MRNA-002

MRNA-002 is an investigational genomic medicine developed by Arbor Biotechnologies for the treatment of primary hyperoxaluria type 1 (PH1). PH1 is a rare, life-threatening genetic disorder caused by a deficiency of the enzyme alanine-glyoxylate aminotransferase, which leads to the overproduction of oxalate and subsequent kidney damage. MRNA-002 utilizes a CRISPR-based gene editing approach to target and permanently knock out the *HAO1* gene in hepatocytes. The *HAO1* gene encodes glycolate oxidase; by disrupting this enzyme, the therapy reduces the production of glyoxylate, the immediate precursor to oxalate, thereby lowering toxic oxalate levels. The therapeutic components, including a proprietary CRISPR nuclease and guide RNA, are delivered as mRNA encapsulated within lipid nanoparticles (LNPs) for targeted delivery to the liver via intravenous administration.

Other names

Arbor PH1 programHAO1 CRISPR therapyHAO-1 CRISPR therapyHAO 1 CRISPR therapy