CME Presentations

NORD Gene Therapy


Presentations


Speakers

  • Kevin Flanigan, MD

    Kevin Flanigan, MD

    Kevin Flanigan, MD

    Director, Center for Gene Therapy

    Kevin Flanigan, MD, is director of the Center for Gene Therapy at Nationwide Children’s Hospital, and professor of pediatrics and neurology at the Ohio State University in Columbus. He directs the Nationwide Children’s Neuromuscular Program and the NIAMS P50-funded Centers of Research Translation (CORT) in muscular dystrophy therapeutic development. Dr Flanigan’s research focuses on genotype/phenotype correlations in the muscular dystrophies, with a goal of understanding molecular mechanisms that lead to amelioration of symptoms in order to identify new therapeutic pathways. His laboratory works primarily on AAV-based approaches to gene therapy. Dr Flanigan trained in neurology and neuromuscular medicine at Johns Hopkins University in Baltimore, Maryland.
  • Stephan Grupp, MD, PhD

    Stephan Grupp, MD, PhD

    Stephan Grupp, MD, PhD

    Section Chief, Cellular Therapy and Transplant

    Stephan Grupp, MD, PhD, is the chief of the Cellular Therapy and Transplant Section, director of the Cancer Immunotherapy Program, and medical director of The Cell and Gene Therapy Lab at Children’s Hospital of Philadelphia, as well as the Novotny Professor of Pediatrics at the University of Pennsylvania Perelman School of Medicine. His primary area of research is the use of CAR T and other engineered cell therapies in pediatric cancers and other life-threatening disorders such as sickle cell disease. Dr Grupp led all of the pediatric ALL trials of CTL019. As a result of this work, he presented the clinical perspective at the first US Food and Drug Administration Oncologic Drugs Advisory Committee CAR meeting. Dr Grupp was elected to the National Academy of Medicine in 2019.
  • Alessandra Biffi, MD

    Alessandra Biffi, MD

    Alessandra Biffi, MD

    Chief of the Pediatric Hematology, Oncology, and Stem Cell Transplant Division

    Alessandra Biffi, MD, is chief of the Pediatric Hematology, Oncology, and Stem Cell Transplant Division at Padua University and the Padua University Hospital, and coordinates the research area on onco-hematology, stem cell transplant, and gene therapy at the Pediatric Research Institute in Padua, Italy. She is also a member of the pediatric faculty at Harvard Medical School and affiliated with the Gene Therapy Program of the Dana-Farber/Boston Children’s Cancer Center in Boston, Massachusetts. Dr Biffi’s research is devoted to enhancing the efficacy of HSC-based therapeutic approaches for metabolic disorders with severe nervous system involvement. She has published more than 100 peer-reviewed manuscripts and several textbook chapters, and she received an ERC Consolidator Grant award.
  • Julie A. Parsons, MD

    Julie A. Parsons, MD

    Julie A. Parsons, MD

    Professor of Clinical Pediatrics and Neurology

    Julie A. Parsons, MD, is a professor of clinical pediatrics and neurology at the University of Colorado School of Medicine in Aurora, the Haberfeld Family Endowed Chair in Pediatric Neuromuscular Disorders, and co-director of the neuromuscular clinic at Children’s Hospital Colorado. Her clinical interests include neuromuscular diseases such as muscular dystrophies, spinal muscular atrophy (SMA), and myotonic dystrophies. She is an active participant and principal investigator for many clinical trials, and is a member of organizations that include the Child Neurology Society and the American Academy of Neurology. Dr Parsons earned her MD from the University of Colorado School of Medicine, followed by residencies in pediatrics and child neurology and a neuromuscular medicine fellowship.
  • Perry Shieh, MD, PhD

    Perry Shieh, MD, PhD

    Perry Shieh, MD, PhD

    Professor of Neurology and Pediatrics

    Perry Shieh, MD, PhD, is professor of neurology and pediatrics at the David Geffen School of Medicine at University of California, Los Angeles. His principal clinical interests include myasthenia gravis, inflammatory myopathy, spinal muscular atrophy, muscular dystrophy, electromyography, and muscle histopathology. Dr Shieh has served as an investigator in numerous clinical trials for neuromuscular conditions and has a particular interest in gene therapy. He received his medical degree and his PhD in neuroscience from Johns Hopkins University in Baltimore, Maryland. Dr Shieh completed fellowship training in clinical neurophysiology/EMG at Brigham and Women’s Hospital/Harvard Medical School in Boston, Massachusetts.
  • John Kessler, MD

