News

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…

Over $18 million has been given to support research into how best to assist young people with sickle cell disease (SCD) moving from pediatric to adult care. The award comes from the board of governors of the Patient-Centered Outcomes Research Institute (PCORI). Transiting to adult care can be difficult, as children…

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…

New therapies to treat sickle cell disease (SCD) may receive a boost after CSL Behring‘s recent $91 million acquisition of Calimmune. CSL Behring will now develop Calimmune’s pre-clinical product, CAL-H — a gene therapy candidate based on hematopoietic stem cells (HSC), or those that generate all the blood cells in…

With drug overdoses now the leading cause of accidental death in the U.S., efforts are underway in many states to limit abuse and access to prescription opioids —  steps that, some say, could cause unnecessary suffering among sickle cell disease (SCD) patients who rely on these medications to treat their symptoms. “I’m seeing…

Researchers at Brown University have developed computer models that recreate what happens with hemoglobin molecules inside red blood cells  affected by sickle cell disease. Their study, “Mesoscopic Adaptive Resolution Scheme toward Understanding of Interactions between Sickle Cell Fibers,” appeared in the Biophysical Journal. It describes a virtual model authors hope…

The new Stanford Center for Definitive and Curative Medicine will foster the development of stem cell and gene therapies for genetic diseases, including sickle cell anemia. More than 280 million people around the world have diseases with genetic causes, experts estimate. While research has identified the underlying causes of several, scientists have developed…

The Children’s National Health System hospital in Washington has recruited two of the country’s leading hematology and sickle cell disease experts to lead its cutting-edge blood disorders program. Dr Suvankar Majumdar will head the hospital’s Division of Hematology, one of the country’s largest and most comprehensive blood disorders programs. It is…

The U.S. Food and Drug Administration (FDA) approved Imbruvica (ibrutinib) for treatment of adult patients with chronic graft-versus-host-disease (cGVHD) who failed to respond to other forms of systemic therapy, AbbVie announced. FDA’s decision means Imbruvica is the first and only therapy specifically approved for adults with cGVHD, a serious outcome that…

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…