Base-edited T lymphocyte cells

Gene therapy

NNational Institute of Allergy and Infectious Diseases (NIAID)

Base-edited T lymphocyte cells is one of 34 biologic programs targeting Severe Combined Immunodeficiency. It is currently in development.

Indications

Total Trials

Active Trials

Competitors

Pipeline

Status by Indication[3][1]

Indication

Severe Combined ImmunodeficiencyActive, not recruiting

Chronic Granulomatous DiseaseActive, not recruiting

Severe Combined ImmunodeficiencyPhase 1/Phase 2

Active, not recruiting

Chronic Granulomatous DiseasePhase 1/Phase 2

Active, not recruiting

Competition

Competitive Landscape

42 competitors in Chronic Granulomatous Disease

#DrugCompanyPhaseTrials

Advil

Interferon-Gamma

1 3

Fludara

Sanofi

Phase 2

2 4

Neoral

Novartis

Phase 2

1 5

Busulfex

Akero Therapeutics

Phase 2

5 6

Cytoxan

BBaxter Healthcare Corporation

Phase 2

4 7

Promacta

Actemra

Sporanox

SSebela Pharmaceuticals Development LLC

Phase 2

1 10

Noxafil

CCidara Therapeutics Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA)

Phase 2

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q4 2027

Phase 1/2Recruiting

Gene Therapy

NCT06959771

Q4 2032

PreclinicalRecruiting

Chronic Granulomatous Disease (CGD)

NCT06325709

Clinical

Trials by Indication[1]

Severe Combined Immunodeficiency1 trial

Base-Edited Hematopoietic Stem/Progenitor Cell X-Linked Severe Combined Immunodeficiency Gene Therapy

Enrolling by invitation·NNational Institute of Allergy and Infectious Diseases (NIAID)·Phase 1/2·NCT06851767·18 enrolled

Gene Therapy1 trial

Base Editing Hematopoietic Stem Cell and T Cell Gene Therapy for CD40L-HyperIgM Syndrome: Single Patient Study

Recruiting·NNational Institute of Allergy and Infectious Diseases (NIAID)·Phase 1/2·NCT06959771·1 enrolled

Chronic Granulomatous Disease (CGD)1 trial

Phase 1/2 Trial of Base Editing for Mutation Repair in Hematopoietic Stem & Progenitor Cells for X-linked Chronic Granulomatous Disease

Recruiting·NNational Institute of Allergy and Infectious Diseases (NIAID)·Preclinical·NCT06325709·10 enrolled

Sources

References

[1]
ClinicalTrials.gov — 3 trial records for Base-edited T lymphocyte cells. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — Base-edited T lymphocyte cells compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for Base-edited T lymphocyte cells. EMBL-EBI & Wellcome Sanger Institute.
[4]
SEC EDGAR — National Institute of Allergy and Infectious Diseases (NIAID) company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Gene therapy[2]
Company: NNational Institute of Allergy and Infectious Diseases (NIAID)

Base-edited T lymphocyte cells

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q4 2027

Q4 2032

Trials by Indication[1]

Severe Combined Immunodeficiency1 trial

Gene Therapy1 trial

Chronic Granulomatous Disease (CGD)1 trial

References

Drug Info

Quick Facts

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