Conference Coverage from ISTH 2019 in Melbourne

Conference Coverage from ISTH 2019 in Melbourne

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Conference Coverage from ISTH 2019 in Melbourne, featuring expert interviews on the latest advances in Gene Therapy.

 

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Johnny Mahlangu, BSc, MBBCh, MMed, FCPath

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

Johnny Mahlangu, BSc, MBBCh, MMed, FCPath is a Personal Professor in Haematology and Head of School of Pathology in the Faculty of Health Sciences of the University of the Witwatersrand and the National Health Laboratory Service in Johannesburg, South Africa. He is also Director of the International Haemophilia Training Centre and Consultant Clinical Haematologist at the Charlotte Maxeke Johannesburg Academic Hospital, Johannesburg. Dr Mahlangu received his undergraduate and postgraduate training in science and medicine at the University of the Witwatersrand with haematology specialist and clinical haematology sub-specialist qualifications through the Colleges of Medicine of South Africa. He has published many journal articles and abstracts and has delivered lectures, workshops, and presentations at national and international meetings. His main area of research is novel therapies in bleeding disorders in which he has served as Principal Investigator for over 70 international multicenter studies. Professor Mahlangu is the current President of the College of Pathologists in South Africa, board member of the South African Medical Research Council, Wits Health Consortium and Poliomyelitis Research Foundation and Chair of the International Society of Thrombosis and Haemostasis (ISTH) Scientific and Standardization Committee on Factor VIII, FIX and rare bleeding disorders.


K. John Pasi, MChB, PhD, FRCP, FRCPath, FRCPCH

K. John Pasi, MB ChB, PhD, FRCP, FRCPath, FRCPCH
Queen Mary, University of London | QMUL • Centre for Haematology - London, United Kingdom

John Pasi is Professor of Haemostasis and Thrombosis at The Royal London Hospital, Barts and the London School of Medicine and Dentistry and Haemophilia Centre Director. His clinical practice spans both adults and children and covers all aspects of haemostasis and thrombosis. His research interests cover many aspects of inherited and acquired bleeding and clotting disorders, particularly new bioengineered therapies for haemophilia, novel therapies for haemophilia including gene therapy and RNAi technologies. He is closely involved in the design and development of clinical trials for new therapies and evolving phase 1-4 programmes. In addition, he has a major interest in the development of robust and harmonised outcome measures for haemophilia including application to research.

He has also been involved in the development of national policy and guideline formulation across a broad range of haemostatic and thrombotic disorders and the development of novel models for haematology training initiatives such as residential academies and preceptorships for both haemostasis and thrombosis. He currently chairs the London Haemophilia Clinical Advisory Group and Commissioning Forum, is a member of NHS England Clinical Reference Group (GRG) for Inherited Bleeding Disorders, leads the link between the CRG and NIHR on research and co-chairs the World Federation of Haemophilia Round table on Gene Therapy.


Glenn F. Pierce, MD, PhD

Glenn F. Pierce, MD, PhD

Glenn Pierce, MD, PhD, currently serves on the World Federation of Hemophilia (WFH) Vice President Medical and WFH USA Board of Directors and National Hemophilia Foundation (NHF) U.S. Medical and Scientific Advisory Council. He is an Entrepreneur in-residence at Third Rock Ventures as well as a consultant for gene therapy and hematology biotech companies. He co-founded Ambys Medicines in 2018, a cell and gene therapy liver regeneration start-up, and serves as the Chief Medical Officer (CMO). Dr. Pierce retired from Biogen in 2014, where he led the R&D of extended half-life FVIII and FIX Fc fusions as CMO. He spearheaded the donation of 1 billion international units (IUs) and initiation of the humanitarian aid collaboration with WFH. Dr. Pierce also led the initiation of the My Life Our Future (MLOF) program to genotype >10,000 individuals in the US bleeding disorder community. Previously, Dr. Pierce served on the NHF Board of Directors and was President of the NHF. Dr. Pierce received an MD and a PhD in Immunology, and did his postgraduate training in pathology and hematology research. He has 30+ years of experience in biotech drug development in the areas of tissue regeneration and hematology, including hemophilia, beginning with Amgen and has been involved in the development of 5 approved products for hemophilia. He splits time between San Francisco and San Diego. Dr. Pierce was born with severe hemophilia A, and was cured in 2008.


