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tREX-1
- Development stage
- Preclinical
- Lead developer
- Cedars-Sinai
- Modality
- RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intravenous
01
Overview
tREX-1 (also known as tREX1) is a bioactive small non-coding RNA (ncRNA) derived from the 5' half of a glutamate transfer RNA (tRNA). Naturally plentiful in cardiosphere-derived cell extracellular vesicles (CDC-EVs), tREX-1 has demonstrated potent disease-modifying immunomodulatory and cardioprotective bioactivity [S45, O139]. In preclinical models of Duchenne muscular dystrophy (DMD), tREX-1 administration preserves cardiac and skeletal muscle function, reduces myocardial fibrosis, and reprograms macrophages toward a reparative, wound-healing phenotype [S45, O139]. A chemically modified version of tREX-1 with phosphorothioate (PS) bonds to improve RNase resistance and potency is known as TT1.
- Other names
- tREX1tREX-1tREX 1
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