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…
November 24, 2022 - 7 minutes
Hemophilia
FDA approves CSL gene therapy for hemophilia B
CSL has revealed that the U.S. Food and Drug Administration (FDA) has approved HEMGENIX (etranacogene…
November 23, 2022 - 5 minutes
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…
November 2, 2022 - 3 minutes
Beyond Biotech podcast 17: BIO-Europe, National Hemophilia Foundation
This week, we have three interviews. Our guests are: Pam Putz, managing director of EBD…
October 7, 2022 - 2 minutes
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…
August 30, 2022 - 2 minutes
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…
August 26, 2022 - 3 minutes
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…
July 22, 2022 - 3 minutes
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…
July 11, 2022 - 4 minutes
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)…
December 20, 2019 - 2 minutes
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…
May 14, 2019 - 3 minutes
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…
May 3, 2018 - 2 minutes
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…
February 28, 2018 - 8 minutes
