Our goal is to transform the treatment of sickle cell disease (SCD), an overlooked rare condition, and help address the urgent needs of the community. Our mission is driven by the historical lack of understanding and attention given to SCD. Although the fundamental cause of SCD has been understood for decades, therapeutic innovation and access to care has significantly lagged.
GBT’s medicine is the first treatment approved by the FDA to target an underlying cause of SCD. We are developing additional investigational treatments that have the potential to better the lives of patients.
We are driven by a deep and urgent desire to improve the care of SCD patients. Beyond developing medicines, we are working to shine a bright light on the disparities and inequalities faced by people living with SCD. To this end, in July 2019 we launched Sickle Cell Speaks, a national campaign focused on breaking down stigmas associated with SCD.
GBT is collaborating with Syros to discover new medicines that induce fetal hemoglobin to treat SCD and beta thalassemia.
GBT’s first medicine for SCD received accelerated approval in the United States three months prior to our PDUFA date.
Priority Review is granted to therapies that the FDA determines have the potential to provide a significant improvement in the safety or effectiveness of the treatment, diagnosis or prevention of a serious condition.
Topline results for all patients enrolled in the Phase 3 HOPE Study were published in The New England Journal of Medicine.
Breakthrough Therapy designation expedites the development and review of drugs that have demonstrated preliminary clinical evidence indicating the potential for substantial improvement over available therapy.
Voxelotor became the first potential treatment for SCD to be accepted for the EU’s new Priority Medicines (PRIME) designation, enabling frequent and early interactions with the EMA to accelerate evaluation and potential marketing approval.
The HOPE (Hemoglobin Oxygen Affinity Modulation to Inhibit HbS
PolymErization) Study was designed to assess the efficacy of voxelotor
in producing meaningful clinical improvements in anemia.
GBT became a public company.
Within 15 months of voxelotor being identified as a potential clinical candidate, the first 8 patients were dosed in a Phase 1/2 clinical trial in SCD.
GBT began testing GBT440 in 2012 to understand its mechanism of action. This molecule would later be named voxelotor.
The company was founded to develop medicines that could make a transformative impact, and SCD was our first focus.
