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ENTR-601-44
- Development stage
- Phase 2
- Lead developer
- Entrada Therapeutics
- Modality
- Small Molecules, 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, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Gene Therapies
- Administration
- Intravenous
01
Overview
ENTR-601-44 is an investigational exon-skipping therapy developed by Entrada Therapeutics for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to exon 44 skipping. It is a phosphorodiamidate morpholino oligonucleotide (PMO) conjugated to Entrada’s proprietary Endosomal Escape Vehicle (EEV) technology, which enhances cellular uptake and endosomal escape of the therapeutic oligonucleotide. The drug works by facilitating the skipping of exon 44 during pre-mRNA splicing of the DMD gene, restoring the mRNA reading frame and enabling production of a truncated but functional dystrophin protein. This approach aims to address the underlying genetic cause of DMD in this patient subset and potentially ameliorate disease progression[1][2][3][4][6][7].
- Other names
- Exon 44 Skipping Oligonucleotide Entrada TherapeuticsExon44 Skipping Oligonucleotide Entrada TherapeuticsExon-44 Skipping Oligonucleotide Entrada Therapeuticsexon skipping oligonucleotide
02
Targets
DMD (Dystrophin)
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