Lentivector Iterations and Pre-Clinical Scale-Up/Toxicity Testing: Targeting Mobilized CD34 + Cells for Correction of Fabry Disease
Fabry disease is a rare lysosomal storage disorder (LSD). We designed multiple recombinant lentivirus vectors (LVs) and tested their ability to engineer expression of human α-galactosidase A (α-gal A) in transduced Fabry patient CD34 hematopoietic cells. We further investigated the safety and effica...
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| Published in | Molecular therapy. Methods & clinical development Vol. 5; no. C; pp. 241 - 258 |
|---|---|
| Main Authors | , , , , , , , , , , , , , , , , , , , , , |
| Format | Journal Article |
| Language | English |
| Published |
United States
Elsevier Limited
16.06.2017
American Society of Gene & Cell Therapy Elsevier |
| Subjects | |
| Online Access | Get full text |
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| Abstract | Fabry disease is a rare lysosomal storage disorder (LSD). We designed multiple recombinant lentivirus vectors (LVs) and tested their ability to engineer expression of human α-galactosidase A (α-gal A) in transduced Fabry patient CD34
hematopoietic cells. We further investigated the safety and efficacy of a clinically directed vector, LV/AGA, in both ex vivo cell culture studies and animal models. Fabry mice transplanted with LV/AGA-transduced hematopoietic cells demonstrated α-gal A activity increases and lipid reductions in multiple tissues at 6 months after transplantation. Next we found that LV/AGA-transduced Fabry patient CD34
hematopoietic cells produced even higher levels of α-gal A activity than normal CD34
hematopoietic cells. We successfully transduced Fabry patient CD34
hematopoietic cells with "near-clinical grade" LV/AGA in small-scale cultures and then validated a clinically directed scale-up transduction process in a GMP-compliant cell processing facility. LV-transduced Fabry patient CD34
hematopoietic cells were subsequently infused into NOD/SCID/Fabry (NSF) mice; α-gal A activity corrections and lipid reductions were observed in several tissues 12 weeks after the xenotransplantation. Additional toxicology studies employing NSF mice xenotransplanted with the therapeutic cell product demonstrated minimal untoward effects. These data supported our successful clinical trial application (CTA) to Health Canada and opening of a "first-in-the-world" gene therapy trial for Fabry disease. |
|---|---|
| AbstractList | Fabry disease is a rare lysosomal storage disorder (LSD). We designed multiple recombinant lentivirus vectors (LVs) and tested their ability to engineer expression of human α-galactosidase A (α-gal A) in transduced Fabry patient CD34
+
hematopoietic cells. We further investigated the safety and efficacy of a clinically directed vector, LV/AGA, in both ex vivo cell culture studies and animal models. Fabry mice transplanted with LV/AGA-transduced hematopoietic cells demonstrated α-gal A activity increases and lipid reductions in multiple tissues at 6 months after transplantation. Next we found that LV/AGA-transduced Fabry patient CD34
+
hematopoietic cells produced even higher levels of α-gal A activity than normal CD34
+
hematopoietic cells. We successfully transduced Fabry patient CD34
+
hematopoietic cells with “near-clinical grade” LV/AGA in small-scale cultures and then validated a clinically directed scale-up transduction process in a GMP-compliant cell processing facility. LV-transduced Fabry patient CD34
+
hematopoietic cells were subsequently infused into NOD/SCID/Fabry (NSF) mice; α-gal A activity corrections and lipid reductions were observed in several tissues 12 weeks after the xenotransplantation. Additional toxicology studies employing NSF mice xenotransplanted with the therapeutic cell product demonstrated minimal untoward effects. These data supported our successful clinical trial application (CTA) to Health Canada and opening of a “first-in-the-world” gene therapy trial for Fabry disease. Fabry disease is a rare lysosomal storage disorder (LSD). We designed multiple recombinant lentivirus vectors (LVs) and tested their ability to engineer expression of human α-galactosidase A (α-gal A) in transduced Fabry patient CD34+ hematopoietic cells. We further investigated the safety and efficacy of a clinically