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Journal Articles

Reni-Cel, an Investigational AsCas12a Gene-Edited Cell Medicine, Led to Sustained Hemoglobin Normalization and Increased Fetal Hemoglobin in Patients with Severe Sickle Cell Disease Treated in the RUBY Trial

Rabi Hanna, Haydar Frangoul, Christopher McKinney, Luis Pineiro, Markus Mapara, Jignesh Dalal, Hemalatha Rangarajan, Harold Atkins, Kai-Hsin Chang, Baisong Mei, Olubunmi Afonja, Mark C. Walters

Blood (2024) 144 (Supplement 1): 4955.

https://doi.org/10.1182/blood-2024-210343

Published: 2024

Journal Articles

Reni-Cel, an Investigational AsCas12a Gene-Edited Cell Medicine, Led to Successful Engraftment, Increased Hemoglobin, and Reduced Transfusion Dependence in Patients with Transfusion-Dependent Beta-Thalassemia Treated in the Edithal Trial

Haydar Frangoul, Rabi Hanna, Mark C. Walters, Kai-Hsin Chang, Michael Jaskolka, Keunpyo Kim, Baisong Mei, Olubunmi Afonja, Alexis Thompson

Blood (2024) 144 (Supplement 1): 7456.

https://doi.org/10.1182/blood-2024-208623

Published: 2024

Journal Articles

Fitusiran prophylaxis in people with hemophilia A or B who switched from prior BPA/CFC prophylaxis: the ATLAS-PPX trial

Blood (2024) 143 (22): 2256–2269.

https://doi.org/10.1182/blood.2023021864

Published: 2024

Includes: Supplemental data

Journal Articles

AsCas12a Gene Editing of HBG1/2 Promoters with EDIT-301 Results in Rapid and Sustained Normalization of Hemoglobin and Increased Fetal Hemoglobin in Patients with Severe Sickle Cell Disease and Transfusion-Dependent Beta-Thalassemia

Rabi Hanna, Haydar Frangoul, Christopher McKinney, Luis Pineiro, Markus Mapara, Jignesh Dalal, Kai-Hsin Chang, Michael Jaskolka, Keunpyo Kim, Daphne L. Farrington, Maha Wally, Baisong Mei, Adebayo Lawal, Olubunmi O. Afonja, Mark C. Walters

Blood (2023) 142 (Supplement 1): 4996.

https://doi.org/10.1182/blood-2023-187397

Published: 2023

Journal Articles

Fitusiran Prophylaxis Improves Health-Related Quality of Life in People with Hemophilia a or B, with or without Inhibitors: Results of ATLAS-PPX Study

Gili Kenet, Beatrice Nolan, Tadashi Matsushita, Guy Young, Ting Quan, Viridiana Cano, Christian Sussebach, Shauna Andersson, Baisong Mei, Marion Afonso, Kaan Kavakli

Blood (2022) 140 (Supplement 1): 7977–7978.

https://doi.org/10.1182/blood-2022-167169

Published: 2022

Journal Articles

Fitusiran, an Investigational siRNA Therapeutic Targeting Antithrombin for the Treatment of Hemophilia: First Results from a Phase 3 Study to Evaluate Efficacy and Safety in People with Hemophilia a or B without Inhibitors (ATLAS-A/B)

Alok Srivastava, Savita Rangarajan, Kaan Kavakli, Robert Klamroth, Gili Kenet, Liane Khoo, Chur-Woo You, Weiqun Xu, Niel Malan, Laurent Frenzel, Catherine N. Bagot, Oleksandra Stasyshyn, Chia-Yau Chang, Stacey Poloskey, Shauna Andersson, Zhiying Qiu, Baisong Mei, Steven W. Pipe

Blood (2021) 138 (Supplement 2): LBA-3.

https://doi.org/10.1182/blood-2021-155018

Published: 2021

Journal Articles

Efficacy and Safety of Fitusiran Prophylaxis, an siRNA Therapeutic, in a Multicenter Phase 3 Study (ATLAS-INH) in People with Hemophilia A or B, with Inhibitors (PwHI)

Guy Young, Alok Srivastava, Kaan Kavakli, Cecil Ross, Jameela Sathar, Huyen Tran, Runhui Wu, Jing Sun, Stacey Poloskey, Zhiying Qui, Salim Kichou, Shauna R. Andersson, Baisong Mei, Savita Rangarajan

Blood (2021) 138 (Supplement 1): 4.

https://doi.org/10.1182/blood-2021-150273

Published: 2021

Journal Articles

Long-Term Durability, Safety and Efficacy of Fitusiran Prophylaxis in People with Hemophilia a or B, with or without Inhibitors - Results from the Phase II Study

Steven W. Pipe, MD, John Pasi, MB, ChB, PhD FRCP, FRCPath, FRCPCH, Toshko Lissitchkov, Margaret V. Ragni, MDMPH, Claude Négrier, Qifeng Yu, Stacey Poloskey, Baisong Mei, Shauna R. Andersson

Blood (2020) 136 (Supplement 1): 3–4.

