Best in Biotech 29 Apr 2025 Six biotech companies leading the charge in hemophilia treatment Discover six hemophilia companies developing new treatments for the genetic bleeding disorder, from gene therapies to RNAi therapies. April 29, 2025 - 10 minutesmins - By Willow Shah-Neville Share WhatsApp Twitter Linkedin Email
News and Trends 9 Jan 2025 Von Willebrand disease: Star Therapeutics, Chugai and Band Therapeutics are breaking new ground Explore advances in von Willebrand disease treatment, including Star Therapeutics’ VGA039, and other key players in the space. January 9, 2025 - 7 minutesmins - By Jules Adam Share WhatsApp Twitter Linkedin Email
Best in Biotech 22 Jan 2024 Eight newly approved drugs that could become blockbusters in 2024 The much-awaited Drugs to Watch list by Clarivate is out. Let’s do a deep dive into the therapies that made the cut in 2024. January 22, 2024 - 9 minutesmins - By Roohi Mariam Peter Share WhatsApp Twitter Linkedin Email
In Depth 16 Nov 2023 uniQure: is the gene therapy pioneer en route to becoming a biotech rebuff? Once thought to be a frontrunner in gene therapy, uniQure’s fame may no longer be coveted, as the biotech looks to reorganize by slashing its workforce, in an attempt to stay afloat. But how did things go awry for the company that was once in its prime in the DNA game? The Danish company, which […] November 16, 2023 - 7 minutesmins - By Roohi Mariam Peter Share WhatsApp Twitter Linkedin Email
News and Trends 30 Jun 2023 FDA approves BioMarin gene therapy for hemophilia A BioMarin Pharmaceutical Inc. has announced that the United States Food and Drug Administration (FDA) has approved ROCTAVIAN (valoctocogene roxaparvovec-rvox) gene therapy for the treatment of adults with severe hemophilia A (congenital factor VIII (FVIII) deficiency with FVIII activity < 1 IU/dL) without antibodies to adeno-associated virus serotype 5 (AAV5) detected by an FDA-approved test. The […] June 30, 2023 - 5 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
In Depth 18 Apr 2023 Gene therapy: a powerful tool for hemophilia B? The approval of HEMGENIX, the first-ever gene therapy for hemophilia B, by the U.S. Food and Drug Administration (FDA) followed by the European Commission, has been momentous in rare disease therapeutic research, paving the way for various gene therapies that are currently being studied. As we observed World Hemophilia Day on April 17, let us […] April 18, 2023 - 10 minutesmins - By Roohi Mariam Peter Share WhatsApp Twitter Linkedin Email
Interview 3 Mar 2023 Interview: how has the first gene therapy for hemophilia B transformed research? Hemophilia B, a rare disease that affects more than 230,000 people worldwide is caused by the lack of or defective clotting protein factor IX often due to a spontaneous mutation in the F9 gene, leading to excessive bleeding. Until recently, routine prophylactic infusions of factor IX replacement therapy to maintain enough clotting factor to prevent […] March 3, 2023 - 8 minutesmins - By Roohi Mariam Peter Share WhatsApp Twitter Linkedin Email
News and Trends 28 Feb 2023 European Commission approves first gene therapy for hemophilia B The European Commission has approved the first gene therapy for the treatment of hemophilia B developed by biotech company CSL Behring. The one-time gene therapy etranacogene dezaparvovec sold under the brand name Hemgenix is manufactured by UniQure Inc. and distributed by CSL Behring. The gene therapy is used to treat severe and moderately severe hemophilia […] February 28, 2023 - 3 minutesmins - By Roohi Mariam Peter Share WhatsApp Twitter Linkedin Email
News and Trends 28 Feb 2023 GC Biopharma acquiring hemophilia rare disease pipeline GC Biopharma Corp., a South Korean provider of biopharmaceutical products, has signed an asset purchase agreement with Catalyst Biosciences to acquire three programs related to the orphan hematology disorders. The agreement includes Marzeptacog alfa (MarzAA), an engineered factor VIIa, which is ready for phase 3 clinical stage development. In its previous clinical development trials, MarzAA […] February 28, 2023 - 2 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 24 Feb 2023 Sanofi hemophilia drug gets FDA approval The U.S. Food and Drug Administration (FDA) has approved Sanofi’s ALTUVIIIO [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein-ehtl], previously referred to as efanesoctocog alfa, a first-in-class, high-sustained factor VIII replacement therapy. ALTUVIIIO is indicated for routine prophylaxis and on-demand treatment to control bleeding episodes, as well as perioperative management (surgery) for adults and children with hemophilia […] February 24, 2023 - 4 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 16 Dec 2022 CSL closer to gene therapy treatments for hemophilia B patients in Europe CSL says the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending conditional marketing authorization (CMA) of etranacogene dezaparvovec. Etranacogene dezaparvovec is a one-time gene therapy for the treatment of appropriate adults with hemophilia B that reduces the rate of annual bleeds with a single infusion. […] December 16, 2022 - 2 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 24 Nov 2022 Gene therapy approval “game changer” in hemophilia B treatment The approval of CSL’s gene therapy Hemgenix has ushered in a new era for hemophilia B treatment. With the addition of other approvals like Rocktavian and Upstaza, 2022 could be a turning point for the broader gene therapy field. Earlier this week, etranacogene dezaparvovec (Hemgenix) made history as the first gene therapy approved by the […] November 24, 2022 - 7 minutesmins - By Jonathan Smith Share WhatsApp Twitter Linkedin Email