directed vector, LV/AGA, in both ex vivo cell culture studies and animal models. Fabry mice transplanted with LV/AGA-transduced hematopoietic cells demonstrated α-gal A activity increases and lipid reductions in multiple tissues at 6 months after transplantation. Next we found that LV/AGA-transduced Fabry patient CD34+ hematopoietic cells produced even higher levels of α-gal A activity than normal CD34+ hematopoietic cells. We successfully transduced Fabry patient CD34+ hematopoietic cells with “near-clinical grade” LV/AGA in small-scale cultures and then validated a clinically directed scale-up transduction process in a GMP-compliant cell processing facility. LV-transduced Fabry patient CD34+ hematopoietic cells were subsequently infused into NOD/SCID/Fabry (NSF) mice; α-gal A activity corrections and lipid reductions were observed in several tissues 12 weeks after the xenotransplantation. Additional toxicology studies employing NSF mice xenotransplanted with the therapeutic cell product demonstrated minimal untoward effects. These data supported our successful clinical trial application (CTA) to Health Canada and opening of a “first-in-the-world” gene therapy trial for Fabry disease. Fabry disease is a rare lysosomal storage disorder (LSD). We designed multiple recombinant lentivirus vectors (LVs) and tested their ability to engineer expression of human α-galactosidase A (α-gal A) in transduced Fabry patient CD34+ hematopoietic cells. We further investigated the safety and efficacy of a clinically directed vector, LV/AGA, in both ex vivo cell culture studies and animal models. Fabry mice transplanted with LV/AGA-transduced hematopoietic cells demonstrated α-gal A activity increases and lipid reductions in multiple tissues at 6 months after transplantation. Next we found that LV/AGA-transduced Fabry patient CD34+ hematopoietic cells produced even higher levels of α-gal A activity than normal CD34+ hematopoietic cells. We successfully transduced Fabry patient CD34+ hematopoietic cells with “near-clinical grade” LV/AGA in small-scale cultures and then validated a clinically directed scale-up transduction process in a GMP-compliant cell processing facility. LV-transduced Fabry patient CD34+ hematopoietic cells were subsequently infused into NOD/SCID/Fabry (NSF) mice; α-gal A activity corrections and lipid reductions were observed in several tissues 12 weeks after the xenotransplantation. Additional toxicology studies employing NSF mice xenotransplanted with the therapeutic cell product demonstrated minimal untoward effects. These data supported our successful clinical trial application (CTA) to Health Canada and opening of a “first-in-the-world” gene therapy trial for Fabry disease. Fabry disease is a rare lysosomal storage disorder (LSD). We designed multiple recombinant lentivirus vectors (LVs) and tested their ability to engineer expression of human α-galactosidase A (α-gal A) in transduced Fabry patient CD34 hematopoietic cells. We further investigated the safety and efficacy of a clinically directed vector, LV/AGA, in both ex vivo cell culture studies and animal models. Fabry mice transplanted with LV/AGA-transduced hematopoietic cells demonstrated α-gal A activity increases and lipid reductions in multiple tissues at 6 months after transplantation. Next we found that LV/AGA-transduced Fabry patient CD34 hematopoietic cells produced even higher levels of α-gal A activity than normal CD34 hematopoietic cells. We successfully transduced Fabry patient CD34 hematopoietic cells with "near-clinical grade" LV/AGA in small-scale cultures and then validated a clinically directed scale-up transduction process in a GMP-compliant cell processing facility. LV-transduced Fabry patient CD34 hematopoietic cells were subsequently infused into NOD/SCID/Fabry (NSF) mice; α-gal A activity corrections and lipid reductions were observed in several tissues 12 weeks after the xenotransplantation. Additional toxicology studies employing NSF mice xenotransplanted with the therapeutic cell product demonstrated minimal untoward effects. These data supported our successful clinical trial application (CTA) to Health Canada and opening of a "first-in-the-world" gene therapy trial for Fabry disease. |
