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aSyn oligonucleotide
- Development stage
- Preclinical
- Lead developer
- Manifold Bio
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → 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, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Oral, Topical, Subcutaneous, Intravenous, Intramuscular, Inhalation, Intranasal, Ophthalmic, Rectal, Vaginal, Transdermal, Intrathecal, Intraperitoneal, Intra-arterial, Parenteral
01
Overview
aSyn oligonucleotide is a preclinical-stage therapeutic program being developed by Manifold Bio for the treatment of Parkinson's disease and other rare synucleinopathies. The drug utilizes a neuron-targeted delivery approach to provide either an antisense oligonucleotide (ASO) or a small interfering RNA (siRNA) directly to the central nervous system. Its primary mechanism of action is the modulation of alpha-synuclein (aSyn) expression, aiming to reduce the levels of this protein and prevent the formation of toxic aggregates that characterize these neurodegenerative diseases. The program is currently undergoing in vivo discovery and optimization to refine its targeting and efficacy.
- Other names
- aSyn oligonucleotideneuron-targeted alpha-synuclein oligonucleotideaSyn ASOaSyn siRNA
02
Targets
Alpha-synuclein messenger RNA
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