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RGLS5579
- Development stage
- Preclinical
- Lead developer
- Regulus Therapeutics
- Modality
- MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long 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, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intratumoral, Intraventricular, Intrathecal
01
Overview
RGLS5579 is a novel oligonucleotide therapeutic developed by Regulus Therapeutics for the treatment of glioblastoma multiforme (GBM). It is designed as an anti-miR (antagomir) that specifically targets and inhibits microRNA-10b (miR-10b), a microRNA that is highly overexpressed in GBM and associated with tumor progression and poor prognosis. RGLS5579 was selected from a library of over 200 candidates for its potency and safety profile. Preclinical studies have demonstrated that RGLS5579 can cross the blood-brain barrier and exert anti-tumor effects, significantly extending survival in orthotopic mouse models of GBM, particularly when used in combination with temozolomide (TMZ).
02
Targets
miR-10b (MicroRNA 10b)
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