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Journal Articles

BAX 335 hemophilia B gene therapy clinical trial results: potential impact of CpG sequences on gene expression

Barbara A. Konkle, Christopher E. Walsh, Miguel A. Escobar, Neil C. Josephson, Guy Young, Annette von Drygalski, Scott W. J. McPhee, R. Jude Samulski, Ivan Bilic, Maurus de la Rosa, Birgit M. Reipert, Hanspeter Rottensteiner, Friedrich Scheiflinger, John C. Chapin, Bruce Ewenstein, Paul E. Monahan

Blood (2021) 137 (6): 763–774.

https://doi.org/10.1182/blood.2019004625

Published: 2021

Includes: Supplemental data

Journal Articles

AAV8-Specific Immune Adsorption Column: A Treatment Option for Patients with Pre-Existing Anti-AAV8 Neutralizing Antibodies

Anita Kruzik, PhD, Roland Raim, PhD, Dirk Voelkel, PhD, Markus Weiller, PhD, Werner Hoellriegl, PhD, Friedrich Scheiflinger, Hanspeter Rottensteiner, Birgit M Reipert, PhD, Maurus de la Rosa

Blood (2019) 134 (Supplement_1): 5922.

https://doi.org/10.1182/blood-2019-126730

Published: 2019

Journal Articles

The Factor VIII Variant X5 Enhances Hemophilia a Gene Therapy Efficiency By Its Improved Secretion

Franziska Horling, PhD, Johannes Lengler, PhD, Wenjing Cao, PhD, Biao Dong, PhD, Bagirath Gangadharan, Maurus De La Rosa, PhD, Werner Hoellriegl, PhD, Birgit M Reipert, PhD, Friedrich Scheiflinger, Weidong Xiao, PhD, Hanspeter Rottensteiner

Blood (2019) 134 (Supplement_1): 3356.

https://doi.org/10.1182/blood-2019-126489

Published: 2019

Journal Articles

Evaluation of the Human Factor IX Gene Therapy Vector TAK-748 in Hemophilia: Results from Non-Clinical Studies in Factor IX Knockout Mice and Rhesus Monkeys

Markus Weiller, Helen Wang, Sogué Coulibaly, Maria Schuster, Hanspeter Rottensteiner, Kefeng Sun, Marinee K Chuah, Thierry Vandendriessche, Friedrich Scheiflinger, Werner Höllriegl

Blood (2019) 134 (Supplement_1): 4633.

https://doi.org/10.1182/blood-2019-124734

Published: 2019

Journal Articles

Data Coming out of the Human Inhibitor PUP Study (HIPS) Reveal 4 Subgroups of Patients with Distinct Antibody Signatures

Blood (2018) 132 (Supplement 1): 3774.

https://doi.org/10.1182/blood-2018-99-115979

Published: 2018

Journal Articles

Bispecific Antibodies with Native Human IgG Structure As Mimetics for FVIII Activity

Sabine Knappe, PhD, Nicolas Fischer, Patrice Robert Douillard, Rudolf Hartmann, PhD, Gerald Schrenk, PhD, Ulla Ravn, Giovanni Magistrelli, Franck Gueneau, Gerard Didelot, Krzysztof Masternak, Marie Kosco-Vilbois, Friedrich Scheiflinger, PhD, Michael Dockal, PhD

Blood (2017) 130 (Supplement 1): 1083.

https://doi.org/10.1182/blood.V130.Suppl_1.1083.1083

Published: 2017

Journal Articles

Elucidating the Excessive Pro-Coagulant Effect of a Sequence Identical Analogue to ACE910 in Combination with Bypassing Agents

Rudolf Hartmann, PhD, Tjerk Feenstra, MSc, Sabine Knappe, PhD, Michael Dockal, PhD, Friedrich Scheiflinger, PhD

Blood (2017) 130 (Supplement 1): 90.

https://doi.org/10.1182/blood.V130.Suppl_1.90.90

Published: 2017

Journal Articles

Influence of Obesity on Factor IX Expression after Gene Therapy in a Mouse Model of Hemophilia B

Sogué Coulibaly, Markus Weiller, Alexander Bauer, Johannes Lengler, Stefan Romeder-Finger, Alexandra Kopić, Karima Benamara, Paul Monahan, Hanspeter Rottensteiner, Friedrich Scheiflinger, PhD, Marietta Turecek, Werner Hoellriegl, MD

Blood (2017) 130 (Supplement 1): 4878.

https://doi.org/10.1182/blood.V130.Suppl_1.4878.4878

Published: 2017

Journal Articles

A Bispecific Antibody Lacks Measurability in Routine Coagulation Assays and Comparability to Factor VIII

