Bleeding Disorder News and Research

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Scientists use new technique to repair fibrotic liver cells within the organ

Advances in stem cell research have made it possible to convert patients' skin cells into heart cells, kidney cells, liver cells and more in the lab dish, giving researchers hope that one day such cells could replace organ transplantation for patients with organ failure.

3 Jun 2016

European Medicines Agency approves first-ever treatment for hereditary factor X deficiency

Bio Products Laboratory, Limited (BPL) today announced that the European Medicines Agency has granted marketing authorisation for Coagadex.

31 Mar 2016

UC San Diego Health participates in nationwide clinical study on hemophilia B gene therapy

The Hemophilia and Thrombosis Treatment Center at UC San Diego Health has joined a nationwide clinical trial testing a potential gene therapy that may one day provide a better and long-lasting treatment for people with hemophilia B.

23 Mar 2016

Idelvion approved for use in children and adults with Hemophilia B

The U.S. Food and Drug Administration today approved Idelvion, Coagulation Factor IX (Recombinant), Albumin Fusion Protein, for use in children and adults with Hemophilia B. Idelvion is the first coagulation factor-albumin fusion protein product to be approved, and the second Factor IX fusion protein product approved in the U.S. that is modified to last longer in the blood.

7 Mar 2016

Gene therapy holds considerable potential for safe, effective treatment of people with factor VII deficiency

Hematology researchers have used a single injection of gene therapy to correct a rare bleeding disorder, factor VII deficiency, in dogs. This success in large animals holds considerable potential for a safe, effective and long-lasting new treatment in humans with the same bleeding disorder.

21 Jan 2016

Wyss Institute scientists develop better assay for testing hemostasis

Scientists at the Wyss Institute for Biologically Inspired Engineering at Harvard University have devised a better assay for testing blood's clotting tendency, also known as hemostasis, which could one day prove lifesaving in a variety of clinical situations in which a patient's health is jeopardized by abnormal blood coagulation and platelet function.

11 Jan 2016

New $8.5M research grant aims to address patients affected by severe hemophilia A

People with hemophilia have prolonged abnormal bleeding as a result of trauma. Hemophilia A, also called factor VIII (FVIII) deficiency, is the most common form of the genetic disorder caused by missing or defective blood clotting protein called factor VIII.

23 Dec 2015

CSL submits rIX-FP new drug application to Japan's Pharmaceuticals and Medical Devices Agency

Global biotherapeutics leader CSL Behring announced today that the company has submitted its new drug application to Japan's Pharmaceuticals and Medical Devices Agency for its investigational fusion protein linking recombinant coagulation factor IX with recombinant albumin (rIX-FP).

22 Dec 2015

New drug approved by FDA for treatment of von Willebrand disease

"VONVENDI [von Willebrand factor (Recombinant)]", the new drug from Baxalta Incorporated, a global biopharmaceutical company recently spun off from Baxter International, has just been approved by the United States Food and Drug Administration.

22 Dec 2015

FDA approves recombinant von Willebrand factor for treating bleeding episodes in adults with VWD

The U.S. Food and Drug Administration today approved Vonvendi, von Willebrand factor (Recombinant), for use in adults 18 years of age and older who have von Willebrand disease (VWD). Vonvendi is the first FDA-approved recombinant von Willebrand factor, and is approved for the on-demand (as needed) treatment and control of bleeding episodes in adults diagnosed with VWD.

8 Dec 2015

Rice scientists solve long-standing mystery about hemophilia protein

Rice University scientists have solved a long-standing mystery about where the body stores and deploys blood-clotting factor VIII, a protein that about 80 percent of hemophiliacs cannot produce due to genetic defects.

3 Nov 2015

Coagadex now approved by FDA for patients with hereditary Factor X deficiency

The U.S. Food and Drug Administration today approved Coagadex, Coagulation Factor X (Human), for hereditary Factor X (10) deficiency. Until today's orphan drug approval, no specific coagulation factor replacement therapy was available for patients with hereditary Factor X deficiency.

21 Oct 2015

First patient enrolled in CSL Behring's rVIIa-FP Phase II/III study to treat patients with hemophilia A or B with inhibitors

CSL Behring announced today that the first patient has been enrolled in its Phase II/III clinical study evaluating the pharmacokinetics (PK), efficacy, and safety of the company's recombinant fusion protein linking coagulation factor VIIa with albumin (rVIIa-FP) for on-demand treatment in patients with congenital hemophilia A or B who have developed an inhibitor to factor VIII or factor IX replacement therapy.

26 Aug 2015

Alnylam presents positive Phase 1 ALN-AT3 trial results in hemophilia at ISTH 2015 Congress

Additional study results from 12 hemophilia A and B subjects demonstrated that subcutaneous administration of ALN-AT3 achieved potent and dose-dependent knockdown of AT of up to 86%. AT knockdown was highly durable, with effects lasting over two months after the last dose, supporting further evaluation of a once-monthly subcutaneous dose regimen.

24 Jun 2015

Apitope's ATX-F8-117 granted FDA Orphan Drug Designation for treatment of haemophilia A patients

Apitope, the drug discovery and development company focused on disease-modifying treatments that reinstate immune tolerance, announced today that pre-clinical product candidate ATX-F8-117 has been granted Orphan Drug Status by the US Food and Drug Administration for the prevention or treatment of inhibitors in haemophilia A patients with inhibitors or at the risk of producing inhibitors.

9 Jun 2015

CSL Behring awarded NHF's 2015 Corporate Leadership Award

The National Hemophilia Foundation has awarded CSL Behring its 2015 Corporate Leadership Award as recognition for the company's longstanding and unwavering commitment to advancing science and improving the care of the bleeding disorders community. The award was accepted by Paul Perreault, CEO and Managing Director, CSL Limited, during the NHF Annual Spring Soiree in New York City on May 21.

24 May 2015

CMC Biologics to supply bulk drug substance for Emergent BioSolutions' IXINITY

CMC Biologics, Inc., a global CMO dedicated to process development and production of biopharmaceuticals for clinical and commercial use, today announced that it will supply bulk drug substance for Emergent BioSolutions' recently FDA-approved product, IXINITY® [coagulation factor IX (recombinant)].

5 May 2015

Scientists identify missing genetic link in common variable immunodeficiency disorder

In the largest genetic study to date of a challenging immunodeficiency disorder, scientists have identified a gene that may be a "missing link" between overactive and underactive immune activity. The gene candidate also plays a key role in autoimmune diseases such as type 1 diabetes, rheumatoid arthritis and allergies.

23 Apr 2015

'Turbocharged' protein holds promise in hemophilia gene therapy

Using gene therapy to produce a mutant human protein with unusually high blood-clotting power, scientists have successfully treated dogs with the bleeding disorder hemophilia, without triggering an unwanted immune response.

13 Mar 2015

Study: New gene therapy safe, effective for patients with hemophilia B

A multi-year, ongoing study suggests that a new kind of gene therapy for hemophilia B could be safe and effective for human patients. Published in the journal Science Translational Medicine, the research showed that a reprogrammed retrovirus could successfully transfer new factor IX (clotting) genes into animals with hemophilia B to dramatically decrease spontaneous bleeding.

12 Mar 2015