GCT Catapult – Selecting appropriate models for non-clinical studies of gene therapies
A review by The Cell and Gene Therapy Catapult sets out the key factors that should be considered when selecting an animal model to be used in non-clinical studies for adeno-associated virus (AAV) based gene therapies.
Currently, six AAV-based gene therapies have been approved in the UK, including treatments for spinal muscular atrophy and haemophilia, with many more experimental therapies being developed.
Before testing an experimental gene therapy in human clinical trials, a developer is currently required by the regulatory authorities to demonstrate that their product is both safe and effective in animal studies. It is vital that developers select an appropriate animal model for these studies as this helps them to ethically and optimally gather the data that is needed to progress to human clinical trials.
In this review, published in Gene Therapy, the non-clinical team at Cell and Gene Therapy Catapult provide guidance on four factors that developers should consider when selecting an animal model to test AAV-based gene therapies.
Published on the 12th of september 2023. Read the full news report here.
OpenEdition vous propose de citer ce billet de la manière suivante :
adelage (20 mai 2024). GCT Catapult – Selecting appropriate models for non-clinical studies of gene therapies. ELSIBI. Consulté le 11 octobre 2026 à l’adresse https://doi.org/10.58079/11osm
