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AMA001

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

Overview

AMA001 is a preclinical gene therapy candidate developed by Amarna Therapeutics for the treatment of hemophilia B. It utilizes Amarna's proprietary SVac (also referred to as SVec) gene delivery platform, which is based on a modified simian virus 40 (SV40) viral vector. The therapy aims to deliver a healthy version of the gene encoding factor IX (FIX) to patient cells to restore the production of functional FIX, thereby addressing the clotting deficiency characteristic of hemophilia B.

Targets

MHC I (MHC class I})

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