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1. In Vivo Delivery of a CD20 CAR Using a CD8-Targeted Fusosome in Southern Pig-Tail Macaques (M. nemestrina) Results in B Cell Depletion

Author

Cunningham, Justine, primary, Chandra, Sundeep, additional, Emmanuel, Akinola, additional, Mazzarelli, Allyse, additional, Passaro, Carmela, additional, Baldwin, Paige, additional, Nguyen-McCarty, Michelle, additional, Rocca, Carrie, additional, Joyce, Shannon, additional, Kim, Joohwan, additional, Vagin, Vasily, additional, Kaczmarek, James, additional, Chavan, Hemant, additional, Jewell, Heather, additional, Lipsitz, Yonatan, additional, Shamashkin, Misha, additional, Hlavaty, Kelan, additional, Rodriguez, Jason, additional, Co, Carl, additional, Cruite, Pattie, additional, Ennajdaoui, Hanane, additional, Duback, Victoria, additional, Elman, Jess, additional, Amatya, Shirisha, additional, Harding, Caspian, additional, Lyubinetsky, Sergey, additional, Patel, Vidur, additional, Pepper, Lauren, additional, Ruzo, Albert, additional, Iovino, Salvatore, additional, Varghese, Bindu, additional, Foster, Aaron E., additional, Gorovits, Boris, additional, Elpek, Kutlu, additional, Laska, Michael, additional, McGill, Trevor, additional, Shah, Jagesh V, additional, Fry, Terry J., additional, and Dambach, Donna, additional

Published
2021
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2. Abstract LB105: In vivo CAR T therapy: targeted in vivo gene delivery of a CAR using a CD8-specific fusogen results in tumor eradication

Author

Emmanuel, Akinola, primary, Cruite, Patty, additional, Ennajdaoui, Hanane, additional, Passaro, Carmela, additional, Baldwin, Paige, additional, Duback, Victoria, additional, Liang, Anna, additional, Elman, Jess, additional, Crocker, Samantha, additional, Amatya, Shirisha, additional, Harding, Caspian, additional, Mazarelli, Allyse, additional, Lyubinetsky, Sergey, additional, Patel, Vidur, additional, Parikh, Avani, additional, Hlavaty, Kelan, additional, Rodriguez, Jason, additional, Pepper, Lauren, additional, Ruzo, Albert, additional, Iovino, Salvatore, additional, Elpek, Kutlu, additional, Laska, Michael, additional, McGill, Trevor, additional, Dambach, Donna, additional, Fry, Terry, additional, and Shah, Jagesh, additional

Published
2021
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3. In VivoDelivery of a CD20 CAR Using a CD8-Targeted Fusosome in Southern Pig-Tail Macaques (M. nemestrina) Results in B Cell Depletion

Author

Cunningham, Justine, Chandra, Sundeep, Emmanuel, Akinola, Mazzarelli, Allyse, Passaro, Carmela, Baldwin, Paige, Nguyen-McCarty, Michelle, Rocca, Carrie, Joyce, Shannon, Kim, Joohwan, Vagin, Vasily, Kaczmarek, James, Chavan, Hemant, Jewell, Heather, Lipsitz, Yonatan, Shamashkin, Misha, Hlavaty, Kelan, Rodriguez, Jason, Co, Carl, Cruite, Pattie, Ennajdaoui, Hanane, Duback, Victoria, Elman, Jess, Amatya, Shirisha, Harding, Caspian, Lyubinetsky, Sergey, Patel, Vidur, Pepper, Lauren, Ruzo, Albert, Iovino, Salvatore, Varghese, Bindu, Foster, Aaron E., Gorovits, Boris, Elpek, Kutlu, Laska, Michael, McGill, Trevor, Shah, Jagesh V, Fry, Terry J., and Dambach, Donna

Abstract

Introduction: Ex vivomanufactured chimeric antigen receptor (CAR) T cell therapies are highly effective for treating B cell malignancies. However, the complexity, cost and time required to manufacture CAR T cells limits access. To overcome conventional ex vivoCAR T limitations, a novel gene therapy platform has been developed that can deliver CAR transgenes directly to T cells through systemic administration of a fusosome, an engineered, target-directed novel paramyxovirus-based integrating vector that binds specific cell surface receptors for gene delivery through membrane fusion. Here, we demonstrate that systemic administration of a CD8a-targeted, integrating vector envelope (i.e., fusogen) encoding an anti-CD20 CAR into Southern pig-tail macaques (M. nemestrina), which is a species permissive to the integrating vector-mediated transduction, results in T cell transduction and B cell depletion with no treatment-related toxicities.

Published
2021
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