Gene Therapy Saves Baby with Rare Liver Disorder in World-First Trial

Published on Spencer Knight via LinkedIn

A 14-month-old boy named Tomas, born with a life-threatening liver disorder called OTC deficiency, is now thriving after receiving gene therapy at Great Ormond Street Hospital. The therapy, delivered by startup iECURE, inserted a functioning OTC gene directly into his liver using a viral vector.

Prior to treatment, Tomas needed a strict diet and daily medications to avoid toxic ammonia buildup. Six months after therapy, he’s medication-free, eating normally, and just celebrated his first birthday. With only 15 babies diagnosed with OTC deficiency each year, this result is both a medical milestone and a message of hope.

Read the full article via BBC News:

👉 Breakthrough for baby with rare illness in world-first gene therapy trial

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