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The Basics of Gene Therapy: Preclinical Development

(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:

This session will review the basics of the preclinical development of a new gene therapy for a rare disorder. The presentation will span the bench to bedside spectrum with a focus on proof-of-concept and preclinical studies needed to initiate a clinical trial.

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.

 Learning Objectives:

At the conclusion of this session, participants should be able to:

• Describe key aspects of preclinical studies prior to Investigational New Drug (IND) application
• Review the key steps in the interaction with the FDA prior to submitting an IND application
• Explain the relevance of an IND number

Moderators:

Lindsay C Burrage, MD, PhD, FACMG

Assistant Professor, Molecular & Human Genetics

Baylor College of Medicine

Dwight Koeberl, MD, PhD, FACMG

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

Duke University Medical Center

Presenters:

Haiyan Fu, PhD

Research Associate Professor, Gene Therapy Center, Department of Pediatrics

University of North Carolina at Chapel Hill

Charles P. Venditti MD, PhD

Metabolic Medicine Branch, Head, Organic Acid Research Section

Senior Investigator, National Human Genome Research Institute

National Institutes of Health

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.