GBT Expands Sickle Cell Disease Pipeline with Exclusive In-license of Two Novel Small Molecule Programs from Sanofi S.A.

SOUTH SAN FRANCISCO, CA, USA I March 16, 2021 I Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today announced it has entered into an agreement with Sanofi S.A. to exclusively in-license worldwide rights to two early-stage research programs in sickle cell disease (SCD): one that pursues a novel anti-sickling mechanism and another that leverages a new approach to reduce inflammation and oxidative stress. These mechanisms are distinct and potentially complementary to that of Oxbryta® (voxelotor) tablets, a novel hemoglobin S polymerization inhibitor approved in the United States for the treatment of SCD in patients ages 12 years and older. The programs, from Sanofi’s Bioverativ subsidiary, supplement GBT’s existing pipeline and support the company’s strategy to address SCD from multiple approaches.

“We envision a future in which sickle cell disease is a well-managed condition with the potential for a functional cure in the form of patient-friendly oral therapies. As we work toward this vision and our goal to transform the treatment and care of people living with this devastating disease, we are advancing our robust internal research programs with disease-modifying potential while continually exploring partnership opportunities across a variety of mechanisms,” said Jung E. Choi, chief business and strategy officer of GBT. “These novel discovery programs represent promising approaches that we believe may have the potential to lead to meaningful improvements for patients.”

Under the terms of the agreement, GBT will conduct all research, development, regulatory and commercialization activities worldwide. Sanofi will receive an upfront payment and is entitled to payments up to approximately $353 million upon achievement of development, regulatory and commercial milestones and single-digit tiered royalties on worldwide net sales.

About Sickle Cell Disease
Sickle cell disease (SCD) affects an estimated 100,000 people in the United States,1 an estimated 52,000 people in Europe,2 and millions of people throughout the world, particularly among those whose ancestors are from sub-Saharan Africa.1 It also affects people of Hispanic, South Asian, Southern European and Middle Eastern ancestry.1 SCD is a lifelong inherited rare blood disorder that impacts hemoglobin, a protein carried by red blood cells that delivers oxygen to tissues and organs throughout the body.3 Due to a genetic mutation, individuals with SCD form abnormal hemoglobin known as sickle hemoglobin. Through a process called hemoglobin polymerization, red blood cells become sickled – deoxygenated, crescent-shaped and rigid.3-5 The sickling process causes hemolytic anemia (low hemoglobin due to red blood cell destruction) and blockages in capillaries and small blood vessels, which impede the flow of blood and oxygen throughout the body. The diminished oxygen delivery to tissues and organs can lead to life-threatening complications, including stroke and irreversible organ damage.4-7

About Global Blood Therapeutics
Global Blood Therapeutics (GBT) is a biopharmaceutical company dedicated to the discovery, development and delivery of life-changing treatments that provide hope to underserved patient communities. Founded in 2011, GBT is delivering on its goal to transform the treatment and care of sickle cell disease (SCD), a lifelong, devastating inherited blood disorder. The company has introduced Oxbryta® (voxelotor), the first FDA-approved treatment that directly inhibits sickle hemoglobin polymerization, the root cause of red blood cell sickling in SCD. GBT is also advancing its pipeline program in SCD with inclacumab, a P-selectin inhibitor in development to address pain crises associated with the disease, and GBT021601 (GBT601), the company’s next-generation hemoglobin S polymerization inhibitor. In addition, GBT’s drug discovery teams are working on new targets to develop the next wave of treatments for SCD. To learn more, please visit and follow the company on Twitter @GBT_news.


  1. Centers for Disease Control and Prevention website. Sickle Cell Disease (SCD). Accessed June 3, 2019.
  2. European Medicines Agency. Accessed June 12, 2020.
  3. National Heart, Lung, and Blood Institute website. Sickle Cell Disease. Accessed August 5, 2019.
  4. Rees DC, et al. Lancet. 2010;376(9757):2018-2031.
  5. Kato GJ, et al. Nat Rev Dis Primers. 2018;4:18010.
  6. Kato GJ, et al. J Clin Invest. 2017;127(3):750-760.
  7. Caboot JB, et al. Paediatr Respir Rev. 2014;15(1):17-23.

SOURCE: Global Blood Therapeutics

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