VivoSeq
Immunology

Severe Combined Immunodeficiency

16

Pipeline Programs↗

5

Active Trials↗

11

Companies Involved↗

Severe Combined Immunodeficiency is an emerging landscape with 16 programs across 11 companies. The most active players are Taiga Biotechnologies, Inc., Eastern Cooperative Oncology Group, Akero Therapeutics. 2 therapies are approved.

Intelligence

Pipeline Metrics

Competitive Density

44/100

Pipeline Velocity

0/100

Mech. Concentration

14/100

Completion Rate

57/100

Attrition Rate

7/100

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.

T

Taiga Biotechnologies, Inc.

1 program

Phase 1E

Eastern Cooperative Oncology Group

1 program

Phase 1Akero Therapeutics logo

Akero Therapeutics

1 program

Phase 1Roche logo

Roche

1 program

Phase 1G

Genzyme, a Sanofi Company

1 program

Phase 2Alnylam Pharmaceuticals logo

Alnylam Pharmaceuticals

1 program

Phase 2

Pipeline

Programs by Phase

Analysis

Modality Breakdown

69%

Small molecule

11 of 16 classified programs — the dominant modality here.

31%ALL OTHERSMonoclonal antibody213%Protein / enzyme213%Gene therapy16%

Small molecule leads; the ring shows all 16 classified programs, each share of the total. Modality classified for 100% of 18 pipeline programs.

Analysis

Trial Status

14

clinical trials

Completed4 · 29%
Recruiting3 · 21%
Unknown status2 · 14%
Suspended2 · 14%
Enrolling by invitation1 · 7%
Active, not recruiting1 · 7%
Other1 · 7%

Clinical Trials

Trial Activity

14 trials

Lentiviral Gene Therapy for X-linked Severe Combined Immunodeficiency
Recruiting·GGreat Ormond Street Hospital for Children NHS Foundation Trust·Phase 1·NCT03601286·5 enrolled
The Determination of Genetic Basis Of Immunodeficiency
Recruiting·National Heart, Lung, and Blood Institute (NHLBI)·N/A·NCT00055172·100 enrolled
Gene Therapy Via Intravenous Injection of Lentiviral Vector (Ivlv-ADA) for the Treatment of Adenosine Deaminase-severe Combined Immunodeficiency (ADA-SCID)
Recruiting·SShenzhen Geno-Immune Medical Institute·Preclinical·NCT03645460·10 enrolled
Haplocompatible Transplant Using TCRα/β Depletion Followed by CD45RA-Depleted Donor Lymphocyte Infusions for Severe Combined Immunodeficiency (SCID)
Active, not recruiting·SSt. Jude Children's Research Hospital·Phase 1/2·NCT03597594·4 enrolled
Base-Edited Hematopoietic Stem/Progenitor Cell X-Linked Severe Combined Immunodeficiency Gene Therapy
Enrolling by invitation·National Institute of Allergy and Infectious Diseases (NIAID)·Phase 1/2·NCT06851767·18 enrolled
Total-Body Irradiation Followed By Cyclosporine and Mycophenolate Mofetil in Treating Patients With Severe Combined Immunodeficiency Undergoing Donor Bone Marrow Transplant
Completed·FFred Hutchinson Cancer Center·Phase 1·NCT00008450·6 enrolled
Transplantation of Hematopoietic Cells in Children With Severe Combined Immunodeficiency Syndrome
Completed·SSt. Jude Children's Research Hospital·Phase 1·NCT00152100·4 enrolled
Stem Cell Gene Therapy to Treat X-Linked Severe Combined Immunodeficiency (XSCID)
Completed·National Institute of Allergy and Infectious Diseases (NIAID)·Phase 1·NCT00028236·3 enrolled
Gene Transfer for Severe Combined Immunodeficiency, X-linked (SCID-X1) Using a Self-inactivating (SIN) Gammaretroviral Vector
Completed·—·Phase 1/2·NCT01129544·8 enrolled
Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants
Suspended·SSt. Jude Children's Research Hospital·Phase 1/2·NCT01512888·28 enrolled
Phase I/II Clinical Trial of Autologous Hematopoietic Stem Cell Gene Therapy in RAG1-Deficient Severe Combined Immunodeficiency
Suspended·GGenewity Holding B.V.·Preclinical·NCT04797260·10 enrolled
Treatment for Growth Failure in Patients With X-Linked Severe Combined Immunodeficiency: Phase 2 Study of Insulin-Like Growth Factor-1
Terminated·National Institute of Allergy and Infectious Diseases (NIAID)·Phase 1/2·NCT00490100·6 enrolled
Safety and Early Efficacy Study of TBX-1400 in Patients With Severe Combined Immunodeficiency
Unknown status·TTaiga Biotechnologies, Inc.·Phase 1·NCT02860559·8 enrolled
Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined Immunodeficiency
Unknown status·National Institute of Allergy and Infectious Diseases (NIAID)·Phase 1/2·NCT03315078·13 enrolled

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