Other
GM2 Gangliosidosis
6
Pipeline Programs↗
4
Active Trials↗
4
Companies Involved↗
GM2 gangliosidosis is an early-stage landscape with 6 programs across 4 companies. The most active players are Eastern Cooperative Oncology Group, Johnson & Johnson, Medexus Pharma, Inc..
Intelligence
Pipeline Metrics
Competitive Density
Pipeline Velocity
Mech. Concentration
Completion Rate
Attrition Rate
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.
Pipeline
Programs by Phase
Preclinical
No programs
Phase 12
Phase 24
Phase 3
No programs
Approved
No programs
Analysis
Trial Status
7
clinical trials
Completed2 · 29%
Active, not recruiting2 · 29%
Recruiting1 · 14%
Withdrawn1 · 14%
Not yet recruiting1 · 14%
Clinical Trials
Trial Activity
7 trials
A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease
Recruiting·—·Phase 2·NCT07399704·21 enrolled
Phase 2 Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients with GM2 Gangliosidosis or Niemann-Pick Type C Disease
Active, not recruiting·—·Phase 2·NCT05758922·13 enrolled
First-in-Human Study of TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 Gangliosidosis
Active, not recruiting·—·Phase 1/2·NCT04798235·3 enrolled
Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)