https://doi.org/10.1182/blood-2020-136501

Published: 2020

Journal Articles

Fitusiran Treatment Impacts on Health-Related Quality of Life in Subjects with Hemophilia A and B with Inhibitors Assessed with the Haemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL)

Sylvia Von Mackensen, Pratima Chowdary, Sarah Mangles, Qifeng Yu, Baisong Mei, Shauna R. Andersson, Pronabesh Dasmahapatra, MBBS, MPH

Blood (2020) 136 (Supplement 1): 23–24.

https://doi.org/10.1182/blood-2020-142266

Published: 2020

Journal Articles

Longitudinal Assessment of Thrombin Generation in Patients with Hemophilia Receiving Fitusiran Prophylaxis: Phase II Study Results

Claude Négrier, Margaret V. Ragni, MDMPH, John Pasi, MB, ChB, PhD FRCP, FRCPath, FRCPCH, Steven W. Pipe, MD, Gili Kenet, Savita Rangarajan, MBBS,MDFRCP,FRCPath, Salim Kichou, Baisong Mei, Shauna R. Andersson

Blood (2020) 136 (Supplement 1): 36–37.

https://doi.org/10.1182/blood-2020-136536

Published: 2020

Journal Articles

BIVV001, a new class of factor VIII replacement for hemophilia A that is independent of von Willebrand factor in primates and mice

Blood (2020) 135 (17): 1484–1496.

https://doi.org/10.1182/blood.2019001292

Published: 2020

Includes: Supplemental data

Journal Articles

Fitusiran, an RNAi Therapeutic Targeting Antithrombin to Restore Hemostatic Balance in Patients with Hemophilia a or B with or without Inhibitors: Management of Acute Bleeding Events

Steven Pipe, MD, Margaret V. Ragni, MD MPH, Claude Négrier, MD PhD, Qifeng Yu, PhD, Naghmana Bajwa, MD, John Caminis, MD, Baisong Mei, MD PhD, Shauna R. Andersson, MD PhD

Blood (2019) 134 (Supplement_1): 1138.

https://doi.org/10.1182/blood-2019-124108

Published: 2019

Journal Articles

Perioperative Management of Hemostasis with Recombinant FIX Fc Fusion Protein in Subjects Undergoing Surgery in the B-YOND Study

Margareth Castro Ozelo, MD PhD, Johnny Mahlangu, Godfrey Chan, Margareta Holmstrom, Nicolas Novitzky, David J Perry, Huixing Yuan, Lynda M Cristiano, Baisong Mei

Blood (2015) 126 (23): 1083.

https://doi.org/10.1182/blood.V126.23.1083.1083

Published: 2015

Journal Articles

Extended-Interval Prophylaxis with rFIXFc in Adults and Adolescents with Hemophilia B: Interim Results of the B-YOND Extension Study

Johnny Mahlangu, Amy D Shapiro, MD, K John Pasi, Margaret V Ragni, Margareth Castro Ozelo, MD PhD, Johannes Oldenburg, Tadashi Matsushita, MD PhD, Ross I Baker, MD, Huixing Yuan, Alejandra Ramirez-Santiago, Glenn F Pierce, Geoffrey Allen, Baisong Mei

Blood (2015) 126 (23): 1088.

https://doi.org/10.1182/blood.V126.23.1088.1088

Published: 2015

Journal Articles

Clinical Outcomes in Children with Hemophilia B Treated Long Term with rFIXFc: Interim Results of the B-YOND Extension Study

Roshni Kulkarni, Beatrice Nolan, Carolyn M. Bennett, Kathelijn Fischer, David J Perry, Christopher Barnes, Huixing Yuan, Alejandra Ramirez-Santiago, Glenn F Pierce, Baisong Mei

Blood (2015) 126 (23): 1093.

https://doi.org/10.1182/blood.V126.23.1093.1093

Published: 2015

Journal Articles

Analysis of Target Joint Bleeding with Prophylactic Use of Recombinant Factor IX Fc Fusion Protein in Patients with Severe Hemophilia B

Amy D Shapiro, MD, David J Perry, Ross I Baker, MD, Elisa Tsao, Baisong Mei

Blood (2015) 126 (23): 2294.

https://doi.org/10.1182/blood.V126.23.2294.2294

Published: 2015

Journal Articles

Low Bleeding Rates with Increase or Maintenance of Physical Activity in Patients Treated with Recombinant Factor IX Fc Fusion Protein (rFIXFc) in the B-LONG and Kids B-LONG Studies

Roshni Kulkarni, Amy D Shapiro, MD, Jerzy Windyga, Margaret V Ragni, K John Pasi, Margareth Castro Ozelo, MD PhD, Elisa Tsao, Baisong Mei

Blood (2015) 126 (23): 2307.

https://doi.org/10.1182/blood.V126.23.2307.2307

Published: 2015

Journal Articles

Predicting FIX Activity in Prophylaxis Patients Using Recombinant FIX Fc Fusion Protein for Treatment of Bleeding Episodes

