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Journal Articles

Improvement in Red Blood Cell Physiology in Children with Sickle Cell Anemia Receiving Voxelotor

Satheesh Chonat, MD, Earl Fields, Hannah Baratz, Amanda Watt, Mira Pochron, PhD, Sandy Dixon, Margaret Tonda, PharmD, Joshua Lehrer-Graiwer, MD MPhil, FACC, Clark Brown, MD PhD, David R. Archer, PhD

Blood (2019) 134 (Supplement_1): 2281.

https://doi.org/10.1182/blood-2019-129351

Published: 2019

Journal Articles

Peri-Procedural Management and Outcomes of Patients with Hemophilia on Emicizumab Prophylaxis

Isabella McCary, BS, Christine Guelcher, PPCNP-BC, Jan Kuhn, BA, BSN, MSN, Regina Butler, RN-BC, Michael F. Guerrera, MD, Gita Massey, Leslie J. Raffini, MD

Blood (2019) 134 (Supplement_1): 2396.

https://doi.org/10.1182/blood-2019-126944

Published: 2019

Journal Articles

Pozelimab, a Human Antibody Against Complement Factor C5, Demonstrates Robust Inhibition of Alternative Complement Activity Both in Normal Human Serum and in Phase I Normal Healthy Volunteers

Kishor Devalaraja-Narashimha, PhD DVM, Yan G Ni, PhD, Cong Huang, Ming-Dauh Wang, PhD, Umesh Chaudhari, MD, Srinivasa Prasad, PhD, Olivier Harari, MD PhD, Andrew J Rankin, PhD, Lori Morton, PhD, Jonathan Weyne, MD

Blood (2019) 134 (Supplement_1): 2278.

https://doi.org/10.1182/blood-2019-129865

Published: 2019

Journal Articles

Phase 1 Study of Bisthianostat, an Orally Efficacious Pan-HDAC Inhibitor: Part Results of Safety, Pharmacokinetics and Efficacy in Patients with Relapsed or Refractory Multiple Myeloma

Hong-Hui Huang, PhD MD, Jian Hou, PhD MD, Yang-Ming Zhang, PhD, Yu-Bo Zhou, PhD, Li Jia, PhD, Fa-Jun Nan, PhD

Blood (2019) 134 (Supplement_1): 5591.

https://doi.org/10.1182/blood-2019-129683

Published: 2019

Journal Articles

Preclinical Development of an Anti-CD38 Antibody-Drug Conjugate for Treatment of Hematological Malignancies

Lingna Li, PhD, Wenyong Tong, PhD, Megan Lau, BS, Katherine Fells, PhD, Tong Zhu, PhD, Yingqing Sun, PhD, Ernest Kovacs, PhD, Alisher Khasanov, PhD, Zheng Yan, PhD, Dalun Deng, PhD, Ken Takeshita, MD, Gunnar F. Kaufmann, PhD, Henry Ji, PhD, Hui Li, PhD, Hong Zhang, PhD

Blood (2019) 134 (Supplement_1): 5621.

https://doi.org/10.1182/blood-2019-132062

Published: 2019

Journal Articles

A Phase I Study of Single-Agent Inotuzumab Ozogamicin in Pediatric CD22-Positive Relapsed/Refractory Acute Lymphoblastic Leukemia: Preliminary Results of the ITCC-059 Study

Blood (2019) 134 (Supplement_1): 2629.

https://doi.org/10.1182/blood-2019-122411

Published: 2019

Journal Articles

Preliminary Results from a Phase I Study of SHC014748M in Patients with Relapsed or Refractory Indolent B-Cell Lymphomas

Wei Xu, MD PhD, Jie Jin, MD PhD, Keshu Zhou, MD, Li Wang, MD, Huayuan Zhu, Jiejing Qian, Jianying Huang, Fuling Zhou, MD, Xiaohong Xu, Zhiqiang Wang, Wei Guo, Jianyong Li, MD

Blood (2019) 134 (Supplement_1): 4000.

https://doi.org/10.1182/blood-2019-126707

Published: 2019

Journal Articles

TNB-486, a Novel Fully Human Bispecific CD19 x CD3 Antibody That Kills CD19-Positive Tumor Cells with Minimal Cytokine Secretion

