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PNAi-g3139

Development stage: Unknown
Lead developer: GSK
Modality: MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration: Intravenous

Overview

PNAi-g3139 is a DNA interference (DNAi) duplex, a single-stranded DNA-like oligonucleotide developed by ProNAi Therapeutics. It is designed to inhibit gene transcription by specifically targeting promoter DNA. The drug targets B-cell lymphoma 2 (BCL-2) to inhibit its transcription, primarily for the treatment of cancer, including hematologic malignancies. Its delivery utilizes Marina Biotech's SMARTICLES® technology, which was licensed to ProNAi Therapeutics. Phase 1 trials for DNAi, including PNAi-g3139, were underway by 2012.

Targets

BCL-2 (BCL-2 family)HSPG (Basement membrane-specific heparan sulfate proteoglycan core protein (perlecan))

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