VivoSeq
Immunology

Wiskott-Aldrich Syndrome

8

Pipeline Programs↗

0

Active Trials↗

6

Companies Involved↗

Wiskott-Aldrich syndrome is an early-stage landscape with 8 programs across 6 companies. The most active players are Essen Biotech, The Korean Society of Pediatric Hematology Oncology, Amgen. 3 therapies are approved.

Intelligence

Pipeline Metrics

Competitive Density

33/100

Pipeline Velocity

0/100

Mech. Concentration

100/100

Completion Rate

83/100

Attrition Rate

14/100

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.

E

Essen Biotech

1 program

Phase 1T

The Korean Society of Pediatric Hematology Oncology

1 program

Phase 2Amgen logo

Amgen

1 program

Phase 2GSK logo

GSK

1 program

Phase 2Alnylam Pharmaceuticals logo

Alnylam Pharmaceuticals

1 program

ApprovedAkero Therapeutics logo

Akero Therapeutics

1 program

Approved

Pipeline

Programs by Phase

Analysis

Modality Breakdown

78%

Small molecule

7 of 9 classified programs — the dominant modality here.

22%ALL OTHERSProtein / enzyme222%

Small molecule leads; the ring shows all 9 classified programs, each share of the total. Modality classified for 100% of 10 pipeline programs.

Analysis

Trial Status

7

clinical trials

Completed5 · 71%
Terminated1 · 14%
Unknown status1 · 14%

Clinical Trials

Trial Activity

7 trials

Haploidentical Hematopoietic Stem Cell Transplantation Patients With Wiskott-Aldrich Syndrome
Completed·SSt. Jude Children's Research Hospital·Phase 1·NCT00160355·4 enrolled
Interleukin-2 Treatment for Wiskott-Aldrich Syndrome
Completed·—·Phase 1·NCT00774358·9 enrolled
Romiplostim Treatment for Thrombocytopenia in Patients With Wiskott-Aldrich Syndrome.
Completed·FFederal Research Institute of Pediatric Hematology, Oncology and Immunology·N/A·NCT04350164·67 enrolled
Study of Reduced Toxicity Myeloablative Conditioning Regimen for Wiskott-Aldrich Syndrome (WAS)
Completed·TThe Korean Society of Pediatric Hematology Oncology·Phase 1/2·NCT00885833·5 enrolled
Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for the Wiskott-Aldrich Syndrome
Completed·—·Phase 1/2·NCT01410825·5 enrolled
Thrombocytopenia and Bleeding in Wiskott-Aldrich Syndrome (WAS) Patients
Terminated·WWeill Medical College of Cornell University·Phase 2·NCT00909363·24 enrolled
Efficacy and Safety of Romiplostim Versus Eltrombopag in the Treatment of Thrombocytopenia in Patients With Wiskott-Aldrich Syndrome
Unknown status·FFederal Research Institute of Pediatric Hematology, Oncology and Immunology·Phase 2·NCT04371939·30 enrolled

Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)