Drug intelligence / Profile preview

F5R2

Development stage
Preclinical
Modality
RNA Aptamers → RNA Therapeutics → Nucleic Acid Therapeutics, DNA Aptamers → DNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intraperitoneal, Intravenous
01

Overview

F5R2 is a DNA aptamer specifically developed to bind aggregated α-synuclein (alpha-synuclein) with high affinity. It inhibits α-synuclein aggregation, promotes lysosomal degradation of α-synuclein aggregates in neurons, and protects against α-synuclein-induced mitochondrial dysfunction. F5R2 is highly specific for aggregated forms of human α-synuclein, does not recognize endogenous mouse monomeric α-synuclein, and demonstrates no significant off-target effects with Aβ42 (amyloid beta) oligomers. Developed by aptamer selection (SELEX), F5R2 is under investigation as a potential disease-modifying therapy for Parkinson’s disease and other synucleinopathies[1][3].

02

Targets

aggregated α-syn (Alpha-synuclein (α-syn))

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