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Journal Articles

Results from the Completed Hgb-205 Trial of Lentiglobin for β-Thalassemia and Lentiglobin for Sickle Cell Disease Gene Therapy

Blood (2019) 134 (Supplement_1): 3358.

https://doi.org/10.1182/blood-2019-127393

Published: 2019

Journal Articles

Exploring the Drivers of Potential Clinical Benefit in Initial Patients Treated in the Hgb-206 Study of Lentiglobin for Sickle Cell Disease (SCD) Gene Therapy

Mark C. Walters, MD, John F. Tisdale, MD, Janet L. Kwiatkowski, MD MSCE, Lakshmanan Krishnamurti, MD, Markus Y. Mapara, MD PhD, Manfred Schmidt, PhD, Alexandra L. Miller, Francis J. Pierciey, Jr., Wenmei Huang, PhD MS, Jean-Antoine Ribeil, MD PhD, Julie Kanter, MD, Alexis A. Thompson, MD MPH

Blood (2019) 134 (Supplement_1): 2061.

https://doi.org/10.1182/blood-2019-128814

Published: 2019

Journal Articles

Resolution of Sickle Cell Disease Manifestations in Patients Treated with Lentiglobin Gene Therapy: Updated Results from the Phase 1/2 Hgb-206 Group C Study

Julie Kanter, MD, John F. Tisdale, MD, Markus Y. Mapara, MD PhD, Janet L. Kwiatkowski, MD MSCE, Lakshmanan Krishnamurti, MD, Manfred Schmidt, PhD, Alexandra L. Miller, Francis J. Pierciey, Jr., Wenmei Huang, PhD MS, Jean-Antoine Ribeil, MD PhD, Alexis A. Thompson, MD MPH, Mark C. Walters, MD

Blood (2019) 134 (Supplement_1): 990.

https://doi.org/10.1182/blood-2019-128894

Published: 2019

Journal Articles

GDF15 and Erythroferrone Mark Erythropoietic Response to ACE-011 (Sotatercept) in Thalassemia

Blood (2018) 132 (Supplement 1): 3633.

https://doi.org/10.1182/blood-2018-99-111770

Published: 2018

Journal Articles

Outcomes for Initial Patient Cohorts with up to 33 Months of Follow-up in the Hgb-206 Phase 1 Trial

Julie Kanter, MD, John F. Tisdale, MD, Janet L. Kwiatkowski, MD, Lakshmanan Krishnamurti, MD, Markus Y. Mapara, MD PhD, Manfred Schmidt, PhD, Alexandra L. Miller, Francis J. Pierciey, Jr., Weiliang Shi, PhD, Jean-Antoine Ribeil, MD PhD, Mark C. Walters, MD, Alexis A. Thompson, MD MPH

Blood (2018) 132 (Supplement 1): 1080.

https://doi.org/10.1182/blood-2018-99-113477

Published: 2018

Journal Articles

Current Results of Lentiglobin Gene Therapy in Patients with Severe Sickle Cell Disease Treated Under a Refined Protocol in the Phase 1 Hgb-206 Study

John F. Tisdale, MD, Julie Kanter, MD, Markus Y. Mapara, MD PhD, Janet L. Kwiatkowski, MD, Lakshmanan Krishnamurti, MD, Manfred Schmidt, PhD, Alexandra L. Miller, Francis J. Pierciey, Jr., Weiliang Shi, PhD, Jean-Antoine Ribeil, MD PhD, Mohammed Asmal, MD PhD, Alexis A. Thompson, MD MPH, Mark C. Walters, MD

Blood (2018) 132 (Supplement 1): 1026.

https://doi.org/10.1182/blood-2018-99-113480

Published: 2018

Journal Articles

Analysis of RBC Properties in Patients with SCD Treated with Lentiglobin Gene Therapy

