Hemophilia Archives

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

In Depth 28 Nov 2017

Reviewing Gene Therapy: How Close is Biotech to Genetic Cures?

Over 10,000 human diseases are caused by exactly one faulty gene. Gene therapy promises to fix each of them. Here’s a review of biotech’s progress. Biotech is arguably built on genetic medicine. When Genentech‘s insulin produced by genetically engineered bacteria kicked off biotech fever in the 1980s, the concept of directly repairing defective human DNA wasn’t […]

November 28, 2017 - 8 minutesmins - By Evelyn Warner

News and Trends 2 Nov 2017

Close to Launching Gene Therapy, Dutch Biotech Raises Over $91M on the Nasdaq

Update (2/11/2017): uniQure has closed its public offering raising over $91m (€78M) on the Nasdaq, taking advantage of a massive stock jump triggered by the announcement that an improved version of its gene therapy for hemophilia B will enter Phase III trials. Originally published on 24/10/2017 UniQure launched a public offering, perfectly timed with a rise in […]

November 2, 2017 - 3 minutesmins - By Clara Rodríguez Fernández

News and Trends 19 Oct 2017

Dutch Biotech plans Pivotal Clinical Trial with First Gene Therapy for Hemophilia

uniQure is getting ready to start a Phase III clinical trial in 2018 with a new version of its gene therapy for hemophilia B. uniQure has officially launched a plan to bring the first gene therapy for hemophilia to the market. After multiple meetings with the FDA and the EMA, the Dutch biotech has received permission […]

October 19, 2017 - 2 minutesmins - By Clara Rodríguez Fernández

Bicycle Therapeutics Bioverativ Hemophilia blood disorders

News and Trends 6 Sep 2017

A €344M Partnership will Develop Bicycle Drugs for Hemophilia

Bicycle Therapeutics has partnered with Bioverativ to develop a new class of drugs for patients with rare blood disorders like hemophilia and sickle cell disease. Bicycle Therapeutics, a money-magnet biotech founded by Greg Winter, has closed a deal with US firm Bioverativ for the discovery, development and commercialization of bicycle drugs for rare blood disorders. […]

September 6, 2017 - 2 minutesmins - By Clara Rodríguez Fernández

novimmune shire hemophilia antibody roche

News and Trends 18 Jul 2017

Shire In-Licenses Swiss Antibody for Hemophilia A to Compete with Roche

Shire has bought exclusive worldwide rights to develop and commercialize a bispecific antibody from Novimmune that could improve the treatment of hemophilia A. Novimmune is a biotech from Geneva that specializes in developing antibodies. Back in 2015, it struck a research collaboration with Baxalta, which has since been acquired Shire. The Irish pharma has now decided to […]

July 18, 2017 - 2 minutesmins - By Clara Rodríguez Fernández

News and Trends 10 Jul 2017

Sanofi and US Partner Launch Phase III Trial in Hemophilia with RNAi

Alnylam and its partner Sanofi are starting a Phase III program to enter its RNAi drug fitusiran in the competitive hemophilia market. Sanofi and its partner Alnylam have announced the start of a Phase III program that seeks to recruit 250 patients to test its RNAi drug fitusiran in patients with both hemophilia A and […]

July 10, 2017 - 3 minutesmins - By Clara Rodríguez Fernández

Trevor Baglin XO1 SuperX Virtual Biotech

Interview 19 Jun 2017

Discussing the Virtual Biotech Model with XO1 Co-Founder Trevor Baglin

Academic, entrepreneur and now venture partner Trevor Baglin discusses his successful experience working with the unique model of virtual biotech. Trevor Baglin, along with Jim Huntington and David Grainger, is one of the members of the team behind XO1, a virtual biotech founded in 2013 and acquired by Janssen Pharmaceuticals just two years later […]

June 19, 2017 - 7 minutesmins - By Clara Rodríguez Fernández

News and Trends 25 Apr 2017

EMA designates uniQure’s gene therapy for hemophilia a priority

Following a Breakthrough Designation from the FDA earlier this year, the Dutch biotech has scored the European equivalent for AMT-060. Since its claim to fame, Glybera, fell from grace to become a commercial failure, uniQure has been on the rebound. The company recently has undergone several management shakeups and a strategy review, and it just made the decision to withdraw […]

April 25, 2017 - 3 minutesmins - By Evelyn Warner

News and Trends 28 Feb 2017

It’s Global Rare Disease Day! What Cures is Biotech Cooking Up?

We’ve compiled a list of some of the latest advances of biotech in the field of rare disease to contribute to Rare Disease Day 2017. The European Organization for Rare Diseases (EURORDIS) is once again coordinating Rare Disease Day, which takes place on the last day of February every year. The aim is to raise awareness of […]

February 28, 2017 - 5 minutesmins - By Clara Rodríguez Fernández

News and Trends 30 Jan 2017

The FDA gives Fast Track Priority to uniQure’s Hemophilia B Gene Therapy

UniQure has received Breakthrough Therapy Designation from the FDA, granting fast track to its gene therapy for hemophilia B. UniQure is one of the companies leading the development of gene therapy for hemophilia. Now, the FDA has granted Breakthrough Therapy Designation to its candidate AMT-060 for hemophilia B. This is awarded to selected therapies that have […]

January 30, 2017 - 2 minutesmins - By Clara Rodríguez Fernández

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Hemophilia

This Gene Therapy for Hemophilia Can Reduce Bleeding by 70%

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

Reviewing Gene Therapy: How Close is Biotech to Genetic Cures?

Close to Launching Gene Therapy, Dutch Biotech Raises Over $91M on the Nasdaq

Dutch Biotech plans Pivotal Clinical Trial with First Gene Therapy for Hemophilia

A €344M Partnership will Develop Bicycle Drugs for Hemophilia

Shire In-Licenses Swiss Antibody for Hemophilia A to Compete with Roche

Sanofi and US Partner Launch Phase III Trial in Hemophilia with RNAi

Discussing the Virtual Biotech Model with XO1 Co-Founder Trevor Baglin

EMA designates uniQure’s gene therapy for hemophilia a priority

It’s Global Rare Disease Day! What Cures is Biotech Cooking Up?

The FDA gives Fast Track Priority to uniQure’s Hemophilia B Gene Therapy

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Catalonia: A biotech hub going from strength to strength, with Barcelona at its core

Top biotech deals of February 2025

Grab your free report on 3D organoids and explore their future potential

These 7 biotechs are shaping the next generation of endometriosis care

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

Catalonia: A biotech hub going from strength to strength, with Barcelona at its core

Top biotech deals of February 2025

Grab your free report on 3D organoids and explore their future potential

These 7 biotechs are shaping the next generation of endometriosis care

Connect with decision-makers and researchers worldwide for collaborative opportunities

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Advertise with us

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