Hemophilia A News and Research

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CHOP-led research increases numbers of patients who could be treated with gene therapy

Gene therapy researchers have produced a bioengineered decoy that fools the immune system and prevents it from mistakenly defeating the benefits delivered by a corrective gene.

18 Jul 2013

Bayer starts enrollment in BAY94-9027 Phase II/III trial for hemophilia A in children

Bayer HealthCare today announced the company has started to enroll patients in an international Phase II/III trial to evaluate its investigational compound BAY94-9027 for the treatment of hemophilia A in children.

8 Jul 2013

Data shows individualized regimens can help optimize prophylaxis treatment in hemophilia patients

Baxter International Inc. today announced data featuring ADVATE [Antihemophilic Factor (Recombinant), Plasma/Albumin-Free Method], a leading recombinant treatment available worldwide, during the 24th Annual Congress of the International Society on Thrombosis and Haemostasis (ISTH) in Amsterdam, The Netherlands.

From Baxter International Inc. 5 Jul 2013

Biogen, Sobi present new data supporting clinical, safety profile of ELOCTATE for hemophilia A

Biogen Idec nd Swedish Orphan Biovitrum AB (publ) presented new data that support the clinical and safety profile of their long-lasting recombinant factor VIII candidate ELOCTATE for hemophilia A.

4 Jul 2013

Biogen, Sobi present new findings from Phase 3 trial of ALPROLIX at XXIV ISTH Congress

Biogen Idec and Swedish Orphan Biovitrum AB (publ) today presented new findings for their long-lasting recombinant factor IX candidate ALPROLIX for hemophilia B at the XXIV International Society on Thrombosis and Haemostasis Congress in Amsterdam, The Netherlands.

3 Jul 2013

Alnylam Pharmaceuticals presents new pre-clinical data of ALN-AT3 at ISTH meeting

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has presented new pre-clinical data from its RNAi therapeutic program for the treatment of hemophilia and rare bleeding disorders.

2 Jul 2013

Duke researchers find novel way to repair gene responsible for Duchenne muscular dystrophy

Using a novel genetic 'editing' technique, Duke University biomedical engineers have been able to repair a defect responsible for one of the most common inherited disorders, Duchenne muscular dystrophy, in cell samples from Duchenne patients.

5 Jun 2013

New technique selectively represses unwanted immune reactions without disabling immune system

The human immune system is remarkably efficient, but sometimes its attack is misdirected, leading to allergies, autoimmune diseases and rejection of transplant organs and therapeutic drugs. Current immune suppressants have major drawbacks, but a team from The Scripps Research Institute (TSRI) has demonstrated a new technique that may lead to a better way to selectively repress unwanted immune reactions without disabling the immune system as a whole.

4 Jun 2013

UIC's Center for Clinical and Translational Science selects six research projects for new pilot grants

The University of Illinois at Chicago's Center for Clinical and Translational Science has selected six research projects to receive pilot grants in 2013.

15 May 2013

Biogen Idec announces FDA's acceptance of ELOCTATE BLA for treatment of hemophilia A

Biogen Idec announced today that the U.S. Food and Drug Administration has accepted the company's Biologics License Application for the marketing approval of ELOCTATE (recombinant factor VIII Fc fusion protein) for the treatment of hemophilia A.

13 May 2013

GZS releases WebTracker-Hemophilia EHR patient registry system

GZS, Inc., a leader in developing disease-specific and condition-specific Electronic Medical Records/Electronic Health Records and patient registry software solutions, has announced the worldwide release of the WebTracker-Hemophilia EHR patient registry solution used under the new brand name HemaGlobal.

2 Apr 2013

Translational Research's April issue examines progress, outlook of gene therapy research

The April issue of Translational Research examines the progress and outlook of gene therapy research, with a specific focus on the clinical applicability of gene therapy today.

22 Mar 2013

Biogen announces FDA acceptance of rFIXFc BLA for treatment of hemophilia B

Biogen Idec announced today that the U.S. Food and Drug Administration (FDA) has accepted the company's Biologics License Application (BLA) for the marketing approval of recombinant factor IX Fc fusion protein (rFIXFc) for the treatment of hemophilia B and granted the company a standard review timeline.

5 Mar 2013

Baxter files FEIBA NF BLA with FDA for prophylactic treatment of hemophilia A or B

Baxter International Inc. today announced that the company has submitted a biologics license application (BLA) supplement to the U.S. Food and Drug Administration (FDA) for the approval of prophylaxis treatment of FEIBA NF [Anti-Inhibitor Coagulant Complex], Nanofiltered and Vapor Heated, in patients with hemophilia A or B and inhibitors.

From Baxter International Inc. 26 Feb 2013

Research roundup: Patients hesitant to focus on costs; Increase in primary care demand is uneven

One way to contain health care spending is to get patients involved in weighing costs of treatment options with their doctors. Researchers recruited 211 medically insured participants to assess their willingness to consider costs when choosing treatments. The authors found that participants generally "preferred better care, even when the relative benefit it offered was marginal, and even when told that the second-best choice still met the threshold of 'good enough' care from a clinician's perspective.

26 Feb 2013

Ipsen, Inspiration Biopharmaceuticals sell IB1001 hemophilia B product to Cangene

Inspiration Biopharmaceuticals and Ipsen today announced the closing of the sale of the proprietary hemophilia B product, IB1001 (recombinant FIX), to Cangene Corporation.

20 Feb 2013

New approach could make gene therapy dramatically more effective for HIV patients

A research team from Case Western Reserve University School of Medicine has discovered an approach that could make gene therapy dramatically more effective for patients.

16 Feb 2013

Biogen Idec, Sobi release data from rFVIIIFc and rFIXFc phase 3 trials on hemophilia

Biogen Idec and Swedish Orphan Biovitrum (Sobi) released data that confirmed the ability of investigational recombinant factors VIII Fc fusion protein (rFVIIIFc) and IX Fc fusion protein (rFIXFc) to provide long-lasting protection from bleeding with fewer injections than are required with the current standard of care for people with hemophilia.

10 Feb 2013

Sangamo BioSciences fourth quarter revenues increase to $8.9 million

Sangamo BioSciences, Inc. today reported fourth quarter and full year 2012 financial results and accomplishments.

7 Feb 2013

Inspiration, Ipsen and Baxter enter Asset Purchase Agreement for OBI-1

Ipsen and Inspiration Biopharmaceuticals Inc. today announced they entered into an Asset Purchase Agreement (APA) whereby Baxter International (Baxter) agrees to acquire the worldwide rights to OBI-1, a recombinant porcine factor VIII (rpFVIII) in development for congenital hemophilia A with inhibitors and acquired hemophilia A, and Ipsen's industrial facility in Milford (Boston, MA).

24 Jan 2013