Drug intelligence / Profile preview

AMA002

Development stage
Preclinical
Lead developer
Amarna Therapeutics
Modality
Viral Vectors → Gene Addition/Replacement → Gene Therapies
Administration
Intravenous
01

Overview

AMA002 (formerly AMR-103) is an investigational gene therapy candidate developed by Amarna Therapeutics for the treatment of Multiple Sclerosis (MS). The therapy utilizes Amarna's proprietary SVec platform, which is based on a non-replicative, non-immunogenic viral vector derived from the Simian Virus 40 (SV40). AMA002 is designed to deliver the gene encoding myelin oligodendrocyte glycoprotein (MOG) specifically to hepatocytes in the liver. The expression of MOG in the liver is intended to induce peripheral immune tolerance by promoting the expansion of antigen-specific regulatory T cells (Tregs). These Tregs then suppress the autoimmune attack against the myelin sheath in the central nervous system, potentially halting disease progression and allowing for neural repair. This approach represents a novel strategy for treating autoimmune diseases by restoring self-tolerance rather than employing broad systemic immunosuppression.

02

Targets

MOG (Myelin oligodendrocyte glycoprotein)

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