GBT supports the sickle cell disease program of the ASH Research Collaborative Data Hub

WASHINGTON and SOUTH SAN FRANCISCO, California, December 2, 2021 / PRNewswire / – The ASH Research Collaborative (ASH RC), a nonprofit organization established in 2018 by the American Society of Hematology (ASH) and Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) has announced today that GBT will provide a grant of $ 400,000 to ASH RC in support of the ASH RC Data Hub Sickle Cell Disease (SCD) program. With the Data Hub SCD program, ASH RC seeks to advance research and outcomes for people living with SCD by expanding its current data collection of real research-grade data to include more than half of people living with SCD in United States.

“We have a common mission with GBT to help transform the treatment of sickle cell anemia,” said Martin S. Tallman, MD, of Memorial Sloan Kettering Cancer Center, who is president of the ASH Research Collaborative and the American Society of Hematology. “By working closely with the SCD community, ASH RC is committed to effectively capturing real-world longitudinal data to better understand the natural history of sickle cell disease and to create a high-quality national data repository for accelerate research and improve clinical care. We aim to give the SCD community a meaningful voice in creating one of the world’s largest repositories of real, science-grade data in SCD. We appreciate the support from GBT to help us work to improve the care and outcomes for our patients with this rare disease. “

ASH RC fosters collaborative partnerships to advance advances in hematology and improve the lives of those affected by blood diseases. The foundation of ASH RC is its Data Hub, a multi-stakeholder real-world data program that facilitates the exchange of information on hematological conditions to support scientific research, discovery and quality improvement. Nearly 30 US clinical sites providing SCD care currently participate in the Data Hub and submit HIPAA compliant data representing 6,000 people living with SCD. The Data Hub is actively recruiting up to 110 additional clinical sites, and ASH RC believes it is well positioned to collect data on more than 50,000 people in the United States living with SCD.

GBT is the first biopharmaceutical company to provide financial support to ASH RC via a grant that will help onboard clinical sites and integrate their data into the Data Hub. These sites are organized into 19 consortia also registered in the ASH RC SCD clinical trials network. The goal of the SCD Clinical Trials Network is to accelerate research that advances the treatment and care of people affected by SCD.

“Comprehensive real-world data can empower people with sickle cell disease and their healthcare providers to make decisions about their care, while also guiding the development of new treatments and guidelines that we hope will transform CDS into a well-managed disease, ”said Kim smith whitley, MD, executive vice president and head of research and development at GBT. “GBT is proud to support ASH RC and its core mission of collecting real-world data to help meet the urgent needs of people with sickle cell disease. There has never been a more important time to advance research initiatives with the potential to improve care. and address long-standing health equity gaps for the SCD community. “

The Data Hub can collect a wide variety of data, including electronic medical record data, clinical and laboratory data, genomic or molecular correlates, patient-reported outcomes, and aggregated population data. This data is obtained from inpatient and outpatient clinical sites, industry or government datasets, registries, other US or international sources, and directly from patients. By using cutting-edge technology to automate clinical data entry and linkages to other data sources, the Data Hub focuses on reducing the burden of data entry. Patient information collected in the Data Hub complies with state and local privacy laws and regulations to protect patient privacy.

To learn more, please visit www.ashresearchcollaborative.org.

About sickle cell anemia
Sickle cell disease affects more than 100,000 people in the United States,1 around 52,000 people in Europe,2 and millions of people around the world, especially among those whose ancestors originated from sub-Saharan Africa.3 It also affects people of Hispanic, South Asian, South European, and Middle Eastern ancestry.3 SCD is a rare inherited blood disorder that affects hemoglobin, a protein carried by red blood cells that provides oxygen to tissues and organs throughout the body.4 Due to a genetic mutation, people with sickle cell anemia form an abnormal hemoglobin called sickle hemoglobin. Through a process called hemoglobin polymerization, red blood cells become sickles – deoxygenated, crescent-shaped, and rigid.4-6 The sickling process causes hemolytic anemia (low hemoglobin level due to the destruction of red blood cells) and blockages in capillaries and small blood vessels, which impede the flow of blood and oxygen throughout the body. . Decreased oxygen supply to tissues and organs can lead to life-threatening complications, including stroke and irreversible organ damage.5-8

About the ASH Research Collaborative
The ASH Research Collaborative (ASH RC) is a non-profit organization created by the American Society of Hematology (ASH) to improve the lives of people affected by blood diseases by fostering collaborative partnerships to accelerate progress in hematology. The basis of ASH RC is its data center and clinical trials network. Through ASH RC’s state-of-the-art data sharing platforms and patient-centric approach to recruiting, designing and executing clinical trials for SCD, ASH RC enables companies to develop new treatments more quickly and efficiently to help people with blood problems and to maximize the value of these treatments after approval. The ASH RC aims to transform research and practice in malignant and non-malignant hematologic diseases around the world, for the benefit of patients and the hematologic community.

About Global Blood Therapeutics
Global Blood Therapeutics, Inc. (GBT) is a biopharmaceutical company dedicated to the discovery, development and delivery of life-changing treatments that give hope to underserved patient communities. Founded in 2011, GBT is achieving its goal of transforming the treatment and care of sickle cell anemia (SCD), a devastating, inherited blood disorder that lasts for life. The company introduced Oxbryta® (voxelotor), the first FDA-approved treatment that directly inhibits the polymerization of sickle hemoglobin, the root cause of sickle red blood cells in sickle cell disease. GBT is also advancing its pipeline program in SCD with inclacumab, a P-selectin inhibitor in Phase 3 development to treat pain attacks associated with disease, and GBT021601 (GBT601), the polymerization inhibitor of the company’s next-generation hemoglobin S. In addition, GBT’s drug discovery teams are working on new targets to develop the next wave of potential treatments for SCD. For more information, visit www.gbt.com and follow the company on Twitter @GBT_news.

The references

  1. Centers for Disease Control and Prevention website. Research on sickle cell disease. https://www.cdc.gov/ncbddd/hemoglobinopathies/scdc-understanding-sickle-cell-disease.html. Accessed December 1, 2021.
  2. European Medicines Agency. https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3182125. Accessed June 12, 2020.
  3. Centers for Disease Control and Prevention website. Sickle cell disease (SCD). https://www.cdc.gov/ncbddd/sicklecell/data.html. Accessed June 3, 2019.
  4. National Heart, Lung and Blood Institute website. Sickle cell disease. https://www.nhlbi.nih.gov/health-topics/sickle-cell-disease. Accessed August 5, 2019.
  5. Rees DC, et al. Lancet. 2010; 376 (9757): 2018-2031.
  6. Kato GJ, et al. Nat Rev Dis Primers. 2018; 4: 18010.
  7. Kato GJ, et al. J Clin Invest. 2017; 127 (3): 750-760.
  8. Caboot JB, et al. Paediatr Respir Rev. 2014; 15 (1): 17-23.

SOURCE American Society of Hematology


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Hector Hedgepeth

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