Opti-AAV | Towards a gene therapy for age-related macular degeneration (AMD)

Summary
While the blood neural barriers represent significant hurdles for the development of gene therapies for neurological and ophthalmological conditions it is now becoming clear that many of these conditions actually present with disrupted barrier integrity as the driving force of the pathology. Included in these diseases is age related macular degeneration (AMD), in which we have shown that the blood retina barrier (BRB) is sub-clinically disrupted and is a key driving force for disease progression (ERC funded Retina Rhythm project). Specifically related to this Proof of Concept grant, a novel form of therapy for the end stage of AMD, namely geographic atrophy (GA), is now desperately needed. A targeted approach to locally delivering adeno associated virus (AAV) to endothelial cells of the BRB would offer the opportunity to restore barrier function and prevent disease. However, the current tools available to achieve this localized approach to gene therapy are not optimum. Here we will use an exosome encapsulated and endothelial cell specific AAV to achieve robust transduction of retinal endothelial cells. This modified and optimized AAV (Opti-AAV) will represent a novel tool in translating recently identified biological mechanisms into real and meaningful therapies for patients.
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More information & hyperlinks
Web resources: https://cordis.europa.eu/project/id/101069382
Start date: 01-07-2022
End date: 31-12-2023
Total budget - Public funding: - 150 000,00 Euro
Cordis data

Original description

While the blood neural barriers represent significant hurdles for the development of gene therapies for neurological and ophthalmological conditions it is now becoming clear that many of these conditions actually present with disrupted barrier integrity as the driving force of the pathology. Included in these diseases is age related macular degeneration (AMD), in which we have shown that the blood retina barrier (BRB) is sub-clinically disrupted and is a key driving force for disease progression (ERC funded Retina Rhythm project). Specifically related to this Proof of Concept grant, a novel form of therapy for the end stage of AMD, namely geographic atrophy (GA), is now desperately needed. A targeted approach to locally delivering adeno associated virus (AAV) to endothelial cells of the BRB would offer the opportunity to restore barrier function and prevent disease. However, the current tools available to achieve this localized approach to gene therapy are not optimum. Here we will use an exosome encapsulated and endothelial cell specific AAV to achieve robust transduction of retinal endothelial cells. This modified and optimized AAV (Opti-AAV) will represent a novel tool in translating recently identified biological mechanisms into real and meaningful therapies for patients.

Status

SIGNED

Call topic

ERC-2022-POC1

Update Date

09-02-2023
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Horizon Europe
HORIZON.1 Excellent Science
HORIZON.1.1 European Research Council (ERC)
HORIZON.1.1.0 Cross-cutting call topics
ERC-2022-POC1 ERC PROOF OF CONCEPT GRANTS1
HORIZON.1.1.1 Frontier science
ERC-2022-POC1 ERC PROOF OF CONCEPT GRANTS1