uniQure Archives

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

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

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

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

Best in Biotech 20 Dec 2021

The Billion-Euro European Biotech Companies in 2022

Here’s a list of the public European biotech companies that are worth more than a billion euros in 2022. European biotech is growing steadily and this year we welcome a few new names to the list of billion-euro biotechs. However, we also had to say goodbye to some of them, including Cellectis, Autolus, and Orchard […]

December 20, 2021 - 12 minutesmins - By Larissa Warneck-Silvestrin

Best in Biotech 2 Sep 2021

The top 15 biotech companies to find in the Netherlands

The Netherlands has established itself as a biotech and life sciences hub, possessing leading academic and research institutions and an excellent business infrastructure that makes it an attractive place for biotech companies to grow and thrive. In this article, we take a look at some of the top biotechs in the country. The Netherlands is […]

September 2, 2021 - 7 minutesmins - By Clara Rodríguez Fernández

Best in Biotech 28 Dec 2020

A Year to Remember: The Biggest European Biotech News in 2020

The Covid-19 pandemic caused huge disruption for European biotech in 2020, but the industry pulled through. Let’s look back at some of the top moments in the last 12 months. After the many drug approvals, startup foundations, and venture capital highs we saw in 2019, it looked like 2020 was going to be a comfortable […]

December 28, 2020 - 12 minutesmins - By Jonathan Smith

News and Trends 17 Dec 2020

Covid-19 Leaves Lasting Impact on Hematology at ASH 2020 Conference

Under the heavy shadow of the Covid-19 pandemic, last week’s annual conference of the American Society of Hematology (ASH) displayed the achievements of many innovative treatments for blood conditions, including gene and cell therapies. A sweeping new wave of Covid-19 infections that is forcing new lockdowns across the northern hemisphere is already reshaping the biotech […]

December 17, 2020 - 6 minutesmins - By Victor Kotsev

News and Trends 26 Jun 2020

CSL Behring Nabs UniQure’s Hemophilia Gene Therapy for up to €1.8B

Big pharma CSL Behring will pay the Dutch gene therapy developer uniQure an upfront of €400M for the global rights to a phase III-stage gene therapy for the blood disorder hemophilia B. Under the terms of the agreement, uniQure will complete an ongoing phase III trial of the gene therapy, with topline results expected […]

June 26, 2020 - 3 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

huntingtons disease gene therapy uniqure2 1

News and Trends 23 Jan 2019

Update: The FDA Green-lights Clinical Trials for Huntington’s Disease Gene Therapy

Update (23/01/2019): The FDA has given uniQure regulatory clearance to begin a phase I/II trial of its gene therapy for Huntington’s disease. uniQure is planning to launch the trial in the US in late 2019. Published on 23/01/2018: The EMA has given uniQure’s gene therapy candidate for Huntington’s disease Orphan Medicinal Product Designation, which the […]

January 23, 2019 - 3 minutesmins - By Alex Dale

News and Trends 6 Dec 2018

Update: Liver Gene Therapy 8 Times Stronger Than Usual

Update (06/12/2018): uniQure has incorporated Synpromics’ strong liver promoter into a preclinical gene therapy candidate for treating hemophilia A. Published on 08/10/2018 Synpromics and uniQure have unveiled new technology for precise genetic control that could make gene therapy safer and more precise. The team has developed a gene therapy system that is highly selective for […]

December 6, 2018 - 2 minutesmins - By Clara Rodríguez Fernández

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