Stable and Durable Factor VIII Expression in Subjects Dosed with Investigational Hemophilia A Gene Therapy
More than two years after receiving Spark Therapeutics’ investigational adeno-associated virus (AAV)-mediated gene therapy (SPK-8011), five adult subjects with hemophilia A exhibited stable factor VIII (FVIII) expression, with no evidence of FVIII inhibitors or a cellular immune response.
New Site Tracks Vaccines and Drugs/Treatments in Development for COVID-19
BioRender, an online tool for creating scientific figures, has created a robust, interactive COVID VacTracker that is maintained regularly and updated daily.
CoVIg-19 Plasma Alliance Expands Membership and Clinical Trial Collaboration
The CoVIg-19 Plasma Alliance now includes 11 plasma companies, and now also includes global organizations from outside the plasma industry.
BCG Vaccine Being Studied as Potential Protection Against COVID-19
Because research has shown the Bacillus Calmette-Guérin (BCG) tuberculosis vaccine boosts production of immune cells, it is currently being trialed in people as potential protection against COVID-19.
Studies Show Influenza and Pneumonia Vaccines May Reduce Risk of Developing Alzheimer’s
Two studies show the influenza (flu) and pneumonia vaccines lessen the risk of developing Alzheimer’s disease (AD) in the future.
FDA Approves Uplizna to Treat Rare Autoimmune Disease of the CNS
The U.S. Food and Drug Administration (FDA) has approved inebilizumab-cdon (Uplizna) to treat neuromyelitis optica spectrum disorder (NMOSD) in adult patients with the anti-AQP4 antibody.
Proposed Payment Rules and Court Rulings
Two court rulings and segments of the proposed OPPS/ASC and PFS rules may require action from providers to recover expenses caused by the COVID-19 pandemic.
Rituxan Superior to Mycophenolate Mofetil in Sustaining Complete Remission in Pemphigus Vulgaris Patients
Results from Genentech’s Phase III PEMPHIX study evaluating the efficacy and safety of Rituxan (rituximab) compared to mycophenolate mofetil (MMF) in adults with moderate to severe pemphigus vulgaris (PV) showed the study met the primary endpoint at week 52 and demonstrated Rituxan is superior to MMF.
Large-Dose Albumin Supplementation Does Not Reduce Mortality or Need for Critical Care Interventions: Retrospective Review
Investigators conducted a retrospective review of 38 cases treated at Taipei Veterans General Hospital between January 2007 and December 2018 to determine whether more aggressive albumin supplementation can benefit major burn patients with persistent hypoalbuminemia.
Bleed Protection with up to 14-Day Dosing of Long-Acting Recombinant Factor IX Product in Selected Children with Hemophilia B
In pharmacokinetic studies, a recombinant fusion protein genetically linking human coagulation factor IX with human albumin (rIX-FP) (IDELVION, CSL Behring) has been shown to have an approximately five-fold longer half-life compared with standard recombinant factor IX products.
