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ENTR-601-51
- Development stage
- Preclinical
- Lead developer
- Entrada Therapeutics
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Molecules, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intravenous
01
Overview
ENTR-601-51 is an investigational antisense oligonucleotide therapy developed by Entrada Therapeutics for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to exon 51 skipping. The drug utilizes Entrada's proprietary Endosomal Escape Vehicle (EEV) technology, which is designed to enhance cellular uptake and endosomal escape of the therapeutic oligonucleotide. By promoting exon 51 skipping during mRNA splicing, ENTR-601-51 aims to restore the reading frame of the DMD gene transcript, enabling production of a truncated but functional dystrophin protein. This approach targets the underlying genetic cause of DMD in patients with specific mutations suitable for exon 51 skipping[6][7].
02
Targets
DMD (Dystrophin)
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