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AVB-SNCA
- Development stage
- Preclinical
- Lead developer
- AviadoBio
- Modality
- Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intravenous
01
Overview
AVB-SNCA is an investigational, preclinical gene therapy designed to lower SNCA expression in the brain for the potential treatment of synucleinopathies, including Parkinson's disease, Lewy body disease, and multiple system atrophy. Developed by AviadoBio, the therapy utilizes the proprietary vMiX™ RNA interference (RNAi) gene-silencing platform delivered via an adeno-associated virus (AAV) vector. It delivers a microRNA (miRNA) payload designed to target and silence SNCA mRNA, thereby reducing the expression of the disease-causing alpha-synuclein protein. AVB-SNCA is designed as a potential one-time, disease-modifying treatment administered intravenously, leveraging blood-brain barrier-crossing capsids and a neuron-specific promoter to focus activity within the central nervous system.
02
Targets
SNCA (Alpha-synuclein)
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