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DTX701

Development stage: Preclinical
Lead developer: Ultragenyx Pharmaceutical
Modality: Gene Therapies
Administration: Intravenous

Overview

DTX701 is an adeno-associated virus (AAV) gene therapy developed by Dimension Therapeutics, in collaboration with the University of Pennsylvania, for the preclinical development of a treatment for Wilson disease. The therapy aims to address the genetic defect underlying Wilson disease, which is characterized by impaired copper metabolism. Dimension Therapeutics licensed REGENXBIO's AAV technology platform for the development of this and other gene therapies. Following its preclinical development, Dimension Therapeutics was acquired by Ultragenyx Pharmaceutical.

Targets

ATP7B (Copper ion transporter ATPase 7B)

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