2022 ACMG Summer Gene Therapy Education Series OnDemand
Advancements in Gene Therapy Options for Rare Diseases – This course is supported by an independent medical education grant from Pfizer, Inc.
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4 Courses
Expires on 06/30/2024
ACMG Member: $0.00
Non-Member: $50.00
Postdoc/Trainee (M): $0.00
Postdoc/Trainee (NM): $25.00
Student (M): $0.00
Student (NM): $25.00
Credit Offered
4 CME (AMA) Credits
4 CME (Other) Credits
4 Certificate of Participation Credits

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(2022 ACMG Summer Gene Therapy Education Series: Advancements in Gene Therapy Options for Rare Diseases – This course is supported by an independent medical education grant from Pfizer, Inc.)


Date of Release: June 16, 2022

Expiration Date: June 30, 2024

Credits offered: CME, NSGC Category 2 (Self-report)

Estimate time of completion: 1 hour per webinar

Course must be completed by the expiration date

Series Information:

The 2022 ACMG Summer Gene Therapy Education Series: Advancements in Gene Therapy Options for Rare Diseases is designed to provide a basic background on gene therapy for all healthcare professionals. Experts will present new pathways in gene therapies and demonstrate best practices for patient care from existing guidelines from ACMG and ASGCT.


Topics include:

  • Gene Basics of Gene Therapy: Preclinical Development
  • Gene Therapy Targets: Inborn Errors of Metabolism
  • Gene Therapy Targets: Broadening the Scope of Therapy
  • Gene Therapy: Ethical, Social and Economic Issues

Learning Objectives:
At the conclusion of the webinar series, participants should be able to:

1. Consider gene therapy options for their patients

2. Determine patient eligibility for gene therapy

3. Discuss gene therapy with their patients

4. Counsel their patients regarding gene therapy options

5. Address patient safety concerns of viral vector technologies

Target Audience: This summer webinar series is targeted to genetics professionals in research, lab, and clinical settings, as well nongenetic healthcare professionals caring for patients with rare disease.


Accredited Continuing Education Information:

Continuing Medical Education (CME AMA & CME Other)



The American College of Medical Genetics and Genomics is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.


Credit Designation

The American College of Medical Genetics and Genomics designates this enduring activity for a maximum of 4 AMA PRA Category 1 Credit™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

National Society of Genetic Counselors (NSGC Self-report)

This activity meets requirements to apply for Category 2 CEUs from the National Society of Genetic Counselors (NSGC). Please complete the evaluation and submit your certificate to NSGC, using the instructions for “How To Submit a Category 2 CEU Application in the NSGC CEU Portal.” ACMG is the accredited sponsor. You must apply within 30 days of the activity date. NSGC will charge a processing fee of $25 for this activity. ACMG does not report attendance to NSGC for this activity.

Claiming your Educational Credits

This activity consists of: View content, take a post-test, the test may be taken as often as necessary to achieve a passing score of 80% or better is required to receive credit. If you do not achieve a passing score, the program will identify which questions you answered incorrectly so that you can review the module and try again. Complete the evaluation form.

Accredited Continuing Education Financial Disclosure

The American College of Medical Genetics and Genomics (ACMG) is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide Accredited Continuing Education (ACE) for physicians. ACMG is an organization committed to improvement of patient care and general health by the incorporation of genetics and genomics into clinical practice.

ACMG has implemented the following procedures to ensure the independence of ACE activities from commercial influence/promotional bias, the Accreditation Council for Continuing Medical Education (ACCME) requires that providers (ACMG) must be able to demonstrate that: 1) everyone in a position to control the content of an ACE activity has disclosed all financial relationships that they have had in the past 24 months with ineligible* companies; 2) ACMG has implemented a mechanism to mitigate relevant financial relationships; and 3) all relevant financial relationships with ineligible companies are disclosed to the learners before the beginning of the educational activity. The learners must also be informed if no relevant financial relationships exist.
*Ineligible companies are defined as those whose primary business is producing, marketing, selling, re-selling, or distributing healthcare products used by or on patients.


ACMG Education Policies

Please review the policies below regarding the ACMG Education program


All of the relevant financial relationships listed for these individuals have been mitigated.



Program Committee Member


Peer Reviewer

Financial Disclosure Relationship/Company

Stephanie Austin, MS, CGC



  • Employee: Amicus Therapeutics, Ins.

John A. Bernat, MD, PhD, FACMG



  • Research support for clinical trials: Avrobio, BioMarin Pharmaceutical Inc., Idorsia Pharmaceuticals, Pfizer, Inc., Protalix Biotherapeutics, Sangamo Therapeutics, Sanofi, Takeda Pharmaceutical, Travere Therapeutics, Inc.
  • SAB: Sanofi, Takeda Pharmaceutical

Lindsay C. Burrage, MD, PhD, FACMG

Nothing to disclose

Irene J. Chang, MD, FACMG


Nothing to disclose

Haiyan Fu, PhD



Nothing to disclose

Brett H. Graham, MD, PhD, FACMG

Nothing to disclose

Dwight Koeberl, MD, PhD, FACMG


  • Stock: Asklepios BioPharmaceutical (AskBio)
  • Consultant: Moderna
  • DSMB: Baxalta

Gerald S. Lipshutz, MD, PhD



  • DSMB: Astellas Pharma
  • Consultant: Taysha

Loren D. Pena, MD, PhD, FACMG



Consultant: Novartis Gene Therapies, Inc., Orphazyme, Sanofi US Services, Inc., Ultragenyx Pharmaceutical Inc.

Jane Radford, MHA, CHCP



Nothing to disclose

Kuntal Sen, MBBS, FACMG

Nothing to disclose

Amy Siemon, MS


Nothing to disclose

Charles P. Venditti MD, PhD



Grant/Contract: Selecta Biosciences

Jerry Vockley, MD, PhD, FACMG


  • Grants/Contracts: LogicBio Therapeutics, Homology Pharmaceuticals, Reneo Pharmaceuticals, Hemoshear Pharmaceuticals, BioMarin Pharmaceutical Inc., CoA Therapeutics, Nestle HealthCare Nutrition Inc., Synlogic Pharmaceuticals, 4D Molecular Therapeutics, Moderna Pharmaceuticals, Ultragenyx Pharmaceutical Inc., Aeglea BioTherapeutics, Stealth Pharmaceuticals
  • Consultant: BioMarin Pharmaceutical Inc., Applied Therapeutics, LogicBio Therapeutics, Agios Pharmaceuticals, Inc., Axcella Health, Orphan Labs, Sangamo Therapeutics, Synlogic Pharmaceuticals, Sanofi Pasteur Biologics, LLC, Ecrol Capital, Frictionless Solutions
  • Stock: American Gene Technologies

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