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1-Azepan-1-yl-2-phenyl-2-(4-thioxo-1,4-dihydro-pyrazolo[3,4-d]pyrimidin-5-yl)ethanone adduct

Development stage: Unknown
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, Small Molecules, 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

Overview

This compound is an experimental nucleic acid-based molecule classified as a biotech/oligonucleotide. It is not approved for clinical use and has no known marketed indications. The molecule features an azepane ring linked to a phenylethanone moiety and a thioxo-substituted pyrazolopyrimidine core. It has been studied as an inhibitor of the mitochondrial enzyme "3-hydroxyacyl-CoA dehydrogenase type II" (also known as ABAD/HSD10), which is implicated in neurodegenerative disease pathways such as Alzheimer's disease. Its mechanism involves binding to this enzyme and potentially modulating its activity; however pharmacological action remains uncharacterized in humans[8][2]. No clinical trials or approvals are reported.

Other names: [[[(2R,3S,4R,5R)-5-(6-aminopurin-9-yl)-3,4-dioxidooxolan-2-yl]methoxy–oxidophosphoryl][(2R,3S,4R,5R)-5-[ (4S)-4-[5-[ (1S)-2-(azepan–1–yl)–2–oxo–1–phenylethyl ] – 4-sulfanylidenepyrazolo[3,4-d]pyrimidin – 2 – yl ] – 3-carbamoyl – 4H-pyridin – 1 - yl ] - 3 , 4 - dihydroxyoxolan - 2 - yl ] methyl phosphate(((2R , 3S , 4R , 5R ) -5 -(6-amino purin -9-y l ) -3 ,CHEBI:45941

Targets

HSD11B2 (11β-HSD2)

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