Drug intelligence / Profile preview

MOG1

Development stage
Preclinical
Lead developer
Mogrify
Modality
Viral Vectors → Gene Addition/Replacement → Gene Therapies
Administration
Ophthalmic
01

Overview

MOG1 is an experimental gene therapy-like candidate developed by Mogrify for the treatment of retinal degeneration. It utilizes the company's proprietary MOGRIFY platform to perform *in vivo* direct cellular reprogramming, a process that converts one mature cell type into another without passing through a pluripotent state. The therapy involves the delivery of specific transcription factors via a viral vector to the retina, where they induce the conversion of endogenous cells into functional retinal cells, such as photoreceptors or retinal pigment epithelial cells. This approach aims to restore vision by regenerating lost cell populations directly within the eye, offering a potential alternative to cell transplantation or traditional gene replacement therapies.

02

Targets

SCN5A (Sodium channel protein type 5 subunit alpha)

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