Latest Clinical Trial Updates for Hemophilia B Gene Therapy

Latest Clinical Trial Updates for Hemophilia B Gene Therapy

Listen as Dr. Margareth Ozelo from the University of Campinas in Brazil discusses the latest clinical trial updates in Gene Therapy for Hemophilia B. 

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.

 

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CME Information


Activity Title

Latest Clinical Trial Updates for Hemophilia B Gene Therapy

Topic

Gene Therapy in Hemophilia

Accreditation Type

AMA PRA Category 1 Credit(s)™

Release Date

October 8, 2021

Expiration Date

October 9, 2021

Estimated Time to Complete Activity

15 minutes

LEARNING OBJECTIVE
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
  • Appropriately discuss pros and cons of gene therapy with patients and caregivers, including available resources for further education

FACULTY PRESENTER
Margareth Ozelo, MD, PhD
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

FACULTY 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

Wolfgang A. Miesbach, MD, PhD
Goethe University Hospital
Frankurt/Main, Germany

Margareth Ozelo, MD, PhD
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
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). TFF 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 0.25 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.

DISCLOSURE POLICY
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:

  • 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.
  • 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 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:

  • 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 also conducts contract research for BioMarin, Novo Nordisk, Pfizer, Roche, Sanofi, Spark, and Takeda.

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.

DISCLAIMER
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.

COPYRIGHT INFORMATION
Copyright © 2020 The France Foundation. Any unauthorized use of any materials on the site may violate copyright, trademark, and other laws. You may view, copy, and download information or software ("Materials") found on the Site subject to the following terms, conditions, and exceptions:

  • The materials are to be used solely for personal, noncommercial, informational and educational purposes. The materials are not to be modified. They are to be distributed in the format provided with the source clearly identified. The copyright information or other proprietary notices may not be removed, changed, or altered.
  • Materials may not be published, uploaded, posted, transmitted (other than as set forth herein), without The France Foundation's prior written permission.


PRIVACY POLICY

The France Foundation protects the privacy of personal and other information regarding participants and educational collaborators. The France Foundation will not release personally identifiable information to a third party without the individual's consent, except such information as is required for reporting purposes to the ACCME.

The France Foundation maintains physical, electronic, and procedural safeguards that comply with federal regulations to protect against the loss, misuse or alteration of information that we have collected from you.

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..

Margareth C. Ozelo, MD, PhD
Margareth C. Ozelo, MD, PhD
University of Campinas
Hemocentro UNICAMP, Brazil
Campinas, São Paulo, Brazil

Dr. Margareth C. Ozelo is Associate Professor of Internal Medicine Department, and Director of the Haematology Division from University of Campinas (UNICAMP) in Campinas, São Paulo, Brazil. She received her medical degree (1994), specialist training in haematology and transfusion medicine (1994-1997), and PhD (2004) at UNICAMP before undertaking a postdoctoral fellowship in David Lillicrap’s laboratory, at Queen’s University in Kingston, Ontario, Canada, focused in gene therapy for haemophilia (2006–2009).

Dr. Ozelo is also the Director of the WFH International Haemophilia Training Centre (IHTC) from Hemocentro UNICAMP. In 2012, she was nominated as a member of the Novo Nordisk Haemophilia Foundation Council and was member of the Executive Committee of the World Federation of Haemophilia from 2012 to 2014.

Dr Ozelo is involved with several research projects, including, gene therapy for haemophilia, risk factors for inhibitor development, immune tolerance induction, and the management of musculoskeletal complications of haemophilia, and inherited platelet disorders.

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Supported by educational grants from Bayer, BioMarin, Freeline Therapeutics Limited, Pfizer Inc., Shire, Spark Therapeutics, and uniQure, Inc.

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