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Journal Articles

Impacts of Total Body Irradiation in Allogeneic Umbilical Cord Blood Hematopoietic Stem Cell Transplantation

Jeffrey J. Pu, Kristin N. Berger, Hao Wang, Wei Fu, Elizabeth L Miller, Larisa Broglie, Heiko Konig, Frederick Goldman, Julie-An Talano, Sherif S. Farag, Melanie Comito

Blood (2021) 138 (Supplement 1): 4852.

https://doi.org/10.1182/blood-2021-152893

Published: 2021

Journal Articles

ROR1 targeting with the antibody-drug conjugate VLS-101 is effective in Richter syndrome patient–derived xenograft mouse models

Tiziana Vaisitti, Francesca Arruga, Nicoletta Vitale, Thanh-Trang Lee, Mira Ko, Amy Chadburn, Esteban Braggio, Arianna Di Napoli, Andrea Iannello, John N. Allan, Langdon L. Miller, Brian J. Lannutti, Richard R. Furman, Katti A. Jessen, Silvia Deaglio

Blood (2021) 137 (24): 3365–3377.

https://doi.org/10.1182/blood.2020008404

Published: 2021

Includes: Supplemental data

Journal Articles

Resolution of Serious Vaso-Occlusive Pain Crises and Reduction in Patient-Reported Pain Intensity: Results from the Ongoing Phase 1/2 HGB-206 Group C Study of LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy

Alexis A. Thompson, MD, Mark C. Walters, MD, Markus Y Mapara, MD PhD, Janet L. Kwiatkowski, MD MSCE, Lakshmanan Krishnamurti, MD, Banu Aygun, MD, Kimberly A. Kasow, DO, Stacey Rifkin-Zenenberg, DO, Manfred Schmidt, PhD, Jay DelCarpini, Francis J. Pierciey, Jr., Alexandra L. Miller, Meghan E. Gallagher, MSc, Ren Chen, Sunita Goyal, MD, Julie Kanter, MD, John F. Tisdale, MD

Blood (2020) 136 (Supplement 1): 16–17.

https://doi.org/10.1182/blood-2020-134940

Published: 2020

Journal Articles

VLS-101, a ROR1-Targeting Antibody-Drug Conjugate, Demonstrates a Predictable Safety Profile and Clinical Efficacy in Patients with Heavily Pretreated Mantle Cell Lymphoma and Diffuse Large B-Cell Lymphoma

Blood (2020) 136 (Supplement 1): 13–14.

https://doi.org/10.1182/blood-2020-136373

Published: 2020

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

Vls-101 Is a Novel Therapeutic Antibody-Drug Conjugate (ADC) Targeting Receptor Tyrosine Kinase-like Orphan Receptor 1 (ROR1) in Richter's Syndrome (RS)

Tiziana Vaisitti, PhD, Katti Jessen, PhD, Thanh-Trang Vo, PhD, Mira Ko, Francesca Arruga, PhD, Nicoletta Vitale, PhD, Esteban Braggio, PhD, Arianna Di Napoli, Amy Chadburn, MD, John N. Allan, MD, Richard R. Furman, MD, Langdon L. Miller, Brian J Lanutti, PhD, Silvia Deaglio, MD PhD

Blood (2019) 134 (Supplement_1): 2856.

https://doi.org/10.1182/blood-2019-126827

Published: 2019

Journal Articles

LAM-003, a Novel Oral Heat Shock Protein 90 Inhibitor for Treatment of Acute Myeloid Leukemia, Including Wild-Type and FMS-like Tyrosine Kinase 3 (FLT3)-Mutant Disease

Neil Beeharry, Sean Landrette, PhD, Jeff Grotzke, PhD, Sophia Gayle, PhD, Marylens Hernandez, PhD, Stephanie Halene, MD, Peter R Young, PhD, Langdon L Miller, MD, Tian Xu, PhD, Jonathan Rothberg, PhD, Henri Lichenstein, PhD

Blood (2019) 134 (Supplement_1): 2664.

https://doi.org/10.1182/blood-2019-125770

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

Analysis of Platelet Alpha and Dense Granule Release Using Small Volumes of Blood

Joseph Cho, MD PhD, Melissa Tjota, MD PhD, Jocelyn Gutierrez, BSMT, Krzysztof Mikrut, BSMT(ASCP), Geoffrey D. Wool, MD PhD, Jonathan L Miller, MD PhD

Blood (2019) 134 (Supplement_1): 2342.

