iECURE

DNA vector
#Gene editing #Gene therapy #iECURE #Phase II trial

Study indicates gene editing could restore OTC enzyme activity

Gene editing company iECURE has shared data from the ongoing OTC-HOPE Phase I/II study of ECUR-506, which suggest partial restoration of functional ornithine transcarbamylase (OTC) enzyme activity in the first patient dosed.  “Long-term restoration of ornithine transcarbamylase activity has the potential to allow infants afflicted with neonatal… Read More

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Cell and gene therapy
#FDA #Gene editing #Gene therapy #iECURE #In vivo #Paediatrics

FDA approves first in vivo gene insertion programme in infants

iECURE has been given the go-ahead to begin US trials of its investigational gene editing-based therapy in newborn males with neonatal onset Ornithine Transcarbamylase (OTC) deficiency. The Food and Drug Administration (FDA) Investigational New Drug (IND) clearance for ECUR-506 makes it the first in vivo gene… Read More

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