Nicolas Hebert, Elisa Magrin, PhD, Annarita Miccio, PhD, Kiger Laurent, PhD, Nguyen-Peyre Kim-Anh, PhD, Laure Joseph, MD, Alessandra Magnani, MD PhD, Chloé Couzin, MD, Wassim El Nemer, PhD, France Pirenne, MD PhD, Olivier Negre, PhD, Jean-Antoine Ribeil, MD PhD, Isabelle André-Schmutz, PhD, Pablo Bartolucci, MD PhD, Marina Cavazzana, MD PhD

Blood (2018) 132 (Supplement 1): 2195.

https://doi.org/10.1182/blood-2018-99-115973

Published: 2018

Journal Articles

Prospective Controlled Trial on Endurance Exercise Training in Adult Sickle Cell Disease Patients

Barnabas Gellen, Laurent Messonnier, Angele Merlet, Etienne Audureau, Thomas Rupp, Sandrine Peyrot, Cyril Martin, PhD, Jean-Antoine Ribeil, MD PhD, jean Benoit Arlet, Frédéric Galactéros, MDPhD, Leonard Feasson, Pablo Bartolucci, MD PhD

Blood (2017) 130 (Supplement 1): 991.

https://doi.org/10.1182/blood.V130.Suppl_1.991.991

Published: 2017

Journal Articles

Hemoglobin S Screening Using the « Sickle Scan » - Biomedomics System. the Necker-Enfants Malades Hospital Experience

Louis MINE, Thao Nguyen-Khoa, Birch Allaf, Jean-Antoine Ribeil, MD PhD, Christelle Remus, Valérie Gauthereau, Sarah Enouz, Jason S. KIM, Xiaoxi Yang, Eliane Gluckman, MD PhD, Arnold Munnich, Marina Cavazzana, MD PhD, Robert Girot, MD

Blood (2016) 128 (22): 1308.

https://doi.org/10.1182/blood.V128.22.1308.1308

Published: 2016

Journal Articles

Hydroxycarbamide (HU), Sickle Cell Disease and Pregnancy, a Multicentric Retrospective Study

Laure Joseph, MD, Anoosha Habibi, MD, Giovanna Cannas, MD, Stephanie Ngo, MD, Sylvain Le Jeune, MD, Ersi Voskaridou, MD, Elena Foïs, MD, Caroline Makowski, MD, Nathalie Lemonne, MD, Narcisse Elenga, MD, Yves Ville, MD PhD, Alexandra Benachi, MDPhD, Robert Girot, MD, Marina Cavazzana, MD PhD, Jean-Antoine Ribeil, MD PhD

Blood (2016) 128 (22): 1309.

https://doi.org/10.1182/blood.V128.22.1309.1309

Published: 2016

Journal Articles

Update from the Hgb-205 Phase 1/2 Clinical Study of Lentiglobin Gene Therapy: Sustained Clinical Benefit in Severe Hemoglobinopathies

Blood (2016) 128 (22): 2311.

https://doi.org/10.1182/blood.V128.22.2311.2311

Published: 2016

Journal Articles

Arterio-Venous Fistula Is an Applicable Vascular Access for Erythracytapheresis in Patients with Sickle Cell Disease

Blood (2016) 128 (22): 4856.

https://doi.org/10.1182/blood.V128.22.4856.4856

Published: 2016

Journal Articles

Comparison of the Safety Profile of Sickle Cell Disease Patients Treated with Hydroxycarbamide in Off-Label Versus in-Label Prescriptions in the Escort-HU Non Interventional, Prospective, Observational Open-Label Cohort Study

Ersi Voskaridou, MD, Stephan Lobitz, MD, Uwe Kordes, MD MRCP, Regine Grosse, MD, Valentine Brousse, MD, Malika Benkerrou, MD, Mariane De Montalembert, MD PhD, Jean-Antoine Ribeil, MD PhD, Frederic Galacteros, MD PhD

Blood (2016) 128 (22): 2497.

https://doi.org/10.1182/blood.V128.22.2497.2497

Published: 2016

Journal Articles

Assessment of Cardiac Iron Overload in Chonically Transfused Patients with Thalassemia, Sickle Cell Anemia, and Myelodysplastic Syndromes

