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TRK-250

Development stage: Phase 1
Lead developer: Toray Industries
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, Protein Replacement 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, 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: Inhalation

Overview

TRK-250 is a novel single-stranded oligonucleotide drug designed as a small interfering RNA (siRNA) agent that targets and selectively suppresses the expression of transforming growth factor-beta 1 (TGF-β1) at the gene expression level. TGF-β1 is a key growth factor involved in the progression of pulmonary fibrosis, particularly idiopathic pulmonary fibrosis (IPF). By inhibiting TGF-β1, TRK-250 aims to prevent or slow down fibrotic changes in lung tissue. The drug utilizes BONAC’s proprietary nucleic acid platform to enhance molecular stability and is formulated for aerosol inhalation, allowing direct delivery to the lungs. Clinical studies have shown that both single and multiple inhaled doses are safe and well tolerated in IPF patients, with minimal systemic exposure detected[1][2][4][5][6].

Targets

TGFB1 (Transforming growth factor Beta-1 proprotein)

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