Harbani Malik, PhD, Ben Buelow, MD PhD, Udaya Rangaswamy, PhD, Aarti Balasubramani, Andrew Boudreau, Kevin Dang, Laura Davison, PhD, Shelley Force Aldred, Katherine Marie Harris, MD, Suhasini Iyer, PhD, Brett Jorgensen, PhD, Duy Pham, MBA, Kirthana Prabhakar, Ute Schellenberger, PhD, Harshad Ugamraj, Nathan Trinklein, PhD, Wim Van Schooten, PhD

Blood (2019) 134 (Supplement_1): 4070.

https://doi.org/10.1182/blood-2019-123226

Published: 2019

Journal Articles

Third Party Type 2 Innate Lymphoid Cells Treat and Prevent GI Tract Acute GvHD

Danny W. Bruce, PhD, Oleg Kolupaev, PhD, Hemamalini Bommiasamy, PhD, Heather Stefanski, MD PhD, Bruce R. Blazar, MD, James M. Coghill, Jr., MD, Jonathan S Serody, MD

Blood (2019) 134 (Supplement_1): 3205.

https://doi.org/10.1182/blood-2019-126848

Published: 2019

Journal Articles

Phase 1 Single (SAD) and Multiple Ascending Dose (MAD) Studies of the Safety, Tolerability, Pharmacokinetics (PK) and Pharmacodynamics (PD) of FT-4202, an Allosteric Activator of Pyruvate Kinase-R, in Healthy and Sickle Cell Disease Subjects

Theodosia A. Kalfa, MD PhD, Frans A Kuypers, PhD, Marilyn J. Telen, MD, Punam Malik, MD, Diamantis G. Konstantinidis, PhD BSc, Jeremy H. Estepp, MD, Hyon J. Kim, MD PhD, Santosh L. Saraf, MD, Lindsey Wilson, Maria D Ribadeneira, PhD, Sanjeev Forsyth, Patricia Schroeder, Adam Drake, Olga Polyanskaya, Patrick Kelly, MD, Lukasz Biernat, MD

Blood (2019) 134 (Supplement_1): 616.

https://doi.org/10.1182/blood-2019-121889

Published: 2019

Journal Articles

Improved Outcomes for Patients Receiving High-Doses of IL-21 Ex Vivo Expanded NK Cells after Haploidentical Transplantation (haploSCT): Long-Term Follow-up of a Phase 1/2 Clinical Trial with Comparison to CIBMTR Controls

Stefan O. Ciurea, MD, Roland Bassett, Doris Soebbing, Gabriela Rondon, MD, Kai Cao, MS, MD, Amanda L. Olson, MD, Qaiser Bashir, MBBS, Elizabeth J. Shpall, MD, Steven Devine, Marcelo C. Pasquini, MD, Andrew St. Martin, MS, Dean Anthony Lee, MD PhD, Richard E Champlin

Blood (2019) 134 (Supplement_1): 700.

https://doi.org/10.1182/blood-2019-123936

Published: 2019

Journal Articles

Hyper-Sialylated IgG M254, an Innovative Therapeutic Candidate, Evaluated in Healthy Volunteers and in Patients with Immune Thrombocytopenia Purpura: Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics

Santiago Arroyo, MD PhD, Renger G Tiessen, MD, William S Denney, PhD, Jim Jin, MS, Mattheus Paulus (Thijs) van Iersel, MD, Heidi Zeitz, MD CCRA, Anthony M Manning, PhD, Martin R. Schipperus, MD PhD, James B. Bussel, MD

Blood (2019) 134 (Supplement_1): 1090.

https://doi.org/10.1182/blood-2019-125993

Published: 2019

Journal Articles

Dreamm-5: Platform Trial Evaluating Belantamab Mafodotin (a BCMA-directed Immuno-conjugate) in Combination with Novel Agents in Relapsed or Refractory Multiple Myeloma (RRMM)

Paul G. Richardson, MD, Swethajit Biswas, Beata Holkova, Nicola Jackson, Thelma Netherway, Weichao Bao, Geraldine Ferron-Brady, Anne Yeakey, Chris Shelton, Rocio Montes De Oca, PhD, Chris Ahlers, Sonia Franco, Marc Ballas, Elaine Paul, Katarina Luptakova, Ira Gupta, Joanna Opalinska

