SIG: Cell and Gene Therapy

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  • 1.  28 May Meeting Minutes

    Posted 7 days ago

    Hi All,

    See below for full meeting minutes, a few call outs to make at the top, please let us know if you are interested in any of the following opportunities to support the SIG:

    • Small working group review of the SIG mandate and update to core mission
    • Volunteers for SIG leadership for 2026-2027, formal transition to occur after the ISPE annual meeting

    Please note that our next scheduled meeting will be during the ISPE meeting in Milan on Tue 1 Sep at 11:30 CET and we'll try to get a room to support virtual attendance, Zoom link to follow.

    Best,

    Kristen

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    Pre-Conference Course Update

    • ISPE CGT SIG successfully secured pre-conference course for annual meeting
      • Title: "Evaluating Long-Term Safety and Effectiveness of Gene Therapies Using Real World Data"
      • Saturday May 29th afternoon, 4-hour session
      • Built on published manuscript (30+ citations in 14 months) and successful webinar (300+ attendees)
    • Course faculty includes:
      • Peter Mol (EMA/University of Netherlands) - regulatory requirements
      • Conny Berlin (Novartis) - patient engagement perspective
      • Lourens - feasibility factors from EMA reportsQuazi - prevention/mitigation measuresCase studies of successful/unsuccessful RWD studies
    • Highly interactive format planned vs traditional lecture style
    • Potential for recurring course pending attendance/feedback
    • Future iterations could involve broader SIG community as faculty

    Long-Term Follow-Up Manuscript Progress

    New manuscript focusing on data quality in post-authorization RWD studies

    • Core team: Nahila, Lisette, Andreja Ilic (Novartis Medical Safety), Carla, Lourens (5 people currently)
    • Scope: Understanding registry experiences with gene therapy long-term follow-up
      • Target registries across 3 domains:
        1. EBMT/CIBMTR - cell-based gene therapies, sickle cell disease
        2. World Federation of Hemophilia - in vivo gene therapies
        3. Treat-NMD - neurological conditions registry network
    • Methodology:
      • Literature review + qualitative interviews with registry holders
      • Registry representatives as co-authors
      • Patient interviews if possible (pediatric to adult transition focus)
    • Master's student joining for groundwork/interviews starting summer
    • Timeline: Complete by end of year
    • IRB approval needed for patient interviews

    SIG Policy & Mandate Review

    • Current mission statement from 2022 establishment needs updating
    • Proposal to critically review mandate with small working group
      • Include new leadership (Jamie/Lourens stepping down August)
      • Develop clearer rules/procedures for SIG operations
      • Better align with actual activities vs backward engineering reports
    • Request for volunteers to participate in review process

    Leadership Transition

    • Jamie and Lourens stepping down from leadership roles in August
    • Handoff planned for annual meeting or early September
    • Both will remain involved in SIG activities (manuscript completion, etc.)
    • Call for new leadership volunteers from broader SIG community
    • Conference room requested for SIG meeting in Milan


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    Kristen Hahn MPH, PhD
    PicnicHealth
    Old Lyme CT
    +1 (860) 575-3411
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