About OXBRYTA® (voxelotor) OXBRYTA (voxelotor) is an oral, once-daily therapy for patients with sickle cell disease (SCD). OXBRYTA works by increasing hemoglobin’s affinity for oxygen.

GBT601 is a sickle hemoglobin polymerization inhibitor being tested in a phase 2/3 trial. The preliminary results were for 35 patients from the first two doses tested, 100 mg and 150 mg.

Patients 1 and 2 reached normal hemoglobin levels five months post-treatment with EDIT-301, and both maintained normal hemoglobin levels at 10- and 6-month follow-ups, respectively.

At low oxygen tension, hemoglobin molecule undergoes a process of polymerization, which is thought to play a critical role in the process of sickling. Hydroxyurea (HU) was the first approved ...

Mitapivat, etavopivat, and the hemoglobin S polymerization inhibitor voxelotor (Oxbryta) have emerged as new treatment options for SCD, the authors noted. Gene therapies have also been studied for ...

A number of potential strategies can be exploited to either inhibit or alter the kinetics of Hb S polymerization (11 – 14). These include disruption of intermolecular contacts in the sickle fiber, ...

Hamad Medical Corporation, Doha, Qatar Sickle cell disease (SCD) is a heterogeneous group of inherited disorders characterized by the production of sickle hemoglobin which is less soluble than an ...

GBT’s pipeline also includes GBT601 (formerly GBT021601), an oral, once-daily, next-generation sickle hemoglobin (HbS) polymerization inhibitor under study in Phase I and Phase II/III trials.

Both Oxbryta and GBT601, also known as GBT021601, work by increasing the ability of hemoglobin — the protein inside red blood cells that is responsible for transporting oxygen — to bind oxygen. This ...

Both Oxbryta and GBT601 work by increasing hemoglobin’s ability to bind to oxygen, thereby preventing its clumping, or polymerization, and the subsequent sickling and destruction of red blood cells — ...