Drug intelligence / Profile preview

EMC023

Development stage
Unknown
Lead developer
Exegenesis Bio
Modality
AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
Administration
Intramuscular
01

Overview

EMC023 is a proprietary adeno-associated virus (AAV) capsid developed by Exegenesis Bio. It is designed to specifically target and transduce skeletal muscle tissues with higher efficiency than current benchmark AAV muscle-specific capsids. In preclinical non-human primate studies, EMC023 demonstrated up to a threefold increase in vector genome copies and up to a thirteenfold increase in transgene mRNA levels within skeletal muscle compared to existing AAV capsids. The primary application of this technology is as a delivery vehicle for gene therapies targeting diseases that affect skeletal muscle, such as neuromuscular disorders. EMC023 itself is not the therapeutic agent but rather the engineered viral vector used for gene delivery[3][8].

02

Targets

ACVR2B (Activin receptor type IIB)

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