Blood (2019) 134 (Supplement_1): 1857.

https://doi.org/10.1182/blood-2019-129879

Published: 2019

Journal Articles

Phase 1, Open-Label, Dose Escalation Study of I-131-CLR1404 (CLR 131) in Patients with Relapsed or Refractory Multiple Myeloma

Jarrod Longcor, Kate Oliver, BS

Blood (2019) 134 (Supplement_1): 1864.

https://doi.org/10.1182/blood-2019-131806

Published: 2019

Journal Articles

Phase II Multicenter, Not Comparative, Study of Multiple Doses of NEPA (netupitant + palonosetron) in Preventing Chemotherapy-Induced Nausea and Vomiting (CINV) in Non-Hodgkin Lymphoma Patients Eligible for Autologous Hematopoietic Stem Cell Transplantation Receiving Multiple Days / High Dose Chemotherapy Regimens

Nicola Di Renzo, MD, Maurizio Musso, MD, Rosanna Scimè, MD, Giuseppe Milone, MD PhD, Tommasina Perrone, MD, Clara De Risi, MD, Domenico Pastore, MD PhD, Attilio Guarini, MD, Andrea Mengarelli, MD, Fabio Benedetti, MD, Patrizio Mazza, MD, Vera Capria, MD, Patrizia Chiusolo, Luca Cupelli, MD, Vincenzo Federico, MD, Valentina Bozzoli, PhD MD, Anna Rita Messa, Giorgina Specchia, MD

Blood (2019) 134 (Supplement_1): 1974.

https://doi.org/10.1182/blood-2019-125071

Published: 2019

Journal Articles

Rozanolixizumab, an Anti-FcRn Antibody: Final Results from a Phase II, Multiple-Dose Study in Patients with Primary Immune Thrombocytopenia

Tadeusz Robak, MD, Maciej Kaźmierczak, Isidro Jarque, Vasile Musteata, MD PhD, Jacek Trelinski, Nichola Cooper, MD MRCP, Peter Kiessling, Ute Massow, Franz Woltering, Rose Snipes, Juan Ke, Grant Langdon, Birgit Haier, James B. Bussel, MD, Stephen Jolles

Blood (2019) 134 (Supplement_1): 897.

https://doi.org/10.1182/blood-2019-129839

Published: 2019

Journal Articles

An Updated Safety and Efficacy Results of Phase 1 Study of HQP1351, a Novel 3 rd Generation of BCR-ABL Tyrosine Kinase Inhibitor (TKI), in Patients with TKI Resistant Chronic Myeloid Leukemia

Qian Jiang, MD, Xiaojun Huang, MD, Zi Chen, MD, Qian Niu, Lichuang Men, Hengbang Wang, PhD, Jiao JI, PhD MD, Bo Huang, Dayu Shi, Ting Zhao, Yue Hou, Dajun Yang, MD PhD, Yifan Zhai, MD PhD

Blood (2019) 134 (Supplement_1): 493.

https://doi.org/10.1182/blood-2019-124295

Published: 2019

Journal Articles

A Novel Non-Covalent Modulator of Hemoglobin Improves Anemia and Reduces Sickling in a Mouse Model of Sickle Cell Disease

Kelly M. Knee, PhD, Reema Jasuja, PhD, Amey Barakat, MS, Dharani Rao, Zane Wenzel, MS, Parag Sahasrabudhe, Jatin Narula, PhD, Jay Jasti, Jeanne S. Chang, Kevin Beaumont, David W. Piotrowski, Jay M. Janz, PhD

Blood (2019) 134 (Supplement_1): 207.

https://doi.org/10.1182/blood-2019-128270

Published: 2019

Journal Articles

A phase 1/2 ascending dose study and open-label extension study of voxelotor in patients with sickle cell disease

Jo Howard, Claire Jane Hemmaway, Paul Telfer, D. Mark Layton, John Porter, Moji Awogbade, Timothy Mant, Daniel D. Gretler, Kobina Dufu, Athiwat Hutchaleelaha, Mira Patel, Vincent Siu, Sandra Dixon, Noel Landsman, Margaret Tonda, Joshua Lehrer-Graiwer

