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Targeting Rare Liver Diseases with Gene Editing Therapies
Manage episode 462646256 series 60790
Earlier this year iECURE reported that an infant with the rare and deadly liver disease OTC deficiency had a complete response to its experimental gene editing therapy. It is believed to be the time that an infant was treated with an in vivo, liver-directed gene editor. The treatment restored ammonia levels in the child’s blood to normal and the child is off of ammonia scavenger medicines and is eating a normal diet. We spoke to Joe Truitt, CEO of iECURE, about the company’s experimental therapy for OTC, how it works, and its approach to in vivo gene editors.
528 эпизодов
Manage episode 462646256 series 60790
Earlier this year iECURE reported that an infant with the rare and deadly liver disease OTC deficiency had a complete response to its experimental gene editing therapy. It is believed to be the time that an infant was treated with an in vivo, liver-directed gene editor. The treatment restored ammonia levels in the child’s blood to normal and the child is off of ammonia scavenger medicines and is eating a normal diet. We spoke to Joe Truitt, CEO of iECURE, about the company’s experimental therapy for OTC, how it works, and its approach to in vivo gene editors.
528 эпизодов
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