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IND-1233-ASO
- Development stage
- Preclinical
- Lead developer
- nLife Therapeutics
- Modality
- MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intranasal, Parenteral
01
Overview
**IND-1233-ASO** is an investigational, indatraline-conjugated antisense oligonucleotide designed to reduce expression of **alpha-synuclein** by sequence-specifically targeting alpha-synuclein mRNA. The indatraline ligand is a triple monoamine-transporter inhibitor used as a neuronal-targeting moiety to preferentially deliver the ASO into dopaminergic, serotonergic, and noradrenergic neurons through dopamine, serotonin, and norepinephrine transporters. In preclinical mouse and nonhuman-primate studies, intranasal or intracerebroventricular administration reduced alpha-synuclein synthesis and accumulation in vulnerable monoaminergic brain regions and improved dopamine neurotransmission in Parkinson's disease models. ([mdpi.com](https://www.mdpi.com/1422-0067/22/6/2939))
- Other names
- indatraline-conjugated 1233-ASOindatraline-conjugated1233-ASOindatraline-conjugated-1233-ASO
02
Targets
SNCA (Alpha-synuclein)SERT (Sodium-dependent serotonin transporter)DAT (Dopamine plasma membrane transport protein)NET (Norepinephrine Transporter)
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