Startseite Kongressberichte 2020 Virtual Edition of the 25th Annual Congress of the European Hematology Association Oral Sessions Immunology Immunotherapy: Immunotherapy:

Immunotherapy - Clinical

 
Selim Corbacioglu, Maria Domenica Cappellini, John Chapin, et al.
 
The authors of the abstract conclude: 
The first 2 patients treated with CTX001 demonstrated successful engraftment. The patient with TDT has been transfusion free since D+30 after CTX001 infusion (12.4 g/dL HbF at 12 mo). The patient with severe SCD has had no VOCs in the 6 mo following CTX001 infusion (HbF 47.3% of total Hb at 6 mo). This is the first scientific report of patients with a human genetic disease treated successfully with CRISPR-Cas9 and indicates that CTX001 is a promising approach for the treatment of hemoglobinopathies. Data will be updated for the presentation.

Submitted on behalf of the CLIMB THAL-111 and CLIMB SCD-121 Investigators.

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Maria Ester Bernardo, Bernhard Gentner, Francesca Tucci, et al.
 
The authors of the abstract conclude: 
These preliminary results suggest that HSPC-GT accomplishes extensive metabolic correction of peripheral and central compartments. Our study compares favorably with allo-HSCT in terms of safety and biological efficacy and highlights the therapeutic potential of HSPC-GT for the treatment of MPSIH.
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Julie Kanter, John F. Tisdale, Markus Y. Mapara, et al.
 
The authors of the abstract conclude: 
The safety profile of LentiGlobin for SCD gene therapy remains consistent with single-agent busulfan conditioning and underlying SCD. At ≥6 months post-treatment, HGB-206 Group C patients maintained median total Hb at ≥10 g/dL with stable, nearly pancellular, HbAT87Q expression and reduction of median HbS contribution to ≤60%. Absence of ACS and serious VOCs, and reduction in hemolysis suggest suppression of sickle-related complications. Longer follow-up and data on additional patients will be presented.
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Fabio Ciceri, Bernhard Gentner, Farina Francesca, et al.
 
The authors of the abstract conclude: 
These preliminary results indicate feasibility of engrafting a pre-determined fraction of Temferon cells in the BM of GBM patients, which stabilizes after D+60 & remains detectable until the latest follow up. Updated results will be presented.
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Jolie R Schafer, Soumen Khatua, Vanessa Valim, et al.
 
The authors of the abstract conclude: 
Across cell sources, disease types, and treatment settings, hyperfunctional NK cells expanded ex vivo with IL-21 have significant and clinically meaningful numeric expansion and functional persistence.