Abstract: SA-PO0776
Patient and Carer Feedback on a Nephrology Gene Therapy Study Concept
Session Information
- Glomerular Research: Design, Registries, Surveys, and Epidemiology
November 08, 2025 | Location: Exhibit Hall, Convention Center
Abstract Time: 10:00 AM - 12:00 PM
Category: Glomerular Diseases
- 1402 Glomerular Diseases: Clinical, Outcomes, and Therapeutics
Authors
- Harrison, Pille, Purespring Therapeutics Limited, London, England, United Kingdom
- Marsala, John, IgA Nephropathy Foundation, Wall, New Jersey, United States
- Harper, Heather Kate, National Institute for Health and Care Research, London, England, United Kingdom
- Munday, Davina, National Institute for Health and Care Research, London, England, United Kingdom
- Pickering, Emily, National Institute for Health and Care Research, London, England, United Kingdom
- Smerdon, Charlotte J, Purespring Therapeutics Limited, London, England, United Kingdom
- Rowland, Alicia, Purespring Therapeutics Limited, London, England, United Kingdom
- Erlandsson, Fredrik, Purespring Therapeutics Limited, London, England, United Kingdom
Background
There is limited experience with gene therapies in nephrology. The first gene therapy trials specifically targeting kidney cells are likely to open for enrolment in 2025 and 2026. Patient input is required to optimize and appropriately design clinical trials and novel treatments.
Methods
Virtual insight sessions were conducted to understand IgAN patients’ and patient carers’ perceptions of a gene therapy trial. Each session involved 6 participants and was independently conducted by the IgA Nephropathy Foundation (US) and the National Institute for Health and Care Research (UK). Participants received detailed information on the risks and requirements of participation in a phase 1 gene therapy trial, and feedback was collected and summarized.
Results
IgAN patients reported fatigue, anxiety, and side effects of treatment as the main impact of their disease on daily life. Several participants shared feelings of concern about participating in a phase 1 gene therapy trial, especially with regards to side effects, but there was also excitement around trying a completely new approach. There was strong support for once-and-done therapy being more attractive than current treatments. Questions were raised about identifying the right patients for gene therapy to ensure efficacy in every treated patient. A 4-day in-patient stay would not deter most patients from enrolling, especially if visits and calls from friends and family was allowed. Some patients considered reimbursement of certain costs critical. A follow-up research kidney biopsy may be accepted by most if informative, but patients wanted thorough information on associated risks. There was mixed reception to doing a baseline biopsy, and to receiving a course of corticosteroids to minimize gene therapy side effects.
Conclusion
Patient input was generally supportive of a planned phase 1/2 gene therapy trial, and suggested improvements related to the trial design, conduct, and patient facing materials. Minimizing biopsy collection and corticosteroid use in gene therapy trials may increase enrolment. Unknown side effects and potential lack of efficacy in subsets of patients were the main patient concerns, highlighting the need for further research.
Funding
- Commercial Support – Purespring Therapeutics