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ANB-010

Development stage: Unknown
Lead developer: Biocad
Modality: Recombinant Proteins and Enzymes, Gene Silencing → Gene Therapies, Gene Editing → Gene Therapies, Gene Addition/Replacement → Gene Therapies
Administration: Intravenous

Overview

**ANB-010 is an investigational gene therapy consisting of a recombinant adeno-associated virus (rAAV) vector carrying the human clotting factor VIII (FVIII) gene, under development for the treatment of hemophilia A.** It is administered as a single intravenous dose, aiming to provide a long-lasting (years) restoration of FVIII production in patients with severe deficiency. According to studies sponsored by BIOCAD, ANB-010 is designed to enable endogenous production of FVIII following transduction of patient liver cells by the rAAV vector[1][2][3][4].

Targets

F8 (Coagulation Factor VIIIa)

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