https://doi.org/10.1182/blood-2019-131258

Published: 2019

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

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

Phase 1 Clinical Safety, Pharmacokinetics (PK), and Activity of Apilimod Dimesylate (LAM-002A), a First-in-Class Inhibitor of Phosphatidylinositol-3-Phosphate 5-Kinase (PIKfyve), in Patients with Relapsed or Refractory B-Cell Malignancies

Wael A Harb, MD, Catherine S Diefenbach, MD, Nehal Lakhani, MD PhD, Sarah C Rutherford, MD, Marshall T Schreeder, MD, Stephen M Ansell, MD PhD, Taimur Sher, MD, David M Aboulafia, MD, Jonathon B Cohen, MDMS, Darrell Nix, BS, PhD, Sean Landrette, PhD, Kate Flanders, BS, Langdon L Miller, MD, Henri Lichenstein, PhD, Jeremy S. Abramson, MD MMSc

Blood (2017) 130 (Supplement 1): 4119.

https://doi.org/10.1182/blood.V130.Suppl_1.4119.4119

Published: 2017

Journal Articles

The Let-7 Chronoregulatory Cascade Components May Act Independently to Regulate Fetal Hemoglobin in Human Adult Erythroblasts

Jaira F. de Vasconcellos, Colleen Byrnes, Y. Terry Lee, May Li, Antoinette Rabel, Richard L. Proia, Jeffery L. Miller

Blood (2017) 130 (Supplement 1): 944.

https://doi.org/10.1182/blood.V130.Suppl_1.944.944

Published: 2017

Journal Articles

A Phase 1-2 Dose-Escalation and Cohort- Expansion Study of eFT508, a Selective, Orally Bioavailable Inhibitor of MNK1 and MNK2, in Patients with Hematological Malignancies

Ajay K. Gopal, MD, Rod Ramchandren, MD, Nashat Y. Gabrail, Manish R Patel, MD, Ding Wang, MDPhD, Langdon L Miller, MD, Vikas Goel, PhD, Gary G Chiang, PhD, Kevin R Webster, PhD, Sam Sperry, PhD, Debra T Vallner, PhDMBA, Cara Casseday, BSc MBA, Jeremy Barton, MBBS

Blood (2017) 130 (Supplement 1): 4624.

https://doi.org/10.1182/blood.V130.Suppl_1.4624.4624

Published: 2017

Journal Articles

IGF2BP1 Protein Binding of Erythroblast Transcription Factor mRNA: A Cytoplasm-Localized Mechanism for Globin Gene Regulation

Jaira F. de Vasconcellos, Y. Terry Lee, Colleen Byrnes, Benjamin A. Clarke, Pauline C. Xu, Antoinette Rabel, Richard L. Proia, Jeffery L. Miller

Blood (2016) 128 (22): 1009.

https://doi.org/10.1182/blood.V128.22.1009.1009

Published: 2016

Journal Articles

IGF2BP3 Moderately Increases Fetal Hemoglobin Levels in Human Adult Erythroblasts

Jaira F. de Vasconcellos, Colleen Byrnes, Y. Terry Lee, Pauline C. Xu, Antoinette Rabel, Jeffery L. Miller

Blood (2016) 128 (22): 2464.

https://doi.org/10.1182/blood.V128.22.2464.2464

Published: 2016

Journal Articles

The 1000 Genomes Project: Prediction of Known and Novel Blood Group Continental Distribution By Next Generation Sequencing

Celina Montemayor Garcia, MDPhD, Panagiota Karagianni, PhD, Erika M Reese, MSL(ASCP)CMSBBCM, Danielle A Smellie, MD, Debrean A. Loy, MT(ASCP), Kimberly A Levy, MSL(ASCP)CMSBBCM, Magdalene Nwokocha, MD, Marina Bueno, MSL(ASCP)CMSBBCM, Jeffery L Miller, MD, Harvey G. Klein, MD

Blood (2016) 128 (22): 23.

https://doi.org/10.1182/blood.V128.22.23.23

Published: 2016

Journal Articles

Hemoglobin S Oxidation Promotes Plasma-Derived Microparticle Membrane Alterations and Toxicity

Sirsendu Jana, PhD, Michael B Strader, PhD, Tigist Kassa, PhD, Fantao Meng, PhD, Wayne Hicks, PhD, Ivan Tarandovskiy, PhD, Silvia De Paoli, PhD, Jan Simak, PhD, Jeffery L Miller, MD, Laurel Mendelsohn, BSc., Jim Nichols, RSN, Swee Lay Thein, MD, John D. Belcher, PhD, Gregory M Vercellotti, MD, Abdu Alayash, PhD

