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Gene Therapy Targets: Inborn Errors of Metabolism

(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.)

ACMG Genetics Academy – Live Webinar

Session Description:

 The goal of this session is to discuss selected inborn errors of metabolism as candidates for gene therapy. Given that inborn errors of metabolism are single gene defects that each cause a well-characterized enzyme deficiency, these disorders are ideal candidates for gene replacement therapy. Proof-of-concept studies and clinical translational research will be discussed for several disorders with an emphasis on recent developments and common themes.

Learning Objectives:

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

1. Describe the basis for biochemical correction of muscle in Pompe disease involving receptor mediated uptake of GAA.
2. List two advantages of liver-targeted gene therapy for Pompe disease.
3. Discuss the problems posed by antibody responses against the AAV vector and against GAA.
4. Recognize the underlying pathophysiology and potential toxins in Arginase Deficiency.
5. Develop an appreciation for the neuropathology that occurs in Arginase Deficiency 

Moderator:

Stephanie Austin, MS, MA, CGC

Genetic Counselor, formerly at Duke University

Medical Science Liaison and Associate Director at Amicus Therapeutics

 
Co-moderator & Presenter:

Dwight Koeberl, MD, PhD, FACMG

Professor, Department of Pediatrics/Division of Medical Genetics, 
Department of Molecular Genetics and Metabolism, Medical Director, Biochemical Genetics Laboratory

Duke University Medical Center

Presenter:

Gerald S. Lipshutz, MD

Professor-in-Residence, Member, Intellectual and Developmental Disabilities Research Center
University of California UCLA
Department of Molecular & Medical Pharmacology, University of California, Los Angeles

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. There will be four live multidisciplinary team webinars. Experts will present new pathways in gene therapies and demonstrate best practices for patient care from existing guidelines from ACMG and ASGCT. Each webinar will be 60 minutes with the last 10 minutes reserved for panel discussion/Q&A.

The four webinars’ dates and topics include:

• June 9              Gene therapy basics: vector options and preclinical development
• June 23            Gene therapy targets: inborn errors of metabolism
• July 7               Gene therapy targets: broadening the scope of therapy
• July 21             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. 

Accreditation
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 live activity for a maximum of 1.0 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Claiming your Educational Credits

Complete the activity and carefully complete the evaluation form. The deadline to claim educational credits is within 30 days from the date of the activity. Educational credit requests after this date will not be accepted.

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

• Financial Disclosures and Mitigation Procedure
• ACMG Content Validation Policy
• ACMG Content Branding Policy

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