GS1191, a gene therapy in the pipeline of Gritgen Therapeutics, increased factor VIII (FVIII) activity in the blood of people with hemophilia AÂ taking part in a small clinical trial in China, resulting in fewer bleeding episodes. The fully enrolled investigator-initiated trial (ChiCTR2300073179) is testing the safety and…
News
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…
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…
Hemgenix (etranacogene dezaparvovec), the first and only gene therapy approved for adults with hemophilia B, has won this year’s Prix Galien USA award in the category of Best Product for Rare/Orphan Diseases. The award — the nation’s top prize for leading-edge advances in life sciences — was received…
The first participant has been dosed in a Phase 2b clinical trial that’s assessing the safety and efficacy of SerpinPC in people with hemophilia B who have inhibitors, according to an announcement from its developer, Centessa Pharmaceuticals. “We are excited to be further evaluating the potential of…
Health Canada has approved the gene therapy Hemgenix (etranacogene dezaparvovec) for treating adults with hemophilia B who rely on routine prophylactic therapies to prevent or reduce bleeding episodes. Hemgenix is now the first and only gene therapy available to this patient group. “The approval of Hemgenix in Canada…
This year’s Hope Conference, a national educational event presented by Hope Charities for the bleeding disorders community, is set for Nov. 2-4 in Florida, with an online option as well. The annual conference, to be presented at the Baytowne Conference Center, in Miramar Beach, is designed to bring together…
People with hemophilia are living longer, but Black males still are more likely to die from the disease, and at a younger age, compared with white people, a U.S. study finds. More research is needed to understand the reason for this difference and to improve care and survival rates…
Venture philanthropy fund Pathway to Cures (P2C)) has invested $500,000 in Five Liters, which is developing noninvasive, nonpharmacological neuromodulation therapies for treating inheritable blood and bleeding disorders, such as hemophilia and von Willebrand disease (VWD). “Investing in Five Liters’ early-stage research is a critical step in addressing unmet…
Two longtime bleeding disorders advocates, who are the parents of a son with hemophilia B, have made a founding donation to Pathway to Cures — dubbed P2C — the new venture philanthropy fund of the National Bleeding Disorders Foundation (NBDF). The transformational gift was added to the estate…
Recent Posts
- Little interest in gene therapy for many with severe hemophilia: German study
- With bleeding disorders, how do we know when pain isn’t ‘just pain’?
- 17-year-old advocate shares her NMOSD story to raise awareness
- Accommodations won’t keep us from enjoying a day at the museum
- I unpack the past with hemophilia and discover the strength of my adult son