A Phase 1 trial evaluating the safety and pharmacological properties of TAK-755 — Takeda’s experimental therapy for painful vaso-occlusive crises (VOCs) — in adults with sickle cell disease (SCD) is actively recruiting patients for its intermediate dose group. SCD patients, ages 18–65 years, who had no VOCs in the…
Imara has announced that it will change the main goal of its ongoing Phase 2b Ardent trial, which is assessing the safety and efficacy of IMR-687 for the treatment of sickle cell disease (SCD). The trial’s new main goal is the annual rate of vaso-occlusive crises (VOCs) —…
IMR-687 (tovinontrine), an experimental oral therapy for sickle cell disease (SCD), reduced the rate of painful vaso-occlusive crisis (VOC), lowered hospitalization due to VOCs, and extended the time to a first VOC in SCD patients, according to new data from an open-label extension of a Phase 2 clinical trial.
Prompted by the disparities in care between children and adults with sickle cell disease (SCD) and inspired to raise awareness about rare diseases, Cory Lewis has made it his personal mission to improve health for the African-American sickle cell community. Lewis, who is a 27-year-old leasing agent and has…
GBT601 and inclacumab, experimental therapies from Global Blood Therapeutics (GBT) for sickle cell disease (SCD) and associated vaso-occlusive crises (VOCs), were generally safe and showed signs of biological activity in healthy volunteers, according to data from Phase 1 clinical trials. These findings will be presented by GBT through two…
Long-term treatment with Oxbryta (voxelotor) safely and effectively reduced anemia and red blood cell destruction (hemolysis), while keeping a low rate of painful vaso-occlusive crises (VOCs), in adolescents and adults with sickle cell disease (SCD). That’s according to nearly three years of data from the Phase 3 HOPE…
Through a new partnership between Emmaus Life Sciences and UpScript, patients with sickle cell disease (SCD) will soon be able to consult with a physician from home and have greater access to Endari (L-glutamine). Through telehealth — using computers and mobile devices to manage and receive…
The first participant has been enrolled in the Phase 1/2 clinical trial CEDAR evaluating Graphite Bio’s investigational gene editing therapy GPH101, which is designed to directly correct the genetic mutation that causes sickle cell disease (SCD). “We are thrilled that our first patient is now enrolled in our CEDAR…
The U.S. Food and Drug Administration (FDA) has cleared Beam Therapeutics to start clinical testing of BEAM-101, the company’s investigational gene editing cell therapy for sickle cell disease (SCD). “BEAM-101 has the potential to offer a one-time treatment for patients with sickle cell disease, and this clearance enables…
Enrollment is underway for a Phase 1/2 trial evaluating the safety and efficacy of EDIT-301, an experimental gene editing cell therapy for people with sickle cell disease (SCD). The trial, called RUBY (NCT04853576), had been cleared for launch by the U.S. Food and Drug Administration earlier this…
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