News

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…

Introducing a “natural” genetic mutation into the blood cells of people with sickle cell and like diseases, using CRISPR  gene editing, was seen to restore the production of fetal hemoglobin and offer a way to treat these disorders, researchers report. Their study was published in the journal Blood, in an article titled “KLF1 drives the expression…

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…

Couples who wish to know the risk of passing on a genetic disease such as sickle cell anemia to their children may now do so with QHerit, a new screening test by Quest Diagnostics. The QHerit Pan-Ethnic Expanded Carrier Screen test covers 22 genetic diseases and was produced…

Doctors treating pediatric patients with sickle cell disease (SCD) for pain associated with a vaso-occlusive crisis (VOC) often have few optimal therapy options. But a recent study reviewed a technique that shows potential for mitigating pain in child SCD patients who are experiencing VOC.

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…

Endari (L-glutamine) today became the first therapy in almost 20 years to win approval from the U.S. Food and Drug Administration to treat sickle cell disease. In giving the green light to Endari use in patients starting at age 5, the FDA also made the oral drug the first ever…

Patients with sickle cell anemia who do not have alpha-thalassemia and the genetic rs1427407 T variant of the BCL11A gene may be at higher risk for hemolysis (rupture of red blood cells) and stroke, according to the results of studies of three independent groups. These findings were featured in…

The European Medicines Agency (EMA) has granted GBT440 — Global Blood Therapeutics’ lead drug candidate for sickle cell disease (SCD) — access to its Priority Medicines (PRIME) program. GBT440 is an oral, once-daily drug being developed to specifically inhibit hemoglobin polymerization and the consequent sickling of red blood cells, the hallmark…