News

Researchers at the University of York in the U.K. have been awarded a $3.1 million (£2.3 million) grant to accelerate research supporting stem cell gene therapy to treat sickle cell disease (SCD). The grant was awarded by the nonprofit Bill & Melinda Gates Foundation to track genetic changes in…

Agios Pharmaceuticals has launched a Phase 2/3 trial called RISE UP to test its investigational therapy mitapivat (AG-348) in people with sickle cell disease (SCD). The trial (NCT05031780), which is not yet recruiting, aims to enroll about 237 people with SCD, ages 16 and older. To be…

The National Organization for Rare Disorders (NORD) asks Americans to plan ahead to participate in the Light Up for Rare campaign to raise awareness of rare diseases. NORD is the U.S. sponsor for Rare Disease Day on Feb. 28. The annual awareness day spotlights approximately 7,000…

Regina Hartfield is set to become the president and CEO of the Sickle Cell Disease Association of America (SCDAA) on Jan. 14. She brings with her experience ranging from project manager at Memorial Sloan-Kettering Cancer Center and community relations coordinator at The New York Botanical Garden to, most recently,…

A clinical program for lovotibeglogene autotemcel (lovo-cel), an investigational gene therapy for sickle cell disease (SCD), has been suspended for patients under 18 by the U.S. Food and Drug Administration (FDA). Bluebird Bio announced the partial suspension of its program due to an ongoing investigation into persistent, nontransfusion-dependent…

The U.S. Food and Drug Administration (FDA) has agreed to expand the use of Oxbryta (voxelotor) — the first approved therapy targeting the underlying cause of sickle cell disease (SCD) — to children as young as 4. The therapy previously was approved for SCD patients ages 12 and older.

Higher doses of FTX-6058, an investigational oral treatment for sickle cell disease (SCD), effectively raised hemoglobin messenger RNA (mRNA) levels above those found at lower doses in healthy volunteers participating in a Phase 1 trial, new data suggests. Increases in hemoglobin mRNA — the molecule cells use as…

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.