Perspectives on the FDA platform technology designation program for the approval of gene therapies: a Swiss multi-stakeholder exploratory interview study
The introduction of the platform technology designation program in the US is a step towards increasing treatment options for ultra-rare diseases. While there is potential for platform designation to enable development in this area, its success will depend on addressing the outlined challenges.
Authors
- Kelly E. Ormond (ORCID: https://orcid.org/0000-0002-1033-0818)
- yuehu han
Institutions
- ETH Zurich (CH)
Publication Details
- Journal
- Orphanet Journal of Rare Diseases
- Published
- 2025-10-14
- DOI
- https://doi.org/10.1186/s13023-025-04038-y
- Primary Topic
- CRISPR and Genetic Engineering
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Eidgenössische Technische Hochschule Zürich
- National Institutes of Health