AAV-ENPP1-Fc

**AAV-ENPP1-Fc** is an experimental **AAV-based gene therapy** designed to treat **ENPP1 deficiency**, a rare inherited mineralization disorder that includes generalized arterial calcification of infancy and autosomal recessive hypophosphatemic rickets type 2 phenotypes. The construct is described as an adeno-associated viral vector encoding a **modified human ENPP1-Fc fusion protein** under a **liver-specific promoter**, intended as a **one-dose systemic therapy** that drives hepatic production of soluble ENPP1 activity. By restoring extracellular **ectonucleotide pyrophosphatase/phosphodiesterase 1** function, the therapy is intended to increase **pyrophosphate** generation and thereby inhibit ectopic calcification while improving bone abnormalities associated with ENPP1 loss. Publicly available information links this program to **Inozyme Pharma** in preclinical mouse studies; it appears to be a research-stage program rather than a clinically advanced asset.

Other names

ENPP1-Fc expressing AAV vectorENPP-1-Fc expressing AAV vectorENPP 1-Fc expressing AAV vector