Expanded Capacity AAV Retinal Gene Therapy Enabled by Efficient RNA-Joining Technology
Grant Award Details
Grant Type:
Grant Number:
DISC2-14187
Investigator(s):
Award Value:
$0
Status:
Closed
Grant Application Details
Application Title:
Expanded Capacity AAV Retinal Gene Therapy Enabled by Efficient RNA-Joining Technology
Public Abstract:
Research Objective
A novel AAV-based gene therapy candidate for use in treatment of inherited ocular disease.
Impact
AAV vectors have a limited cargo capacity (<5kb), preventing their use in the treatment of many untreatable genetic diseases caused by large genes.
Major Proposed Activities
A novel AAV-based gene therapy candidate for use in treatment of inherited ocular disease.
Impact
AAV vectors have a limited cargo capacity (<5kb), preventing their use in the treatment of many untreatable genetic diseases caused by large genes.
Major Proposed Activities
- In vitro optimization and selection of gene therapy candidates
- In vivo testing to establish efficacy of gene therapy candidates
- Demonstration of disease-modifying activity in mouse genetic model of ocular disease
- Evaluation of safety for a dual vector AAV approach
- Testing activity in human photoreceptors
Statement of Benefit to California:
Of the 280 genetic mutations that cause inherited retinal disease (IRD), there is an approved treatment for just one. Our innovative technology provides a solution for challenges encountered by conventional AAV-based gene therapy approaches. Because our technology is agnostic to gene, the advancement of this therapy will be an important step forward for developing treatment for a wide variety of genetic diseases, including those that impact the diverse California population.