RARE Daily

GBT In-Licenses Two Sickle Cell Small Molecule Programs from Sanofi

March 16, 2021

Rare Daily Staff

Global Blood Therapeutics entered into an agreement with Sanofi to exclusively in-license worldwide rights to two early-stage research programs in sickle cell disease: 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 GBT’s Oxbryta, a novel hemoglobin S polymerization small molecule inhibitor approved in the United States for the treatment of Sickle Cell Disease 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,” said Jung 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.

Sickle cell disease (SCD) is an inherited rare blood disorder that impacts hemoglobin, a protein carried by red blood cells that delivers oxygen to tissues and organs throughout the body. 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. 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.

SCD affects an estimated 100,000 people in the United States, an estimated 52,000 people in Europe, and millions of people throughout the world, particularly among those whose ancestors are from sub-Saharan Africa. It also affects people of Hispanic, South Asian, Southern European and Middle Eastern ancestry.

Photo: Jung Choi, chief business and strategy officer of GBT

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