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Educational Session

Biotherapies, Cellular Therapies, and Immunotherapies

AM25-ST-14-L - Allogeneic Transplant, Gene Therapy, or the Evolving Drug Landscape in Sickle Cell Disease: Helping Patients Choose Between Options

Saturday, October 25, 2025
10:45 AM - 12:15 PM  PT
Location: 30CDE

    Program Chair(s)

  • Patricia Shi, MD (she/her/hers)

    Vice-President
    New York Blood Center
    Rye, New York

    Disclosure(s): No financial relationships to disclose

    • Speaker(s)

  • ST

    Swee Lay Thein, MD, DSc., FMedSci

    Senior Investigator
    Sickle Cell Branch, NHLBI, NIH, Maryland

    Disclosure(s): No financial relationships to disclose

  • Adetola Kassim, MD, MS

    Professor of Medicine; Director, Adult Sickle Cell Disease Program; Clinical Director, Adult Stem C
    Vanderbilt University
    Nashville, Tennessee

    Disclosure(s): No financial relationships to disclose

  • Julie Kanter, MD (she/her/hers)

    Co-director; Professor
    The University of Alabama at Birmingham
    Mount Olive

    Disclosure(s): No relevant disclosure to display

Proposed Session Description: Allogeneic transplant outcomes using HLA-haploidentical donors have improved to the point that it is a potentially curative option for many patients with sickle cell disease. At the same time, there are currently two FDA options for autologous gene therapy for SCD and more on the horizon. Still more to consider is that, although voxelotor was withdrawn from the market, new Hb-raising drugs that seem more promising, such as the pyruvate kinase activators, are in Phase III clinical trials. Drug-based BCL11A inhibition is also being investigated. In this session, the state of the art and ongoing clinical trials in haploidentical transplant, gene therapy and non-curative options will be described: our experts will offer their perspective on how patients and providers should choose between these three options given their risks and benefits. Transfusion history considerations with each therapy will also be reviewed. The session will conclude with a roundtable discussion of what clinicians, including transfusion medicine specialists, need to consider to best guide their patients with sickle cell disease.

Learning Objectives:

  • Identify the risks with and appropriate patient profiles for current and future FDA-approved gene therapies.
  • Identify the risks with and appropriate patient profiles for haploidentical transplant.
  • Discuss the drugs that are expected to be approved in the next 2-5 years for sickle cell disease and their potential efficacy compared to transplant.