfer With Valoctocogene Roxaparvovec: Results From Gener8-1

Johnny Mahlangu, BSc, MBBCh, MMed, FCPath

Results from B-LIEVE, a Phase 1/2 Dose-Confirmation Study of FLT180a AAV Gene Therapy in Patients With Hemophilia B

Results from B-LIEVE, a Phase 1/2 Dose-Confirmation Study of FLT180a AAV Gene Therapy in Patients With Hemophilia B

Guy Young, MD

Improvements in Health-Related Quality of Life in Adults With Severe or Moderately Severe Hemophilia B After Receiving Etranacogene Dezaparvovec Gene Therapy

Improvements in Health-Related Quality of Life in Adults With Severe or Moderately Severe Hemophilia B After Receiving Etranacogene Dezaparvovec Gene Therapy

Steven W. Pipe, M

Highlights from the ASGCT 25th Annual Meeting

CME INFORMATION

Stable Hemostatic Correction and Improved Hemophilia-Related Quality of Life: Final Analysis from the Pivotal Phase 3 HOPE-B Trial of Etranacogene Dezaparvovec

Stable Hemostatic Correction and Improved Hemophilia-Related Quality of Life: Final Analysis from the Pivotal Phase 3 HOPE-B Trial of Etranacogene Dezaparvovec

Steven W. Pipe, MD

Delivery of CRISPR/Cas9 mRNA LNPs to Repair a Small Deletion in FVIII Gene in Hemophilia A Mice

Delivery of CRISPR/Cas9 mRNA LNPs to Repair a Small Deletion in FVIII Gene in Hemophilia A Mice

Chun-Yu Chen, PhD

A Factor VIII Variant With Elimination of the N-glycosylation Site at 2118 Reduced Anti-FVIII Immune Responses in Gene Therapy Treated Hemophilia A Mice

A Factor VIII Variant With Elimination of the N-glycosylation Site at 2118 Reduced Anti-FVIII Immune Responses in Gene Therapy Treated Hemophilia A Mice

Carol H. Miao, MD

Stabilin-2 Promoter Modulates the Immune Response to FVIII After Lentiviral Vector Delivery in Hemophilic Mice

Stabilin-2 Promoter Modulates the Immune Response to FVIII After Lentiviral Vector Delivery in Hemophilic Mice

Antonia Follenzi, MD, PhD

Highlights From The WFH 2022 World Congress

Using the World Federation of Haemophilia Gene Therapy Registry for the Long-term Follow-up of Haemophilia Patients Treated with Gene Therapy

Using the World Federation of Haemophilia Gene Therapy Registry for the Long-term Follow-up of Haemophilia Patients Treated with Gene Therapy

Barbara Konkle, MD

Available for CME Credit!

Topic: Gene Therapy in Hemophilia
Accreditation Type: AMA PRA Category 1 Credit(s)™
Release Date: June 6, 2022
Expiration Date: June 5, 2023
Estimated Time to Complete Activity: 15 minutes

CME INFORMATION

Highlights From the 15th Annual Congress of EAHAD

Dose Selection and Study Design for B-LIEVE, A Phase 1/2 Dose Confirmation Clinical Trial of FLT180A Gene Therapy for Patients with Hemophilia B

Dose Selection and Study Design for B-LIEVE, A Phase 1/2 Dose Confirmation Clinical Trial of FLT180A Gene Therapy for Patients with Hemophilia B

Pratima Chowdary, MRCP, FRCPath

Final Analysis From the Pivotal Phase 3 HOPE-B Gene Therapy Trial: Stable Steady-state Efficacy and Safety of Etranacogene Dezaparvovec in Adults With Severe or Moderately Severe Hemophilia B

Final Analysis From the Pivotal Phase 3 HOPE-B Gene Therapy Trial

Wolfgang Miesbach, MD

Efficacy and Safety of Valoctocogene Roxaparvovec Gene Transfer for Severe Haemophilia A: Results From the GENEr8-1 Two-Year Analysis

Efficacy and Safety of Valoctocogene Roxaparvovec Gene Transfer for Severe Haemophilia A: Results From the GENEr8-1 Two-Year Analysis

Steven W. Pipe, MD

Impact of Valoctocogene Roxaparvovec Gene Transfer for Severe Haemophilia A on Health-related Quality of Life

Impact of Valoctocogene Roxaparvovec Gene Transfer for Severe Haemophilia A on Health-related Quality of Life

