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
1 drugs in development
By Therapeutic Area
top 1
R&D Momentum
32/100
Pipeline Velocity
6/100
Trial Completion Rate
N/A
Pipeline Concentration
50/100
Pipeline Freshness
82/100
Strategic Signals
Activity
Last 14 days
A Phase 1/2 Open-Label Study to Evaluate the Safety, Tolerability and Efficacy of a Single Dose of ELP-02 Delivered Via Lumbar Intrathecal Administration in Charcot-Marie-Tooth-4J (CMT4J)
A Prospective Natural History and Outcome Measure Discovery Study of Charcot-Marie-Tooth Disease, Type 4J
A Phase 1/2 Open-label Intrathecal Administration of MELPIDA to Determine Its Safety and Efficacy for Patients With Spastic Paraplegia Type 50 (SPG50) Caused by Mutation in the AP4M1 Gene.
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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)