Blood (2016) 128 (22): 856.

https://doi.org/10.1182/blood.V128.22.856.856

Published: 2016

Journal Articles

First-in-Human Phase 1a Study of the Safety, Pharmacokinetics, and Pharmacodynamics of the Noncovalent Bruton Tyrosine Kinase (BTK) Inhibitor SNS-062 in Healthy Subjects

Linda L. Neuman, MD MBA, Renee Ward, MD PhD, David Arnold, Daniel L. Combs, BS, Deena Gruver, MS, Wendy Hill, MBA, Josué Mfopou Kunjom, MD PhD, Langdon L. Miller, MD, Judith A. Fox, PhD

Blood (2016) 128 (22): 2032.

https://doi.org/10.1182/blood.V128.22.2032.2032

Published: 2016

Journal Articles

A phase 2 study of idelalisib plus rituximab in treatment-naïve older patients with chronic lymphocytic leukemia

Susan M. O'Brien, Nicole Lamanna, Thomas J. Kipps, Ian Flinn, Andrew D. Zelenetz, Jan A. Burger, Michael Keating, Siddhartha Mitra, Leanne Holes, Albert S. Yu, David M. Johnson, Langdon L. Miller, Yeonhee Kim, Roger D. Dansey, Ronald L. Dubowy, Steven E. Coutre

Blood (2015) 126 (25): 2686–2694.

https://doi.org/10.1182/blood-2015-03-630947

Published: 2015

Includes: Supplemental data

NARROW

Impacts of Total Body Irradiation in Allogeneic Umbilical Cord Blood Hematopoietic Stem Cell Transplantation

ROR1 targeting with the antibody-drug conjugate VLS-101 is effective in Richter syndrome patient–derived xenograft mouse models

Resolution of Serious Vaso-Occlusive Pain Crises and Reduction in Patient-Reported Pain Intensity: Results from the Ongoing Phase 1/2 HGB-206 Group C Study of LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy

VLS-101, a ROR1-Targeting Antibody-Drug Conjugate, Demonstrates a Predictable Safety Profile and Clinical Efficacy in Patients with Heavily Pretreated Mantle Cell Lymphoma and Diffuse Large B-Cell Lymphoma

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

Vls-101 Is a Novel Therapeutic Antibody-Drug Conjugate (ADC) Targeting Receptor Tyrosine Kinase-like Orphan Receptor 1 (ROR1) in Richter's Syndrome (RS)

LAM-003, a Novel Oral Heat Shock Protein 90 Inhibitor for Treatment of Acute Myeloid Leukemia, Including Wild-Type and FMS-like Tyrosine Kinase 3 (FLT3)-Mutant Disease

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

Analysis of Platelet Alpha and Dense Granule Release Using Small Volumes of Blood

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

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

Phase 1 Clinical Safety, Pharmacokinetics (PK), and Activity of Apilimod Dimesylate (LAM-002A), a First-in-Class Inhibitor of Phosphatidylinositol-3-Phosphate 5-Kinase (PIKfyve), in Patients with Relapsed or Refractory B-Cell Malignancies

The Let-7 Chronoregulatory Cascade Components May Act Independently to Regulate Fetal Hemoglobin in Human Adult Erythroblasts

A Phase 1-2 Dose-Escalation and Cohort- Expansion Study of eFT508, a Selective, Orally Bioavailable Inhibitor of MNK1 and MNK2, in Patients with Hematological Malignancies

IGF2BP1 Protein Binding of Erythroblast Transcription Factor mRNA: A Cytoplasm-Localized Mechanism for Globin Gene Regulation

IGF2BP3 Moderately Increases Fetal Hemoglobin Levels in Human Adult Erythroblasts

The 1000 Genomes Project: Prediction of Known and Novel Blood Group Continental Distribution By Next Generation Sequencing

Hemoglobin S Oxidation Promotes Plasma-Derived Microparticle Membrane Alterations and Toxicity

First-in-Human Phase 1a Study of the Safety, Pharmacokinetics, and Pharmacodynamics of the Noncovalent Bruton Tyrosine Kinase (BTK) Inhibitor SNS-062 in Healthy Subjects

A phase 2 study of idelalisib plus rituximab in treatment-naïve older patients with chronic lymphocytic leukemia

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American Society of Hematology

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American Society of Hematology

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