Mariane De Montalembert, MD PhD, Jean-Antoine Ribeil, MD PhD, Valentine Brousse, Agnes Guerci-Bresler, MD, Aspasia Stamatoullas, MD, Jean Pierre Vannier, MD, Agnes Lahary, MD, Mohamed Touati, MD, Krimo Bouabdallah, MD, Marina Cavazzana, MD PhD, Emmanuelle Chauzit, MD, Amandine Baptiste, MD, Thibaud Lefebvre, MD, Herve Puy, MD PhD, Caroline Elie, MD PhD, Zoubida Karim, PhD, Olivier Ernst, MD PhD, Christian Rose

Blood (2015) 126 (23): 2151.

https://doi.org/10.1182/blood.V126.23.2151.2151

Published: 2015

Journal Articles

Outcomes of Gene Therapy for Severe Sickle Disease and Beta-Thalassemia Major Via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex Vivo with a Lentiviral Beta AT87Q-Globin Vector

Blood (2015) 126 (23): 202.

https://doi.org/10.1182/blood.V126.23.202.202

Published: 2015

Journal Articles

XPO1 (Exportin-1) Is a Major Regulator of Human Erythroid Differentiation. Potential Clinical Applications to Decrease Ineffective Erythropoiesis of Beta-Thalassemia

Flavia Guillem, PhD, Michael Dussiot, PhD, Sebastien Causse, PhD, Guillaume Marcion, Emilie-Fleur Gautier, PhD, Julien Rossignol, MD, Jean benoit Arlet, MD PhD, Mathilde Lamarque, MD, Patrick Mayeux, PhD, Frederique Verdier, PhD, Xiuli An, MD PhD, Jean-Antoine Ribeil, MD PhD, Carmen Garrido, PhD, Genevieve Courtois, PhD, Narla Mohandas, D.Sc., Olivier Hermine

Blood (2015) 126 (23): 2368.

https://doi.org/10.1182/blood.V126.23.2368.2368

Published: 2015

Journal Articles

Study Hgb-205: Outcomes of Gene Therapy for Hemoglobinopathies Via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex Vivo with a Lentiviral β Α-T87Q -Globin Vector (LentiGlobin ® BB305 Drug Product)

Blood (2014) 124 (21): 4797.

https://doi.org/10.1182/blood.V124.21.4797.4797

Published: 2014

Journal Articles

Implementation of a European Cohort to Follow Sickle Cell Children and Adults Treated with Hydroxycarbamide

Mariane De Montalembert, MD, Frédéric Galacteros, Jean Antoine Ribeil, MD PhD, Uwe Kordes, MD MRCP, Jean Benoit Arlet, MD PhD, M Dimopoulou, MD, V Komninaka, MD, Pablo Bartolucci, MD PhD, Anoosha Habibi, Valentine Brousse, MD, Justine Gellen, MD, Stephen Lobitz, MD, Ersi Voskaridou, MD

Blood (2014) 124 (21): 564.

https://doi.org/10.1182/blood.V124.21.564.564

Published: 2014

Journal Articles

A Phase 2a, Open-Label, Dose-Finding Study To Determine The Safety and Tolerability Of Sotatercept (ACE-011) In Adults With Beta (β)-Thalassemia: Interim Results

Maria-Domenica Cappellini, MD, John Porter, MD, Raffaella Origa, MD, Gian Luca Forni, MD, Adberrahmane Laadem, MD, Frédéric Galacteros, MD PhD, Dimana Miteva, PhD, Victoria Sung, PhD, Rajesh Chopra, MD, Jean-Benoît Arlet, MD, PhD, Jean-Antoine Ribeil, MD, PhD, Kenneth Klesczewski, PhD, Kenneth Attie, MD, Maciej Garbowski, MD, Olivier Hermine, MD, PhD

Blood (2013) 122 (21): 3448.

https://doi.org/10.1182/blood.V122.21.3448.3448

Published: 2013

Journal Articles

Modulation of Activin Signaling by RAP-011 (ActRIIA-IgG1) Improve Anemia, Increases Hemoglobin Levels and Corrects Ineffective Erythropoiesis in β-Thalassemia

