Drug intelligence / Profile preview

aptoll

Development stage
Phase 2
Lead developer
aptaTargets
Modality
Spiegelmers → RNA Aptamers → RNA Therapeutics → Nucleic Acid Therapeutics, DNA Vaccines → Plasmid DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Vaccine mRNA → mRNA 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, Modified DNA Aptamers → DNA Aptamers → DNA Therapeutics → Nucleic Acid Therapeutics, Gene Therapy Plasmids → Plasmid DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Protein Replacement mRNA → mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Gene Editing mRNA → mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Natural RNA Aptamers → RNA Aptamers → RNA Therapeutics → Nucleic Acid Therapeutics, Unmodified DNA Aptamers → DNA Aptamers → DNA Therapeutics → Nucleic Acid Therapeutics, miRNA Mimics → MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Long Non-coding RNA (lncRNA) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, miRNA Inhibitors → MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small 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
Administration
Intravenous
01

Overview

ApTOLL is a first-in-class neuroprotective drug developed by AptaTargets. It is an unmodified single-stranded DNA aptamer (ssDNA) that acts as a highly specific antagonist of Toll-like receptor 4 (TLR4), a key receptor in the innate immune system. By binding to and inhibiting TLR4, ApTOLL blocks the inflammatory cascade triggered after acute ischemic events such as stroke or myocardial infarction, thereby reducing inflammation-induced cell damage and improving functional outcomes. The drug has demonstrated significant neuroprotective effects in preclinical models and clinical trials for acute ischemic stroke, including reduced mortality and infarct volume. It is administered intravenously and is being investigated for use in both acute ischemic stroke (AIS) and ST-elevation myocardial infarction (STEMI)[1][2][3][5][7][8].

Brand names
ApTOLL
Other names
Aptamer-4FtAptamer4FtAptamer 4Ft
02

Targets

TLR4 (Toll-like receptor 4)

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