Johnny Mahlangu, BSc, MBBCh, MMed, FCPath

Highlights From NHF's 16th Workshop on Novel Technologies and Gene Transfer for Hemophilia

AAV integration summary and implications

AAV Integration Summary and Implications

Frederic D. Bushman, PhD

Breaking transgene tolerance via immunosuppression

Breaking Transgene Tolerance via Immunosuppression

Valder R. Arruda, MD, PhD

Targeting Sinusoial Endothelium

Targeting Sinusoial Endothelium

Antonia Follenzi, MD, PhD

Highlights From the 63rd ASH Annual Meeting

A Post-Hoc Analysis of the GENEr8-1 Phase 3 Trial

Relationship Between Endogenous, Transgene FVIII Expression and Bleeding Events Following Valoctocogene Roxaparvovec Gene Transfer for Severe Hemophilia A: A Post-Hoc Analysis of the GENEr8-1 Phase 3 Trial

Steven W. Pipe, MD

Bleeding Data Across Baseline FIX Expression Levels in People With Hemophilia B: An Analysis Using the ‘Factor Expression Study

Bleeding Data Across Baseline FIX Expression Levels in People With Hemophilia B: An Analysis Using the ‘Factor Expression Study

Steven W. Pipe, MD

Follow-up of More Than 5 Years in a Cohort of Patients With Hemophilia B Treated With Fidanacogene Elaparvovec Adeno-Associated Virus Gene Therapy

Follow-up of More Than 5 Years in a Cohort of Patients With Hemophilia B Treated With Fidanacogene Elaparvovec Adeno-Associated Virus Gene Therapy

Ben J. Samelson-Jones, MD, PhD

Factor IX Expression Within the Normal Range Prevents Spontaneous Bleeds Requiring Treatment Following FLT180a Gene Therapy in Patients With Severe Hemophilia B: Long-term Follow-up Study of the B-Amaze Program

Factor IX Expression Within the Normal Range Prevents Spontaneous Bleeds Requiring Treatment Following FLT180a Gene Therapy in Patients With Severe Hemophilia B: Long-term Follow-up Study of the B-Amaze Program

Pratima Chowdary, MRCP, FRCPath

Highlights From the ESGCT 28th Annual Congress

Monitoring of Vector Integration Sites in In Vivo Gene Therapy Approaches by Liquid-Biopsy-Integration-Site Sequencing

Monitoring of Vector Integration Sites in In Vivo Gene Therapy Approaches by Liquid-Biopsy-Integration-Site Sequencing

Daniela Cesana, PhD

Overcoming anti-AAV pre-existing immunity to achieve safe and efficient gene transfer in clinical settings.

Overcoming Anti-AAV Pre-existing Immunity to Achieve Safe and Efficient Gene Transfer in Clinical Settings.

Giuseppe Ronzitti, PhD

Lentiviral Gene Therapy to the Liver for Hemophilia and Beyond

Lentiviral Gene Therapy to the Liver for Hemophilia and Beyond

Alessio Cantore, PhD

Current Update of Clinical trials on Gene Therapy for Hemophilia

Current Update of Clinical trials on Gene Therapy for Hemophilia

Wolfgang Miesbach, MD

Highlights From the ISTH 2021 Congress

CME INFORMATION

Efficacy and Safety of Valoctocogene Roxaparvovec Adeno-associated Virus Gene Transfer for Severe Hemophilia A: Results From the Phase 3 GENEr8-1 Trial

Efficacy and Safety of Valoctocogene Roxaparvovec Adeno-associated Virus Gene Transfer for Severe Hemophilia A: Results From the Phase 3 GENEr8-1 Trial

Margareth C. Ozelo, MD, PhD

Liver Safety Case Report From the Phase 3 HOPE-B Gene Therapy Trial in Adults With Hemophilia B

Liver Safety Case Report From the Phase 3 HOPE-B Gene Therapy Trial in Adults With Hemophilia B

Steven W. Pipe, MD

52 Week Efficacy and Safety of Etranacogene Dezaparvovec in Adults With Severe or Moderate-severe Hemophilia B: Data From the Phase 3 HOPE-B Gene Therapy Trial

52 Week Efficacy and Safety of Etranacogene Dezaparvovec in Adults With Severe or Moderate-severe Hemophilia B: Data From the Phase 3 HOPE-B Gene Therapy Trial