Michael Dussiot, Thiago Maciel, PhD, Aurelie Fricot, Joel Veiga, Etienne Paubelle, Emmanuel Payen, Yves Beuzard, MD, Jean-Antoine Ribeil, MD, PhD, Jean-Benoît Arlet, MD, Genevieve Courtois, PhD, Tom O Daniel, MD, PhD, Rajesh Chopra, MD, PhD, Victoria Sung, Ph.D., Olivier Hermine, MD, PhD, Ivan Cruz Moura, PhD

Blood (2012) 120 (21): 247.

https://doi.org/10.1182/blood.V120.21.247.247

Published: 2012

NARROW

Results from the Completed Hgb-205 Trial of Lentiglobin for β-Thalassemia and Lentiglobin for Sickle Cell Disease Gene Therapy

Exploring the Drivers of Potential Clinical Benefit in Initial Patients Treated in the Hgb-206 Study of Lentiglobin for Sickle Cell Disease (SCD) Gene Therapy

Resolution of Sickle Cell Disease Manifestations in Patients Treated with Lentiglobin Gene Therapy: Updated Results from the Phase 1/2 Hgb-206 Group C Study

GDF15 and Erythroferrone Mark Erythropoietic Response to ACE-011 (Sotatercept) in Thalassemia

Outcomes for Initial Patient Cohorts with up to 33 Months of Follow-up in the Hgb-206 Phase 1 Trial

Current Results of Lentiglobin Gene Therapy in Patients with Severe Sickle Cell Disease Treated Under a Refined Protocol in the Phase 1 Hgb-206 Study

Analysis of RBC Properties in Patients with SCD Treated with Lentiglobin Gene Therapy

Prospective Controlled Trial on Endurance Exercise Training in Adult Sickle Cell Disease Patients

Hemoglobin S Screening Using the « Sickle Scan » - Biomedomics System. the Necker-Enfants Malades Hospital Experience

Hydroxycarbamide (HU), Sickle Cell Disease and Pregnancy, a Multicentric Retrospective Study

Update from the Hgb-205 Phase 1/2 Clinical Study of Lentiglobin Gene Therapy: Sustained Clinical Benefit in Severe Hemoglobinopathies

Arterio-Venous Fistula Is an Applicable Vascular Access for Erythracytapheresis in Patients with Sickle Cell Disease

Comparison of the Safety Profile of Sickle Cell Disease Patients Treated with Hydroxycarbamide in Off-Label Versus in-Label Prescriptions in the Escort-HU Non Interventional, Prospective, Observational Open-Label Cohort Study

Assessment of Cardiac Iron Overload in Chonically Transfused Patients with Thalassemia, Sickle Cell Anemia, and Myelodysplastic Syndromes

Outcomes of Gene Therapy for Severe Sickle Disease and Beta-Thalassemia Major Via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex Vivo with a Lentiviral Beta AT87Q-Globin Vector

XPO1 (Exportin-1) Is a Major Regulator of Human Erythroid Differentiation. Potential Clinical Applications to Decrease Ineffective Erythropoiesis of Beta-Thalassemia

Study Hgb-205: Outcomes of Gene Therapy for Hemoglobinopathies Via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex Vivo with a Lentiviral β Α-T87Q -Globin Vector (LentiGlobin ® BB305 Drug Product)

Implementation of a European Cohort to Follow Sickle Cell Children and Adults Treated with Hydroxycarbamide

A Phase 2a, Open-Label, Dose-Finding Study To Determine The Safety and Tolerability Of Sotatercept (ACE-011) In Adults With Beta (β)-Thalassemia: Interim Results

Modulation of Activin Signaling by RAP-011 (ActRIIA-IgG1) Improve Anemia, Increases Hemoglobin Levels and Corrects Ineffective Erythropoiesis in β-Thalassemia

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American Society of Hematology

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American Society of Hematology

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