| Author | Barber, Dwayne L Boutin, Michel Nagree, Murtaza S Auray-Blais, Christiane Turner, Patricia V Dworski, Shaalee Medin, Jeffrey A Schambach, Axel Prokopishyn, Nicole L Rothe, Michael Sirrs, Sandra Abaoui, Mona West, Michael L Au, Bryan C Keating, Armand Huang, Ju López-Vásquez, Lucía Foley, Ronan Rupar, C Anthony Rip, Jack W Khan, Aneal Klassen, John |
| AuthorAffiliation | 9 Department of Pathology and Laboratory Medicine, Western University, London, ON N6A 5C1, Canada 3 Department of Laboratory Medicine and Pathobiology, University of Toronto, Toronto, ON M5S 1A8, Canada 5 Institute of Medical Science, University of Toronto, Toronto, ON M5S 1A8, Canada 16 Medical College of Wisconsin, Milwaukee, WI 53226, USA 13 Department of Pathobiology, University of Guelph, Guelph, ON N1G 2W1, Canada 4 Department of Medical Biophysics, University of Toronto, Toronto, ON M5G 1L7, Canada 15 Juravinski Hospital and Cancer Centre, Hamilton, ON L8V 5C2, Canada 6 Department of Pathology and Laboratory Medicine, University of Calgary and Cellular Therapy Laboratory, Calgary Lab Services, Calgary, AB T2N 1N4, Canada 1 University Health Network, Toronto, ON M5G 1L7, Canada 11 Division of Nephrology, Department of Medicine, Dalhousie University, Halifax, NS B3H 1V8, Canada 14 Division of Endocrinology, Department of Medicine, University of British Columbia, Vancouver, BC V5Z 1M9, Canad |
| AuthorAffiliation_xml | – name: 10 Division of Hematology/Oncology, Boston Children’s Hospital, Harvard Medical School, Boston, MA 02115, USA – name: 5 Institute of Medical Science, University of Toronto, Toronto, ON M5S 1A8, Canada – name: 8 Institute of Experimental Hematology, Hannover Medical School, 30625 Hannover, Germany – name: 13 Department of Pathobiology, University of Guelph, Guelph, ON N1G 2W1, Canada – name: 1 University Health Network, Toronto, ON M5G 1L7, Canada – name: 11 Division of Nephrology, Department of Medicine, Dalhousie University, Halifax, NS B3H 1V8, Canada – name: 3 Department of Laboratory Medicine and Pathobiology, University of Toronto, Toronto, ON M5S 1A8, Canada – name: 4 Department of Medical Biophysics, University of Toronto, Toronto, ON M5G 1L7, Canada – name: 6 Department of Pathology and Laboratory Medicine, University of Calgary and Cellular Therapy Laboratory, Calgary Lab Services, Calgary, AB T2N 1N4, Canada – name: 7 Division of Medical Genetics, Department of Pediatrics, Université de Sherbrooke, Sherbrooke, QC J1K 2R1, Canada – name: 14 Division of Endocrinology, Department of Medicine, University of British Columbia, Vancouver, BC V5Z 1M9, Canada – name: 9 Department of Pathology and Laboratory Medicine, Western University, London, ON N6A 5C1, Canada – name: 12 Department of Hematology, University of Calgary, Foothills Hospital, Calgary, AB T2N 2T9, Canada – name: 15 Juravinski Hospital and Cancer Centre, Hamilton, ON L8V 5C2, Canada – name: 2 Department of Medical Genetics, Alberta Children’s Hospital Research Institute, Cumming School of Medicine, University of Calgary, Calgary, AB T2N 4N1, Canada – name: 16 Medical College of Wisconsin, Milwaukee, WI 53226, USA |
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| BackLink | https://www.ncbi.nlm.nih.gov/pubmed/28603745$$D View this record in MEDLINE/PubMed |
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| Keywords | xenotransplantation α-galactosidase A hematopoietic stem/progenitor cell lysosomal storage disease GMP production |
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| Snippet | Fabry disease is a rare lysosomal storage disorder (LSD). We designed multiple recombinant lentivirus vectors (LVs) and tested their ability to engineer... |
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| StartPage | 241 |
| SubjectTerms | Animal models CD34 antigen Cell culture Enzymes Expression vectors Fabry's disease FDA approval Flow cytometry Gene therapy Genomes GMP production Good Manufacturing Practice hematopoietic stem/progenitor cell lysosomal storage disease Males Medical screening Metabolism Original Patients Toxicity testing Transplantation Xenografts xenotransplantation α-galactosidase A |
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| Title | Lentivector Iterations and Pre-Clinical Scale-Up/Toxicity Testing: Targeting Mobilized CD34 + Cells for Correction of Fabry Disease |
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