Research

Advancing the way sickle cell disease is treated

Sickle cell disease (SCD) is a rare inherited blood disorder that has historically been overlooked by the entire health system for decades.

  • The sickle cell community represents one of the most underserved populations in the United States. People with SCD often lack access to specialists, and dedicated patient services.
  • In the United States, SCD is most common in minority populations and in people of lower socioeconomic status.1,2
  • The life expectancy for people with SCD is approximately 30 years shorter than average.3 SCD greatly impacts a patient’s life, including their ability to work, attend school and look after their families, as well as the lives of their caregivers.

GBT is committed to partnering with key industry stakeholders to evolve clinical trial strategies to address the specific needs of people with SCD and other rare disease populations aimed at accelerating the development of new therapies. We are proud to serve as a catalyst for change for the sickle cell community.

Evolving care beyond the treatment paradigm

In addition to the disparities and inequalities in care, people living with SCD face significant misconceptions and stigmas,4 which can further exacerbate the emotional and social challenges posed by this disease. GBT is working to help dispel these stigmas to help improve the lives of those living with SCD.

Sickle cell disease can take a devastating toll

In addition to being life-threatening, SCD places a significant burden on patients and their families

More about SCD

Changing the SCD treatment paradigm

We are committed to advancing care by developing and delivering innovative medicines

See our pipeline

Review key publications

Our key results have been presented and published at medical conferences and in peer-reviewed publications

Access our publications
  1. Genetics Home Reference. Sickle Cell Disease. https://ghr.nlm.nih.gov/condition/sickle-cell-disease#statistics.
  2. Panepinto, JA, et al. Qual Life Res. 2009 Feb; 18(1): 5–13.
  3. Piel et al. N Engl J Med. 2017;376:1561.
  4. Sivamuth, Krupa MD. Get Healthy Stay Healthy. Overcoming the Stigma of Sickle Cell Disease. 2015 (v1.0)