VivoSeq
Neurology

Gaucher Disease Type 1

7

Pipeline Programs↗

3

Active Trials↗

5

Companies Involved↗

Gaucher disease type 1 is an early-stage landscape with 7 programs across 5 companies. The most active players are Roche, Sanofi, Genzyme, a Sanofi Company. 1 therapy is approved.

Intelligence

Pipeline Metrics

Competitive Density

34/100

Pipeline Velocity

6/100

Mech. Concentration

100/100

Completion Rate

0/100

Attrition Rate

25/100

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.

Roche logo

Roche

1 program

Phase 1Sanofi logo

Sanofi

1 program

Phase 2G

Genzyme, a Sanofi Company

3 programs

Phase 2A

Arbor Pharmaceuticals, Inc.

1 program

ApprovedS

Spur Therapeutics

0 programs

Preclinical

Pipeline

Programs by Phase

Analysis

Modality Breakdown

80%

Small molecule

4 of 5 classified programs — the dominant modality here.

20%ALL OTHERSProtein / enzyme120%

Small molecule leads; the ring shows all 5 classified programs, each share of the total. Modality classified for 86% of 7 pipeline programs.

Analysis

Trial Status

4

clinical trials

Terminated1 · 25%
Not yet recruiting1 · 25%
Recruiting1 · 25%
Active, not recruiting1 · 25%

Clinical Trials

Trial Activity

Study of Arimoclomol in Patients Diagnosed With Gaucher Disease Type 1 or 3
Terminated·ZZevraDenmark·Phase 2·NCT03746587·39 enrolled
Efficacy and Safety of Eliglustat in Chinese Pediatric Patients With Gaucher Disease Type 1 and Type 3
Not yet recruiting·PPeking Union Medical College Hospital·Phase 2·NCT06523517·5 enrolled
A Phase 3 Safety and Efficacy Trial of FLT201 Gene Therapy in Patients With Gaucher Disease Type 1
Recruiting·SSpur Therapeutics·Phase 3·NCT07223944·45 enrolled
A 4-part, Open-label, Multicenter, Multinational Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamic, and Exploratory Efficacy of Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension
Active, not recruiting·Sanofi logoSanofi·Phase 2·NCT02843035·12 enrolled

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