Drugs / GNT0003
GNT0003
Trials 2
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 21 trial | ||||||
| Phase 2 | NCT06518005 | Nov 2024 → Sep 2026 overdue | Crigler-Najjar syndrome | Genethon | Recruiting | No outcome recorded |
| Phase not applicable1 trial | ||||||
| — | NCT03466463 | Mar 2018 → Mar 2026 overdue | Crigler-Najjar syndrome | Genethon | Recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 4
| Date | Issuer | Release |
|---|---|---|
| 2026-05-04 | Genethon | Results Genethon to Present Efficacy Data from 3 Major Clinical Trials of Its Gene Therapy Products at the 2026 American Society of Gene & Cell Therapy Annual Meeting May 11-15 in Boston, MA genethon.com ↗
This is the first time gene therapy has been successfully administered to a Crigler–Najjar syndrome patient with antibodies against AAV8. |
| 2025-10-10 | Hansa Biopharma AB | Results Data from GNT-018-IDES trial supports feasibility of imlifidase as pretreatment in gene therapy treatment for patients with Crigler–Najjar syndrome who are immune to AAV hansabiopharma.com ↗
This encouraging result, achieved in a clinical trial, is a significant advance in the treatment of patients with immunity to AAVs who were previously ineligible for clinical trials and existing gene therapy treatments. |
| 2025-10-10 | Genethon | Results Data from GNT-018-IDES trial supports feasibility of imlifidase as pretreatment in gene therapy treatment for patients with Crigler–Najjar syndrome who are immune to AAV genethon.com ↗
This encouraging result, achieved in a clinical trial, is a significant advance in the treatment of patients with immunity to AAVs who were previously ineligible for clinical trials and existing gene therapy treatments. |
| 2021-10-20 | Genethon | Results Genethon Presents Significant Advances in Gene Therapy at European Society for Gene and Cell Therapy, Oct 19-22, 2021 genethon.com ↗
These results suggest that in patients with Crigler Najjar syndrome, GNT0003 at the dose of 5×10 12 vg/kg is safe and restores UGT1A1 expression to levels allowing safe phototherapy withdrawal. |
All press releases naming this drug 4 releases
Evidence & citations 5 cited values
Every value below carries the sentence it was read from. 3 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | GNT0003 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT03466463 ↗ |
| Action | Restore | “aims to correct the dysfunction of the mutated gene by achieving durable expression of a functional copy of the affected gene” NCT06518005 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “an adeno-associated virus serotype 8 vector encoding UGT1A1” PMID 37585628 ↗ Aug 2023 |
| Route | Intravenous | “a single intravenous infusion” PMID 37585628 ↗ Aug 2023 |
| Target | UGT1A1 | “an adeno-associated virus serotype 8 vector encoding UGT1A1” PMID 37585628 ↗ Aug 2023 |