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Journal Articles

Long-Term Persistence of Antibodies to Adeno-Associated Viral Vectors Following Gene Therapy with scAAV8-LP1-Fixco

Jenny H. McIntosh, Saira Mohamed, Lydia Ireland, Christopher L Morton, Ulrike M. Reiss, Pratima Chowdary, Edward G. D. Tuddenham, Andrew M. Davidoff, Amit C. Nathwani

Blood (2023) 142 (Supplement 1): 2255.

https://doi.org/10.1182/blood-2023-188383

Published: 2023

Journal Articles

NVG-222: A First-in-Class Autoregulating Half-Life Extended ROR1xCD3 T Cell Engager Heralding a New Class of Safer Drugs

David Granger, Vincent Muczynski, Patricia Henne, Annalisa Baccaro, Dinusha Fernando, Mittal Shah, John Devlin, Kieran O'Donovan, Amit C. Nathwani

Blood (2023) 142 (Supplement 1): 2820.

https://doi.org/10.1182/blood-2023-189058

Published: 2023

Journal Articles

AAV-Mediated Expression of a Single-Chain FVIII Mimetic Antibody Efficiently Corrects Bleeding in Haemophilia a Mice

Vincent Muczynski, Eric Bun, Lewis Tanner, Jenny H. McIntosh, Olivier D. Christophe, Amit C. Nathwani

Blood (2023) 142 (Supplement 1): 482.

https://doi.org/10.1182/blood-2023-186285

Published: 2023

Journal Articles

Time Limited Exposure to a ROR1 Targeting Bispecific T Cell Engager (NVG-111) Leads to Durable Responses in Subjects with Relapsed Refractory Chronic Lymphocytic Leukemia (CLL) and Mantle Cell Lymphoma (MCL)

William Townsend, Sarah Leong, Mittal Shah, Toby Batten, David Tucker, Bryson Pottinger, Shankaranarayana Paneesha, Dima El-Sharkawi, Toby A. Eyre, Ho Pui Jeff Lam, Jiexin Zheng, Sarah Cook, David Granger, David Ahern, Kieran O'Donovan, Amit C. Nathwani, Parag Jasani

Blood (2023) 142 (Supplement 1): 329.

https://doi.org/10.1182/blood-2023-188607

Published: 2023

Journal Articles

Stable Therapeutic Transgenic FIX Levels for More Than 10 Years in Subjects with Severe Hemophilia B Who Received scAAV2/8-LP1-Hfixco Adeno-Associated Virus Gene Therapy

Blood (2023) 142 (Supplement 1): 1056.

https://doi.org/10.1182/blood-2023-186891

Published: 2023

Journal Articles

GO-8: Stable Expression of Factor VIII over 5 Years Following Adeno-Associated Gene Transfer in Subjects with Hemophilia a Using a Novel Human Factor VIII Variant

Blood (2023) 142 (Supplement 1): 3624.

https://doi.org/10.1182/blood-2023-180803

Published: 2023

Journal Articles

First-in-Human Phase I Trial of a ROR1 Targeting Bispecific T Cell Engager (NVG-111) in Combination with Ibrutinib or As Monotherapy in Subjects with Relapsed Refractory Chronic Lymphocytic Leukaemia (CLL) and Mantle Cell Lymphoma (MCL)

William Townsend, Sarah Leong, David Tucker, Bryson Pottinger, Shankara Paneesha, Dima El-Sharkawi, Toby A. Eyre, Toby Batten, Mittal Shah, Sarah Cook, David Granger, Kieran O'Donovan, Amit C. Nathwani, Parag Jasani

Blood (2022) 140 (Supplement 1): 4162–4163.

https://doi.org/10.1182/blood-2022-169658

Published: 2022

Journal Articles

Pre-Clinical Development of a First in Class Half-Life Extended T Cell Engager Targeting ROR-1

David Granger, Patricia Henne, Annalisa Baccaro, Dinusha Fernando, Jasmine Gore, Ephraim Gbajumo, Vincent Muczynski, Mittal Shah, Kieran O'Donovan, Kerry Chester, Amit C. Nathwani

Blood (2022) 140 (Supplement 1): 7810–7811.

https://doi.org/10.1182/blood-2022-168961

Published: 2022

Journal Articles

A FVIII-Mimetic Bispecific Antibody with an Embedded Self-Regulation Mechanism Reduces the Risk of Prothrombotic Events for the Treatment of Haemophilia a

Vincent Muczynski, Angel Smith, Thibaud Sefiane, Olivier D. Christophe, Peter J. Lenting, David Granger, Kerry Chester, Amit C. Nathwani

Blood (2022) 140 (Supplement 1): 677–678.

https://doi.org/10.1182/blood-2022-169482

Published: 2022

Journal Articles

The cost-effectiveness of gene therapy for severe hemophilia B: a microsimulation study from the United States perspective

