A Single Dose of CD117 Antibody Drug Conjugate Enables Hematopoietic Stem Cell Based Gene Therapy in Nonhuman Primates

Naoya Uchida; John F. Tisdale; Robert E. Donahue; Bradley R. Pearse; Sean M. McDonough; Jennifer L. Proctor; Allen E Krouse; Nathaniel Linde; Aylin Bonifacino; Rajiv Panwar; Ganapathy N. Sarma; Lena Kien; Kellie Latimer; Junia Dushime; Sharon L. Hyzy; Melissa L. Brooks; Rahul Palchaudhuri; Qing Li; Pranoti Sawant; Charlotte F. McDonagh; Anthony E. Boitano; Michael P. Cooke

doi:10.1016/j.bbmt.2019.12.139

A Single Dose of CD117 Antibody Drug Conjugate Enables Hematopoietic Stem Cell Based Gene Therapy in Nonhuman Primates

Biology of Blood and Marrow Transplantation ◽

10.1016/j.bbmt.2019.12.139 ◽

2020 ◽

Vol 26 (3) ◽

pp. S6

Author(s):

Naoya Uchida ◽

John F. Tisdale ◽

Robert E. Donahue ◽

Bradley R. Pearse ◽

Sean M. McDonough ◽

...

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Single Dose ◽

Hematopoietic Stem Cell ◽

Nonhuman Primates ◽

Antibody Drug Conjugate ◽

Hematopoietic Stem ◽

Drug Conjugate ◽

Antibody Drug

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Successful Allogeneic Hematopoietic Stem Cell Transplantation in Mice Mediated By Well-Tolerated Conditioning Regimens Based on CD45-Targeted Antibody Drug Conjugate: Implications for Haplo Transplantation

Biology of Blood and Marrow Transplantation ◽

10.1016/j.bbmt.2019.12.713 ◽

2020 ◽

Vol 26 (3) ◽

pp. S160

Author(s):

Sharon L. Hyzy ◽

Rahul Palchaudhuri ◽

Jennifer L. Proctor ◽

Bradley R. Pearse ◽

Ganapathy N. Sarma ◽

...

Keyword(s):

Stem Cell ◽

Hematopoietic Stem Cell Transplantation ◽

Stem Cell Transplantation ◽

Cell Transplantation ◽

Hematopoietic Stem Cell ◽

Antibody Drug Conjugate ◽

Hematopoietic Stem ◽

Drug Conjugate ◽

Conditioning Regimens ◽

Antibody Drug

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Targeted CD45 Antibody Drug Conjugate Enables Full Mismatch Allogeneic Hematopoietic Stem Cell Transplantation in a Murine HSCT Model As a Single Agent

Transplantation and Cellular Therapy ◽

10.1016/s2666-6367(21)00268-2 ◽

2021 ◽

Vol 27 (3) ◽

pp. S217

Author(s):

Sharon L. Hyzy ◽

Jennifer L. Proctor ◽

Geoffrey O. Gillard ◽

Katelyn J. Hammond ◽

Ganapathy N. Sarma ◽

...

Keyword(s):

Stem Cell ◽

Hematopoietic Stem Cell Transplantation ◽

Stem Cell Transplantation ◽

Cell Transplantation ◽

Hematopoietic Stem Cell ◽

Single Agent ◽

Antibody Drug Conjugate ◽

Hematopoietic Stem ◽

Drug Conjugate ◽

Antibody Drug

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A Non-Genotoxic Antibody Drug Conjugate Targeting C-Kit for Hematopoietic Stem Cell Transplant Conditioning

Biology of Blood and Marrow Transplantation ◽

10.1016/j.bbmt.2017.12.598 ◽

2018 ◽

Vol 24 (3) ◽

pp. S47-S48 ◽

Cited By ~ 3

Author(s):

Adam J. Hartigan ◽

Brad R. Pearse ◽

Sean M. McDonough ◽

Jennifer L. Proctor ◽

Hillary L. Adams ◽

...

