VivoSeq
Rare Disease

Batten Disease

3

Pipeline Programs

2

Active Trials

3

Companies Involved

Batten Disease is an early-stage landscape with 3 programs across 3 companies. The most active players are Roche, Pfizer, BioMarin Pharmaceutical.

Pipeline Metrics

Competitive Density

24/100

Pipeline Velocity

0/100

Mech. Concentration

100/100

Completion Rate

N/A

Attrition Rate

0/100

Competitive Landscape

Bubble size = clinical trials

Approved
Phase 3
Phase 2
Phase 1
Preclinical

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

Programs by Phase

Phase 11
Phase 3
No programs
Approved
No programs

Modality Breakdown

2programs
Small molecule150%
Protein / enzyme150%

Modality classified for 67% of 3 pipeline programs.

Trial Status

2

clinical trials

Recruiting1 · 50%
Active, not recruiting1 · 50%

Trial Activity

GENEr8-2: Cerliponase alfa Earlier Intervention
Recruiting·BioMarin Pharmaceutical logoBioMarin Pharmaceutical·Phase 2·NCT05700006·30 enrolled
GENEr8-1 Extension: Cerliponase alfa in CLN2
Active, not recruiting·BioMarin Pharmaceutical logoBioMarin Pharmaceutical·Phase 3·NCT04628000·24 enrolled

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