    John Kessler, MD

    John Kessler, MD

    Director

    John A. Kessler, MD, is the Davee Professor of Stem Cell Biology, director of the Feinberg Neuroscience Institute, and director of the Northwestern University Stem Cell Institute in Chicago, Illinois. He was chairman of the Davee Department of Neurology for 12 years and is an active clinician and stem cell biologist. Dr Kessler is currently the principal investigator on 4 NIH grants in the field of stem cell research and is the principal investigator in multicenter gene therapy trials for the treatment of neuropathy and ALS. He is the author of more than 300 scientific publications, an editor of several journals, and on the Board of Trustees of two hospitals. Dr Kessler received his medical degree from Cornell University Medical College in New York, New York.
  • Kimberly Goodspeed, MD

    Kimberly Goodspeed, MD

    Kimberly Goodspeed, MD

    Assistant Professor of Pediatrics, Neurology Division

    Kimberly Goodspeed, MD, received her medical degree at the University of Texas Medical School at Houston (now McGovern Medical School) and completed a neurodevelopmental disabilities residency UT Southwestern/Children’s Medical Center. She joined UTSW as faculty in the Department of Pediatrics within the Division of Neurology and holds joint appointments in the Department of Neurology and the Department of Psychiatry. Her clinical practice is at the Center for Autism and Developmental Disabilities where she cares for patients with autism spectrum disorder and intellectual disability. Dr Goodspeed’s research interests are in phenotyping and clinical trial readiness studies of rare genetic variants of neurodevelopmental disorders in preparation for future precision therapy clinical trials.

CME Information

The Time Is Now: Understanding Gene Therapy, Applications, and Implications


PIM LogoPQH Logo

LEARNING OBJECTIVES

At the conclusion of this activity, participants will be able to:
  • Explain the potential of gene‐based treatment approaches to change the disease course for patients with genetic conditions
  • Discuss different types of current and emerging gene‐based therapies
  • Summarize the key steps of development programs for gene therapies, identifying how and why some programs may differ from others
  • Explain the role of the FDA in providing regulatory oversight from development to post marketing
  • Cite evidence for the use of gene‐based therapies that are approved or pending approval for the management of medical disorders
  • Counsel patients about clinical‐trial opportunities
  • Discuss the role of genetic counselors in educating patients and caregivers on the decision‐making processes for access to gene therapy, financial support, and post‐treatment care
  • Identify specialists and specialty care centers that patients may be referred to for further evaluation
Jointly provided by Postgraduate Institute for Medicine (PIM) and PlatformQ Health Education, LLC (PQHE) working in collaboration with the National Organization for Rare Disorders (NORD).

ACKNOWLEDGEMENT

This activity is supported by independent educational grants from Astellas Gene Therapies; AVROBIO, Inc.; Biogen; CRISPR Therapeutics, Inc.; Helixmith Co., Ltd; Orchard Therapeutics; REGENXBIO Inc.; and uniQure, Inc.

TUITION

Complimentary

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JOINT ACCREDITATION STATEMENT

In support of improving patient care, this activity has been planned and implemented by the Postgraduate Institute for Medicine and PlatformQ Health Education, LLC. Postgraduate Institute for Medicine is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.

LIVE CREDIT DESIGNATION STATEMENTS

Physician Continuing Medical Education

The Postgraduate Institute for Medicine designates this live activity for a maximum of 4.0 AMA PRA Category 1 Credit(s)™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

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Successful completion of this CME activity, which includes participation in the evaluation component, enables the participant to earn up to 4.0 MOC points in the American Board of Internal Medicine's (ABIM) Maintenance of Certification (MOC) program. Participants will earn MOC points 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.

Please note, MOC credit is reported on a monthly basis.

To claim and receive MOC credits, learners must provide:
  • ABIM ID number
  • First and last name
  • Degree and specialty
  • DOB (MMDD)
  • Activity completion date

Nursing Continuing Education

The maximum number of hours awarded for this Continuing Nursing Education activity is 4.0 contact hours.

Physician Assistant Education

AAPA Logo

Postgraduate Institute for Medicine has been authorized by the American Academy of PAs (AAPA) to award AAPA Category 1 CME credit for activities planned in accordance with AAPA CME Criteria. This activity is designated for 4.0 AAPA Category 1 CME credits. PAs should only claim credit commensurate with the extent of their participation.

