Other
Gene Therapy
0
Pipeline Programs↗
16
Active Trials↗
8
Companies Involved↗
Gene Therapy is an early-stage landscape with 0 programs across 8 companies. The most active players are Adverum Biotechnologies, Inc., AbbVie, 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
No pipeline programs currently tracked for this disease.
Analysis
Trial Status
38
clinical trials
Completed11 · 29%
Recruiting9 · 24%
Unknown status6 · 16%
Terminated4 · 11%
Active, not recruiting3 · 8%
Not yet recruiting2 · 5%
Other3 · 8%
Clinical Trials
Trial Activity
38 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
Base Editing Hematopoietic Stem Cell and T Cell Gene Therapy for CD40L-HyperIgM Syndrome: Single Patient Study
Recruiting·National Institute of Allergy and Infectious Diseases (NIAID)·Phase 1/2·NCT06959771·1 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
Page 1 of 3
Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)