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…

For the first time, as part of a U.S. clinical trial, the cell therapy BE-101 has been administered to someone with hemophilia B. Be Biopharma, the company developing BE-101, announced the dosing of the first participant in a first-in-human Phase 1/2 clinical trial called BeCoMe-9 (NCT06611436). The…

Catalyst Biosciences recently announced that top-line data from the Phase 1/2 trial of CB 2679d/ISU304 in patients with hemophilia B will be presented at the 11th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD), Feb. 7-9 in Madrid, Spain. The oral presentation, titled…

What happens when a symptomatic hemophilia carrier needs minor surgery? Will her increased risk of bleeding be recognized? The story of Kim H. came to my attention on Facebook. She is 39 years old and lives in Pennsylvania. I recently interviewed Kim over Zoom, as her story is important.

As I transition to a new job in a new city, I’m meeting new colleagues at work. In these early days of getting to know everyone, I share only what I’m asked. I’ll share more of my story in time, especially since joining a team requires trust and acceptance, but…

My bleeding disorders, hemophilia B and von Willebrand disease, have significantly affected my family. As I’ve aged, my appreciation for my family has grown tremendously. I’ve come to realize that hemophilia‘s impact isn’t just limited to me; it’s also affected my family in profound ways. True, patients…

I thought the worst days were behind me in helping my youngest son navigate his bleeding disorder. Caeleb is 16 years old and a junior in high school. During his younger years, an inhibitor wreaked havoc on my son, causing extreme complications and joint bleeds that took an…

A new online service allows patients with bleeding disorders like hemophilia to share important information about their treatment and bleeds with their healthcare team in real time. MORE: Five tips for living healthily with hemophilia.  The HemaGo XChange portal was created by Novo Nordisk A/S as an update to their existing…

Adults with moderate-to-severe hemophilia engage in less physical activity and have reduced physical fitness relative to healthy adults, according to a recent study. Data also indicated these patients had more indicators of abdominal fat accumulation and more often had high blood pressure than healthy people, which could overall reflect…

Gene therapy company uniQure released new data showing that its lead therapy AMT-060 for severe and moderately severe hemophilia B is safe and effective for up to two years, with adults requiring fewer doses of replacement therapy and showing a marked decrease in the spontaneous bleeding rate. The results support uniQure’s clinical…