Pharmacist Continuing Education

Postgraduate Institute for Medicine is accredited by the Accreditation Council for Pharmacy Education as a provider of continuing pharmacy education. Postgraduate Institute for Medicine designates this continuing education activity for 4 contact hours (0.04 CEUs) of the Accreditation Council for Pharmacy Education.

Gene Transfer, Editing, Silencing and RNA Based‐Approaches: What, When and How?
(Universal Activity Number - JA4008162-9999-21-2657-L01-P)
Type of Activity: Application

Clinical Development and Regulatory Oversight of Cell and Gene-Based Therapies from Development to Post‐Marketing
(Universal Activity Number - JA4008162-9999-21-2658-L01-P)
Type of Activity: Application

Examples of Diseases that Utilize Gene‐Based Therapy
(Universal Activity Number - JA4008162-9999-21-2659-L01-P)
Type of Activity: Application

Post‐Gene Therapy: Implications for Patients’ Management
(Universal Activity Number - JA4008162-9999-21-2660-L01-P)
Type of Activity: Application

Pharmacists will need to provide their National Association of Boards of Pharmacy (NABP) e-Profile ID and date of birth (MMDD) to ensure the data is submitted to the ACPE and NABP properly. If you need to obtain your NABP e-Profile, please visit www.nabp.net.

APA Education (American Psychology Association)

Continuing Education (CE) credits for psychologists are provided through the co-sponsorship of the American Psychological Association (APA) Office of Continuing Education in Psychology (CEP). The APA CEP Office maintains responsibility for the content of the programs. This program offers 4.0 continuing education credit for psychologists.

Social Work Education

ACE Logo

As a Jointly Accredited Organization, Postgraduate Institute for Medicine is approved to offer social work continuing education by the Association of Social Work Boards (ASWB) Approved Continuing Education (ACE) program. Organizations, not individual courses, are approved under this program. State and provincial regulatory boards have the final authority to determine whether an individual course may be accepted for continuing education credit. Postgraduate Institute for Medicine maintains responsibility for this course. Social workers completing this course receive 4.0 clinical continuing education credits.

ENDURING CREDIT DESIGNATION STATEMENTS

Physician Continuing Medical Education

The Postgraduate Institute for Medicine designates this enduring activity for a maximum of 4.0 AMA PRA Category 1 Credit(s)™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

ABIM Logo

Successful completion of this CME activity, which includes participation in the evaluation component, enables the participant to earn up to 4.0 MOC points in the American Board of Internal Medicine's (ABIM) Maintenance of Certification (MOC) program. Participants will earn MOC points 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.

Please note, MOC credit is reported on a monthly basis.

To claim and receive MOC credits, learners must provide:
  • ABIM ID number
  • First and last name
  • Degree and specialty
  • DOB (MMDD)
  • Activity completion date

Nursing Continuing Education

The maximum number of hours awarded for this Continuing Nursing Education activity is 4.0 contact hours.

Physician Assistant Education

AAPA Logo

Postgraduate Institute for Medicine has been authorized by the American Academy of PAs (AAPA) to award AAPA Category 1 CME credit for activities planned in accordance with AAPA CME Criteria. This activity is designated for 4.0 AAPA Category 1 CME credits. PAs should only claim credit commensurate with the extent of their participation.

Pharmacist Continuing Education

Postgraduate Institute for Medicine is accredited by the Accreditation Council for Pharmacy Education as a provider of continuing pharmacy education. Postgraduate Institute for Medicine designates this continuing education activity for 4 contact hours (0.04 CEUs) of the Accreditation Council for Pharmacy Education.

(Universal Activity Number - JA4008162-9999-21-2661-H01-P) Type of Activity: Application

Pharmacists will need to provide their National Association of Boards of Pharmacy (NABP) e-Profile ID and date of birth (MMDD) to ensure the data is submitted to the ACPE and NABP properly. If you need to obtain your NABP e-Profile, please visit www.nabp.net.

APA Education (American Psychology Association)

Continuing Education (CE) credits for psychologists are provided through the co-sponsorship of the American Psychological Association (APA) Office of Continuing Education in Psychology (CEP). The APA CEP Office maintains responsibility for the content of the programs. This program offers 4.0 continuing education credit for psychologists.

