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AAV-miRNA

Development stage: Preclinical
Lead developer: Spark Therapeutics
Modality: Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
Administration: Parenteral

Overview

AAV-miRNA is an adeno-associated virus (AAV) mediated gene therapy that delivers an artificial microRNA (miRNA) designed to target and reduce the expression of human alpha-synuclein (α-syn). Developed by Spark Therapeutics, this RNA interference (RNAi)-based approach aims to treat synucleinopathies, including Parkinson’s disease and Multiple System Atrophy, by lowering the levels of soluble α-syn mRNA and protein. Preclinical data indicates that the therapy, delivered via a proprietary AAV capsid, can achieve significant knockdown of α-syn in the brain, providing neuroprotection and preventing the loss of dopamine-producing neurons in disease models.

Other names: AAV-mediated artificial miRNA targeting human alpha-synuclein

Targets

Alpha-synuclein messenger RNA

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