Author Archives: Ana Pena, PhD

Bronchodilators used to treat asthma could worsen sickle cell disease (SCD) in children and teens, and contribute to a greater risk of vaso-occlusive crisis (VOC), a study suggests. The study, “Salbutamol Worsens the Autonomic Nervous System Dysfunction of Children With Sickle Cell Disease”…

FT-4202, a potential disease-modifying oral treatment for sickle cell disease being developed by Forma Therapeutics, has been given fast track and rare pediatric disease designations by the U.S. Food and Drug Administration (FDA). A fast track status is meant to accelerate the development and review…

Jasper Therapeutics has raised an additional $14.1 million to finance the development of its investigational conditioning therapy, JSP191, as a safer and more effective treatment to prepare patients for stem cell transplant. The investment, led by Roche Venture Fund and sponsored by other investors, increases the company…

The U.S. Food and Drug Administration (FDA) has awarded $2 million to support a Phase 2 clinical trial testing whether daily supplementation of vitamin D reduces the risk of respiratory complications in children and adolescents with sickle cell disease (SCD). Gary M. Brittenham, MD, a pediatrician…

A multidisciplinary plan implemented at Yale New Haven Children’s Hospital was able to cut by more than half the number of days young sickle cell disease (SCD) patients were hospitalized for a vaso-occlusive crisis and lower their readmission rates, a study reports.

ReachBio Research Labs has created a drug screening platform to discover therapeutic compounds that trigger the production of fetal hemoglobin (HbF) to treat sickle cell disease (SCD) and other blood disorders. SCD belongs to a group of inherited disorders known as hemoglobinopathies, primarily caused by mutations in…

The U.S. Food and Drug Administration (FDA) accepted Novartis’ Biologics License Application (BLA) seeking marketing permission for crizanlizumab (SEG101), a potential treatment for vaso-occlusive crises (VOCs) in people with sickle cell disease (SCD). The agency granted the file priority review, to accelerate the product’s evaluation. “The…

Chronic pain in adults with sickle cell disease (SCD), a frequent symptom of the disease, is associated with alterations in the connection of brain regions involved with pain perception, a study indicates. When SCD patients with chronic pain were compared to those without it, multiple differences were found…

IMR-687, an investigational therapy for sickle cell disease, has been granted Fast Track designation by the U.S. Food and Drug Administration (FDA). It’s currently being evaluated in a Phase 2a trial in the U.S. and U.K. The Fast Track process is meant to get new therapeutics that treat…

The California Institute for Regenerative Medicine (CIRM) and the National Heart, Lung and Blood Institute (NHLBI) have entered into a “landmark” collaboration to co-fund and help speed the development of cell and gene therapies to cure sickle cell disease (SCD), according to a press release.