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Journal Articles

One Year Follow-up on the First Patient Treated with Nula-Cel: An Autologous CRISPR/Cas9 Gene Corrected CD34+ Cell Product to Treat Sickle Cell Disease

Blood (2023) 142 (Supplement 1): 5000.

https://doi.org/10.1182/blood-2023-188963

Published: 2023

Journal Articles

Impact of GBT440 on Unconjugated Bilirubin and Jaundice in Sickle Cell Disease: A Patient Case Report

Paul Telfer, FRCPath, MD, Josh Lehrer, MD MPhil, FACC, Irene Agodoa, MD, Marzena Jurek, MS, Kathleen M Fox, PhD, Laurie Burke, BPharm, MPH, Noel Landsman, Dip.Nur (SA), Timothy Mant, MB, FFPM, FRCP

Blood (2017) 130 (Supplement 1): 4779.

https://doi.org/10.1182/blood.V130.Suppl_1.4779.4779

Published: 2017

Journal Articles

The Pharmacokinetics (PK) of GBT440 Following Single Doses in Pediatric Patients with Sickle Cell Disease (SCD)

Carla B Washington, PhD, Michelle Green, PhD, Adlette C. Inati, MD, Clark Brown, MD PhD, Carolyn C Hoppe, MD, Winfred Wang, MD, Ganesh Balaratnam, MD, Sandra Dixon, MS, Erica Fong, Athiwat Hutchaleelaha, PhD, Margaret E Tonda, PharmD, Josh Lehrer, MD MPhil, FACC

Blood (2017) 130 (Supplement 1): 980.

https://doi.org/10.1182/blood.V130.Suppl_1.980.980

Published: 2017

Journal Articles

Initial Results from a Cohort in a Phase 2a Study (GBT440-007) Evaluating Adolescents with Sickle Cell Disease Treated with Multiple Doses of GBT440, a HbS Polymerization Inhibitor

Carolyn C Hoppe, MD, Adlette C. Inati, MD, Clark Brown, MD PhD, Winfred Wang, MD, Victor R. Gordeuk, MD, Robert Liem, MD, Gerald Woods, MD, Connie M. Piccone, MD, Erica Fong, Ganesh Balaratnam, MD, Sandy Dixon, Margaret E Tonda, PharmD, Carla B Washington, PhD, Yifah Yaron, MD PhD, Josh Lehrer, MD MPhil, FACC

Blood (2017) 130 (Supplement 1): 689.

https://doi.org/10.1182/blood.V130.Suppl_1.689.689

Published: 2017

Journal Articles

Absorption, Metabolism and Excretion of GBT440, a Novel Hemoglobin S (HbS) Polymerization Inhibitor for the Treatment of Sickle Cell Disease (SCD), in Healthy Male Subjects

Peter Rademacher, PhD, Athiwat Hutchaleelaha, PhD, Carla Washington, Josh Lehrer, Eleanor Ramos, MD

Blood (2016) 128 (22): 2487.

https://doi.org/10.1182/blood.V128.22.2487.2487

Published: 2016

Journal Articles

Long-Term Dosing in Sickle Cell Disease Subjects with GBT440, a Novel HbS Polymerization Inhibitor

Josh Lehrer-Graiwer, MDMPhil, FACC, Jo Howard, MB, BChir, Claire Jane Hemmaway, MD, Moji Awogbade, Paul Telfer, FRCPath,MD, Mark Layton, MB, FRCP, John B. Porter, MD, Timothy Mant, MB, FFPM, FRCP, Kobina Dufu, PhD, Athiwat Hutchaleelaha, PhD, Donna Oksenberg, PhD, Mira Patel, PhD, Margaret Eleanor Tonda, PharmD, Kenneth Bridges, MD, Eleanor Ramos, MD

Blood (2016) 128 (22): 2488.

https://doi.org/10.1182/blood.V128.22.2488.2488

Published: 2016

Journal Articles

The 10-Item Sickle Cell Disease Severity Measure (SCDSM-10): A Novel Measure of Daily SCD Symptom Severity Developed to Assess Benefit of GBT440, an Experimental HbS Polymerization Inhibitor

Laurie Burke, BPharm, MPH, Jeremy Charles Hobart, MD PhD FRCP, MBBS, BSc, Kathleen Fox, PhD, Josh Lehrer-Graiwer, MD MPhil, FACC, Kenneth Bridges, MD, Martine Kraus, PhD, Eleanor Ramos, MD

Blood (2016) 128 (22): 4760.

https://doi.org/10.1182/blood.V128.22.4760.4760

Published: 2016

Journal Articles

GBT440, a Potent Anti-Sickling Hemoglobin Modifier Reduces Hemolysis, Improves Anemia and Nearly Eliminates Sickle Cells in Peripheral Blood of Patients with Sickle Cell Disease

Josh Lehrer-Graiwer, MDMPhil, FACC, Jo Howard, MD, Claire Jane Hemmaway, MD, Moji Awogbade, Paul Telfer, FRCPath,MD, Mark Layton, MB, FRCP, Timothy Mant, MB, FFPM, FRCP, Kobina Dufu, PhD, Athiwat Hutchaleelaha, PhD, Teri Koller, MBA, Donna Oksenberg, PhD, Mira Patel, PhD, Eleanor Ramos, MD

Blood (2015) 126 (23): 542.

https://doi.org/10.1182/blood.V126.23.542.542

Published: 2015

NARROW

One Year Follow-up on the First Patient Treated with Nula-Cel: An Autologous CRISPR/Cas9 Gene Corrected CD34+ Cell Product to Treat Sickle Cell Disease

Impact of GBT440 on Unconjugated Bilirubin and Jaundice in Sickle Cell Disease: A Patient Case Report

The Pharmacokinetics (PK) of GBT440 Following Single Doses in Pediatric Patients with Sickle Cell Disease (SCD)

Initial Results from a Cohort in a Phase 2a Study (GBT440-007) Evaluating Adolescents with Sickle Cell Disease Treated with Multiple Doses of GBT440, a HbS Polymerization Inhibitor

Absorption, Metabolism and Excretion of GBT440, a Novel Hemoglobin S (HbS) Polymerization Inhibitor for the Treatment of Sickle Cell Disease (SCD), in Healthy Male Subjects

Long-Term Dosing in Sickle Cell Disease Subjects with GBT440, a Novel HbS Polymerization Inhibitor

The 10-Item Sickle Cell Disease Severity Measure (SCDSM-10): A Novel Measure of Daily SCD Symptom Severity Developed to Assess Benefit of GBT440, an Experimental HbS Polymerization Inhibitor

GBT440, a Potent Anti-Sickling Hemoglobin Modifier Reduces Hemolysis, Improves Anemia and Nearly Eliminates Sickle Cells in Peripheral Blood of Patients with Sickle Cell Disease

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American Society of Hematology

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ASH Publications

American Society of Hematology

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