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EXG-7001 (Elixirgen Therapeutics, Inc.)
- Development stage
- Preclinical
- Lead developer
- Elixirgen Therapeutics
- Modality
- mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intramuscular
01
Overview
EXG-7001 (mRNA-DMD) is an investigational, locally administered messenger RNA (mRNA) therapeutic being developed by Elixirgen Therapeutics, in collaboration with Nippon Shinyaku, for the treatment of Duchenne muscular dystrophy (DMD). Utilizing Elixirgen's proprietary Bobcat mRNA technology, which features a unique 5'- and 3'-untranslated region (UTR) design, EXG-7001 is engineered to deliver a high-quality, single-strand mRNA encoding the complete, full-length human dystrophin protein. This mutation-independent approach aims to restore the full-length dystrophin protein in muscle cells, potentially offering superior stability, a longer half-life, and more robust restoration of muscle function compared to existing exon-skipping or microdystrophin gene therapies that only produce truncated versions of the protein. EXG-7001 is currently in preclinical development.
- Other names
- Bobcat mRNA-DMDmRNA-DMD
02
Targets
DMD (Dystrophin)
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