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IND-499-siRNA
- Development stage
- Preclinical
- Lead developer
- Institut d'Investigacions Biomèdiques August Pi i Sunyer
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intranasal
01
Overview
**IND-499-siRNA** is a preclinical, indatraline-conjugated small interfering RNA designed to selectively silence **SNCA** messenger RNA encoding alpha-synuclein in monoaminergic neurons. The covalently attached triple monoamine-transporter inhibitor indatraline is intended to promote uptake into dopaminergic, serotonergic, and noradrenergic neurons following intranasal administration. In mouse studies, intranasal dosing reduced alpha-synuclein messenger RNA and protein in brainstem monoamine nuclei without evidence of monoaminergic neurodegeneration, supporting investigation as a disease-modifying approach for Parkinson's disease. ([pubmed.ncbi.nlm.nih.gov](https://pubmed.ncbi.nlm.nih.gov/29273501/))
02
Targets
SNCA (Alpha-synuclein)
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