Blood (2019) 133 (17): 1865–1875.

https://doi.org/10.1182/blood-2018-08-868893

Published: 2019

Journal Articles

Efficacy and Safety of 1500 mg Voxelotor in a Phase 2a Study (GBT440-007) in Adolescents with Sickle Cell Disease

Clark Brown, Carolyn Hoppe, Adlette Inati, Miguel R. Abboud, Winfred Wang, MD, Robert Liem, MD, Gerald Woods, Lewis L. Hsu, Victor R. Gordeuk, MD, Connie Piccone, Richard A. Drachtman, Erica Fong, Sandra Dixon, Margaret Tonda, PharmD, Carla Washington, Joshua Lehrer-Graiwer

Blood (2018) 132 (Supplement 1): 509.

https://doi.org/10.1182/blood-2018-99-117510

Published: 2018

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Improvement in Red Blood Cell Physiology in Children with Sickle Cell Anemia Receiving Voxelotor

Peri-Procedural Management and Outcomes of Patients with Hemophilia on Emicizumab Prophylaxis

Pozelimab, a Human Antibody Against Complement Factor C5, Demonstrates Robust Inhibition of Alternative Complement Activity Both in Normal Human Serum and in Phase I Normal Healthy Volunteers

Phase 1 Study of Bisthianostat, an Orally Efficacious Pan-HDAC Inhibitor: Part Results of Safety, Pharmacokinetics and Efficacy in Patients with Relapsed or Refractory Multiple Myeloma

Preclinical Development of an Anti-CD38 Antibody-Drug Conjugate for Treatment of Hematological Malignancies

A Phase I Study of Single-Agent Inotuzumab Ozogamicin in Pediatric CD22-Positive Relapsed/Refractory Acute Lymphoblastic Leukemia: Preliminary Results of the ITCC-059 Study

Preliminary Results from a Phase I Study of SHC014748M in Patients with Relapsed or Refractory Indolent B-Cell Lymphomas

TNB-486, a Novel Fully Human Bispecific CD19 x CD3 Antibody That Kills CD19-Positive Tumor Cells with Minimal Cytokine Secretion

Third Party Type 2 Innate Lymphoid Cells Treat and Prevent GI Tract Acute GvHD

Phase 1 Single (SAD) and Multiple Ascending Dose (MAD) Studies of the Safety, Tolerability, Pharmacokinetics (PK) and Pharmacodynamics (PD) of FT-4202, an Allosteric Activator of Pyruvate Kinase-R, in Healthy and Sickle Cell Disease Subjects

Improved Outcomes for Patients Receiving High-Doses of IL-21 Ex Vivo Expanded NK Cells after Haploidentical Transplantation (haploSCT): Long-Term Follow-up of a Phase 1/2 Clinical Trial with Comparison to CIBMTR Controls

Hyper-Sialylated IgG M254, an Innovative Therapeutic Candidate, Evaluated in Healthy Volunteers and in Patients with Immune Thrombocytopenia Purpura: Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics

Dreamm-5: Platform Trial Evaluating Belantamab Mafodotin (a BCMA-directed Immuno-conjugate) in Combination with Novel Agents in Relapsed or Refractory Multiple Myeloma (RRMM)

Phase 1, Open-Label, Dose Escalation Study of I-131-CLR1404 (CLR 131) in Patients with Relapsed or Refractory Multiple Myeloma

Rozanolixizumab, an Anti-FcRn Antibody: Final Results from a Phase II, Multiple-Dose Study in Patients with Primary Immune Thrombocytopenia

An Updated Safety and Efficacy Results of Phase 1 Study of HQP1351, a Novel 3 rd Generation of BCR-ABL Tyrosine Kinase Inhibitor (TKI), in Patients with TKI Resistant Chronic Myeloid Leukemia

A Novel Non-Covalent Modulator of Hemoglobin Improves Anemia and Reduces Sickling in a Mouse Model of Sickle Cell Disease

A phase 1/2 ascending dose study and open-label extension study of voxelotor in patients with sickle cell disease

Efficacy and Safety of 1500 mg Voxelotor in a Phase 2a Study (GBT440-007) in Adolescents with Sickle Cell Disease

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