Posted by Michael Wonder on 23 Aug 2022
iECURE receives FDA rare paediatric disease designation for GTP-506, an investigational gene editing product candidate for the treatment of ornithine transcarbamylase deficiency
23 August 2022 - GTP-506, a potential single-dose gene editing therapy designed to restore metabolic function in patients suffering with OTC deficiency, a rare urea cycle disorder.
iECURE today announced that the US FDA has granted rare paediatric disease designation to its lead product candidate GTP-506 for the treatment of ornithine transcarbamylase deficiency, a rare genetic condition that can lead to irreversible neurological impairment, seizures, coma and death, in a paediatric population.