Steven W. Pipe, MD

Investigation of Early Outcomes Following Adeno-associated Viral Gene Therapy in a Canine Hemophilia Model

Investigation of Early Outcomes Following Adeno-associated Viral Gene Therapy in a Canine Hemophilia Model

David Lillicrap, MD, FRCPC

Clinical Outcomes in Adults With Hemophilia B With and Without Pre-existing Neutralizing Antibodies to AAV5: 6 Month Data From the Phase 3 Etranacogene Dezaparvovec HOPE-B Gene Therapy Trial

Clinical Outcomes in Adults With Hemophilia B With and Without Pre-existing Neutralizing Antibodies to AAV5: 6 Month Data From the Phase 3 Etranacogene Dezaparvovec HOPE-B Gene Therapy Trial

Wolfgang A. Miesbach, MD, PhD

Evolution of AAV Vector Gene Therapy Is Ongoing in Hemophilia. Will the Unique Features of BAY 2599023 Address the Outstanding Needs?

Evolution of AAV Vector Gene Therapy Is Ongoing in Hemophilia. Will the Unique Features of BAY 2599023 Address the Outstanding Needs?

Steven W. Pipe, MD

Highlights From the EAHAD 2021 Virtual Congress

CME INFORMATION

Efficacy and Safety of Etranacogene Dezaparvovec in Adults With Severe or Moderate-severe Hemophilia B: First Data From the Phase 3 Hope-B Gene Therapy Trial

Efficacy and Safety of Etranacogene Dezaparvovec in Adults With Severe or Moderate-severe Hemophilia B: First Data From the Phase 3 Hope-B Gene Therapy Trial

Steven W. Pipe, MD

Characterisation of Adeno-Associated Virus Vector Persistence After Long-Term Follow-up in the Haemophilia a Dog Model

Characterisation of Adeno-Associated Virus Vector Persistence After Long-Term Follow-up in the Haemophilia a Dog Model

Paul Batty, MBBS, PhD

Etranacogene Dezaparvovec (AAV5-Padua hFIX variant), an Enhanced Vector for Gene Transfer in Adults With Severe or Moderate-Severe Hemophilia B: Two Year Data From a Phase 2b Trial

Etranacogene Dezaparvovec (AAV5-Padua hFIX variant), an Enhanced Vector for Gene Transfer in Adults With Severe or Moderate-Severe Hemophilia B: Two Year Data From a Phase 2b Trial

Annette von Drygalski, MD, PharmD

AMT-060 Gene Therapy in Adults With Severe or Moderate-Severe Hemophilia B Confirm Stable FIX Expression and Durable Reductions in Bleeding and Factor IX Consumption for Up to 5 Years

AMT-060 Gene Therapy in Adults With Severe or Moderate-Severe Hemophilia B Confirm Stable FIX Expression and Durable Reductions in Bleeding and Factor IX Consumption for Up to 5 Years

Frank W.G. Leebeek, MD, PhD

Follow-up on a Novel Adeno-Associated Virus (AAV) Gene Therapy (FLT180a) Achieving Normal FIX Activity Levels in Severe Hemophilia B (HB) Patients (B-AMAZE Study)

Follow-up on a Novel Adeno-Associated Virus (AAV) Gene Therapy (FLT180a) Achieving Normal FIX Activity Levels in Severe Hemophilia B (HB) Patients (B-AMAZE Study)

Pratima Chowdary, MRCP, FRCPath

ASH Conference Briefings

EARN CME CREDIT

First-in-Human Gene Therapy Study of AAVhu37 Capsid Vector Technology in Severe Hemophilia A – BAY 2599023 has Broad Patient Eligibility and Stable and Sustained Long-Term Expression of FVIII

First-in-Human Gene Therapy Study of AAVhu37 Capsid Vector Technology in Severe Hemophilia A – BAY 2599023 has Broad Patient Eligibility and Stable and Sustained Long-Term Expression of FVIII

Steven W. Pipe, MD

The World Federation of Hemophilia Gene Therapy Registry

The World Federation of Hemophilia Gene Therapy Registry

Barbara A. Konkle, MD

AMT-060 Gene Therapy in Adults With Severe or Moderate-Severe Hemophilia B Confirm Stable FIX Expression and Durable Reductions in Bleeding and Factor IX Consumption for Up to 5 Years

AMT-060 Gene Therapy in Adults With Severe or Moderate-Severe Hemophilia B Confirm Stable FIX Expression and Durable Reductions in Bleeding and Factor IX Consumption for Up to 5 Years