Thierry VandenDriessche, PhD

Thierry VandenDriessche, PhD
Vrije University Brussels - Brussels, Belgium

Thierry VandenDriessche, PhD is currently holding several academic appointments. He is a tenured Full Professor at the Vrije Universiteit Brussel (VUB, Belgium), Faculty of Medicine & Pharmacy, where he is Founding Director of the Department of Gene Therapy and Regenerative Medicine. He is also Professor at the University of Leuven (Belgium) at the Faculty of Medicine in the Department of Cardiovascular Sciences. Finally, he was the 2017 Francqui Chair Laureate at the University of Ghent (Belgium). Professor Dr. VandenDriessche obtained his PhD at the Free University of Brussels in 1992 in the field of gene therapy for cancer and was a visiting fellow at the Weizmann Institute for Science (Israel). He continued his research in gene therapy as a post-doctoral fellow at the National Institutes of Health (NIH, USA) where he started his research on gene therapy for hemophilia. He was subsequently recruited to the University of Leuven and the Flanders Institute of Biotechnology (VIB) and returned to the NIH as a sabbatical fellow. He previously served as President of the European Society of Gene & Cell Therapy and was member of the Board of Directors of the American Society of Gene & Cell Therapy and has been committee member of the World Federation of Hemophilia and U.S. National Hemophilia Foundation. Professor Dr. VandenDriessche has published over 130 peer-reviewed publications, including many in high-impact factor journals, and has received several awards for his work. His main research focuses on gene therapy and gene editing for hereditary diseases, particularly hemophilia.

Activity Title:Conference Coverage from ISTH 2019 in Melbourne

Topic: Gene Therapy in Hemophilia

Accreditation Type: AMA PRA Category 1 Credit(s)™

Release Date: September 30, 2019

Expiration Date: September 29, 2020

Estimated Time to Complete Activity: 30 minutes

CME Information


Activity Title

Conference Coverage from ISTH 2019 in Melbourne

Topic

Gene Therapy in Hemophilia

Accreditation Type

AMA PRA Category 1 Credit(s)™

Release Date

September 30, 2019

Expiration Date

September 29, 2020

Estimated Time to Complete Activity

30 minutes

 

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

  • Outline the current and emerging 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

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

K. John Pasi, MB ChB, PhD, FRCP, FRCPath, FRCPCH
Queen Mary, University of London QMUL
Centre for Haematology
London, United Kingdom

Glenn F. Pierce, MD, PhD
World Federation of Hemophilia
United States

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 online posttest. A score of 100% is required to successfully complete this activity. The participant may take the test until successfully passed.
  5. 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.
  6. 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 provided by The France Foundation.

TARGET AUDIENCE
This activity is intended for physicians (hematologists), nurse practitioners, physician assistants, nurses managing patients with hemophilia and 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 and International Society of Thrombosis and Haemostasis. 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 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 reviewers, editors, staff, CME committee, or other members at The France Foundation who control content have no relevant financial relationships to disclose.

The reviewers, editors, staff, or other members at the International Society of Thrombosis and Haemostasis 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.

