Hemophilia Archives

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…

June 30, 2023 - 5 minutesmins - By Jim Cornall

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…

April 18, 2023 - 10 minutesmins - By Roohi Mariam Peter

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…

March 3, 2023 - 8 minutesmins - By Roohi Mariam Peter

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…

February 28, 2023 - 3 minutesmins - By Roohi Mariam Peter

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…

February 28, 2023 - 2 minutesmins - By Jim Cornall

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. …

February 24, 2023 - 4 minutesmins - By Jim Cornall

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…

December 16, 2022 - 2 minutesmins - By Jim Cornall

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…

November 24, 2022 - 7 minutesmins - By Jonathan Smith

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…

November 23, 2022 - 5 minutesmins - By Jim Cornall

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…

November 2, 2022 - 3 minutesmins - By Liza Laws

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…

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…

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…

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…

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…

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…

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…

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…

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…

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…

December 26, 2017 - 6 minutesmins - By Evelyn Warner

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