/
Other
Gene Therapy
10
Pipeline Programs↗
14
Active Trials↗
9
Companies Involved↗
Gene Therapy is an early-stage landscape with 10 programs across 9 companies. The most active players are BioMarin Pharmaceutical, Atamyo Therapeutics.
Intelligence
Pipeline Metrics
Competitive Density
Pipeline Velocity
Mech. Concentration
Completion Rate
Attrition Rate
Competition
Competitive Landscape
Bubble size = clinical trials
Approved
Phase 3
Phase 2
Phase 1
Preclinical
Key Players
Top Companies Companies ranked by the number of pipeline programs they run for this disease.
Pipeline
Programs by Phase
Preclinical3
Phase 25
Phase 3
No programs
Approved
No programs
Analysis
Modality Breakdown
9programs
Small molecule556%
Gene therapy444%
Modality classified for 100% of 10 pipeline programs.
Analysis
Trial Status
34
clinical trials
Completed10 · 29%
Recruiting7 · 21%
Unknown status5 · 15%
Terminated4 · 12%
Active, not recruiting3 · 9%
Not yet recruiting2 · 6%
Other3 · 9%
Clinical Trials
Trial Activity
34 trials
Lentiviral Gene Therapy for p47 AR-CGD
Recruiting·GGreat Ormond Street Hospital for Children NHS Foundation Trust·Phase 1/2·NCT05207657·5 enrolled
Gene Therapy for Crigler Najjar Syndrome Type I (AlphaCN)
Recruiting·Federal State Budget Institution Research Center for Obstetrics, Gynecology and Perinatology Ministry of Healthcare·Phase 1/2·NCT06641154·5 enrolled
An Expanded Clinical Study Evaluating the AAV2-RPE65 Gene Therapy(LX101) in Patients With LCA
Not yet recruiting·SShanghai General Hospital, Shanghai Jiao Tong University School of Medicine·N/A·NCT06024057·3 enrolled
Gene Therapy for IGHMBP2-Related Diseases
Enrolling by invitation·—·Phase 1/2·NCT05152823·10 enrolled
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Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)