VivoSeq
Other

Sandhoff Disease

6

Pipeline Programs↗

1

Active Trials↗

3

Companies Involved↗

Sandhoff disease is an early-stage landscape with 6 programs across 3 companies. The most active players are Eastern Cooperative Oncology Group, Glaxo Dba Glaxosmithkline, Akero Therapeutics. 2 therapies are approved.

Intelligence

Pipeline Metrics

Competitive Density

30/100

Pipeline Velocity

0/100

Mech. Concentration

100/100

Completion Rate

50/100

Attrition Rate

33/100

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.

E

Eastern Cooperative Oncology Group

1 program

Phase 1G

Glaxo Dba Glaxosmithkline

1 program

Phase 2Akero Therapeutics logo

Akero Therapeutics

1 program

Approved

Pipeline

Programs by Phase

Analysis

Modality Breakdown

83%

Small molecule

5 of 6 classified programs — the dominant modality here.

17%ALL OTHERSGene therapy117%

Small molecule leads; the ring shows all 6 classified programs, each share of the total. Modality classified for 100% of 6 pipeline programs.

Analysis

Trial Status

3

clinical trials

Active, not recruiting1 · 33%
Completed1 · 33%
Terminated1 · 33%

Clinical Trials

Trial Activity

Long-Term Follow-Up of Subjects Treated With AXO-AAV-GM2 for Tay-Sachs or Sandhoff Disease
Active, not recruiting·—·N/A·NCT06614569·7 enrolled
Pyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff Disease)
Completed·TThe Hospital for Sick Children·Phase 1/2·NCT01102686·20 enrolled
A Dose-escalation and Safety & Efficacy Study of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease
Terminated·—·Phase 1·NCT04669535·9 enrolled

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