Gene Therapy for Hemophilia A and B Compared to Available Treatments
Listen as Dr. Daniel Hart from Barts and The London School of Medicine and Dentistry and Dr. Maria Elisa Mancuso from the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center discuss gene therapy for hemophilia A and B compared to available treatments.
This podcast is part of a comprehensive educational resource designed by leading experts for the global hemophilia community to help you stay abreast of the evolving science and latest clinical advancements in gene therapy in hemophilia. Visit www.genetherapy.isth.org for more information.
Gene Therapy for Hemophilia A and B Compared to Available Treatments
Gene Therapy in Hemophilia
AMA PRA Category 1 Credit(s)™
December 6, 2021
December 5, 2022
Estimated Time to Complete Activity
Upon completion of the activity, participants should be able to:
- Describe gene therapy correctly, including basic terms and concepts
- Discuss up-to-date information on the current and emerging approaches for treating hemophilia, including the various approaches of gene therapy
- Review the pros and cons of gene therapy with patients and caregivers, including available resources for further education
Daniel Hart, BSc, MBChB, MRCP, FRCPath, PhD
Barts and The London School of Medicine and Dentistry
Maria Elisa Mancuso, MD, PhD
Angelo Bianchi Bonomi Hemophilia and Thrombosis Center
David Lillicrap, MD, FRCPC
Johnny Mahlangu, BSc, MBBCh, MMed, FCPath
Faculty of the Health Sciences–University of the Witwatersrand and National Health Laboratory Service
Johannesburg, South Africa
Wolfgang A. Miesbach, MD, PhD
Goethe University Hospital
Prof. Discipline of Hematology and Transfusion Medicine
Department of Internal Medicine
School of Medical Sciences, University of Campinas
Hemocentro UNICAMP, Brazil
Campinas, São Paulo, Brazil
Glenn F. Pierce, MD, PhD
World Federation of Hemophilia (WFH)
United States of America
Steven W. Pipe, MD
University of Michigan
Ann Arbor, Michigan
Flora Peyvandi, MD, PhD
University of Milan
Alok Srivastava, MD, FRACP, FRCPA, FRCP
Christian Medical College
Thierry VandenDriessche, PhD
Vrije University Brussels
METHOD OF PARTICIPATION / HOW TO RECEIVE CREDIT
- There are no fees for participating in and receiving credit for this activity.
- Review the activity objectives and CME/CE information.
- Complete the CME/CE activity
- 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.
- 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.
STATEMENT OF NEED
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.
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.
The faculty listed below report that they have no relevant financial relationships to disclose:
- Alok Srivastava, MD, FRACP, FRCPA, FRCP
The faculty listed below report that they have relevant financial relationships to disclose:
- David Lillicrap, MD, FRCPC, serves as a consultant for BioMarin, CSL-Behring, Sanofi, and Takeda. He receives research funding from Bayer, BioMarin, Bioverativ, CSL-Behring, and Octapharma, and Sanofi.
- 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.
- Wolfgang Miesbach, MD, receives grant research support from Bayer, Biotest, CSL Behring, LFB, Novo Nordisk, Octapharma, Pfizer, and Takeda/Shire. He serves on the speakers bureau for Bayer, Biomarin, Biotest, CSL Behring, Chugai, LFB, Novo Nordisk, Octapharma, Pfizer, Roche, and Takeda/Shire. Dr. Miesbach serves on advisory boards for Bayer, BioMarin, Biotest, CSL Behring, Chugai, Freeline, LFB, Novo Nordisk, Octapharma, Pfizer, Roche, Sanofi, Takeda/Shire, and uniQure.
- Margareth Ozelo, MD, PhD, serves as consultant for BioMarin, Novo Nordisk, Pfizer, Roche, Sanofi, and Takeda. She serves on the speakers bureau for Bayer, BioMarin, Novo Nordisk, Roche, and Takeda. Dr. Ozelo conducts contract research for BioMarin, Novo Nordisk, Pfizer, Roche, Sanofi, Spark, and Takeda.
- Flora Peyvandi, MD, PhD, serves as a consultant for Sanofi and Sobi. She serves on the speakers bureau for Roche, Sanofi, Sobi, 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 Presenter Disclosures
The following faculty presenters reports that they have relevant financial relationships to disclose:
- Daniel Hart, BSc, MBChB, MRCP, FRCPath, PhD, serves as a consultant for SOBI/Flori, Sanofi, and Takeda. He conducts contract research for BioMarin, Octapharma, Roche, Sigilon, and uniQure.
- Maria Elisa Mancuso, MD, PhD, conducts contract research for Bayer, Catalyst Bioscience, and Novo Nordisk.
All conflicts of interest disclosures for the faculty presenter and planners were mitigated.
DISCLOSURE OF UNLABELED USE
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.
COMMERCIAL SUPPORT ACKNOWLEDGMENT
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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