Stepping Into the Future: Hemophilia Gene Therapy Clinical Trial Updates

Stepping Into the Future: Hemophilia Gene Therapy Clinical Trial Updates

Faculty: Flora Peyvandi, MD, PhD, Glenn Pierce, MD, PhD, and Steven W. Pipe, MD

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CME Information


Activity Title

Stepping Into the Future: Hemophilia Gene Therapy Clinical Trial Updates

Topic

Gene Therapy in Hemophilia

Accreditation Type

AMA PRA Category 1 Credit(s)™

Release Date

September 30, 2020

Expiration Date

September 29, 2021

Estimated Time to Complete Activity

60 minutes

 

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

  • Outline the current and emerging gene therapy approaches for treating hemophilia, including various approaches of gene therapy
  • Identify key characteristics of current clinical trials in gene therapy for both hemophilia A and hemophilia B
  • Identify and critically evaluate important safety and efficacy clinical trial outcomes under consideration for gene therapy as a treatment for hemophilia
  • Recommend appropriate patients for ongoing and emerging trials
  • Appropriately discuss pros and cons of gene therapy with patients and caregivers, including available resources for further education
  • Recognize key concerns and unknowns related to the future of gene therapy for hemophilia

FACULTY
Flora Peyvandi, MD, PhD
Professor of Internal Medicine at the University of Milan
Director of the Angelo Bianchi Bonomi Hemophilia and Thrombosis Centre
Fondazione IRCCS Ca’ Granda, Ospedale Maggiore Policlinico
Milan, Italy

Glenn Pierce, MD, PhD
Vice President, Medical
World Federation of Hemophilia
Montreal, Canada

Steven W. Pipe, MD
Professor of Pediatrics and Pathology
Laurence A. Boxer Research Professor of Pediatrics and Communicable Diseases
Medical Director of Pediatric Hemophilia and Coagulation Disorders Director of the Special Coagulation Laboratory
University of Michigan
Ann Arbor, Michigan

PLANNERS
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

METHOD OF PARTICIPATION / HOW TO RECEIVE CREDIT

  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.

CLICK HERE TO VIEW TECHNICAL REQUIREMENTS


ACCREDITED PROVIDER

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

TARGET AUDIENCE
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.

ACCREDITATION STATEMENT
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). The France Foundation is accredited by the ACCME to provide continuing medical education for physicians .

CREDIT DESIGNATION
Physicians:
The France Foundation designates this enduring activity for a maximum of 0.50 AMA PRA Category 1 Credit(s)™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Nurses: Nurses 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.

DISCLOSURE POLICY
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 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–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, Bauer, 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:

  • 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 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.
  • Steven W. Pipe, MD, serves as a consultant for ApcinteX, Bauer, 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.

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 U.S. 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.

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

DISCLAIMER
The France Foundation and International Society of Thrombosis and Haemostasis 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, International Society of Thrombosis and Haemostasis, and the commercial supporter(s) assume no liability for the information herein.

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PRIVACY POLICY

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Additional information regarding The France Foundation's Privacy Policy can be viewed at www.francefoundation.com/privacy-policy.


CONTACT INFORMATION
If you have questions about this CME activity, please contact The France Foundation at 860-434-1650 or This email address is being protected from spambots. You need JavaScript enabled to view it..