VivoSeq
Other

Wilson Disease

9

Pipeline Programs↗

4

Active Trials↗

5

Companies Involved↗

Wilson disease is an early-stage landscape with 9 programs across 5 companies. The most active players are AstraZeneca, Nobelpharma, Revance Therapeutics, Inc.. 2 therapies are approved with 3 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

37/100

Pipeline Velocity

11/100

Mech. Concentration

100/100

Completion Rate

80/100

Attrition Rate

13/100

Competition

Competitive Landscape

Bubble size = clinical trials

Approved
Phase 3
Phase 2
Phase 1
Preclinical

Key Players

Top Companies Companies ranked by the number of pipeline programs they run for this disease.

AstraZeneca logo

AstraZeneca

3 programs

Phase 3N

Nobelpharma

1 program

ApprovedR

Revance Therapeutics, Inc.

1 program

PreclinicalUltragenyx logo

Ultragenyx

0 programs

PreclinicalL

Lingyi Biotech Co., Ltd.

0 programs

Preclinical

Pipeline

Programs by Phase

Analysis

Trial Status

16

clinical trials

Completed8 · 50%
Terminated2 · 13%
Recruiting2 · 13%
No longer available1 · 6%
Unknown status1 · 6%
Active, not recruiting1 · 6%
Other1 · 6%

Clinical Trials

Trial Activity

16 trials

A Clinical Study on the Treatment of Wilson Disease With ATP7B mRNA/LNP (DSL101)
Recruiting·DDSciLab Co., Ltd.·Phase 1·NCT07240896·18 enrolled
Natural History of Wilson Disease: Registry for Patients With Wilson Disease
Recruiting·YYale University·N/A·NCT03334292·300 enrolled
A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease
Active, not recruiting·Ultragenyx logoUltragenyx·Phase 1/2·NCT04884815·82 enrolled
A Multicenter, Open, Single-arm, Single-dose, Phase I/II Study Evaluating the Safety, Tolerability, and Efficacy of LY-M003 Injection in Adult Patients With Wilson's Disease
Not yet recruiting·LLingyi Biotech Co., Ltd.·Preclinical·NCT07641140·18 enrolled
Single Daily Dosage of Trientine for Maintenance Treatment for Wilson Disease
Completed·YYale University·N/A·NCT01472874·8 enrolled
Study of Zinc for Wilson Disease
Completed·NNational Center for Research Resources (NCRR)·Phase 4·NCT00004338·300 enrolled
The Clinical Study of Botulinum Toxin Type A Injection in the Treatment of Wilson Disease
Completed·TThe First Affiliated Hospital of Anhui University of Traditional Chinese Medicine·N/A·NCT06762509·60 enrolled
The Assessment of Copper Parameters in Wilson Disease Participants on Standard of Care Treatment
Completed·AstraZeneca logoAstraZeneca·N/A·NCT02763215·64 enrolled
Copper and Molybdenum Balance in Participants With Wilson Disease Treated With ALXN1840
Completed·AstraZeneca logoAstraZeneca·Phase 2·NCT04573309·9 enrolled
Study of Tetrathiomolybdate in Patients With Wilson Disease
Completed·NNational Center for Research Resources (NCRR)·Phase 3·NCT00004339·90 enrolled
Copper Concentration & Histopathologic Changes in Liver Biopsy in Participants With Wilson Disease Treated With ALXN1840
Completed·AstraZeneca logoAstraZeneca·Phase 2·NCT04422431·31 enrolled
Efficacy and Safety Study of WTX101 (ALXN1840) in Adult Wilson Disease Patients
Completed·AstraZeneca logoAstraZeneca·Phase 2·NCT02273596·29 enrolled
Study of ALXN1840 Versus Standard of Care in Pediatric Participants With Wilson Disease
Terminated·AstraZeneca logoAstraZeneca·Phase 3·NCT05047523·40 enrolled
Efficacy and Safety of ALXN1840 Administered for 48 Weeks Versus Standard of Care in Participants With Wilson Disease
Terminated·AstraZeneca logoAstraZeneca·Phase 3·NCT03403205·214 enrolled
Early Access Program for ALXN1840 in Patients With Wilson Disease
No longer available·AstraZeneca logoAstraZeneca·N/A·NCT05686564
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Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)