ASC Therapeutics has joined forces with Charles River to scale the manufacturing of ASC618, its second-generation virus-based gene therapy for hemophilia A. The agreement expands upon their current collaboration, begun in 2019 that focused on achieving Good Manufacturing Practice (GMP)-virus manufacturing and establishing processes for adeno-associated virus (AAV) production…
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A therapy designed to promote the growth of regulatory T-cells (Tregs) in the body prevented inhibitor development in a mouse model of hemophilia A that was treated with factor replacement therapy, a study showed. Inhibitors, a type of neutralizing antibody some patients develop that can make replacement therapies…
When administered at the highest dose, the investigational gene therapy SB-525 (giroctocogene fitelparvovec) prevented bleeds and the use of clotting factor VIII (FVIII) in men with severe hemophilia A, according to follow-up data from the Alta Phase 1/2 clinical trial. All five men given the highest dose…
The development of neutralizing antibodies, or inhibitors, against the clotting factor in replacement therapies administered to people with hemophilia may depend on the type of product used, a recent study has found. In previously untreated people with severe hemophilia A, or those who lack…
BioMarin Pharmaceuticals is seeking marketing approval in Europe for its investigational gene therapy, valoctocogene roxaparvovec, for the treatment of adults with severe hemophilia A. The company has submitted a marketing authorization application (MAA) to the European Medicines Agency (EMA) for the experimental gene therapy, formerly…
Switching to a different clotting factor VIII (FVIII) product was not linked to a greater risk of developing neutralizing antibodies against FVIII, or FVIII inhibitors, in people with hemophilia A, a recent study reported. Changing treatment also had no impact on a patient’s immune profile or on therapy efficacy.
I recently asked my husband, Jared, who has hemophilia, a this-or-that question: “Would you rather have many insignificant bleeds several times a month, or big bleeds several times a year that keep you bedridden for an entire month?” Jared chose the former, and I agreed. As a busy mom and entrepreneur,…
Treatment with Octapharma‘s Nuwiq is associated with a low risk of developing inhibitors — antibodies that prevent the medication from working properly — in previously untreated patients (PUPs) with hemophilia A, according to the results of a clinical trial. These findings were published in the journal Thrombosis…
A partnership between ARUP Laboratories and Medicover will improve access to AAV5 DetectCDx, the former’s companion diagnostic test to assess hemophilia A patients’ eligibility to Roctavian (valoctocogene roxaparvovec-rvox) in the European Union (EU). The partnership, which allows testing to be done at one of Medicover’s…
My last home had a great fence. It was a wall of concrete blocks surrounding my backyard and stood 7 feet high. With two dogs who bark at the wind blowing, the wall deterred them from jumping and kept some of their barking at bay. However, there was an iron…