NPHP1 gene therapy

NPHP1 gene therapy is an investigational gene replacement treatment being developed by Odylia Therapeutics for the treatment of NPHP1-associated diseases, most notably Nephronophthisis. Nephronophthisis is a rare autosomal recessive cystic kidney disease that often leads to end-stage renal disease in children and young adults. The therapy aims to deliver a functional copy of the NPHP1 gene to target cells, potentially restoring the function of nephrocystin-1, a protein involved in ciliary function and cellular adhesion. This approach is intended to slow or prevent the progression of renal fibrosis and cyst formation characteristic of the disease.

Other names

NPHP1 gene replacement therapyNPHP-1 gene replacement therapyNPHP 1 gene replacement therapy