Drug intelligence / Profile preview

ENTR-801

Development stage
Preclinical
Lead developer
Entrada Therapeutics
Modality
Recombinant Proteins and Enzymes, Peptides
Administration
Intravenous
01

Overview

ENTR-801 is an investigational therapeutic developed by Entrada Therapeutics, utilizing the company's proprietary Endosomal Escape Vehicle (EEV) platform. The drug is a recombinant protein-based EEV conjugate designed to inhibit myostatin (Growth Differentiation Factor 8), a key negative regulator of skeletal muscle mass. By facilitating the efficient delivery of the inhibitory cargo into target muscle cells and ensuring its escape from endosomes into the cytosol, ENTR-801 aims to promote muscle growth and improve physical function. It is currently being evaluated in preclinical studies for the treatment of Inclusion Body Myositis (IBM) and other muscle-wasting conditions, representing a novel approach to intracellular protein delivery for neuromuscular diseases.

02

Targets

USH2A (Usherin)

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