Drug intelligence / Profile preview

scAAV2-EMC

Development stage
Preclinical
Lead developer
Nanoscope Therapeutics
Modality
Gene Addition/Replacement → Gene Therapies, Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies
Administration
Ophthalmic
01

Overview

scAAV2-EMC is an investigational gene therapy candidate developed by Nanoscope Technologies for the treatment of glaucoma and chronic ocular hypertension. It utilizes a self-complementary adeno-associated virus serotype 2 (scAAV2) vector to deliver a transgene encoding an engineered mechanosensitive channel (EMC) under the control of the Matrix Gla protein (MGP) promoter. The therapy is designed for intracameral administration to target the trabecular meshwork (TM). Once expressed, the EMC acts as an autonomous, pressure-sensing valve that activates cellular responses to enhance aqueous humor outflow through the conventional pathway. This mechanism aims to provide sustained reduction of intraocular pressure (IOP) and neuroprotection to retinal ganglion cells (RGCs), potentially offering a long-term alternative to daily eye drop medications and addressing challenges related to patient compliance.

Other names
scAAV2-engineered mechanosensitive channelscAAV-2-engineered mechanosensitive channelscAAV 2-engineered mechanosensitive channel
02

Targets

HSPG (Basement membrane-specific heparan sulfate proteoglycan core protein (perlecan))GPR108 (G protein-coupled receptor 108)MSC (Engineered mechanosensitive ion channel)

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