News

Vertex Pharmaceuticals and CRISPR Therapeutics recently announced a joint collaboration to co-develop and co-commercialize CTX001 as a gene-editing treatment of diseases of hemoglobin, including β-thalassemia and sickle cell disease (SCD). The announcement follows the presentation of preclinical data for CTX001 at the American Society of Hematology…

Intellia Therapeutics has used gene editing to replace an oxygen-carrying component in blood that is defective in sickle cell disease. The work involved editing the BCL11A gene in a mouse model of the disease to produce an effective version of the oxygen-carrying component, hemoglobin. Intellia discussed the project at the…

Sickle cell disease patients who have relatively low pain levels use opioids more when they are sad, anxious, or experience other negative emotions, a study indicates. The Johns Hopkins University research was based on entries in 45 patients’ electronic diaries. It suggested that patients misused opioids when they increased their intake…

A new online platform, called oneSCDvoice.com, will help those affected by sickle cell disease (SCD) by offering free access to comprehensive support and a robust knowledge network . rareLife solutions, the health tech, design and research team behind oneSCDvoice and other custom rare disease platforms, will launch the modular community…

Anyah Randolph, a teenager with sickle cell disease (SCD), was recently able to join her school class on a trip to Europe – and even climb the Eiffel Tower in Paris after the elevator broke – thanks to the help of hydroxyurea and Loyola Medicine pediatricians. Anyah had…

Following a review of studies published in the last decade, experts have updated their recommendations for managing a sickle cell disease-related eye complication known as sickle cell retinopathy (SCR). Their paper, “Sickle cell retinopathy: improving care with a multidisciplinary approach,” appeared in the Journal of Multidisciplinary Healthcare. SCR, a…

An independent review board has found Global Blood Therapeutics’ sickle cell disease treatment voxelotor safe, and recommended that its Phase 3 clinical trial continue. The hallmark of sickle cell disease is damage to a red blood cell protein called hemoglobin that carries oxygen to organs and tissue. The damage causes the cells…

The National Organization for Rare Disorders (NORD) says it’s “disappointed and dismayed” after the House of Representatives voted 227-205 last week to repeal the Orphan Drug Tax Credit as part of a U.S. tax reform package. A similar package before the Senate Finance Committee does not repeal the credit…

Howard University researchers have discovered a link between increases in Veillonella gut bacteria and higher levels of pain in sickle cell disease patients. The team presented their findings at the American Physiological Society’s Physiological and Pathophysiological Consequences of Sickle Cell Disease conference in Washington, Nov. 6-8. Sickle cell disease is a blood…

Global Blood Therapeutics (GBT) reported a case study in which a sickle cell disease (SCD) patient was granted compassionate access to the company’s ongoing Phase 3 HOPE study of voxelotor (NCT03036813). The patient had severe, transfusion-refractory anemia and experienced rapid improvements with the therapy.