A first patient has been dosed in a Phase 1/2a clinical trial that’s assessing the safety and preliminary efficacy of ASC618, a second-generation gene therapy for hemophilia A. The Phase1/2a study (NCT04676048), which has been in the works since 2021, aims to enroll about 12 men with severe…

Eligible patients with severe hemophilia A in Germany should now have improved access to Roctavian (valoctocogene roxaparvovec-rvox) — a gene therapy intended to reduce bleed risk in certain adults with the bleeding disorder — given a new agreement between its developer BioMarin Pharmaceutical and the German National Association…

Last month, I had the privilege and the honor of being one of 60 attendees at the Mild Matters Summit in Tulsa, Oklahoma, organized by the Hemophilia Federation of America (HFA). The summit aimed to empower those with mild bleeding disorders by providing essential information and tools to…

The holiday season is upon us. Often, we have our meal around a table with people we love, and we give thanks for one another’s company. The holidays are all about rituals — preparing feasts, gathering to exchange gifts, and focusing on the things that unite us. Year after…

A phone call in the wee hours of the morning makes the heart skip a beat, as it often brings bad news. A baby crying in the middle of the night means a sleep-deprived parent going through the motions of changing diapers and warming bottles. When the quiet hours of…

Adding an antibody targeting the anticoagulant protein S to factor IX (FIX) replacement therapy accelerated blood clotting in samples from children with hemophilia B, including those with severe disease, a study showed. Greater production of the blood clotting protein thrombin also was seen in these samples, while blocking…

AAV5 DetectCDx, a companion diagnostic test used to determine patient eligibility for the hemophilia A gene therapy Roctavian (valoctocogene roxaparvovec-rvox), has been cleared for use under more stringent European Union rules coming into effect in a few years. Specifically, the test gained the Conformité Européenne (CE) mark under…

Whenever I scroll through my Instagram feed, I often reflect on the “what ifs” in my life. For example, what if my life had unfolded exactly as I had envisioned it during my youth? As a child, I was a dreamer with countless aspirations. I envisioned living in a different…

Genevant Sciences is teaming up with Novo Nordisk to develop a gene-editing treatment for hemophilia A that combines Genevant’s proprietary lipid nanoparticle (LNP) platform with cutting-edge mRNA-based megaTAL technology. The partners will seek to advance a treatment involving gene editing — used to correct, add, or delete…

As a former band director, I understand the importance of each musical instrument in an ensemble. The percussion keeps the rhythm steady. The low brass offers the foundation of the music, and the trumpets, flutes, and clarinets play the melodies. Each section contributes to the overall piece. Music is an…