Keyword(s):

Stem Cell ◽

Hematopoietic Stem Cell ◽

Hematopoietic Stem Cell Transplant ◽

Stem Cell Transplant ◽

Cell Transplant ◽

Antibody Drug Conjugate ◽

Hematopoietic Stem ◽

Drug Conjugate ◽

Antibody Drug

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A Non-Genotoxic Anti-CD117 Antibody Drug Conjugate (ADC) Designed for Patient Conditioning Prior to Stem Cell Transplant and HSC-Based Gene Therapy Has a Broad Therapeutic Window across Species

Biology of Blood and Marrow Transplantation ◽

10.1016/j.bbmt.2019.12.580 ◽

2020 ◽

Vol 26 (3) ◽

pp. S35-S36

Author(s):

Bradley R. Pearse ◽

Jennifer L. Proctor ◽

Sean M. McDonough ◽

Rajiv Panwar ◽

Ganapathy N. Sarma ◽

...

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Stem Cell Transplant ◽

Therapeutic Window ◽

Cell Transplant ◽

Antibody Drug Conjugate ◽

Drug Conjugate ◽

Antibody Drug

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Faculty Opinions recommendation of Repetitive busulfan administration after hematopoietic stem cell gene therapy associated with a dominant HDAC7 clone in a nonhuman primate.

Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature ◽

10.3410/f.4242977.4039078 ◽

2010 ◽

Author(s):

Karen E Pollok ◽

Shanbao Cai

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Hematopoietic Stem Cell ◽

Nonhuman Primate ◽

Hematopoietic Stem ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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Faculty Opinions recommendation of Selective hematopoietic stem cell ablation using CD117-antibody-drug-conjugates enables safe and effective transplantation with immunity preservation.

Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature ◽

10.3410/f.735034391.793568487 ◽

2019 ◽

Author(s):

John F DiPersio

Keyword(s):

Stem Cell ◽

Hematopoietic Stem Cell ◽

Hematopoietic Stem ◽

Antibody Drug Conjugates ◽

Drug Conjugates ◽

Cell Ablation ◽

Antibody Drug

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Long-term hematopoietic stem cell gene therapy corrects neuromuscular manifestations in preclinical study of Pompe mice

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2020.12.263 ◽

2021 ◽

Vol 132 (2) ◽

pp. S107

Author(s):

Niek P. van Til ◽

Yildirim Dogan ◽

Cecilia Barese ◽

Zeenath Unnisa ◽

Swaroopa Guda ◽

...

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Hematopoietic Stem Cell ◽

Preclinical Study ◽

Hematopoietic Stem ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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The Landscape of Hematopoietic Stem Cell Transplant and Gene Therapy for X-Linked Adrenoleukodystrophy

Current Treatment Options in Neurology ◽

10.1007/s11940-019-0605-y ◽

2019 ◽

Vol 21 (12) ◽

Cited By ~ 2

Author(s):

Eric J. Mallack ◽

Bela Turk ◽

Helena Yan ◽

Florian S. Eichler

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Hematopoietic Stem Cell ◽

Hematopoietic Stem Cell Transplant ◽

Stem Cell Transplant ◽

Cell Transplant ◽

Hematopoietic Stem

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Update on safety and efficacy of lentiviral hematopoietic stem cell gene therapy (HSC-GT) for metachromatic leukodystrophy (MLD)

European Journal of Paediatric Neurology ◽

10.1016/j.ejpn.2017.04.986 ◽

2017 ◽

Vol 21 ◽

pp. e20

Author(s):

F. Fumagalli ◽

V. Calbi ◽

A. Zambon ◽

F. Ciotti ◽

L. Lorioli ◽

...

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Hematopoietic Stem Cell ◽

Metachromatic Leukodystrophy ◽

Hematopoietic Stem ◽

Safety And Efficacy ◽

Cell Gene ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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Hematopoietic stem cell (HSC) gene therapy for two patients with adenosine deaminase (ADA) deficiency without cytoreductive conditioning; a suggestion for the optimal protocol for HSC gene therapy for ADA deficiency

Blood Cells Molecules and Diseases ◽

10.1016/j.bcmd.2007.10.013 ◽

2008 ◽

Vol 40 (2) ◽

pp. 251

Author(s):

Tadashi Ariga

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Adenosine Deaminase ◽

Hematopoietic Stem Cell ◽

Hematopoietic Stem ◽

Optimal Protocol

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