Webinar
“Spot the difference”: ophthalmology and potency assays for optogenetic gene therapy drugs
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About this webinar:
Gene therapy has emerged as a promising and powerful treatment modality for numerous human diseases, with Adeno-Associated Virus (AAV) vectors recognized as some of the safest and most widely used delivery systems. Such vectors have been proven to be particularly successful in ophthalmology.
In this field, Axxam has recently developed and validated a potency assay on behalf of GenSight Biologics for the AAV-based optogenetic drug GS030-DP for visual restoration in patients with Retinitis Pigmentosa.
In our webinar, we will briefly introduce the principle behind gene therapy and optogenetics, and recapitulate on potency assays; then, we will describe the GS030-DP (GenSight Biologics) case study providing insights on the main steps of potency assay development and validation. Lastly, we will illustrate Axxam’s advanced technological platforms, which allow us to tailor potency assays and develop them also for complex biological medicines in various application fields.
In this webinar you will learn about:
- Gene therapy and optogenetics as therapeutic strategies in the ophthalmology field
- Development of a potency assay for the optogenetic drug GS030-DP on behalf of GenSight Biologics
- Axxam’s technological platforms and expertise in the development of potency assays providing robust solutions for a critical step in the drug development process
Keywords:
Gene Therapy; AAVs; Ophthalmology; Optogenetics; Cell-based Assays; Potency Assays; Stability studies; Drug Discovery; Drug Development; CMC; ICH guidelines.
Speakers:
Viviana Agus –Unit Manager, Unit 2, Cell Biology, Axxam
Lorenza Garribba – Scientist, Cell Biology 2, Assay Development, Axxam
Viviana Agus joined Axxam in 2001, after her Master’s degree in Biology. She worked as a scientist for several years, mainly setting up cell-based assays for different targets: GPCR, ion channels, transporters and hormone receptors. She gained strong experience with automated 384-well screening platforms. In 2015 she started developing a platform of Optogenetic assays to be used in drug discovery and subsequent drug development, taking care of scientific scouting, creation of cellular models, and invention of new dedicated instrumentation. She is currently Unit Manager of one of the Axxam Cell Biology teams, managing both internal research activities and fee-for service projects for clients.
Lorenza Garribba is currently a Scientist in the Cell Biology-Assay Development group at Axxam. Over the last 2 years, she has successfully developed several cell-based assays suitable for high-throughput screening and compound profiling on a wide range of targets: ion channels, GPCRs and ChannelRhodopsins. Lorenza has a Master’s Degree in Biology for Molecular, Cellular and Physiopathological Research (Università degli Studi Roma Tre) and a PhD in Health and Medical Sciences (University of Copenhagen). She has a solid background in the genome instability field and is author and co-author of several relevant publications in the field.