- |||||||||| IMMUNOMAGNETIC SELECTION OF ALLOGENEIC CD34+ CELLS FOR STEM CELL BOOST (Firenze (Spadolini 1)) - Feb 5, 2025 - Abstract #EBMT2025EBMT_1628;
This resulted in the safe administration of SCB in all of our patients and haematopoietic recovery in the majority of them. Despite the small number of patients included in our study and its retrospectivity, our findings align with outcomes reported in previous research.
- |||||||||| IMMUNOMAGNETIC SELECTION OF ALLOGENEIC CD34+ CELLS FOR STEM CELL BOOST (ePoster Area) - Feb 5, 2025 - Abstract #EBMT2025EBMT_1627;
This resulted in the safe administration of SCB in all of our patients and haematopoietic recovery in the majority of them. Despite the small number of patients included in our study and its retrospectivity, our findings align with outcomes reported in previous research.
- |||||||||| POSITIVE SELECTION OF CD34+ FROM CRYOPRESERVED STEM CELL (SC) SOURCE TO TREAT POOR GRAFT FUNCTION (PGF): PRELIMINARY DATA FROM TWO ITALIAN TRANSPLANT CENTERS (Firenze (Spadolini 1)) - Feb 5, 2025 - Abstract #EBMT2025EBMT_1616;
We have demonstrated that the selection of CD34+ cells from a cryopreserved SC source is feasible; this is the proof of principle that immunoselected CD34+cells from a cryopreserved SC source are able to rescue with success the PGF, without serious adverse events. To our knowledge only an other experience of Mayo Clinic group reported 9 similar procedures, but without data about the patient outcome.A joint effort would be desirable to expand the number of reports with this approach in order to better evaluate the clinical benefit of the boost with selected cryopreserved CD34+ cells
- |||||||||| POSITIVE SELECTION OF CD34+ FROM CRYOPRESERVED STEM CELL (SC) SOURCE TO TREAT POOR GRAFT FUNCTION (PGF): PRELIMINARY DATA FROM TWO ITALIAN TRANSPLANT CENTERS (ePoster Area) - Feb 5, 2025 - Abstract #EBMT2025EBMT_1615;
We have demonstrated that the selection of CD34+ cells from a cryopreserved SC source is feasible; this is the proof of principle that immunoselected CD34+cells from a cryopreserved SC source are able to rescue with success the PGF, without serious adverse events. To our knowledge only an other experience of Mayo Clinic group reported 9 similar procedures, but without data about the patient outcome.A joint effort would be desirable to expand the number of reports with this approach in order to better evaluate the clinical benefit of the boost with selected cryopreserved CD34+ cells
- |||||||||| NOVEL FLOW CYTOMETRIC KIT FOR THE IDENTIFICATION, CHARACTERIZATION AND ENUMERATION OF HEMATOPOIETIC STEM CELLS FROM DIFFERENT BLOOD SOURCES (Firenze (Spadolini 1)) - Feb 5, 2025 - Abstract #EBMT2025EBMT_989;
To support the biomedical field of cell manufacturing and the underlying scientific research that would benefit from optimized cell characterization workflows, we developed a novel HSC Characterization Kit (anti-human) that is convenient, rapid, and reliable for the accurate enumeration and characterization of viable CD34+ HSC and subpopulations such as primitive HSC (e.g. CD34+ CD38-CD45RA- CD133+ CD49f+)1). Furthermore, the kit can be used to support investigation into the identification of optimal subpopulations of CD34+ HSCs for genetic engineering, and to characterize their long-term persistence, enabling the fulfilment of their full biological function in vivo.
