News and Trends 23 Nov 2022 FDA approves CSL gene therapy for hemophilia B CSL has revealed that the U.S. Food and Drug Administration (FDA) has approved HEMGENIX (etranacogene dezaparvovec-drlb), the first and only one-time gene therapy for appropriate adults with hemophilia B. HEMGENIX is approved for the treatment of adults with hemophilia B who currently use factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage or […] November 23, 2022 - 5 minutesmins - By Jim Cornall Share WhatsApp Twitter Linkedin Email
News and Trends 2 Nov 2022 Enzyre secures €12M series A funding to help hemophilia patients test from home Dutch company, Enzyre, which is developing ambulant diagnostic technology for blood coagulation testing, today (November 2) announced it has successfully raised €12 million ($11.9 million) in series A financing. The round was led by new investor, Oost NL, with participation from i&i Biotech Fund, Demcon Investment as technology partner and existing investors, Takeda Ventures and […] November 2, 2022 - 3 minutesmins - By Liza Laws Share WhatsApp Twitter Linkedin Email
News and Trends 7 Oct 2022Beyond Biotech podcast 17: BIO-Europe, National Hemophilia Foundation This week, we have three interviews. Our guests are: Pam Putz, managing director of EBD Group EU; André Hofmann, CEO of biosaxony; and Kevin Mills, chief scientific officer at the National Hemophilia Foundation and Teri Willey, managing director of the National Hemophilia Foundation’s Pathway to Cures. We also have our weekly chat with global commercial […] October 7, 2022 Share WhatsApp Twitter Linkedin Email
News and Trends 30 Aug 2022 Hemophilia drug from Sanofi and Sobi has license application accepted for priority review by FDA The Biologics License Application (BLA) for Efanesoctocog alfa, a drug to treat the rare and life-threatening blood disease, hemophilia A, has been accepted for priority review by the U.S. Food and Drug Administration (FDA). Efanesoctocog alfa (BIVV001) is being developed and commercialized as part of a collaboration between Sanofi and Sobi was given the target […] August 30, 2022 - 2 minutesmins - By Liza Laws Share WhatsApp Twitter Linkedin Email
News and Trends 26 Aug 2022 First gene therapy for adults with severe hemophilia approved by EC The European Commission (EC) has granted conditional marketing authorization for a gene therapy to treat severe hemophilia A in adults it was announced yesterday (August 25). BioMarin Pharmaceutical Inc.’s Roctavian (valoctocogene roxaparvovec) treats the congenital factor 8 deficiency in adults patients without a history of factor VIII inhibitors without detectable antibodies to adeno-associated virus serotype […] August 26, 2022 - 3 minutesmins - By Liza Laws Share WhatsApp Twitter Linkedin Email
News and Trends 22 Jul 2022 People with hemophilia B could be protected from bleeding by a single dose of a drug currently being trialed London-based Freeline Therapeutics Holdings plc has just announced that positive data on treating haemophilia has been published by the New England Journal of Medicine (NEJM). This expands upon previous data for up to three-and-a-half years from the phase 1/2 dose-finding B-AMAZE of the company’s AAVS3 gene therapy candidate, FLT180a for people with the debilitating, hereditary […] July 22, 2022 - 3 minutesmins - By Liza Laws Share WhatsApp Twitter Linkedin Email
News and Trends 11 Jul 2022 Study results reveal bleeding episodes are significantly reduced by hemophilia drug People suffering from a severe blood disorder may only have to take a once-monthly drug following successful results from an ongoing study. Global healthcare company, Sanofi, has been conducting a study into fitusiran prophylaxis and results have shown it reduces bleeds by 61 per cent in people with hemophilia A or B, with or without […] July 11, 2022 - 4 minutesmins - By Liza Laws Share WhatsApp Twitter Linkedin Email
News and Trends 20 Dec 2019 Freeline’s Rare Disease Gene Therapies Get €72M Series C Boost The UK gene therapy biotech Freeline Therapeutics is to receive an investment worth €72M (£61.1M) from the UK VC firm Syncona in a Series C round to fund the clinical development of gene therapies for the blood disorder hemophilia B, Fabry disease, and other rare conditions. Of the €72M commitment, Syncona has already wired €36M […] December 20, 2019 - 2 minutesmins - By Jonathan Smith Share WhatsApp Twitter Linkedin Email
News and Trends 14 May 2019 First Potential Gene Therapy for Hemophilia B Restores Blood Clotting in Phase II A gene therapy developed by the Dutch biotech uniQure increased the activity of a blood clotting protein enough to stop bleeds in patients with the blood disorder hemophilia B. Hemophilia B is a rare genetic condition in which patients have a mutation in the blood clotting protein factor IX (FIX), reducing its activity below 6% […] May 14, 2019 - 3 minutesmins - By Jonathan Smith Share WhatsApp Twitter Linkedin Email
News and Trends 3 May 2018 World’s First Gene Therapy Developer Raises $128M for Hemophilia and Huntington’s uniQure has raised a substantial public offering on the NASDAQ. The funding could help the Dutch biotech advance two new gene therapies after the withdrawal of its first treatment. Amsterdam-based uniQure has raised an ambitious $128M (€107M) in a public offering at the beginning of next week. The Dutch biotech develops gene therapies for hemophilia and Huntington’s disease, for […] May 3, 2018 - 2 minutesmins - By Alexander Burik Share WhatsApp Twitter Linkedin Email
News and Trends 28 Feb 2018 Like Finding a Needle in a Haystack: How Are Biotechs Fighting Rare Diseases? Rare diseases affect fewer than 1 in 2,000 people in Europe and less than 200,000 Americans at any given time. A lack of financial incentive has put pharma off but biotechs are keen to step in and develop treatments and cures. Over 6,000 rare diseases affect up to 30 million people in the EU alone, which highlights […] February 28, 2018 - 8 minutesmins - By Alex Dale Share WhatsApp Twitter Linkedin Email
Best in Biotech 26 Dec 2017 Interviews, Reviews and Tops: Our Best Biotech Articles from 2017 We love hearing from our readers, and we listen to what they like! Here are the long reads from our best biotech articles of 2017. We gave you a list of books to read this holiday season, but do you need something lighter for the post-Christmas daze? We’ve collected our most popular long articles of […] December 26, 2017 - 6 minutesmins - By Evelyn Warner Share WhatsApp Twitter Linkedin Email