Hemophilia A News and Research

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Pfizer commences PF-05280602 Phase 1 trial in hemophilia A & B

Catalyst Biosciences, Inc., the leading company in the discovery and development of engineered proteases, today announced that Pfizer Inc. has initiated a Phase 1 clinical trial for PF-05280602, an investigational proprietary, engineered variant of recombinant human Factor VIIa developed by Catalyst Biosciences.

6 Jan 2012

Baxter commences BAX 855 Phase I trial in hemophilia A

Baxter International Inc. today announced the dosing of the first patients in a Phase I clinical trial of its lead investigational candidate, BAX 855, a longer-acting (PEGylated) form of a full-length recombinant factor VIII (rFVIII) protein.

From Baxter International Inc. 5 Jan 2012

FDA grants orphan designation for AMT's hemophilia gene therapy program

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, announced today that the U.S. Food and Drug Administration has granted orphan drug designation to its gene therapy program for the treatment of hemophilia B.

4 Jan 2012

Baxter receives FDA approval for ADVATE to prevent hemophilia A

Baxter International Inc. today announced that the U.S. Food and Drug Administration (FDA) has approved ADVATE [Antihemophilic Factor (Recombinant) Plasma/Albumin Free Method] for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in patients with hemophilia A.

From Baxter International Inc. 17 Dec 2011

Gene therapy for bleeding disorder Hemophilia B

In a landmark study, researchers used gene therapy to successfully treat six patients with severe hemophilia, a blood-clotting disorder. Despite the early nature of the study, it is promising. A single infusion using the new treatment worked in some patients for more than a year, boosting their clotting ability significantly.

12 Dec 2011

Research examines novel treatment techniques for hemophilia B, VTE

Bleeding and clotting disorders affect a large number of patients annually and, while accurate diagnosis is important, timely and effective treatment for these disorders is key, as they have the potential to quickly become life-threatening. Research examining novel treatment techniques for bleeding and clotting disorders will be presented today at the 53rd Annual Meeting of the American Society of Hematology.

12 Dec 2011

Gene therapy can reduce painful bleeding episodes in patients with hemophilia B

Symptoms improved significantly in adults with the bleeding disorder hemophilia B following a single treatment with gene therapy developed by researchers at St. Jude Children's Research Hospital in Memphis and demonstrated to be safe in a clinical trial conducted at the University College London (UCL) in the U.K.

12 Dec 2011

Biogen, Samsung partner to develop and market biosimilars

Samsung and Biogen Idec today announced that they have entered into an agreement to invest $300 million to establish a joint venture to develop, manufacture and market biosimilars. Samsung will take a leading role in the joint venture, with Biogen Idec contributing its expertise in protein engineering and biologics manufacturing.

5 Dec 2011

American Plasma Users Coalition awarded $40,000 advocacy grant from CSL Behring

CSL Behring announced today that it has awarded a $40,000 advocacy grant to the American Plasma Users Coalition (A-PLUS) through its Local Empowerment for Advocacy Development (LEAD) program. LEAD grants support the grassroots advocacy efforts of organizations that help people who use plasma-derived or recombinant therapies to manage rare and serious medical disorders.

3 Dec 2011

FDA approves Mylan's Tranexamic Acid Injection ANDA

Mylan Inc. today announced that its business Mylan Institutional has received final approval from the U.S. Food and Drug Administration for its Abbreviated New Drug Application (ANDA) for Tranexamic Acid Injection, 100 mg/mL, packaged in 1,000 mg/10 mL Single-dose Vials.

3 Dec 2011

Inspiration commences enrollment in second OBI-1 Accur8 clinical trial program for congenital hemophilia A

Inspiration Biopharmaceuticals, Inc. today announced the initiation of patient enrollment in the second of two pivotal studies in the Company's OBI-1 Accur8 clinical trial program.

28 Nov 2011

Inspiration initiates second OBI-1 phase III clinical trial in congenital hemophilia A with inhibitors

Ipsen today announced that its partner Inspiration Biopharmaceuticals, Inc. has initiated the treatment of the first patient in the second of two pivotal studies from the OBI-1's Accur8 clinical trial program.

28 Nov 2011

$GE Healthcare, Neste Jacobs enter alliance to increase self-sufficiency in blood plasma fractionation$

GE Healthcare, Neste Jacobs enter alliance to increase self-sufficiency in blood plasma fractionation

GE Healthcare, the healthcare business of GE and Neste Jacobs, a leading global expert in plasma fractionation plant engineering design and construction, announced today that the companies have formed a strategic alliance to assist countries worldwide to become self-sufficient in the manufacture of blood plasma products.

21 Nov 2011

EMA grants orphan designation to AMT for hemophilia B gene therapy

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, announced today that the European Medicines Agency (EMA) has granted orphan designation for its gene therapy program for the treatment of hemophilia B. Orphan designation in the European Union provides several benefits including 10 years of market exclusivity from product launch and access to the central authorization procedure.

16 Nov 2011

Public health officials should address needs of people with blood disorders

Public health should focus not only on reducing the burden of common diseases but also address the needs of people with blood disorders , experts say in a supplement to December's American Journal of Preventive Medicine.

16 Nov 2011

EPO-secreting engineered vessels could reverse anemia

Patients who rely on recombinant, protein-based drugs must often endure frequent injections, often several times a week, or intravenous therapy. Researchers at Children's Hospital Boston demonstrate the possibility that blood vessels, made from genetically engineered cells, could secrete the drug on demand directly into the bloodstream.

15 Nov 2011

Gene therapy and stem cell transplantation successfully reverse hemophilia A

For the first time, researchers have combined gene therapy and stem cell transplantation to successfully reverse the severe, crippling bleeding disorder hemophilia A in large animals, opening the door to the development of new therapies for human patients.

3 Nov 2011

FEIBA drug safe and effective for patients with hemophilia A

An international research team led by Dr. Cindy Leissinger of Tulane University School of Medicine, along with Dr. Alessandro Gringeri from the University of Milan, has found that a drug commonly used to treat bleeding events in people with a type of severe hemophilia can also be used to prevent such events from happening in the first place.

3 Nov 2011

Genetically engineered clotting factor shows promise against hemophilia

A genetically engineered clotting factor that controlled hemophilia in an animal study offers a novel potential treatment for human hemophilia and a broad range of other bleeding problems.

24 Oct 2011

Nature publishes Sangamo's gene correction strategy for A1AT deficiency

Sangamo BioSciences, Inc. (Nasdaq: SGMO) announced the publication of a preclinical study demonstrating highly specific, functional correction of the alpha 1-antitrypsin (A1AT) gene defect in patient-derived induced pluripotent stem cells (iPSCs) using zinc finger nucleases.

14 Oct 2011