Drug intelligence / Profile preview

rintatolimod

Development stage
Phase 3
Lead developer
AIM ImmunoTech
Modality
Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, 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
01

Overview

Rintatolimod is an investigational immunomodulator and antiviral agent developed for the treatment of myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS) and other chronic diseases. It is a mismatched double-stranded RNA (dsRNA) that acts as a selective toll-like receptor 3 (TLR3) agonist. By binding to TLR3 receptors located on endosomal membranes within cells, rintatolimod stimulates the innate immune system and induces interferon production. This activation leads to broad-spectrum immune responses against pathogens such as viruses and cancer cells. The drug also activates the intracellular enzyme RNase L, which degrades both viral and cellular RNA to inhibit viral replication and promote apoptosis in infected or abnormal cells. Rintatolimod has shown clinical activity in improving exercise tolerance in certain subsets of ME/CFS patients with disease duration between 2–8 years.

Brand names
AmpligenAtvogen
Other names
Poly I:Poly C12UPoly(I):poly(C12U)poly(I) poly(C12,U)Vaccine-adjuvant-poly-I-polyC12UVaccine-adjuvant-poly-I-polyC-12UVaccine-adjuvant-poly-I-polyC 12UVaccine-adjuvant-rintatolimod
02

Targets

TLR3 (Toll-like receptor 3)RNASEL (Ribonuclease L)

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