A Platform for Gene Therapy Delivery: Adeno-Associated Virus (AAV) Vectors

Target Audience

This activity is designed to meet the educational needs of geneticists, nephrologists, hepatologists, pediatricians, neonatologists, pathologists, and other HCPs who often contribute to the multidisciplinary team when managing patients with genetic disorders.

Program Overview

The goal of gene therapy is to treat diseases at the genetic level by replacing deficient enzymes in target tissues, therefore, changing the course of the disease. Simply stated, it is the transfer of genetic material for disease therapy or prevention. Gene therapy with adeno-associated virus (AAV) vectors has demonstrated safety and long-term efficacy in several trials across target organs, including eye, liver, skeletal muscle, and the central nervous system.

The goal of this CME-certified activity is to increase physicians' knowledge of clinical considerations relating to the use of adeno-associated virus (AAV) vectors in gene therapy, and its impact on the treatment of several rare disorders.

Learning Objectives

Upon completion of this activity, participants should be better able to:

• Analyze gene‐based treatment approaches that have the potential to change the disease course for patients with genetic disorders
• Review the characteristics of AAV that allow it to be used as a vector for gene therapy
• Evaluate evidence for the use of gene therapies, including AAV-based, that are approved or pending approval for the management of rare disorders
• Counsel patients about clinical-trial opportunities