Modèle de coopération médico-pharmaceutique comme contributeur au succès du conditionnement de la greffe allogénique de cellules souches hématopoïétiques chez l’adulte : un regard croisé sur nos organisations

Bourget, Philippe; Falaschi, Ludivine; Suarez, Félipe; Galland, Valérie; Blot, Dominique; Trompette, Caroline; Sibon, David; Fontbrune, Flore Sicre de; Merlette, Christophe; Vidal, Fabrice; Corriol, Odile; Giraud, Bérénice; Broissand, Christine; Clément, R.; Hermine, Olivier

doi:10.1684/bdc.2012.1589

Cited by 3 publications

(1 citation statement)

References 41 publications

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“…However, the additional use of immunosuppressive agent (e.g., cyclophosphamide) is not required in this autologous setting. Monitoring of busulfan conditioning to optimize HSC transplantation is an important component of the success 156 and is meticulously performed. When the drug product manufacture is complete, the subject receives myeloablative conditioning with IV busulfan at a starting dose of 3.2 mg/kg/day for 4 days with pharmacokinetics analysis.…”

Section: Clinical Trials: Interim Results From Francementioning

confidence: 99%

Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the β^A(T87Q)-GlobinGene

et al. 2016

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β-globin gene disorders are the most prevalent inherited diseases worldwide and result from abnormal β-globin synthesis or structure. Novel therapeutic approaches are being developed in an effort to move beyond palliative management. Gene therapy, by ex vivo lentiviral transfer of a therapeutic β-globin gene derivative (βAT87Q-globin) to hematopoietic stem cells, driven by cis-regulatory elements that confer high, erythroid-specific expression, has been evaluated in human clinical trials over the past 8 years. βAT87Q-globin is used both as a strong inhibitor of HbS polymerization and as a biomarker. While long-term studies are underway in multiple centers in Europe and in the United States, proof-of-principle of efficacy and safety has already been obtained in multiple patients with β-thalassemia and sickle cell disease.

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Section: Clinical Trials: Interim Results From Francementioning

confidence: 99%

Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the β^A(T87Q)-GlobinGene

et al. 2016

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Hemoglobin disorders: lentiviral gene therapy in the starting blocks to enter clinical practice

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Giorgi

Leboulch

et al. 2018

Experimental Hematology

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Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia

et al. 2018

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Modèle de coopération médico-pharmaceutique comme contributeur au succès du conditionnement de la greffe allogénique de cellules souches hématopoïétiques chez l’adulte : un regard croisé sur nos organisations

Cited by 3 publications

References 41 publications

Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the β^A(T87Q)-GlobinGene

Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the β^A(T87Q)-GlobinGene

Hemoglobin disorders: lentiviral gene therapy in the starting blocks to enter clinical practice

Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia

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Modèle de coopération médico-pharmaceutique comme contributeur au succès du conditionnement de la greffe allogénique de cellules souches hématopoïétiques chez l’adulte : un regard croisé sur nos organisations

Cited by 3 publications

References 41 publications

Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the βA(T87Q)-GlobinGene

Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the βA(T87Q)-GlobinGene

Hemoglobin disorders: lentiviral gene therapy in the starting blocks to enter clinical practice

Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia

Contact Info

Product

Resources

About

Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the β^A(T87Q)-GlobinGene

Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the β^A(T87Q)-GlobinGene