Nancy S. Bolous, Yichen Chen, Huiqi Wang, Andrew M. Davidoff, Meenakshi Devidas, Timothy W. Jacobs, Michael M. Meagher, Amit C. Nathwani, Ellis J. Neufeld, Bryan A. Piras, Carlos Rodriguez-Galindo, Ulrike M. Reiss, Nickhill Bhakta

Blood (2021) 138 (18): 1677–1690.

https://doi.org/10.1182/blood.2021010864

Published: 2021

Includes: Supplemental data

Journal Articles

Evaluating ROR1 As a Target in Childhood Leukemia

Paraskevi Diamanti, PhD, Bethan Kathleen Bailey, BSc, Obinna Ebere Iheanacho, MD, Amit C. Nathwani, MD PhD, Allison Blair, PhD

Blood (2019) 134 (Supplement_1): 2635.

https://doi.org/10.1182/blood-2019-126734

Published: 2019

Journal Articles

Liver-Directed AAV Gene Therapy for Gaucher Disease

Carlos J Miranda, PhD, Miriam Canavese, PhD, Elisa Chisari, MS, Jalpa Pandya, MPhil, Clement Cocita, PhD, Maria Portillo, MRes, Jenny McIntosh, PhD, Azadeh Kia, PhD, Jonathan H. Foley, PhD, Allison Dane, PhD, Jey M Jeyakumar, PhD, Rose Sheridan, PhD, Romuald Corbau, Amit C. Nathwani, MD PhD

Blood (2019) 134 (Supplement_1): 3354.

https://doi.org/10.1182/blood-2019-124280

Published: 2019

Journal Articles

Enhancing FVIII Expression By Modifying B-Domain Sequence for Hemophilia A Gene Therapy

Doyoung Lee, PhD, Jenny McIntosh, PhD, Allison Dane, PhD, Cecilia Rosales, PhD, Anne Riddell, Edward Tuddenham, MD, Amit C Nathwani, MD PhD

Blood (2018) 132 (Supplement 1): 3484.

https://doi.org/10.1182/blood-2018-99-113830

Published: 2018

Journal Articles

A Novel Lysine to Arginine Substitution at Position 301 Enhances Activity of Factor IX

Daniel Verhoef, Jonathan H. Foley, Andrew Goodale, Emma Macrae, Jenny McIntosh, PhD, Romuald Corbau, PhD, Keith Gomez, Anne Riddell, Amit C Nathwani, MD PhD

Blood (2018) 132 (Supplement 1): 3772.

https://doi.org/10.1182/blood-2018-99-112832

Published: 2018

Journal Articles

Adeno-Associated Mediated Gene Transfer for Hemophilia B:8 Year Follow up and Impact of Removing "Empty Viral Particles" on Safety and Efficacy of Gene Transfer

Amit C Nathwani, MD PhD, Ulrike Reiss, MD, Edward Tuddenham, MD, Pratima Chowdary, MD, Jenny McIntosh, PhD, Anne Riddell, Jun Pie, Johnny N Mahlangu, MD, Michael Recht, MD PhD, Yu-Min Shen, MD, Kathleen G. Halka, MD, Michael M Meagher, PhD, Arthur W. Nienhuis, MD, Andrew M. Davidoff, MD, Sarah Mangles, Chris L Morton, Zhou Junfang, Vlad Calin Radulescu, MD

Blood (2018) 132 (Supplement 1): 491.

https://doi.org/10.1182/blood-2018-99-118334

Published: 2018

Journal Articles

GO-8: Preliminary Results of a Phase I/II Dose Escalation Trial of Gene Therapy for Haemophilia a Using a Novel Human Factor VIII Variant

Amit C Nathwani, MD PhD, Edward Tuddenham, MD, Pratima Chowdary, MD, Jenny McIntosh, PhD, Doyoung Lee, PhD, Cecilia Rosales, PhD, Mark Phillips, Jun Pie, Zhou Junfang, Michael M Meagher, PhD, Ulrike Reiss, MD, Andrew M. Davidoff, MD, Chris L Morton, Anne Riddell

Blood (2018) 132 (Supplement 1): 489.

https://doi.org/10.1182/blood-2018-99-118256

Published: 2018

Journal Articles

Efficacy Evaluation of Liver-Directed Gene Therapy in Fabry Mice

Azadeh Kia, PhD, Jenny McIntosh, PhD, Cecilia Rosales, PhD, Paniz Hosseini, Rose Sheridan, Justyna Spiewak, Kevin Mills, Romuald Corbau, PhD, Amit C Nathwani, MD PhD

Blood (2018) 132 (Supplement 1): 2209.

https://doi.org/10.1182/blood-2018-99-116632

Published: 2018

Journal Articles

Preclinical Evaluation of an Engineered AAV Capsid in Non-Human Primates for the Treatment of Haemophilia B

