Epileptic Encephalopathy

2
Pipeline Programs
2
Companies
1
Clinical Trials
1 recruiting
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
0
0
0
2
0
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

RNA Therapeutic
2100%

Competitive Landscape

2 companies ranked by most advanced pipeline stage

Praxis Precision Medicines
1 program
1
1mg elsunersenPhase 3RNA Therapeutic
Precision BioSciences
1 program
1
1mg elsunersenPhase 3RNA Therapeutic1 trial
Active Trials
NCT07019922Recruiting50Est. Dec 2028

Trial Timeline

Clinical trial activity over time

2025
2026
2027
2028
Precision BioSciences1mg elsunersen

Clinical Trials (1)

Total enrollment: 50 patients across 1 trials

A Clinical Trial of Elsunersen in Pediatric SCN2A-DEE to Assess Efficacy and Safety

Start: Aug 2025Est. completion: Dec 202850 patients
Phase 3Recruiting

Phase Legend

PreclinicalLab & animal studies
Phase 1Safety & dosing
Phase 2Efficacy testing
Phase 3Large-scale trials
On MarketApproved & available

Key Insights

2 late-stage (Phase 3) programs — potential near-term approvals
1 actively recruiting trials targeting 50 patients
2 companies competing in this space