Drug intelligence / Profile preview

AMA003

Development stage
Preclinical
Lead developer
Amarna Therapeutics
Modality
Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics, Gene Editing → Gene Therapies, Gene Addition/Replacement → Gene Therapies
Administration
Parenteral
01

Overview

**AMA003** is a gene therapy under development by Amarna Therapeutics for the treatment of multiple sclerosis (MS). Its mechanism of action involves gene transference and RNA interference, with additional effects as a vascular endothelial growth factor (VEGF) expression inhibitor. The therapy targets self-reactive T lymphocytes implicated in MS pathogenesis. It was previously considered for wet age-related macular degeneration but has been discontinued for that indication[1][2][3].

Other names
AMA-003AMA003AMA 003
02

Targets

MOG (Myelin oligodendrocyte glycoprotein)

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