Nephrogen

Nephrogen is a biotech company developing gene therapies for life-threatening kidney diseases. They aim to deliver curative treatments through a combination of CRISPR/Cas9 gene editing and a novel AAV virus with significantly greater efficiency than existing AAVs to target the disease source. Their initial focus is on autosomal dominant polycystic kidney disease (ADPKD), a genetic disorder causing cyst growth in the kidneys and the most common genetic cause of kidney failure. In ADPKD, mutations occur in the PKD1 or PKD2 genes, leading to abnormal cell growth and cyst formation. Nephrogen's gene therapy aims to introduce functional copies of these genes to halt abnormal growth and prevent cysts, potentially offering a cure for ADPKD.

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