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AM23-ST-01-O - AM23-ST-01-O: Optimizing Apheresis Collections to Enable Hematopoietic Stem Cell Gene Therapies (Enduring)


Expiration Date: Dec 31, 2025


Credits: None available.

Gene therapy can potentially treat or cure a wide range of disease entities and is being used mainly in clinical trials and certain cases as FDA-approved therapies. The starting material for these treatments is often collected via apheresis procedures where a relatively large number of hematopoietic progenitor cells (HPCs) are needed in a short period of time to undergo ex vivo manipulation to generate a therapeutic drug product. This session will provide a brief overview of gene therapy, using sickle cell disease, beta-thalassemia, and active cerebral adrenoleukodystrophy (CALD) as examples to illustrate strategies for gene editing/introduction. We will describe challenges with the mobilization and collection of HPCs and potential solutions for optimization of mobilization/collection of HPCs and discuss the risks and benefits of gene therapy as well as strategies to measure success.The audience for this session includes physicians, laboratory and quality personal involved in HPC mobilization and collection, apheresis centers, and blood banks that may be processing and shipping cells for manufacture.


Learning Objectives:

  • Outline gene therapy with regards to vectors and ex vivo manipulation using sickle cell disease, beta-thalassemia and active cerebral adrenoleukodystrophy as examples.
  • Discuss challenges in mobilization and collection of HPCs in patients with genetic diseases and highlight the importance of a multidisciplinary approach.
  • Describe various strategies for optimization of mobilization and collection of HPCs and in developing protocols and procedures to ensure success.

Moderator(s):

Speaker(s):

Disclosures

  • Reuben Jacob, MD:
    No financial relationships to disclose
  • John Manis, MD:
    No relevant disclosure to display
  • Joseph (Yossi) Schwartz, MD, MPH:
    No financial relationships to disclose

All relevant financial relationships have been mitigated.

By completing the evaluation, you are attesting to watching the presentation in its entirety. A certificate will be immediately provided after submission.

Credits Available


AM23-ST-01-O: Optimizing Apheresis Collections to Enable Hematopoietic Stem Cell Gene Therapies (Enduring) Evaluation