News

Researchers were able to mend the mutated gene that causes sickle cell disease, and successfully transfer the corrected stem cells into mice. Their work could have enormous implications for millions worldwide, as it is proof-of-concept that such a treatment might also work in humans. The study, “CRISPR/Cas9 β-globin gene targeting in human haematopoietic…

Imara is scheduled to present preclinical efficacy and safety data on IMR-687, its lead product candidate for the treatment of sickle cell disease (SCD), at the American Society of Hematology (ASH) 58th Annual Meeting and Exposition in San Diego, Calif., Dec. 3-6. The oral presentation, titled “A Novel,…

The U.S. Food and Drug Administration (FDA) has accepted for review a New Drug Application (NDA) from Emmaus Life Sciences for its orally administered pharmaceutical grade L-glutamine (PGLG) treatment for sickle cell disease. If the FDA approves this new treatment, it would be the first FDA-approved medicine available…

The U.S. Food and Drug Administration (FDA) has approved the use of Immucor’s PreciseType HEA test to screen blood donors for sickle cell trait (SCT), an inherited blood disorder that affects 1 million to 3 million Americans. SCT is not a disease, but people with the disorder…

Researchers have cured mice with a genetic blood disease by correcting mutated genes in blood cell-producing stem cells. The method offers a new therapeutic approach for treating blood disorders, such as sickle cell disease and thalassemia. The gene-editing technique can be administered easily into living animals and notably decreases off-target effects…

Treatment outcomes for people with sickle cell disease (SCD) have improved significantly in the past few decades. Today, over 90 percent of children who are born in Western countries with the genetic condition will live to adulthood. This is a remarkable achievement, as SCD was once synonymous with childhood mortality…

La Jolla’s investigational therapy LJPC-401 (synthetic human hepcidin) to treat sickle cell disease has been recommended for orphan drug designation through a positive opinion issued this week by the European Medicines Agency‘s (EMA) Committee for Orphan Medicinal Products (COMP). “We are encouraged by the positive feedback and continued support of…

National and local policies have led to large reductions in lead poisoning cases overall in the U.S., but severe cases are still common among children. Recently, researchers at the New York City Department of Health and Mental Hygiene (DOHMH) investigated sources of exposure, risk factors, and outcomes for children with severe lead poisoning. Sickle cell…

For the first time, scientists were able to correct the genetic mutation that causes sickle cell disease in stem cells. In a collaborative effort, researchers at UC Berkeley, UC San Francisco Benioff Children’s Hospital Oakland Research Institute (CHORI), and the University of Utah School of Medicine fixed the mutation…

A published case study reports that patients with mildly symptomatic sickle cell disease (SCD) can exceed the U.S. median life expectancy  of 47 years for patients with the disease if it is managed properly. The report published in Blood, the Journal of the American Society of…