SAR444836

SAR444836 is an investigational gene therapy developed for the treatment of phenylketonuria (PKU), a rare inherited metabolic disorder caused by deficiency of the enzyme phenylalanine hydroxylase (PAH). It is an adeno-associated virus (AAV) vector-mediated gene transfer therapy designed to deliver a functional copy of the human PAH gene into hepatocytes in the liver via a single intravenous administration. By restoring PAH activity, SAR444836 aims to reduce blood phenylalanine levels and potentially eliminate the need for dietary Phe restriction in adults with PKU. The drug is currently being evaluated in Phase 1/2 clinical trials[1][3][5][6].

Other names

Adeno-associated virus AAV-based vector with engineered capsid serotype SNY001 harboring human phenylalanine hydroxylase hPAH cDNA