GenScript ProBio and Neoletix Biotechnology have entered into an agreement for clinical and commercial production of a lab-made version of coagulation factor VIII, a product that the companies hope will make the preventive treatment for hemophilia A more accessible to patients in China. Coagulation factor VIII, or FVIII,…

The investigational gene therapy Roctavian continues to effectively and safely prevent bleeding episodes and the need for clotting factor VIII replacement therapy in adults with severe hemophilia A, five-year data from a Phase 1/2 study show. BioMarin Pharmaceutical, the therapy’s developer, plans to share the data in…

Recently, I preached a sermon that focused on advocacy. The basis of the message came from the Gospel of Mark 6:53-56. In the Scripture passage, people carried their friends and loved ones on mats to Jesus’ feet so that those who suffered from illnesses could touch his garment and…

The European Medicines Agency (EMA) has accepted BioMarin Pharmaceutical’s request for accelerated assessment of Roctavian, the company’s investigational gene therapy for severe hemophilia A, for a second time. Last year, BioMarin’s regulatory applications for Roctavian (valoctocogene roxaparvovec) got rejected both in Europe and in the…

Lately, my husband, Jared, and I have been working on home improvement goals. Since the pandemic lockdowns began, couples all over the Philippines have been boasting of their improved homes. With many employees now working from home, several people have both the time and the initiative to renovate and…

The first participant has been dosed in a Phase 1/2 clinical trial evaluating marzeptacog alfa activated (MarzAA) to treat bleeding episodes in patients with factor VII (FVII) deficiency, Glanzmann thrombasthenia, and hemophilia A with inhibitors receiving treatment with…

I learned how to be a caregiver from my mother. Ruby Jane loved my children with complete devotion. While I made her a grandmother, she made me the man I am today. I observed her care for not only family but humanity. She spent years serving as the…

The first participant has been dosed in a Phase 3 clinical trial testing marzeptacog alfa activated (MarzAA), an experimental under-the-skin therapy for hemophilia A and B patients with inhibitors. The study, called Crimson-1 (NCT04489537) and sponsored by the therapy’s developer, Catalyst Biosciences, is currently recruiting patients at sites…

I was recently browsing social media when I randomly clicked on a suggested article titled “34 Things You Need to Give Up to Be Successful.” As an entrepreneur, I get lots of suggested links to articles about success in business and life. I reflected upon what I had just…

Hemophilia A patients with factor VIII (FVIII) inhibitors show deficiencies in regulatory B- and T-cells — immune cells that typically dampen immune and inflammatory responses — but their levels can be restored with successful immune tolerance induction (ITI), a study shows. These findings shed light on how ITI — given…