Hemophilia News and Research

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Hemophilia is a rare, inherited bleeding disorder in which your blood doesn’t clot normally. If you have hemophilia, you may bleed for a longer time than others after an injury. You also may bleed internally, especially in your knees, ankles, and elbows. This bleeding can damage your organs or tissues and, sometimes, be fatal.

Catalyst receives $4.0M milestone payment from Pfizer under hemophilia Factor VIIa program

Catalyst Biosciences, Inc., the leading company in the discovery and development of engineered proteases, today announced the receipt of a $4.0 million milestone payment from Pfizer Inc. under the terms of the collaboration agreement for the development of improved recombinant human Factor VIIa variants for the treatment of hemophilia and other bleeding disorders.

17 Dec 2010

Pain Therapeutics completes special, one-time nondividend cash distribution

Pain Therapeutics, Inc. announced today the completion of a special, one-time nondividend cash distribution of $2.00 per share, or an aggregate of about $86 million. In connection with this event, Nasdaq® will adjust the price of PTIE by $2.00 per share at today's open.

13 Dec 2010

New objective criteria for evaluating hemostatic efficacy in treatment of VWD

Octapharma USA researchers presented new stringent, objective criteria used in the evaluation of hemostatic efficacy in the treatment of von Willebrand disease and reported on their application of these criteria in clinical trials during the recently completed National Hemophilia Foundation Annual Meeting.

9 Dec 2010

rVWF safe in patients with type 3 and severe type 1 von Willebrand disease, suggests interim study data

Recombinant von Willebrand factor (rVWF) may be safe and well tolerated in patients with type 3 and severe type 1 von Willebrand disease, according to interim data from a Phase 1 multicenter, international clinical study presented today at the 52nd Annual Meeting of the American Society of Hematology.

From Baxter International Inc. 7 Dec 2010

No evidence of toxicity in hemophilia B patients enrolled in gene therapy trial

Investigators report no evidence of toxicity in the four hemophilia B patients enrolled to date in a gene therapy trial using a vector under development at St. Jude Children's Research Hospital and UCL (University College London) to correct the inherited bleeding disorder.

7 Dec 2010

First patient dosed with rFVIIIFc in A-LONG trial

Biogen Idec and Swedish Orphan Biovitrum today announced that the first patient has been dosed with the companies' long-lasting recombinant Factor VIII Fc fusion protein (rFVIIIFc) in a global registrational clinical trial. The study, called A-LONG, is an open-label, multicenter, Phase 2/3 study designed to evaluate the safety, pharmacokinetics and efficacy of rFVIIIFc in previously-treated hemophilia A patients.

6 Dec 2010

Baxter licenses Vero cell-based influenza vaccine technology to Takeda

Baxter International Inc. and Takeda Pharmaceutical Company Limited today announced that the parties have completed a Development, License and Technology Transfer Agreement. Under the terms of the agreement, Takeda and Baxter will expand upon their previously announced collaboration to bring Vero cell culture-based influenza vaccines to the Japanese market.

From Baxter International Inc. 2 Dec 2010

AMT, Amgen amend GDNF gene license agreement

Amsterdam Molecular Therapeutics Holding N.V., a leader in the development of gene-based therapies, today announced that it has amended and restated its licensing agreement with Amgen for gene therapy applications incorporating the GDNF (glial cell derived neurotrophic factor) gene, to which Amgen holds rights. Financial terms were not disclosed.

2 Dec 2010

CSL Behring launches next generation HeliTrax System for Apple's devices

CSL Behring announced today the availability of the next generation HeliTrax System as part of a specially designed software platform for Apple's iPhone, iPod touch, or iPad devices. This enhanced system is designed to improve therapy management for people with hemophilia A who use CSL Behring's recombinant factor VIII (rFVIII).

2 Dec 2010

Anacor, UCSF and NYBC collaborate to discover drug therapies for river blindness

Anacor Pharmaceuticals, the University of California San Francisco (UCSF) Sandler Center and the Lindsley F. Kimball Research Institute (LFKRI) of the New York Blood Center (NYBC) today announced the establishment of a research and development collaboration to discover new drug therapies for the treatment of river blindness (onchocerciasis), a parasitic disease that is the second leading cause of infectious blindness worldwide, and is most prevalent in Africa.

1 Dec 2010

Baxter enters definitive agreement to acquire all hemophilia-related assets Archemix

Baxter International Inc. announced today that it has entered into a definitive agreement to acquire all of the hemophilia-related assets of a privately-held biopharmaceutical company, Archemix, and entered into an exclusive license agreement for certain related intellectual property assets.

From Baxter International Inc. 19 Nov 2010

Ipsen initiates patient treatment in phase III OBI-1 study for acquired hemophilia A

Ipsen announced today that its partner Inspiration Biopharmaceuticals, Inc. has initiated treatment of patients in the first of two phase III pivotal clinical studies of OBI-1, an intravenous recombinant porcine factor VIII (FVIII) product, for the treatment of acquired hemophilia A, a rare, though potentially life-threatening bleeding disorder.

