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GenSci124

Development stage: Preclinical
Lead developer: Changchun GeneScience Pharmaceutical
Modality: mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration: Inhalation

Overview

GenSci124 is an inhaled mRNA therapeutic designed for the treatment of cystic fibrosis (CF). Developed by GeneScience Pharmaceuticals (GenSci) and in-licensed from Suzhou Baidi Boyuan Biotechnology, the drug utilizes a non-lipid nanoparticle (non-LNP), non-viral mucosal delivery platform (MucoRD) and aerosol particle engineering technology for liquid nebulization inhalation. GenSci124 delivers mRNA encoding the normal cystic fibrosis transmembrane conductance regulator (CFTR) protein directly to lung epithelial cells, enabling them to produce functional CFTR protein and correct the underlying CFTR dysfunction. It is currently in IND-enabling/preclinical development.

Targets

CFTR (Cystic fibrosis transmembrane conductance regulator)

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