Rudolf Hartmann, PhD, Tjerk Feenstra, MSc, Sabine Knappe, PhD, Gerald Schrenk, PhD, Friedrich Scheiflinger, PhD, Michael Dockal, PhD

Blood (2017) 130 (Supplement 1): 1080.

https://doi.org/10.1182/blood.V130.Suppl_1.1080.1080

Published: 2017

Journal Articles

Synergistic Effects of a Procoagulant Bispecific Antibody and Rescue Therapies on Thrombin Generation- a Potential Safety Risk

Sabine Knappe, Rudolf Hartmann, Brahm Goldstein, MD, Bruce M. Ewenstein, Leonard Valentino, Friedrich Scheiflinger, PhD

Blood (2016) 128 (22): 4952.

https://doi.org/10.1182/blood.V128.22.4952.4952

Published: 2016

Journal Articles

ADAMTS13-mediated thrombolysis of t-PA–resistant occlusions in ischemic stroke in mice

Frederik Denorme, Friederike Langhauser, Linda Desender, Aline Vandenbulcke, Hanspeter Rottensteiner, Barbara Plaimauer, Olivier François, Tommy Andersson, Hans Deckmyn, Friedrich Scheiflinger, Christoph Kleinschnitz, Karen Vanhoorelbeke, Simon F. De Meyer

Blood (2016) 127 (19): 2337–2345.

https://doi.org/10.1182/blood-2015-08-662650

Published: 2016

Includes: Supplemental data

Journal Articles

Pharmacokinetics of BAX 826, a Polysialylated Full-Length rFVIII, in Hemophilia a Mice, Rats, and Cynomolgus Monkeys

Alexandra Schiviz, DVM, Gerald Hoebarth, Martin Wolfsegger, Paolo Rossato, Alfred Weber, Herbert Gritsch, Hanspeter Rottensteiner, PhD, Peter L. Turecek, PhD, Friedrich Scheiflinger, PhD, Werner Hoellriegl, DVM, Marietta Putz, MS

Blood (2015) 126 (23): 1073.

https://doi.org/10.1182/blood.V126.23.1073.1073

Published: 2015

Journal Articles

Non-Proteolytic Regulation of the Multimerization of Von Willebrand Factor By ADAMTS13 Based on a Novel Cysteine Thiol Redundancy

Hanspeter Rottensteiner, PhD, Susanna Skalicky, Birgit Seyfried, PhD, Stefan Kaufmann, Christian Fiedler, Meinhard Hasslacher, PhD, Jing-fei Dong, MD PhD, X. Long Zheng, MD PhD, Barbara Plaimauer, PhD, Friedrich Scheiflinger, PhD

Blood (2015) 126 (23): 2236.

https://doi.org/10.1182/blood.V126.23.2236.2236

Published: 2015

Journal Articles

Temperature-Dependent Irreversible Conformational Change of ADAMTS13 upon Metal Ion Chelation

Rana Grillberger, PhD, Stefan Kaufmann, Barbara Kink, Peter Matthiessen, PhD, Barbara Plaimauer, PhD, Friedrich Scheiflinger, PhD, Hanspeter Rottensteiner, PhD

Blood (2015) 126 (23): 2238.

https://doi.org/10.1182/blood.V126.23.2238.2238

Published: 2015

Journal Articles

The Application of Thrombin Generation Test Is Compromised By Antithrombin III Deficiency

Sabine Knappe, Susanne Till, Gabriele Gerstenbauer, Friedrich Scheiflinger, PhD, Michael Dockal

Blood (2015) 126 (23): 4663.

https://doi.org/10.1182/blood.V126.23.4663.4663

Published: 2015

Journal Articles

Lethality of Mouse Tissue Factor Pathway Inhibitor Gene Disruption Is Rescued By Transgenic Human Tissue Factor Pathway Inhibitor Knock in

Robert Pachlinger, Rudolf Hartmann, Andrea Kolm, Erwin Panholzer, Nadja Ullrich, Fabian Knofl, Maria Schuster, Werner Hoellriegl, DVM, Friedrich Scheiflinger, PhD, Michael Dockal

Blood (2015) 126 (23): 3495.

https://doi.org/10.1182/blood.V126.23.3495.3495

Published: 2015

Journal Articles

Development of BAX 826, a Polysialylated Full-Length rFVIII with Significantly Improved PK Properties

Peter L. Turecek, PhD, Juergen Siekmann, PhD, Artur Mitterer, PhD, Michael Graninger, PhD, Gerald Schrenk, PhD, Peter Matthiessen, PhD, Hanspeter Rottensteiner, PhD, Werner Hoellriegl, DVM, Marietta Putz, MS, Hans Peter Schwarz, MD, Friedrich Scheiflinger, PhD

