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PRX-OTC

Development stage: Preclinical
Lead developer: PhaseRx
Modality: Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes, mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics

Overview

PRX-OTC is a preclinical mRNA therapeutic developed by PhaseRx for the treatment of Ornithine Transcarbamylase Deficiency (OTCD), an orphan liver disease. The drug is designed to act as an intracellular enzyme replacement therapy, utilizing PhaseRx's proprietary Hybrid mRNA Technology platform to deliver mRNA that codes for the deficient ornithine transcarbamylase enzyme. The company raised $18.5 million in an initial public offering (IPO) in May 2016 to fund its preclinical development, manufacturing scale-up, and IND-enabling studies for PRX-OTC.

Targets

ASGPR (Asialoglycoprotein Receptor 1)

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