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Gene Therapy Targets: Broadening the Scope of Therapy

(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 provide a broad overview of the potential for AAV mediated gene transfer to be used as a platform approach for the development of therapies targeting the nervous system. The session will highlight the success in bringing these approaches from preclinical studies to clinical trials and will highlight some of its limitations. Given the expansion of AAV mediated gene transfer approaches being developed and studied in the neuromuscular field, this session will also provide insight to key clinical considerations around patient eligibility and selection, immune monitoring and immune modulation, and assessment of outcomes following gene transfer. The presentation will review outcomes of onasemnogene abeparvovec-xioi (Zolgensma) in spinal muscular atrophy (SMA) and related clinical and ethical issues.

Learning Objectives:

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

1. Discuss basic approaches to deliver a gene to the nervous system.

2. Review safety and immunologic considerations that impact clinical gene replacement therapy.

3. Recognize how gene replacement therapy can lead to shift in care and natural history, using SMA as an example.

Moderators:

Kuntal Sen, MD, FACMG

Attending Pediatric Neurogeneticist

Children’s National Hospital, Washington, DC

Brett Graham, MD, PhD, FACMG

Associate Professor and Associate Director of Clinical Genetics Department of Medical and Molecular Genetics Indiana University School of Medicine, Indianapolis, IN

Presenters:

Diana Bharucha-Goebel, MD

Assistant Professor, Neurology and Pediatrics, Children’s National Health System

Clinical Research Collaborator, National Institutes of Health, NINDS/ Neurogenetics Branch