Blood (2015) 126 (23): 3536.

https://doi.org/10.1182/blood.V126.23.3536.3536

Published: 2015

Journal Articles

Affinity of FVIII-specific antibodies reveals major differences between neutralizing and nonneutralizing antibodies in humans

Christoph J. Hofbauer, Shawn F. J. Whelan, Maria Hirschler, Peter Allacher, Frank M. Horling, John-Philip Lawo, Johannes Oldenburg, Andreas Tiede, Christoph Male, Jerzy Windyga, Andreas Greinacher, Paul N. Knöbl, Gerald Schrenk, Jadranka Koehn, Friedrich Scheiflinger, Birgit M. Reipert

Blood (2015) 125 (7): 1180–1188.

https://doi.org/10.1182/blood-2014-09-598268

Published: 2015

Includes: Supplemental data

Journal Articles

Tissue Factor Pathway Inhibitor Isoforms of Macro- and Microvascular Endothelial Cells

Michael Dockal, PhD, Robert Pachlinger, PhD, Angelina Baldin-Stoyanova, Fabian Knofl, Nadja Ullrich, Friedrich Scheiflinger, PhD

Blood (2014) 124 (21): 4167.

https://doi.org/10.1182/blood.V124.21.4167.4167

Published: 2014

Journal Articles

A TFPI Inhibitory Half Life Extended Fusion Peptide Proves Efficacy in FVIII Knock out Mice and Marmoset Monkeys

Michael Dockal, PhD, Rudolf Hartmann, PhD, Thomas Polakowski, PhD, Erwin Panholzer, Willibald Kammlander, Frank Osterkamp, PhD, Ulrich Reineke, PhD, Alexandra Schiviz, PhD, Werner Hoellriegl, PhD, Friedrich Scheiflinger, PhD

Blood (2014) 124 (21): 1469.

https://doi.org/10.1182/blood.V124.21.1469.1469

Published: 2014

NARROW

BAX 335 hemophilia B gene therapy clinical trial results: potential impact of CpG sequences on gene expression

AAV8-Specific Immune Adsorption Column: A Treatment Option for Patients with Pre-Existing Anti-AAV8 Neutralizing Antibodies

The Factor VIII Variant X5 Enhances Hemophilia a Gene Therapy Efficiency By Its Improved Secretion

Evaluation of the Human Factor IX Gene Therapy Vector TAK-748 in Hemophilia: Results from Non-Clinical Studies in Factor IX Knockout Mice and Rhesus Monkeys

Data Coming out of the Human Inhibitor PUP Study (HIPS) Reveal 4 Subgroups of Patients with Distinct Antibody Signatures

Bispecific Antibodies with Native Human IgG Structure As Mimetics for FVIII Activity

Elucidating the Excessive Pro-Coagulant Effect of a Sequence Identical Analogue to ACE910 in Combination with Bypassing Agents

Influence of Obesity on Factor IX Expression after Gene Therapy in a Mouse Model of Hemophilia B

A Bispecific Antibody Lacks Measurability in Routine Coagulation Assays and Comparability to Factor VIII

Synergistic Effects of a Procoagulant Bispecific Antibody and Rescue Therapies on Thrombin Generation- a Potential Safety Risk

ADAMTS13-mediated thrombolysis of t-PA–resistant occlusions in ischemic stroke in mice

Pharmacokinetics of BAX 826, a Polysialylated Full-Length rFVIII, in Hemophilia a Mice, Rats, and Cynomolgus Monkeys

Non-Proteolytic Regulation of the Multimerization of Von Willebrand Factor By ADAMTS13 Based on a Novel Cysteine Thiol Redundancy

Temperature-Dependent Irreversible Conformational Change of ADAMTS13 upon Metal Ion Chelation

The Application of Thrombin Generation Test Is Compromised By Antithrombin III Deficiency

Lethality of Mouse Tissue Factor Pathway Inhibitor Gene Disruption Is Rescued By Transgenic Human Tissue Factor Pathway Inhibitor Knock in

Development of BAX 826, a Polysialylated Full-Length rFVIII with Significantly Improved PK Properties

Affinity of FVIII-specific antibodies reveals major differences between neutralizing and nonneutralizing antibodies in humans

Tissue Factor Pathway Inhibitor Isoforms of Macro- and Microvascular Endothelial Cells

A TFPI Inhibitory Half Life Extended Fusion Peptide Proves Efficacy in FVIII Knock out Mice and Marmoset Monkeys

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American Society of Hematology

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