News

Imara has opened applications for its second-annual “Real Impact” grants for community-based nonprofit organizations that support people with rare blood disorders, such as sickle cell disease (SCD) and beta-thalassemia. Through this year’s initiative, the biopharmaceutical company is awarding up to $150,000 to U.S. organizations that seek to address…

The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a rare disease pursue personal goals through training and education. For a second year, the #RAREis Scholarship Fund — supported by Horizon Therapeutics – will award 35 one-time scholarships, each…

To inform sickle cell disease (SCD) patients and their caregivers about blood transfusions, which are commonly used to treat the disorder, the Sickle Cell Disease Association of America (SCDAA) is launching a set of educational materials. The pictorial booklet for children, and companion brochure for parents and caregivers,…

Treatment with oral Oxbryta (voxelotor) leads to rapid and sustained rises in hemoglobin levels, reduces red blood cell destruction (hemolysis), and improves overall health in adolescents and adults with sickle cell disease (SCD), according to full, nearly 1.5-year data from the Phase 3 HOPE clinical trial. The findings support the long-term…

A higher 600 mg daily dose of FT-4202, a potential disease-modifying oral treatment for sickle cell disease (SCD), was well-tolerated and safe, and improved several measures of red blood cell health after 14 days of treatment, according to…

Global Blood Therapeutics (GBT) has been granted exclusive global rights to two small-molecule research programs for sickle cell disease (SCD) as part of a newly announced agreement between GBT and Sanofi. The two early stage programs,…

The U.S. Food and Drug Administration approved the start of a first clinical trial of CRISPR_SCD001, the first non-viral and CRISPR/Cas9-based gene editing therapy for sickle cell disease (SCD). Both the therapy and the upcoming Phase 1/2 trial — planned to start this summer — are the result of a…

As anyone affected by a rare disease knows, treating the illness while trying to go about everyday life is an expensive undertaking.  But exactly how expensive — in terms of direct and indirect costs across rare disease populations — might still come as a surprise: almost…

Imara is opening higher dose arms in two Phase 2b trials assessing the safety and efficacy of IMR-687, its potential therapy for sickle cell disease (SCD) and beta-thalassemia, in patients with these inherited blood disorders, the company announced. The decision, which applies to the Ardent (NCT04474314) and…

Graphite Bio has raised $150 million in Series B funding to expand and advance the clinical development of its pipeline of investigational gene-editing therapies — including GPH101, a potentially curative treatment for sickle cell disease (SCD). In addition…