- |||||||||| NOVEL FLOW CYTOMETRIC KIT FOR THE IDENTIFICATION, CHARACTERIZATION AND ENUMERATION OF HEMATOPOIETIC STEM CELLS FROM DIFFERENT BLOOD SOURCES (ePoster Area) - Feb 5, 2025 - Abstract #EBMT2025EBMT_988;
To support the biomedical field of cell manufacturing and the underlying scientific research that would benefit from optimized cell characterization workflows, we developed a novel HSC Characterization Kit (anti-human) that is convenient, rapid, and reliable for the accurate enumeration and characterization of viable CD34+ HSC and subpopulations such as primitive HSC (e.g. CD34+ CD38-CD45RA- CD133+ CD49f+)1). Furthermore, the kit can be used to support investigation into the identification of optimal subpopulations of CD34+ HSCs for genetic engineering, and to characterize their long-term persistence, enabling the fulfilment of their full biological function in vivo.
- |||||||||| Novel Automatic Application for Enrichmentof CD34+ Hematopoietic Stem and Progenitor Cells from Apheresis Products (HCC Exhibit Hall 3; In-Person) - Dec 19, 2024 - Abstract #TCTASTCTCIBMTR2025TCT_ASTCT_CIBMTR_887;
Conclusively, the novel application is an automated process in a closed fluidic system with minimal user interaction and flexible time control that supports the need for individual cell manufacturing. The highly purified CD34+ stem cells can be used either as an injectable stem cell graft or as a starting product for subsequent stem cell engineering approaches.
- |||||||||| EXG34217 / Elixirgen Therap, Neupogen (filgrastim) / Kyowa Kirin, Amgen, Mozobil (plerixafor) / Sanofi
Successful Infusion of Autologous CD34+ Cells Treated with Exg-001 for Ex Vivo Telomere Elongation in a Patient with a Dyskeratosis Congenita () - Dec 16, 2022 - Abstract #TCTASTCTCIBMTR2023TCT_ASTCT_CIBMTR_926;
P1/2
This novel approach uses modified autologous stem cells in the absence of conditioning regimen or immunosuppression and is genotype and mutation independent which is highly desirable in this chemotherapy and radiation sensitive population. These data support further investigation and translation of telomere elongation via ZSCAN4 exposure by EXG-001 in patients with telomere biology disorders as a potential therapeutic intervention for the prevention or treatment of bone marrow failure.
- |||||||||| EXG34217 / Elixirgen Therap, Neupogen (filgrastim) / Kyowa Kirin, Amgen, Mozobil (plerixafor) / Sanofi
Successful Ex Vivo Telomere Elongation with Exg-001 in a Patient with a Dyskeratosis Congenita (ENMCC - 220-222) - Nov 4, 2022 - Abstract #ASH2022ASH_5140;
P1/2
We translated this work in a first-in-human clinical trial (NCT04211714), in which autologous CD34+ hematopoietic stem cells (HSC) were collected by pheresis from an adult subject with dyskeratosis congenita over two days after mobilization with plerixafor and filgrastim with IRB approval and consent...To address the concern of effects on terminal colony forming unit studies were performed and demonstrated maintenance of normal differentiation potential after treatment (Figure 1B; EXG34217)...This novel approach is genotype and mutation independent and uses modified autologous stem cells in the absence of conditioning regimen or immunosuppression which is highly desirable in this radiation and chemotherapy sensitive population. These data support further investigation and translation of telomere elongation via ZSCAN4 exposure by EXG-001 in patients with telomere biology disorders as a potential therapeutic intervention for the prevention or treatment of bone marrow failure.
- |||||||||| [VIRTUAL] Characterization of Peripheral Blood Mononuclear Cells Addback Following CD34 Enrichment, Engraftment and T and NK Cells Immune Reconstitution in Patients with High Risk Sickle Cell Disease (SCD) (IND 14359) (Poster Hall (Virtual Meeting)) - Nov 5, 2020 - Abstract #ASH2020ASH_1741;
P2
PBMNC addback to CD34 enriched HPC products, with a final dose of 2 × 105 CD3 cells/kg, led to stem cell products with a diverse mixture of T, NK, NKT, DC1, and DC2 cells. Immune reconstitution following PBMNC addback to CD34 enriched cells resulted in excellent CD4 and CD8 responses to CMV, ADV and EBV, and rapid functional NK cell reconstitution (Supported by FDA R01FD004090 (MSC)).
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