19 Nov 2010

FDA approves IDE application for Home Hemodialysis system

Baxter International Inc. announced today that the U.S. Food and Drug Administration (FDA) has approved an Investigational Device Exemption (IDE) application for a Home Hemodialysis (HD) system in development through a collaboration between DEKA Research and Development Corp., HHD LLC, and Baxter.

From Baxter International Inc. 19 Nov 2010

Amsterdam 2010 third quarter total expense increases from EUR 4.4 million to EUR 4.7 million

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, today provides its non-audited business update in compliance with the EU transparency directive. This report summarizes material events and AMT's financial position for the third quarter of 2010.

18 Nov 2010

Science and precedent do not support biosimilar approach for plasma protein therapies

The current science and experience does not support the safety and efficaciousness of a biosimilar pathway for plasma protein therapies. At a public hearing last week, the Plasma Protein Therapeutics Association (PPTA) urged the United States Food and Drug Administration (FDA) to make patient safety its top priority by adopting a global approach in its evaluation of plasma protein therapies for the biosimilars process.

15 Nov 2010

NJIT expert to discuss history of sickle cell disease at NIH symposium

The history of sickle cell disease in the 20th century and its link to race is the focus of a Nov. 16, 2010 talk at a national symposium by NJIT Associate Professor Stephen Pemberton, PhD, an expert on the history of diseases. Pemberton will speak and join a panel discussion at the National Institutes of Health (NIH), Bethesda, 100th anniversary symposium commemorating the discovery of sickle cell disease by Chicago physician James B. Herrick.

11 Nov 2010

Inspiration receives two grants under QTDP program to advance development of IB1001, OBI-1 products

Inspiration Biopharmaceuticals, Inc. today announced the Company was awarded two grants, totaling $488,958, under the Qualifying Therapeutic Discovery Projects Grant Program (QTDP).

9 Nov 2010

Pain Therapeutics awarded $2,000,000 U.S. government research grant

Pain Therapeutics, Inc. announced today that it has been awarded over $2,000,000 in research grants by the U.S. government. These grants are planned to support the Company's on-going biomedical research for nervous system disorders and oncology. Pain Therapeutics expects to receive funding from these research grants now through the end of 2011.

2 Nov 2010

Researchers investigate Human-cl rhFVIII factor for severe hemophilia A treatment

Octapharma AG, one of the largest human protein products manufacturers in the world, and Octapharma USA today announced that patients diagnosed with severe hemophilia A have started treatment in a multi-center study that is researching the use of the first recombinant Factor VIII (rFVIII) derived from a human cell line (Human-cl rhFVIII).

2 Nov 2010

NYBC calls for blood donations during Pancreatic and Lung Cancer Awareness Month

November is Pancreatic Cancer Awareness Month and Lung Cancer Awareness Month, and New York Blood Center (NYBC) serving more than 20 million people in New York City, Long Island, the Hudson Valley, New Jersey, and parts of Connecticut and Pennsylvania, calls upon our communities to please join the fight against cancer by donating blood. Healthy people of all blood types and ethnicities are encouraged to donate and help save lives. Blood donations are particularly needed from O-negative, B-negative and A-negative types.

30 Oct 2010

Hemophilia News and Research

Further Reading

Catalyst receives $4.0M milestone payment from Pfizer under hemophilia Factor VIIa program

Pain Therapeutics completes special, one-time nondividend cash distribution

New objective criteria for evaluating hemostatic efficacy in treatment of VWD

rVWF safe in patients with type 3 and severe type 1 von Willebrand disease, suggests interim study data

No evidence of toxicity in hemophilia B patients enrolled in gene therapy trial

First patient dosed with rFVIIIFc in A-LONG trial

Baxter licenses Vero cell-based influenza vaccine technology to Takeda

AMT, Amgen amend GDNF gene license agreement

CSL Behring launches next generation HeliTrax System for Apple's devices

Anacor, UCSF and NYBC collaborate to discover drug therapies for river blindness

Baxter enters definitive agreement to acquire all hemophilia-related assets Archemix

Ipsen initiates patient treatment in phase III OBI-1 study for acquired hemophilia A

FDA approves IDE application for Home Hemodialysis system

Amsterdam 2010 third quarter total expense increases from EUR 4.4 million to EUR 4.7 million

Science and precedent do not support biosimilar approach for plasma protein therapies

NJIT expert to discuss history of sickle cell disease at NIH symposium

Inspiration receives two grants under QTDP program to advance development of IB1001, OBI-1 products

Pain Therapeutics awarded $2,000,000 U.S. government research grant

Researchers investigate Human-cl rhFVIII factor for severe hemophilia A treatment

NYBC calls for blood donations during Pancreatic and Lung Cancer Awareness Month

Achieving and maintaining ISO/IEC 27001 and BS 10012 certifications

Expanding research and clinical options for children with cancer

Examining how pain could play a direct protective role in the gut

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