VivoSeq
Other

GM1 Gangliosidosis

9

Pipeline Programs↗

5

Active Trials↗

5

Companies Involved↗

GM1 gangliosidosis is an early-stage landscape with 9 programs across 5 companies. The most active players are Gemma Biotherapeutics, Pfizer, Prism Pharmaceuticals. 2 therapies are approved with 1 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

38/100

Pipeline Velocity

6/100

Mech. Concentration

50/100

Completion Rate

N/A

Attrition Rate

0/100

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.

G

Gemma Biotherapeutics

1 program

Phase 2Pfizer logo

Pfizer

1 program

Phase 2P

Prism Pharmaceuticals

1 program

Phase 2Roche logo

Roche

1 program

Phase 2Akero Therapeutics logo

Akero Therapeutics

1 program

Approved

Pipeline

Programs by Phase

Analysis

Modality Breakdown

67%

Small molecule

6 of 9 classified programs — the dominant modality here.

33%ALL OTHERSMonoclonal antibody111%Protein / enzyme111%Gene therapy111%

Small molecule leads; the ring shows all 9 classified programs, each share of the total. Modality classified for 100% of 10 pipeline programs.

Analysis

Trial Status

5

clinical trials

Recruiting3 · 60%
Not yet recruiting1 · 20%
Active, not recruiting1 · 20%

Clinical Trials

Trial Activity

A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis
Recruiting·—·Phase 3·NCT07054515·147 enrolled
A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 Gangliosidosis
Recruiting·—·Phase 3·NCT07082543·75 enrolled
A Phase I Study of Prenatal Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis
Not yet recruiting·—·Phase 1·NCT07479953·5 enrolled
A Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis
Recruiting·NNational Human Genome Research Institute (NHGRI)·Phase 1/2·NCT03952637·54 enrolled
Phase 1/2 Open-Label, Multicenter Study to Assess the Safety, Tolerability and Efficacy of a Single Dose of PBGM01 Delivered Into the Cisterna Magna of Pediatric Type 1 (Early Onset) and Type 2a (Late Onset) Infantile GM1 Gangliosidosis
Active, not recruiting·GGemma Biotherapeutics·Preclinical·NCT04713475·26 enrolled

Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)