Drug intelligence / Profile preview

RIG-301

Development stage
Preclinical
Lead developer
RIGImmune
Modality
mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Inhalation
01

Overview

RIG-301 is an inhaled mRNA therapy being developed by RIGImmune for the treatment of cystic fibrosis (CF). It utilizes the proprietary NEED™ (Nano-Emulsion Delivery) technology, which allows for the delivery of RNA compounds without the need for lipid nanoparticle (LNP) encapsulation. The therapy is designed to deliver wild-type Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) mRNA directly to lung bronchial epithelial cells. By providing the genetic template for functional CFTR protein, RIG-301 aims to restore chloride transport and improve lung function in patients regardless of their specific CFTR mutation. Currently in preclinical development, RIG-301 is being prepared for Phase 1 clinical trials, with potential entry into the clinic expected in early 2026.

02

Targets

Cystic fibrosis transmembrane conductance regulator mRNA and translation machinery

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