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GNT-0003
- Development stage
- Phase 2
- Lead developer
- Généthon
- Modality
- Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Administration
- Intravenous
01
Overview
GNT-0003 is an investigational gene therapy developed for the treatment of Crigler-Najjar syndrome, a rare and severe inherited liver disorder caused by deficiency of the enzyme UDP-glucuronosyltransferase 1A1 (UGT1A1). The therapy consists of an adeno-associated virus serotype 8 (AAV8) vector carrying normal copies of the UGT1A1 gene. Administered intravenously, it delivers the functional gene to hepatocytes in order to restore UGT1A1 expression and enable proper bilirubin metabolism. Clinical trials have shown that GNT-0003 can reduce serum bilirubin levels and allow withdrawal from phototherapy in patients with Crigler-Najjar syndrome. The product has received PRIME status from the European Medicines Agency due to its major therapeutic potential[1][2][5][7].
- Other names
- GNT 0003GNT0003GNT-0003AAV8-hUGT1A1AAV-8-hUGT1A1AAV 8-hUGT1A1
02
Targets
UGT1A1 (UDP-glucuronosyltransferase 1A1)
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