SAR421869

SAR421869 is a gene therapy that utilizes a lentiviral vector derived from the equine infectious anemia virus to deliver the human MYO7A (myosin VIIA) gene. It was developed for the treatment of retinitis pigmentosa associated with Usher syndrome type 1B, a rare genetic disorder characterized by hearing loss and progressive vision loss due to retinal degeneration. The drug aims to restore or replace defective myosin VIIA protein function in affected retinal cells through targeted gene transference. Originally developed by Oxford BioMedica and later partnered with Sanofi for further development, this investigational therapy reached up to Phase 2 clinical trials before its development was discontinued[1][2][3][4].

Brand names

UshStat

Other names

MY07A gene therapyMY-07A gene therapyMY 07A gene therapyMyosin VIIA gene therapyUsher syndrome gene therapy