EAHAD Gene Therapy Working Group

EAHAD Gene Therapy Working Group

Listen as Wolfgang Miesbach, MD discusses "EAHAD Gene Therapy Working Group."


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CME Information

Activity Title

EAHAD Gene Therapy Working Group


Gene Therapy in Hemophilia

Accreditation Type

AMA PRA Category 1 Credit(s)™

Release Date

December 18, 2020

Expiration Date

December 17, 2021

Estimated Time to Complete Activity

15 minutes


Upon completion of the activity, participants should be able to:

  • Recognize key concerns and unknowns related to the future of gene therapy for hemophilia

Wolfgang Miesbach, MD
Goethe University Hospital
Frankfurt, Germany

David Lillicrap, MD, FRCPC
Queen’s University–Kingston, Canada

Johnny Mahlangu, BSc, MBBCh, MMed, FCPath
Faculty of the Health Sciences–University of the Witwatersrand and National Health Laboratory Service
Johannesburg, South Africa

Claire McLintock, MBChB, FRACP, FRCPA
Auckland City Hospital
Auckland, New Zealand

Glenn F. Pierce, MD, PhD
World Federation of Hemophilia (WFH)
United States of America

K. John Pasi, MB ChB, PhD, FRCP, FRCPath, FRCPCH
Barts and The London School of Medicine and Dentistry
Queen Mary University of London
London, United Kingdom

Steven W. Pipe, MD
University of Michigan
Ann Arbor, Michigan

Flora Peyvandi, MD, PhD
University of Milan
Milan, Italy

Alok Srivastava, MD, FRACP, FRCPA, FRCP
Christian Medical College
Vellore, India

Thierry VandenDriessche, PhD
Vrije University Brussels
Brussels, Belgium


  1. There are no fees for participating in and receiving credit for this activity.
  2. Review the activity objectives and CME/CE information.
  3. Complete the CME/CE activity
  4. Complete the CME/CE evaluation/attestation form, which provides each participant with the opportunity to comment on how participating in the activity will affect their professional practice; the quality of the instructional process; the perception of enhanced professional effectiveness; the perception of commercial bias; and his/her views on future educational needs.
  5. Credit documentation/reporting:
    • If you are requesting AMA PRA Category 1 Credits™ or a certificate of participation—your CME/CE certificate will be available for download.



This activity is jointly provided by The France Foundation and the International Society on Thrombosis and Haemostasis.

This activity is intended for physicians (hematologists), nurse practitioners, physician assistants, and nurses managing patients with hemophilia. The activity is also intended for scientists with an interest in basic, translational, and clinical research in hemophilia around the world.

As development of gene therapy for hemophilia continues into phase 3 clinical trials, and approval of this therapeutic approach is anticipated, it is essential for all members of the hemophilia care team to be knowledgeable and poised for the integration of this new therapeutic approach into clinical practice.

This activity has been planned and implemented in accordance with the accreditation requirements and policies of the Accreditation Council for Continuing Medical Education (ACCME) through the joint providership of The France Foundation (TFF) and the International Society on Thrombosis and Haemostasis (ISTH). TFF is accredited by the ACCME to provide continuing medical education for physicians.

The France Foundation designates this enduring activity for a maximum of 0.25 AMA PRA Category 1 Credit(s)™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Nurses: Nurses who are certified by the American Nurses Credentialing Center (ANCC) may utilize activities that are certified by ACCME-accredited providers toward their requirement for certification renewal by the ANCC. A certificate of attendance will be provided by The France Foundation, an ACCME accredited provider.

In accordance with the ACCME Standards for Commercial Support, The France Foundation (TFF) and the International Society of Thrombosis and Haemostasis (ISTH) require that individuals in a position to control the content of an educational activity disclose all relevant financial relationships with any commercial interest. TFF and ISTH resolve all conflicts of interest to ensure independence, objectivity, balance, and scientific rigor in all their educational programs. Furthermore, TFF and ISTH seek to verify that all scientific research referred to, reported, or used in a CME/CE activity conforms to the generally accepted standards of experimental design, data collection, and analysis. TFF and ISTH are committed to providing learners with high-quality CME/CE activities that promote improvements in healthcare and not those of a commercial interest.

Activity Staff Disclosures
The planners, reviewers, editors, staff, CME committee, or other members at TFF who control content have no relevant financial relationships to disclose.

The planners, reviewers, editors, staff, CME committee, or other members at the ISTH who control content have no relevant financial relationships to disclose.