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

  • David Lillicrap, MD, FRCPC, has received honoraria for academic consulting from Bioverativ, CSL Behring, and Octapharma Plasma. He has received research funding from Bayer, BioMarin, Bioverative, CSL-Behring, and Octapharma Plasma.
  • Johnny Mahlangu, BSc, MBBCh, MMed, FCPath has held leadership positions with ISTH and WFH. He has served on the speakers’ bureau for Alnylam, Bayer, Biotest, Biogen, ISTH, Novo Nordisk, Pfizer, Sobi, Shire, Roche, and WFH. Dr. Mahlangu has received research funding from Alnylam, Bayer, Biotest, Biogen, ISTH, Novo Nordisk, Pfizer, Sobi, Shire, Roche, and WFH. He has received honoraria from Amgen, Bayer, Biotest, Biogen, Baxalta, CSL- Behring, Catalyst Biosciences, Chugai, Freeline, LFB, Novo Nordisk, Roche, and Spark.
  • K. John Pasi, MChB, PhD, FRCP, FRCPath, FRCPCH, has received research funding from BioMarin GeneR8 programme, uniQure HOPE-B programme, Sanofi - ATLAS fitusiran program. He has received honoraria from Alnylam, ApcinteX, BioMarin, Catalyst Bio, Chugai, Novo Nordisk, Octapharma Plasma, Pfizer, Roche, Sanofi, Shire, and Sobi.
  • Flora Peyvandi, MD, PhD has held leadership positions with EHC, EMA, and EAHAD. She has served on the speakers’ bureau for Bioverativ, CSL-Behring, Grifols, Novo Nordisk, Roche, Sanofi, Sobi, Spark, Sysmex, and Takeda. Dr. Peyvandi is an advisory board member for Sanofi.
  • Glenn F. Pierce, MD, PhD, has received honoraria for academic consulting from BioMarin, Genentech/Roche, Pfizer, St. Jude, and VarmX. He has held leadership positions with Global Blood Therapeutics, NHF MASAC, and World Federation of Hemophilia.
  • Steven W. Pipe, MD, has received honoraria for academic consulting from CSL-Behring, Novo Nordisk and Pfizer. He has held leadership positions with MASAC-National Hemophilia Foundation. Dr. Pipe has received research funding from Siemens and Shire. He has received honoraria from ApcinteX, Bayer, Biomarin, Bioverativ, Catalyst, CSL-Behring, HEMA Biologics, Freeline, Novo Nordisk, Pfizer, Roche/Genentech, Sanofi, Shire, Spark, and uniQure.
  • Alok Srivastava, MD, FRACP, FRCPA, FRCP, has received honoraria for academic consulting from Bayer, Novo Nordisk, Roche, and Shire/Takeda. He has held leadership positions as Chair of Gene Therapy Task Force of SSC of the ISTH and Chair of the Steering Committee of the Asia Pacific Hemophilia Working Group. Dr. Srivastava has received research funding from Alnylam, Bayer, and Shire.
  • Thierry VandenDriessche, PhD, has held leadership positions with NHF and ISTH. He has received research funding from Pfizer and Takeda. Dr. VandenDriessche has received 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:

  • Johnny Mahlangu, BSc, MBBCh, MMed, FCPath has held leadership positions with ISTH and WFH. He has served on the speakers’ bureau for Alnylam, Bayer, Biotest, Biogen, ISTH, Novo Nordisk, Pfizer, Sobi, Shire, Roche, and WFH. Dr. Mahlangu has received research funding from Alnylam, Bayer, Biotest, Biogen, ISTH, Novo Nordisk, Pfizer, Sobi, Shire, Roche, and WFH. He has received honoraria from Amgen, Bayer, Biotest, Biogen, Baxalta, CSL- Behring, Catalyst Biosciences, Chugai, Freeline, LFB, Novo Nordisk, Roche, and Spark.
  • K. John Pasi, MChB, PhD, FRCP, FRCPath, FRCPCH, has received research funding from BioMarin GeneR8 programme, uniQure HOPE-B programme, Sanofi - ATLAS fitusiran program. He has received honoraria from Alnylam, ApcinteX, BioMarin, Catalyst Bio, Chugai, Novo Nordisk, Octapharma Plasma, Pfizer, Roche, Sanofi, Shire, and Sobi.
  • Glenn F. Pierce, MD, PhD, has received honoraria for academic consulting from BioMarin, Genentech/Roche, Pfizer, St. Jude, and VarmX. He has held leadership positions with Global Blood Therapeutics, NHF MASAC, and World Federation of Hemophilia.
  • Thierry VandenDriessche, PhD, has held leadership positions with NHF and ISTH. He has received research funding from Pfizer and Takeda. Dr. VandenDriessche has received honoraria from Baxalta/Shire/Takeda, Bayer, Biotest, and Pfizer.

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 an educational grant from BioMarin, 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.

COPYRIGHT INFORMATION
Copyright © 2019 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..

 

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A History of Hemophilia Treatment: Factor Replacement to Gene Therapy

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