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AAV-APOE2-amiRNA
- Development stage
- Preclinical
- Lead developer
- Voyager Therapeutics
- Modality
- Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intravenous
01
Overview
AAV-APOE2-amiRNA is an investigational bi-functional adeno-associated virus (AAV) gene therapy developed by Voyager Therapeutics for the treatment of Alzheimer's disease. It utilizes Voyager's proprietary VCAP-Gen2 blood-brain barrier (BBB)-penetrant capsid for intravenous delivery. The therapeutic payload is designed to simultaneously knockdown the expression of the high-risk Apolipoprotein E4 (ApoE4) allele using artificial microRNAs (amiRNA) and provide concomitant expression of the protective Apolipoprotein E2 (ApoE2) isoform. This dual-action approach aims to shift the ApoE profile in the brain toward a more neuroprotective state, potentially slowing or preventing the progression of Alzheimer's disease.
02
Targets
APOE2 (Apolipoprotein E2)ALPL (Tissue-nonspecific alkaline phosphatase)
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