Faculty Disclosures–Planners

The faculty listed below report that they have no relevant financial relationships to disclose:

  • Claire McLintock, MBChB, FRACP, FRCPA
  • Alok Srivastava, MD, FRACP, FRCPA, FRCP

The faculty listed below report that they have relevant financial relationships to disclose:

  • David Lillicrap, MD, FRCPC, receives honoraria for academic consulting from Bioverativ, CSL Behring, and Octapharma Plasma. He receives research funding from Bayer, BioMarin, Bioverativ, CSL Behring, and Octapharma Plasma.
  • Johnny Mahlangu, BSc, MBBCh, MMed, FCPath, serves on the speakers bureau and does contract research for CSL Behring, Catalyst Biosciences, Freeline Therapeutics, Novo Nordisk, Roche, Sanofi, Spark, and Takeda.
  • K. John Pasi, MChB, PhD, FRCP, FRCPath, FRCPCH, receives research funding from BioMarin GeneR8 programme, uniQure HOPE-B programme, and Sanofi–ATLAS fitusiran program. He receives honoraria from Alnylam, ApcinteX, BioMarin, Catalyst Bio, Chugai, Novo Nordisk, Octapharma Plasma, Pfizer, Roche, Sanofi, Shire, and Sobi.
  • Flora Peyvandi, MD, PhD, serves as a consultant for Sanofi and Sobi. She serves on the speakers bureau for Bioverativ, Grifols, Roche, Sanofi, Sobi, Spark, and Takeda.
  • Glenn F. Pierce, MD, PhD, receives honoraria for academic consulting from BioMarin, Genentech/Roche, Pfizer, St. Jude, and VarmX. He holds leadership positions with Global Blood Therapeutics, NHF MASAC, and World Federation of Hemophilia.
  • Steven W. Pipe, MD, serves as a consultant for ApcinteX, Bayer, BioMarin, Catalyst Biosciences, CSL Behring, HEMA Biologics, Freeline, Novo Nordisk, Pfizer, Roche/Genentech, Sangamo Therapeutics, Sanofi, Takeda, Spark Therapeutics, and uniQure. He does research for Siemens.
  • Thierry VandenDriessche, PhD, holds leadership positions with NHF and ISTH. He receives research funding from Pfizer and Takeda. Dr. VandenDriessche receives honoraria from Baxalta, Shire, Takeda, Bayer, Biotest, and Pfizer.

Faculty Disclosures–Activity Faculty
The following faculty report that they have relevant financial relationships to disclose:

  • Wolfgang Miesbach, MD, serves as a consultant for Alnylam, Bayer, Biogen Idec, Bioverativ, CSL Behring, BioMarin, Grifols, Freeline, LFB, Novo, Novartis, Noventis, Octopharma, Pfizer, Roche, Sanofi, Sobi, Takeda, and uniQure

TFF and the ISTH require CME faculty (speakers) to disclose when products or procedures being discussed are off label, unlabeled, experimental, and/or investigational. This includes any limitations on the information that is presented, such as data that are preliminary, or that represent ongoing research, interim analyses, and/or unsupported opinion. Faculty in this activity may discuss information about pharmaceutical agents that is outside of US Food and Drug Administration approved labeling. This information is intended solely for continuing medical education and is not intended to promote off-label use of these medications. TFF and the ISTH do not recommend the use of any agent outside of the labeled indications. If you have questions, contact the Medical Affairs Department of the manufacturer for the most recent prescribing information.

This activity is supported by educational grants from BioMarin, Freeline Therapeutics Limited, Pfizer Inc., Shire, Spark Therapeutics, and uniQure, Inc.

TFF and the ISTH present this information for educational purposes only. The content is provided solely by faculty who have been selected for their recognized expertise in their field. Participants have the professional responsibility to ensure that products are prescribed and used appropriately on the basis of their own clinical judgment and accepted standards of care. The France Foundation, the ISTH, and the commercial supporter(s) assume no liability for the information herein.

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Wolfgang Miesbach, MD

Wolfgang Miesbach, MD
Goethe University Hospital - Frankfurt, Germany

Wolfgang Miesbach, MD, is head of the Department of Coagulation Disorders and the Comprehensive Care Haemophilia Centre at the Goethe University Hospital in Frankfurt/Main, Germany. He is member of national and international scientific societies and has authored or co-authored about 120 peer-reviewed publications, review articles, or book chapters.

Professor Miesbach is on the editorial board of several scientific journals. His main research interest is in the field of quality of life investigations, elderly patients with haemophilia and von Willebrand disease, and new treatment options of haemophilia (e.g. gene therapy).