Social Work Education

ACE Logo

As a Jointly Accredited Organization, Postgraduate Institute for Medicine is approved to offer social work continuing education by the Association of Social Work Boards (ASWB) Approved Continuing Education (ACE) program. Organizations, not individual courses, are approved under this program. State and provincial regulatory boards have the final authority to determine whether an individual course may be accepted for continuing education credit. Postgraduate Institute for Medicine maintains responsibility for this course. Social workers completing this course receive 4.0 clinical continuing education credits.

DATE OF RELEASE/EXPIRATION

Live Activity

Thursday, April 22, 2021:
  • 2:00pm – 3:00pm ET: Gene Transfer, Editing, Silencing and RNA Based‐Approaches: What, When and How?
  • 3:00pm – 4:00pm ET: Clinical Development and Regulatory Oversight of Cell and Gene‐Based Therapies from Development to Post‐Marketing
Thursday, April 29, 2021:
  • 12:00pm – 1:00pm ET: Examples of Diseases that Utilize Gene‐Based Therapy
  • 1:00pm – 2:00pm ET: Post‐Gene Therapy: Implications for Patients’ Management

Enduring Material

The enduring material will be available from April 22, 2021 to April 29, 2022

LEARNING OBJECTIVES

At the conclusion of this activity, participants will be able to:

Gene Transfer, Editing, Silencing and RNA Based‐Approaches: What, When and How?
  • Explain the potential of gene‐based treatment approaches to change the disease course for patients with genetic conditions
  • Discuss different types of current and emerging gene‐based therapies
Clinical Development and Regulatory Oversight of Cell and Gene‐Based Therapies from Development to Post‐Marketing
  • Summarize the key steps of development programs for gene therapies, identifying how and why some programs may differ from others
  • Explain the role of the FDA in providing regulatory oversight from development to post marketing
Examples of Diseases that Utilize Gene‐Based Therapy
  • Cite evidence for the use of gene‐based therapies that are approved or pending approval for the management of medical disorders
  • Counsel patients about clinical‐trial opportunities
Post-Gene Therapy: Implications for Patients’ Management
  • Discuss the role of genetic counselors in educating patients and caregivers on the decision‐making processes for access to gene therapy, financial support, and post‐treatment care
  • Identify specialists and specialty care centers that patients may be referred to for further evaluation

TARGET AUDIENCE

This activity is intended for primary care providers, internal-medicine specialists, pediatricians, pulmonologists, cardiologists, endocrinologists, gastroenterologists, hepatologists, neurologists, neuromuscular specialists, infectious-disease specialists, obstetricians/gynecologists, rheumatologists, dermatologists, hematologists, oncologists, neonatologists, genetic counselors, social workers, psychologists, nurse practitioners, physician assistants, nurses, and pharmacists

ESTIMATED TIME TO COMPLETE

This activity consists of 4 sessions which should take approximately 4 hours to complete.

METHOD OF PARTICIPATION

There are no fees to participate in this activity. To participate in the activity, go to www.ClinicalSeriesLive.com. To receive credit, participants must (1) read the target audience, learning objectives, and disclosure statements, (2) complete the educational activity online, and (3) complete the post-test and activity evaluation. To receive AMA PRA Category 1 Credits™, participants must receive a minimum score of 70% on the post-test.

HARDWARE/SOFTWARE REQUIREMENTS

Participants will need a computer with a recent version of Adobe Flash installed, as well as an internet connection sufficient for streaming media.

DISCLOSURE OF CONFLICTS OF INTEREST

Postgraduate Institute for Medicine (PIM) requires instructors, planners, managers and other individuals who are in a position to control the content of this activity to disclose any real or apparent conflict of interest (COI) they may have as related to the content of this activity. All identified COI are thoroughly vetted and resolved according to PIM policy. PIM is committed to providing its learners with high quality CME activities and related materials that promote improvements or quality in healthcare and not a specific proprietary business interest of a commercial interest.

