Developmental and Epileptic Encephalopathy
2
Pipeline Programs
4
Companies
2
Clinical Trials
0
Approved Products
Pipeline by Development Stage
Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
2
0
0
0
0
Early DiscoveryClinical DevelopmentMarket
Drug Modality Breakdown
Gene Therapy
2100%
+ 1 programs with unclassified modality
Competitive Landscape
3 companies ranked by most advanced pipeline stage
Trial Timeline
Clinical trial activity over time
2025
2026
2027
2028
BioTherapeutics Incgene therapy
BiocorpLong-read Whole Genome Sequencing
Clinical Trials (2)
Total enrollment: 32 patients across 2 trials
A Clinical Trial of CAP-002 Gene Therapy in Pediatric Patients With Syntaxin-Binding Protein 1 (STXBP1) Encephalopathy
Start: Jul 2025Est. completion: Dec 202812 patients
Phase 1/2Suspended
Developmental and Epileptic Encephalopathies Diagnosed Via Long-read Genome Sequencing
Start: Jun 2026Est. completion: Jun 202820 patients
N/ANot Yet Recruiting
Phase Legend
Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available
Key Insights
Gene Therapy is the dominant modality (100% of programs)
4 companies competing in this space
The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.