Advances in Gene Therapy for Hemophilia
Recorded Live as a Friday Satellite Symposium preceding the 62nd ASH Annual Meeting and Exposition
Key Considerations: Advances in Gene Therapy for Hemophilia
Gene Therapy in Hemophilia
AMA PRA Category 1 Credit(s)™
December 14, 2020
December 13, 2021
Estimated Time to Complete Activity
EDUCATIONAL ACTIVITY LEARNING OBJECTIVES
Upon completion of the activity, participants should be able to:
- Describe current and emerging approaches for treating hemophilia, including various approaches to gene therapy
- Identify key characteristics of current clinical trials in gene therapy for both hemophilia A and hemophilia B
- Outline strategies for integrating gene therapy into a personalized approach to managing hemophilia
Glenn Pierce, MD, PhD
World Federation of Hemophilia
Lindsey George, MD
The Children’s Hospital of Philadelphia
Alfonso Iorio, MD
Barbara A. Konkle, MD
Washington Center for Bleeding Disorders
University of Washington
MOC Peer Reviewers
Ben Samelson-Jones, MD, PhD
Assistant Professor of Pediatrics
The Children's Hospital of Philadelphia
Theodore O. Bruno, MD
Chief Medical Officer
The France Foundation
Old Lyme, Connecticut
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 1.0 AMA PRA Category 1 Credit(s)™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.
Maintenance of Certification: Successful completion of this CME activity, which includes participation in the evaluation component, enables the participant to earn up to 1.0 MOC point(s) in the American Board of Internal Medicine's (ABIM) Maintenance of Certification (MOC) program. Participants will earn MOC point(s) equivalent to the amount of CME credits claimed for the activity. It is the CME activity provider's responsibility to submit participant completion information to ACCME for the purpose of granting ABIM MOC credit. Aggregate participant data will be shared with commercial supporters of this 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.
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.
- Participate in the CME/CE activity.
- Complete the CME/CE evaluation 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 Credit(s)™ or a certificate of participation—your CME/CE certificate will be available for download.
- If you are requesting MOC credit, your MOC points will be submitted electronically to the ACCME, which will register data and notify certifying boards.
In accordance with the ACCME Standards for Commercial Support, TFF and the 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 the ISTH resolve all conflicts of interest to ensure independence, objectivity, balance, and scientific rigor in all their educational programs. Furthermore, TFF and the 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 the ISTH are committed to providing learners with high-quality CME/CE activities that promote improvements in health care and not those of a commercial interest.
Activity Staff Disclosures
The planners, reviewers, editors, staff, CME committee, or other members at The France Foundation 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–Activity Faculty
The faculty listed below report that they have no relevant financial relationships to disclose:
- Alfonso Iorio, MD
The faculty listed below report that they have relevant financial relationships to disclose:
- Lindsey A. George, MD, is a member of the AVROBIO data safety monitoring board.
- Barbara A. Konkle, MD, has received honoraria for academic consulting from BioMarin, Genentech, Pfizer, Sanofi, Sigilon, and Spark. She holds leadership positions with the Foundation for Women and Girls with Blood Disorders and the World Federation of Hemophilia. Dr. Konkle owns stock in Roche and Siemens. She has received research funding from Bioverativ/Sanofi, Pfizer, Spark, and Sangamo.
- Glenn F. Pierce, MD, PhD, serves as a consultant for Ambys Medicines, BioMarin, CRISPR Therapeutics, Decibel, Geneception, Generation Bio, Grifols, Takeda, and Third Rock Ventures. He holds leadership positions with Global Blood Therapeutics, NHF MASAC, Voyager Therapeutics, and World Federation of Hemophilia.
Faculty Disclosures–MOC Reviewers
- Theodore Bruno, MD, reports that his spouse is employed by Allergan.
- Ben Samelson-Jones, MD, PhD, serves as a consultant for Cabaletta, Frontera, and Genentech. He receices research support from Accugen, Spark and uniQure. Dr. Samelson-Jones receives royalties from Accugen.
DISCLOSURE OF UNLABELED USE
TFF and ISTH require CME faculty (speakers) to disclose when products or procedures being discussed are off label, unlabeled, experimental, and/or investigational, and 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 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.
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COMMERCIAL SUPPORT ACKNOWLEDGMENT
This activity is supported by an educational grants from BioMarin, Pfizer, and uniQure.
The France Foundation and ISTH present this information for educational purposes only. The content is provided solely by faculty who have been selected because of 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, ISTH, and the commercial supporter(s) assume no liability for the information herein.
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Gene therapy holds much promise for individuals with a wide variety of conditions, including types of cancers, AIDS, diabetes, heart disease, and hemophilia. This innovative approach to treatment alters genes inside the body’s cells to stop the disease.
Gene therapy attempts to replace or fix mutated genes and make diseased cells more evident to the immune system. Hemophilia A and B are inherited as part of an X-linked recessive pattern. Hemophilia genetics involve genes found in the X chromosome, and it takes just one faulty gene to lead to this bleeding disorder.Cell and gene therapies are still under study, but the results are promising. Clinical trials have shown success, and the Food and Drug Administration (FDA) has approved one type of cell therapy. In our Key Considerations: Advances in Gene Therapy for Hemophilia led by Glenn F. Pierce, we discuss an overview of the disease, the current state of gene therapy, and more.