Wolfgang A. Miesbach, MD, PhD

First Data From the Phase 3 HOPE-B Gene Therapy Trial: Efficacy and Safety of Etranacogene Dezaparvovec (AAV5-Padua hFIX variant; AMT-061) in Adults With Severe or Moderate-Severe Hemophilia B Treated Irrespective of Pre-Existing Anti-Capsid Neutralizing

First Data From the Phase 3 HOPE-B Gene Therapy Trial: Efficacy and Safety of Etranacogene Dezaparvovec (AAV5-Padua hFIX variant; AMT-061) in Adults With Severe or Moderate-Severe Hemophilia B Treated Irrespective of Pre-Existing Anti-Capsid Neutralizing

Steven W. Pipe, MD

Etranacogene Dezaparvovec (AAV5-Padua hFIX variant), an Enhanced Vector for Gene Transfer in Adults With Severe or Moderate-Severe Hemophilia B: Two Year Data From a Phase 2b Trial

Etranacogene Dezaparvovec (AAV5-Padua hFIX variant), an Enhanced Vector for Gene Transfer in Adults With Severe or Moderate-Severe Hemophilia B: Two Year Data From a Phase 2b Trial

Steven W. Pipe, MD

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

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

Barbara A. Konkle, MD

ISTH Conference Briefings

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

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

Lindsey A. George, 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

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

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)

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

Pratima Chowdary, MRCP, FRCPath

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

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

Paul Batty, MBBS, PhD

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

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

ASGCT Conference Briefings

EARN CME CREDIT

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

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

Glenn F. Pierce, MD, PhD

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

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

Glenn F. Pierce, MD, PhD

Conference Coverage from Orlando

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

EARN CME CREDIT

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

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

Presented by: David Lillicrap, MD and Glenn F. Pierce, MD, PhD
September 13-14, 2019 • Washington, DC

Conference Coverage from ISTH 2019 in Melbourne

Conference Coverage from ISTH 2019 in Melbourne, featuring expert interviews on the latest advances in Gene Therapy.

EARN CME CREDIT

Conference Coverage – ISTH 2019 in Melbourne

The ALTA Study: SB-525 Gene Therapy in Adults with Hemophilia A

Prevalence of Preexisting AAV Antibodies and Implications for Gene Therapy in Hemophilia

Results of the AMT-060 and AMT-061 Gene Therapy Trials in Patients with Hemophilia B

Initial Results of the Phase 1/2 Alta Study: SB-525 Gene Therapy in Adults with Hemophilia A

One Year Follow-up of Patients with Hemophilia B Following SPK-9001 Gene Transfer

AMT-061 Gene Transfer in Adults with Hemophilia B: Early Results of a Phase 2b Trial

Current Status of Gene Therapy for Hemophilia

FLT180a: Next Generation AAV Vector for Hemophilia B

Stable Expression of FIX: Updated Results of the AMT-060 Gene Therapy Trial in Patients with Hemophilia B.

Lentiviral Vector-based Factor VIII and Factor IX Gene Therapy in Non-human Primates

Valoctocogene Roxaparvovec AAV Gene Therapy for Patients with Hemophilia A: New Results

Looking Ahead at Gene Therapy for Hemophilia

Person on the Street - ISTH 2019 in Melbourne

Watch as attendees from the ISTH 2019 Congress in Melbourne, Australia, discuss their reactions to the session on Gene Therapy in Hemophilia.

Product Theater- ISTH 2019 Melbourne

Watch as Flora Peyvandi, MD, PhD and K. John Pasi, MB ChB, PhD, FRCP, FRCPath, FRCPCH announce the launch of Gene Therapy in Hemophilia: An ISTH Education Initiative

Baseline Survey Poster

Poster

Poster Presented at the ISTH 2019 Congress
Sunday, July 7 • 18:30 – 19:30
Abstract/Poster #: PB1724
Melbourne, Australia

In early 2019, ISTH organized a group of world-renowned experts from the global hemophilia community to develop a survey to identify unmet educational needs specific to gene therapy in hemophilia. The survey was distributed online to an international audience. The results demonstrated that many need more education on the fundamentals of gene therapy and a better understanding of gene therapy as a treatment approach for hemophilia A and B.

Educational Roadmap

ISTH's Educational Roadmap–Guiding Education for the Future
Preparing You to Answer These Critical Questions

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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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