Last update 27 Feb 2026

γ-globin gene therapy(CSL Behring LLC)

Overview

Basic Info

Drug Type
Gene therapy, Hematopoietic stem cell therapy
Synonyms
CSL-200
Target
HBG1
Action
stimulants
Mechanism
HBG1 stimulants(hemoglobin subunit gamma 1 stimulants)
Therapeutic Areas
Congenital DisordersHemic and Lymphatic DiseasesRespiratory Diseases+ [1]
Active Indication-
Inactive Indication
Acute Chest SyndromeAnemia, Sickle CellBeta-Thalassemia
Originator Organization
CSL Behring LLCCSL Behring LLC
Active Organization-
Inactive Organization
CSL Behring LLCCSL Behring LLCCincinnati Children's Hospital Medical CenterCincinnati Children's Hospital Medical Center
License Organization-
Drug Highest PhaseDiscontinuedPhase 1
First Approval Date-
Regulation-
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Related

1
Clinical Trials associated with γ-globin gene therapy(CSL Behring LLC)
NCT04091737
/ TerminatedPhase 1
A Phase 1 Pilot Study to Evaluate the Safety and Feasibility of Gene Therapy With CSL200 (Autologous Enriched CD34+ Cell Fraction That Contains CD34+ Cells Transduced With Lentiviral Vector Encoding Human γ-GlobinG16D and Short-Hairpin RNA734) in Adult Subjects With Severe Sickle Cell Disease
100 Clinical Results associated with γ-globin gene therapy(CSL Behring LLC)
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100 Translational Medicine associated with γ-globin gene therapy(CSL Behring LLC)
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100 Patents (Medical) associated with γ-globin gene therapy(CSL Behring LLC)
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100 Deals associated with γ-globin gene therapy(CSL Behring LLC)
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R&D Status

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IndicationHighest PhaseCountry/LocationOrganizationDate
Acute Chest SyndromePhase 1
United States
CSL Behring LLCCSL Behring LLC
02 Oct 2019
Anemia, Sickle CellPhase 1
United States
Cincinnati Children's Hospital Medical CenterCincinnati Children's Hospital Medical Center
27 Sep 2019
Beta-ThalassemiaPhase 1
United States
CSL Behring LLCCSL Behring LLC
27 Sep 2019
Beta-ThalassemiaPhase 1
United States
Cincinnati Children's Hospital Medical CenterCincinnati Children's Hospital Medical Center
27 Sep 2019
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Clinical Result

Indication
Phase
Evaluation
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Clinical Trial

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Approval

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Regulation

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