Drug intelligence / Profile preview

F5R1

Development stage
Preclinical
Lead developer
Aptagen
Modality
miRNA Mimics → MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Natural RNA Aptamers → RNA Aptamers → RNA Therapeutics → Nucleic Acid Therapeutics, DNA Vaccines → Plasmid DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Spiegelmers → RNA Aptamers → RNA Therapeutics → Nucleic Acid Therapeutics, Unmodified DNA Aptamers → DNA Aptamers → DNA Therapeutics → Nucleic Acid Therapeutics, Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Gene Editing mRNA → mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Vaccine mRNA → mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Long Non-coding RNA (lncRNA) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Aptamers → DNA Aptamers → DNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Protein Replacement mRNA → mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, miRNA Inhibitors → MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Gene Therapy Plasmids → Plasmid DNA → DNA Therapeutics → Nucleic Acid Therapeutics
Administration
Not Specified (experimental Studies Used Cellular Delivery And Transfection; Probable Future Use Includes Intracerebral Or Intranasal Administration)
01

Overview

**F5R1** is a DNA aptamer specifically selected for its high affinity and specificity to human alpha-synuclein (α-syn). Aptamers are short, single-stranded nucleic acid ligands that bind to target molecules with high specificity. F5R1 was developed using an optimized SELEX process to target the N- and C-termini of α-synuclein. It has been shown in vitro and in cellular models to inhibit α-syn aggregation, promote lysosomal degradation of α-syn, prevent its association with mitochondria, and considerably reduce cytotoxicity and mitochondrial dysfunction induced by α-syn overexpression, indicating potential utility in treating neurodegenerative disorders such as Parkinson's disease[1].

02

Targets

SNCA (Alpha-synuclein)

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