The faculty reported the following financial relationships or relationships to products or devices they or their spouse/life partner have with commercial interests related to the content of this CME activity:

Name of Faculty or Presenter Reported Financial Relationship
Alessandra Biffi, MD
Professor of Pediatrics
Director, Division of Pediatric Hematology, Oncology and Stem Cell Transplant
University of Padua
Receipt of Intellectual Property Rights/Patent Holder: Altheia Science, Orchard Therapeutics
Consulting Fee: Altheia Science, Orchard Therapeutics
Contracted Research: Altheia Science, Orchard Therapeutics
Ownership Interest less than 5%: Orchard Therapeutics
Ownership Interest greater than 5%: Altheia Science
Kevin Flanigan, MD
Director, Center for Gene Therapy
Robert F. & Edgar T. Wolfe Foundation Endowed Chair in Neuromuscular Research Director, Nationwide Children’s Hospital Center for Research Translation in Muscular Dystrophy Therapeutic Development
Abigail Wexner Research Institute Nationwide Children’s Hospital
Professor of Pediatrics and Neurology
The Ohio State University
Royalty: Audentes
Consulting Fees: Apic Bio, 4D Molecular Theraputics
Contracted Research: Audentes
Ownership Interest less than 5%: 4D Molecular Therapeutics
Kimberly Goodspeed, MD
Assistant Professor of Pediatrics, Neurology Division
University of Texas Southwestern Medical Center Dallas
Salary: Taysha Gene Therapies - Support for work on gene therapy translational research program
Stephan A. Grupp, MD, PhD
Section Chief, Cellular Therapy and Transplant
Director, Cancer Immunotherapy Program
Children’s Hospital of Philadelphia
Novotny Professor of Pediatrics
Perelman School of Medicine at the University of Pennsylvania
Consulting Fees: Adaptimmune, Allogene, CBMG, Cabaletta, Cellectis, GSK, Humanigen, Janssen/JnJ, Jazz, Juno, Novartis, Roche, TCR2, Vertex
Contracted Research: Kite, Novartis, Servier, Vertex
John Kessler, MD
Davee Professor
Davee Department of Neurology
Northwestern University
Contracted Research: Support of clinical trials at 26 institutions including Northwestern. No funds to my lab or to me personally. I am the national PI of the trials but not the site PI at Northwestern.
Julie A. Parsons, MD
Haberfeld Family Endowed Chair in Pediatric Neuromuscular Disorders
Professor of Clinical Pediatrics and Neurology
University of Colorado School of Medicine
Children's Hospital Colorado
Consulting Fees: Biogen, Genentech, Novartis, Scholar Rock
Contracted Research: Biogen, Novartis, Scholar Rock
Perry B. Shieh, MD, PhD, FAAN
Professor of Neurology and Pediatrics
David Geffen School of Medicine at UCLA
University of California, Los Angeles
Consulting Fees: Alexion, Biogen, Genentech, Pfizer, PTC, Sarepta
Fees for Non-CE Services Received Directly from an Ineligible Entity or their Agents: Alexion, Avexis, Biogen, CSL Behring, Genentech, Grifols
Contracted Research: Avexis, Biogen, Pfizer, PTC, Sarepta

The planners and managers reported the following financial relationships or relationships to products or devices they or their spouse/life partner have with commercial interests related to the content of this CME activity:

Name of Faculty or Presenter Reported Financial Relationship
Carole Drexel
Medical Director
PlatformQ Health Education, LLC
None

The PIM planners and managers have nothing to disclose.

The following PlatformQ Health Education, LLC planner, Alissa Yost, hereby states that she or her spouse/life partner do not have any financial relationships or relationships to products or devices with any commercial interest related to the content of this activity of any amount during the past 12 months.

DISCLOSURE OF UNAPPROVED/OFF LABEL USE (if applicable):

This educational activity may contain discussion of published and/or investigational uses of agents that are not indicated by the FDA. The planners of this activity do not recommend the use of any agent outside of the labeled indications.

The opinions expressed in the educational activity are those of the faculty and do not necessarily represent the views of the planners. Please refer to the official prescribing information for each product for discussion of approved indications, contraindications, and warnings.

DISCLAIMER:

Participants have an implied responsibility to use the newly acquired information to enhance patient outcomes and their own professional development. The information presented in this activity is not meant to serve as a guideline for patient management. Any procedures, medications, or other courses of diagnosis or treatment discussed or suggested in this activity should not be used by clinicians without evaluation of their patient’s conditions and possible contraindications and/or dangers in use, review of any applicable manufacturer’s product information, and comparison with recommendations of other authorities.

CONTACT INFORMATION

Accreditation Support: Please contact the Postgraduate Institute for Medicine at www.pimed.com.

Technical Support: For any technical issues or issues with your CME Certificate, please contact OMedLive at 877-394-1306 or at Support@ClinicalSeriesLive.com.