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NGGT003

Development stage: Phase 1
Lead developer: Suzhou Nuojiebei Biotechnology
Modality: Gene Therapies
Administration: Intravenous

Overview

NGGT003 is an adeno-associated virus (AAV)-based gene therapy designed for the treatment of Hemophilia A. It utilizes a liver-specific promoter to drive the expression of a codon-optimized human Factor VIII B-domain deleted (hFVIII-BDD) mutant. Administered via a single intravenous infusion, the therapy aims to establish long-term endogenous production of Factor VIII in the liver, thereby reducing or eliminating the need for exogenous factor replacement therapy and preventing bleeding episodes. It is currently being evaluated in early-phase clinical trials for safety and efficacy.

Targets

F8 (Coagulation Factor VIIIa)

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