Developments, Research, Pipeline, and Applications of Gene Therapy - Annual 2019

ACPE UAN #:  0233-0000-19-014-H04-P

Release Date:  March 25, 2019

Expiration Date:   March 25, 2022

Activity Type:    Knowledge-based

Contact Hours0.75 

Activity Fee:  Member:  $15        Non-Member: $30

Target Audience: Pharmacists who practice in managed care settings.

Activity Description

Gene therapy is a promising treatment option for a number of diseases, including inherited disorders, some types of cancer, and certain viral infections. The first gene therapy approvals in the United States occurred in 2017. Tisagenlecleucel was the first gene therapy to be approved and is indicated for the treatment of lymphoblastic leukemia in pediatric and young adult populations. The first gene therapy approved for the treatment of an inherited rare disease was voretigene neparvovec-rzyl, which treats inherited retinal disease due to mutations in both copies of the RPE65 gene. Additional gene therapies have been approved for use in Europe.

Recent estimates indicate that there are nearly 150 gene therapies currently in development, suggesting that these approvals represent only the tip of the iceberg. Metabolic, ophthalmological, and hematological rare diseases together comprise more than half of the disease targets in the gene therapy pipeline. In this session, presenters will review currently-approved gene therapies and provide an analysis of the gene therapy pipeline.

Learning Objectives:  

At the completion of this activity, participants should be able to:

  1.  Discuss examples of currently-approved gene therapies.
  2.  Explain potential future applications of gene therapy.
  3.  Describe the current status of the research pipeline for investigational gene therapies.

Schedule of Educational Activity

This activity consists of an audio recording, pre-test assessment questions (not required to pass or continue), post-test questions (required to pass with a score of 70% or higher) and a specific activity evaluation. 


The Academy of Managed Care Pharmacy (AMCP) is accredited by the Accreditation Council for Pharmacy Education as a provider of continuing pharmacy education.

As an accredited provider, AMCP strictly adheres to the ACPE Standards for Commercial Support and other relevant guidelines and standards to ensure the independence of continuing pharmacy education (CPE) activities.  It is AMCP’s intent that all CPE activities be independent, objective, balanced, evidence-based and free of any commercial bias.

CPE Requirements:   

Participants should claim continuing pharmacy education (CPE) credit for this home study activity only if they have not claimed credit for the live activity presented at Specialty Connect 2019.

CPE credit will be processed through the AMCP Learn portal and reported directly to CPE Monitor.  Please allow 72 hours for processing and posting in your NABP account.

To receive credit for an online activity you must:

  • Review the full content of the activity.
  • Successfully complete the post-test (required to pass with a score of 70% or higher, it may be taken a maximum of three times).
  • Complete a specific activity evaluation.

Statement of Commercial Support

This educational activity is supported by independent educational grants from AveXis Inc., Genzyme Corporation, Pfizer and Sarepta Therapeutics.


Clifford Goodman, PhD | Senior Vice President & Director | Lewin Center for Comparative Effectiveness Research | Falls Church, Virginia.

System Technical Requirements/Viewing Requirements: System Requirements Courses and learning activities are delivered via your Web browser and Acrobat PDF. For all activities, you should have a basic comfort level using a computer and navigating websites.  View the minimum technical and system requirements for continuing education online learning activities.

Credit Information

Activity Number Credit Amount Accreditation Period
ACPE UAN #0233-0000-19-014-H04-P 0.75 Contact Hours March 25, 2019 - March 25, 2022