SIG: Cell and Gene Therapy

Dear all, 

Thanks to those that were able to attend our hybrid CGT SIG meeting on the 26th August 2025. I am enclosing the notes from the meeting below. 

Date: August 26, 2025
Type: Hybrid Meeting (In-person and Virtual)
Attendees: ~16

Key Discussion Points

1. SIG Leadership and Roles

   • Leadership Transition:

     • Lourens and Jamie will continue as Co-Chairs.

     • Hannah is stepping down as Secretary as she goes on maternity leave.

     • A call was made for a volunteer to assume the role of CGT SIG Secretary.

2. Webinar Planning

   • Webinar Focus: Long-term follow-up (LTFU) of cell and gene therapies, inspired by a recently published overview paper.

   • Current Status: Open to suggestions for speakers.

   • Action Item: Lisette Hoogendoorn to follow up on potential expert speakers.

3. Manuscript Development

   • First Manuscript:

     • Published in March 2025.

     • It has already received four citations.

   • Second Manuscript in Development:

     • Topic: Data quality for real-world long-term follow-up studies in gene therapy.

     • Proposed Focus Areas:

       • Comparative perspectives.

       • Data quality frameworks.

       • Involvement of 2–3 data sources/registries.

     • Potential Disease Areas:

       • Spinal Muscular Atrophy (SMA) and Haemophilia.

     • Discussion Points:

       • Exploration of long-term follow-up of patients treated in clinical trials using real-world data.

       • Future applications of artificial intelligence.

     • Timeline:
       Preliminary outline to be completed by October 2025.

4. Any Other Business (AOB)

   • ACPE Symposium Submission:

     • Keiko informed the group that the CGT SIG proposal was not accepted but plans are underway to submit to an alternative conference.

   • Biologics Course Update:

     • Provided by Quazi Ataher.

     • Included a segment on Cell and Gene Therapies.

     • Attendance: 15–16 students.

     • Observation: 90% of the students had basic or no prior experience with CGTs.

5. Future Meeting Planning

   • Meeting Cadence: Every two months.

   • Next Meeting: September 25, 2025.

     • Location: Virtual.

     • Time: 1:00 p.m (CET)/ 7:00 a.m. Eastern Time (ET)

   • Future meetings: 
     • Thu 27 Nov, Thu 22 Jan, Thu 19 Mar, Thu 28 May, During ISPE Annual Meeting (TBC)

Upcoming Opportunities

• Explore conference submissions (e.g., DIA Global or DIA Asia).

• Investigate patient engagement strategies.

• Consider organizing a dedicated CGT pre-conference course.

Action Items

• Hannah:
  Transition Secretary responsibilities to a new volunteer.
  

• Lisette:
  Finalize the webinar speaker lineup.
  

• Lourens & Team:

• Develop the manuscript outline by October 1, 2025.

• Continue exploring manuscript collaboration opportunities.

• All Members:

• Share speaker suggestions for the webinar.

• Join the webinar working group-contact Lisette at lisette.hoogendoorn@iqvia.com.

• Contribute to manuscript development.

Best wishes,

Hannah (on behalf of Lourens and Jamie)

(CGT SIG Secretary)

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Hannah Furby
F. Hoffmann-La Roche AG
Welwyn Garden City
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