Margarida Azevedo, MSc,  —

A “National Poll of African-Americans on Sickle Cell Disease Awareness” conducted earlier this year has revealed that, while most African-Americans are familiar with sickle cell disease (SCD), only about one-third are aware that it affects people of African descent in a disproportionate way. The findings highlight the need for more…

The U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation to Altemia Soft Gelatin Capsules, developed for the treatment of sickle cell disease (SCD) in children. Altemia is being developed by Sancilio Pharmaceuticals as an oral, once-daily formulation for children aged 5 to 17 years who…

Two pharmaceutical companies, Bioverativ and Bicycle Therapeutics, have agreed to jointly discover, develop and market novel therapies for sickle cell disease (SCD) and hemophilia. The $10 million accord seeks to identify and develop therapeutic molecules based on Bicycle’s bicyclic peptide product platform to treat rare blood disorders. This new modality combines attributes…

Sancilio Pharmaceuticals has completed enrolling patients in a Phase 2 clinical trial evaluating Altemia (docosahexaenoic acid) as a treatment for children with sickle cell anemia (SCA) and sickle cell disease (SCD). The SCOT trial (NCT02973360) is aimed at finding an optimal dose of the therapy as well as…

The Israeli government has awarded Gamida Cell a $3.5 million grant to advance the development of clinical trials for CordIn, a therapy to treat sickle cell disease (SCD) and thalassemia. The money, granted by the Ministry of Economy and Industry’s Israel Innovation Authority (IIA), will also support further development and sales of…

Sema4, an interdisciplinary health information company, has partnered with Helix, a personal genomics company, to launch CarrierCheck — a simple, saliva-based test that screens for 67 conditions, including sickle cell disease (SCD). The test may provide individuals with a snapshot of how their DNA may affect their future…

Global Blood Therapeutics has expanded its ongoing HOPE-KIDS 1 trial (NCT02850406), evaluating the investigative GBT440 in pediatric patients with sickle cell disease, to include a new single-dose cohort in younger children ages 6 to 11. GBT440 is being developed as an oral, once-daily drug candidate for the treatment…

The Health Resources and Services Administration (HRSA), part of the U.S. Department of Health and Human Services, has awarded the Sickle Cell Disease Association of America (SCDAA) a $2.9 million annual grant for the next four years to establish a national infrastructure to ensure sickle cell disease (SCD) patients receive appropriate follow–up care…

The Oncologic Drug Advisory Committee of the U.S. Food and Drug Administration (FDA) will review Emmaus Life Sciences’s New Drug Application (NDA) for Endari to treat sickle cell disease on May 24. “The Advisory Committee meeting is an important step forward in the review process for this promising therapy,”…

The inaugural World Cord Blood Day on Nov. 15 this year will educate parents and healthcare professionals about the current uses for cord blood transplants, a non-controversial source of stem cells which have the ability to treat more than 80 life-threatening diseases, including sickle cell anemia. To date, more…