Self-Study 2025 Release II: Gene Therapy Advances in Hemophilia, Sickle Cell Disease, and Beta-Thalassemia

Type: Individual•Format: On-demand

For inherited classical hematologic diseases like hemophilia, sickle cell disease, and beta-thalassemia, there are treatments available to control symptoms, but none cure or prevent the genetic errors that cause them up until the recent development of gene therapy cures. In this module, we will explore the different mechanisms of action of available agents and their varied impact on target diseases. Readers will also consider the challenges to incorporating gene therapies into routine care of individuals with hemophilia, sickle cell disease, and beta-thalassemia.

Gene Therapy Advances in Hemophilia, Sickle Cell Disease, and Beta-Thalassemia

Author: Jocelyn Mohs, PharmD, BCOP

Learning Objectives

  1. Describe gene therapies used in hemophilia and beta-thalassemia
  2. Evaluate efficacy and safety outcomes from completed gene therapy trials in hemophilia and beta-thalassemia
  3. Examine the cost-effectiveness and challenges for clinical use of gene therapies in hemophilia, sickle cell disease, and beta-thalassemia

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Knowledge Course for Pharmacists

Technology requirements: HOPA Learn requires a modern web browser (Internet Explorer 7+, Mozilla Firefox, Apple Safari, Google Chrome) and the ability to listen to audio with the content.

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HOPA is accredited by the Accreditation Council for Pharmacy Education as a provider of continuing pharmacy education. In order to claim BCOP credit, you must pass the BCOP Post-Test with a 75% or higher.

All CE hours will be transmitted to the CPE Monitor and BPS within 1-2 weeks of course completion.