Hemophilia Archives

News and Trends 7 Oct 2022

Beyond 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 - 2 minutesmins - By Jim Cornall

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

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

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

beta-thalassemia blood APS antiphospholipid syndrome

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

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

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

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

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

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

News and Trends 19 Dec 2017

This Gene Therapy for Hemophilia Can Reduce Bleeding by 70%

UniQure has published clinical data revealing that its gene therapy for hemophilia B is effective for years and lets patients stop weekly replacement therapy. From its headquarters in Amsterdam, uniQure is developing what could be one of the first gene therapies for hemophilia. A publication in the journal Blood yesterday details the results of a […]

December 19, 2017 - 3 minutesmins - By Clara Rodríguez Fernández

Best in Biotech 18 Dec 2017

Smile! Here are the 12 Biggest Biotech Clinical Successes of 2017

Now that we’ve got the bad news out of the way, we’re here to spread some Christmas cheer with the biggest clinical successes in biotech over the past 12 months. It’s unlikely that one whole year will ever pass without a single hiccup, especially in a field as complex as biotech. For that reason, we […]

December 18, 2017 - 9 minutesmins - By Alex Dale

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