Jerry S Powell, MD, Margareth Ozelo, MD PhD, Margaret V. Ragni, MD MPH, John Pasi, MBChB, PhD FRCP, FRCPath, David J. Perry, MD PhD FRCP, FRCPath, Ying Zhu, MPH, Baisong Mei, MD PhD, Glenn F Pierce, MD PhD, Lian Li, PhD, Brian Robinson, MD

Blood (2014) 124 (21): 2842.

https://doi.org/10.1182/blood.V124.21.2842.2842

Published: 2014

Journal Articles

Rational design of a fully active, long-acting PEGylated factor VIII for hemophilia A treatment

Baisong Mei, Clark Pan, Haiyan Jiang, Hendri Tjandra, Jonathan Strauss, Yaoqi Chen, Tongyao Liu, Xin Zhang, Joanne Severs, Jim Newgren, Jianmin Chen, Jian-Ming Gu, Babu Subramanyam, Michael A. Fournel, Glenn F. Pierce, John E. Murphy

Blood (2010) 116 (2): 270–279.

https://doi.org/10.1182/blood-2009-11-254755

Published: 2010

Journal Articles

Site-Specific PEGylation of Factor VIII (PEG-FVIII) Preserves Full Clotting Activity and Extends Therapeutic Efficacy in HemophiliaA Dogs

Tongyao Liu, David Lillicrap, Xin Zhang, Andrea Labelle, Sandra Powell, Baisong Mei, John E. Murphy, Glenn F. Pierce, Haiyan Jiang

Blood (2008) 112 (11): 511.

https://doi.org/10.1182/blood.V112.11.511.511

Published: 2008

NARROW

Reni-Cel, an Investigational AsCas12a Gene-Edited Cell Medicine, Led to Sustained Hemoglobin Normalization and Increased Fetal Hemoglobin in Patients with Severe Sickle Cell Disease Treated in the RUBY Trial

Reni-Cel, an Investigational AsCas12a Gene-Edited Cell Medicine, Led to Successful Engraftment, Increased Hemoglobin, and Reduced Transfusion Dependence in Patients with Transfusion-Dependent Beta-Thalassemia Treated in the Edithal Trial

Fitusiran prophylaxis in people with hemophilia A or B who switched from prior BPA/CFC prophylaxis: the ATLAS-PPX trial

AsCas12a Gene Editing of HBG1/2 Promoters with EDIT-301 Results in Rapid and Sustained Normalization of Hemoglobin and Increased Fetal Hemoglobin in Patients with Severe Sickle Cell Disease and Transfusion-Dependent Beta-Thalassemia

Fitusiran Prophylaxis Improves Health-Related Quality of Life in People with Hemophilia a or B, with or without Inhibitors: Results of ATLAS-PPX Study

Fitusiran, an Investigational siRNA Therapeutic Targeting Antithrombin for the Treatment of Hemophilia: First Results from a Phase 3 Study to Evaluate Efficacy and Safety in People with Hemophilia a or B without Inhibitors (ATLAS-A/B)

Efficacy and Safety of Fitusiran Prophylaxis, an siRNA Therapeutic, in a Multicenter Phase 3 Study (ATLAS-INH) in People with Hemophilia A or B, with Inhibitors (PwHI)

Long-Term Durability, Safety and Efficacy of Fitusiran Prophylaxis in People with Hemophilia a or B, with or without Inhibitors - Results from the Phase II Study

Fitusiran Treatment Impacts on Health-Related Quality of Life in Subjects with Hemophilia A and B with Inhibitors Assessed with the Haemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL)

Longitudinal Assessment of Thrombin Generation in Patients with Hemophilia Receiving Fitusiran Prophylaxis: Phase II Study Results

BIVV001, a new class of factor VIII replacement for hemophilia A that is independent of von Willebrand factor in primates and mice

Fitusiran, an RNAi Therapeutic Targeting Antithrombin to Restore Hemostatic Balance in Patients with Hemophilia a or B with or without Inhibitors: Management of Acute Bleeding Events

Perioperative Management of Hemostasis with Recombinant FIX Fc Fusion Protein in Subjects Undergoing Surgery in the B-YOND Study

Extended-Interval Prophylaxis with rFIXFc in Adults and Adolescents with Hemophilia B: Interim Results of the B-YOND Extension Study

Clinical Outcomes in Children with Hemophilia B Treated Long Term with rFIXFc: Interim Results of the B-YOND Extension Study

Analysis of Target Joint Bleeding with Prophylactic Use of Recombinant Factor IX Fc Fusion Protein in Patients with Severe Hemophilia B

Low Bleeding Rates with Increase or Maintenance of Physical Activity in Patients Treated with Recombinant Factor IX Fc Fusion Protein (rFIXFc) in the B-LONG and Kids B-LONG Studies

Predicting FIX Activity in Prophylaxis Patients Using Recombinant FIX Fc Fusion Protein for Treatment of Bleeding Episodes

Rational design of a fully active, long-acting PEGylated factor VIII for hemophilia A treatment

Site-Specific PEGylation of Factor VIII (PEG-FVIII) Preserves Full Clotting Activity and Extends Therapeutic Efficacy in HemophiliaA Dogs

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American Society of Hematology

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American Society of Hematology

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