GNT0003

Phase 2Small molecule

GGenethon

GNT0003 is one of 4 small molecule programs targeting Crigler-Najjar syndrome. It is currently in Phase 2.

Indications

Total Trials

Active Trials

Competitors

Pipeline

Status by Indication[3][1]

Indication

Crigler-Najjar SyndromeActive, not recruiting

Crigler-Najjar SyndromePhase 2

Active, not recruiting

Competition

Competitive Landscape

3 competitors in Crigler-Najjar Syndrome

#DrugCompanyPhaseTrials

Imlifidase

HHansa Biopharma AB

Phase 2

1 2

Hepastem

CCellaion SA

Phase 2

1 3

Alli

EEngelhard Arzneimittel GmbH & Co.KG

GNT0003

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q3 2030

Phase 2Recruiting

Crigler-Najjar Syndrome

NCT06518005

Clinical

Trials by Indication[1]

Crigler-Najjar syndrome1 trial

Efficacy and Safety of GNT0003 Following Imlifidase Pre-treatment in Severe Crigler-Najjar Syndrome

Recruiting·GGenethon·Phase 2·NCT06518005·3 enrolled

Sources

References

[1]
ClinicalTrials.gov — 1 trial record for GNT0003. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — GNT0003 compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for GNT0003. EMBL-EBI & Wellcome Sanger Institute.
[4]
SEC EDGAR — Genethon company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Small molecule[2]
Highest Phase: Phase 2[1]
Company: GGenethon

GNT0003

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q3 2030

Trials by Indication[1]

Crigler-Najjar syndrome1 trial

References

Drug Info

Quick Facts

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