Allison Dane, PhD, Jenny McIntosh, Doyoung Lee, PhD, Rose Sheridan, Mark Maginn, Romuald Corbau, PhD, Andrew M. Davidoff, MD, Amit C Nathwani, MD PhD

Blood (2018) 132 (Supplement 1): 2197.

https://doi.org/10.1182/blood-2018-99-117969

Published: 2018

Journal Articles

A Single Intravenous Infusion of FLT180a Results in Factor IX Activity Levels of More Than 40% and Has the Potential to Provide a Functional Cure for Patients with Haemophilia B

Pratima Chowdary, MD, Susie Shapiro, MD, Andrew M. Davidoff, MD, Ulrike Reiss, MD, Rebecca Alade, PhD, Geoff Brooks, BSc, Allison Dane, PhD, Jenny McIntosh, PhD, Gerard Short, MBBS, Edward Tuddenham, MD, Amit C Nathwani, MD PhD

Blood (2018) 132 (Supplement 1): 631.

https://doi.org/10.1182/blood-2018-99-118050

Published: 2018

Journal Articles

Cytotoxic CD4+ Cells in Chronic Lymphocytic Leukaemia: An Extended Immunophenotypic Analysis Examining Their Association with Cytomegalovirus Serostatus and Similarities with Cytotoxic CD8+ Cells

Maria A V Marzolini, Ayse Akarca, Karen Teng, Teresa Marafioti, MD, Satyen H Gohil, Amit C Nathwani, MD PhD, Evan Newell, PhD, Sergio A Quezada, Karl S Peggs

Blood (2018) 132 (Supplement 1): 3130.

https://doi.org/10.1182/blood-2018-99-116798

Published: 2018

NARROW

Long-Term Persistence of Antibodies to Adeno-Associated Viral Vectors Following Gene Therapy with scAAV8-LP1-Fixco

NVG-222: A First-in-Class Autoregulating Half-Life Extended ROR1xCD3 T Cell Engager Heralding a New Class of Safer Drugs

AAV-Mediated Expression of a Single-Chain FVIII Mimetic Antibody Efficiently Corrects Bleeding in Haemophilia a Mice

Time Limited Exposure to a ROR1 Targeting Bispecific T Cell Engager (NVG-111) Leads to Durable Responses in Subjects with Relapsed Refractory Chronic Lymphocytic Leukemia (CLL) and Mantle Cell Lymphoma (MCL)

Stable Therapeutic Transgenic FIX Levels for More Than 10 Years in Subjects with Severe Hemophilia B Who Received scAAV2/8-LP1-Hfixco Adeno-Associated Virus Gene Therapy

GO-8: Stable Expression of Factor VIII over 5 Years Following Adeno-Associated Gene Transfer in Subjects with Hemophilia a Using a Novel Human Factor VIII Variant

First-in-Human Phase I Trial of a ROR1 Targeting Bispecific T Cell Engager (NVG-111) in Combination with Ibrutinib or As Monotherapy in Subjects with Relapsed Refractory Chronic Lymphocytic Leukaemia (CLL) and Mantle Cell Lymphoma (MCL)

Pre-Clinical Development of a First in Class Half-Life Extended T Cell Engager Targeting ROR-1

A FVIII-Mimetic Bispecific Antibody with an Embedded Self-Regulation Mechanism Reduces the Risk of Prothrombotic Events for the Treatment of Haemophilia a

The cost-effectiveness of gene therapy for severe hemophilia B: a microsimulation study from the United States perspective

Evaluating ROR1 As a Target in Childhood Leukemia

Liver-Directed AAV Gene Therapy for Gaucher Disease

Enhancing FVIII Expression By Modifying B-Domain Sequence for Hemophilia A Gene Therapy

A Novel Lysine to Arginine Substitution at Position 301 Enhances Activity of Factor IX

Adeno-Associated Mediated Gene Transfer for Hemophilia B:8 Year Follow up and Impact of Removing "Empty Viral Particles" on Safety and Efficacy of Gene Transfer

GO-8: Preliminary Results of a Phase I/II Dose Escalation Trial of Gene Therapy for Haemophilia a Using a Novel Human Factor VIII Variant

Efficacy Evaluation of Liver-Directed Gene Therapy in Fabry Mice

Preclinical Evaluation of an Engineered AAV Capsid in Non-Human Primates for the Treatment of Haemophilia B

A Single Intravenous Infusion of FLT180a Results in Factor IX Activity Levels of More Than 40% and Has the Potential to Provide a Functional Cure for Patients with Haemophilia B

Cytotoxic CD4+ Cells in Chronic Lymphocytic Leukaemia: An Extended Immunophenotypic Analysis Examining Their Association with Cytomegalovirus Serostatus and Similarities with Cytotoxic CD8+ Cells

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American Society of Hematology

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American Society of Hematology

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