Pipeline Programs↗
Distinct drugs in development (Preclinical–Phase 3)
Indications↗
Distinct diseases across all programs
Clinical Trials↗
All sponsored trials (all statuses)
Pipeline
By Phase
2 drugs in development
No phase data available.
By Therapeutic Area
top 1
R&D Momentum
18/100
Pipeline Velocity
6/100
Trial Completion Rate
0/100
Pipeline Concentration
50/100
Pipeline Freshness
71/100
Strategic Signals
Activity
Last 14 days
A Phase 1/2 Open-Label, Multicenter Dose-Ranging and Confirmatory Study to Assess the Safety, Tolerability and Efficacy of PBKR03 Administered to Pediatric Subjects With Early Infantile Krabbe Disease (Globoid Cell Leukodystrophy)
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
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Pipeline
Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)