Hemophilia A News and Research

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EMA accepts Inspiration's IB1001 MAA for treatment of hemophilia B

Inspiration Biopharmaceuticals, Inc. today announced that the European Medicines Agency (EMA) has validated and accepted the filing of the Marketing Authorization Application (MAA) for Inspiration's IB1001, a recombinant factor IX (FIX) product for the treatment and prevention of bleeding in individuals with hemophilia B.

3 Oct 2011

Express Scripts launches suite of services through HealthBridge Pharma & Biotech

Express Scripts has launched a suite of services for pharmaceutical and biotech companies through HealthBridge Pharma & Biotech, providing a single-solution approach to patient care.

1 Oct 2011

Alnylam commences ALN-PCS Phase I trial in severe hypercholesterolemia

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has initiated dosing in its Phase I clinical trial with ALN-PCS, an RNAi therapeutic targeting proprotein convertase subtilisin/kexin type 9, or PCSK9, for the treatment of severe hypercholesterolemia.

27 Sep 2011

Positive results from PROLOR Biotech's Factor VIIa-CTP preclinical study on hemophilia

PROLOR Biotech, Inc.,, today reported positive results from a comparative study of its biobetter longer-acting version of the hemophilia drug Factor VIIa (Factor VIIa-CTP) in hemophilic mice.

15 Sep 2011

AMT, NIH enter exclusive license agreement for use of AAV5-based gene therapy vectors

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, today announced that it has entered into an exclusive license agreement with the US National Institutes of Health (NIH) for use of adeno-associated virus serotype 5 (AAV5)-based gene therapy vectors for liver and brain indications.

6 Sep 2011

UCF, UF receive $5.5 million NIH grant for hemophilia research

At a time when research funding is hard to come by, a University of Central Florida and University of Florida partnership has landed almost $5.5 million in National Institutes of Health highly competitive grants for hemophilia research.

3 Sep 2011

Ipsen, Inspiration partner to launch hemophilia product portfolio in Europe

Ipsen and Inspiration Biopharmaceuticals Inc. today announced they have entered into a strategic partnership agreement, to create a European hemophilia commercial organization, to launch Inspiration's hemophilia product portfolio in Europe.

30 Aug 2011

AMT total net loss decreases 7% to € 8.7 million for first half year of 2011

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, today reported its results for the first half year of 2011.

25 Aug 2011

New book on hemophilia, unintended consequences of medical progress

The Bleeding Disease: Hemophilia and the Unintended Consequences of Medical Progress (John Hopkins University Press) is the new book by NJIT Associate Professor Stephen Pemberton. The book recounts the promising and perilous history of medical and social efforts to manage hemophilia in 20th-century America.

19 Aug 2011

Gene therapy cocktail can treat inherited diseases with 'misfolded' proteins

In a kind of molecular gymnastics, scientists at the University of North Carolina at Chapel Hill School of Medicine have devised a gene therapy cocktail that has the potential to treat some inherited diseases associated with "misfolded" proteins.

16 Aug 2011

Hemophilia Foundation of Michigan announces third annual fundraising walk at the Detroit Zoo

The Hemophilia Foundation of Michigan is pleased to announce: Walkin' on the Wild Side for Hemophilia, its third annual fundraising walk at the Detroit Zoo. Walkin' on the Wild Side for Hemophilia takes place on Sunday, August 28, 2011.

4 Aug 2011

Inspiration presents OBI-1 pivotal study data on hemophilia at 23rd ISTH congress

Ipsen today announced that its partner Inspiration Biopharmaceuticals, Inc. presented data from its clinical development program for OBI-1, an intravenous (IV) recombinant porcine factor VIII product (rpFVIII), intended for the treatment of bleeding in people with hemophilia A with inhibitors and in people with acquired hemophilia.

28 Jul 2011

New preclinical data from Baxter's BAX 499 hemophilia study presented at ISTH

Baxter International Inc. announced today that new data from a series of studies on BAX 499, an investigational compound for potential subcutaneous hemophilia therapy, were presented this week during the 23rd annual congress of the International Society on Thrombosis and Haemostasis (ISTH) in Kyoto, Japan.

From Baxter International Inc. 27 Jul 2011

Biogen Idec reports revenue of $1.2 billion for second quarter 2011

Biogen Idec Inc., a global biotechnology leader in the discovery, development, manufacturing and commercialization of innovative therapies, today announced its second quarter 2011 results.

26 Jul 2011

Swedish Orphan Biovitrum, Biogen Idec announce data of rFVIIIFc Phase 1/2a trial for hemophilia A

Biogen Idec and Swedish Orphan Biovitrum today announced Phase 1/2a trial data showing that the companies' long-lasting fully-recombinant factor VIII Fc fusion protein (rFVIIIFc) was well tolerated and demonstrated an approximately 1.7-fold increase in half-life compared with Advate, a commercially-available factor VIII product, in 16 previously-treated patients with severe hemophilia A.

26 Jul 2011

CSL Behring supports World Federation of Hemophilia with 1 million IUs of von Willebrand factor/Factor VIII

CSL Behring has donated more than 1 million international units (IU) of von Willebrand factor (VWF)/Factor VIII (FVIII) replacement medication to patients through the World Federation of Hemophilia (WFH). The donation supports WFH's progress in improving the diagnosis and treatment of bleeding disorders in developing countries through its Global Alliance for Progress (GAP) program.

18 Jul 2011

IMM researcher wins prestigious Early Career Bayer Hemophilia Award

Vanessa Oliveira, researcher at the Instituto de Medicina Molecular in Portugal won a prestigious and competitive Early Career Bayer Hemophilia Award. Oliveira will develop a strategy to reprogramme the immune system of hemophilia patients to avoid immune targeting of the most used clotting therapeutics.

14 Jul 2011

In vivo genome editing restores hemostasis in mouse model of blood clotting disorder

Using an innovative gene therapy technique called genome editing that hones in on the precise location of mutated DNA, scientists have treated the blood clotting disorder hemophilia in mice.

27 Jun 2011

AMT receives CHMP opinion on Glybera Marketing Authorisation Application

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, today announced that it has received an opinion on its Marketing Authorisation Application (MAA) for Glybera as a potential therapy for Lipoprotein Lipase Deficiency.

24 Jun 2011

European hematologists raise alarm on insufficient research money for blood disorders

European hematologists raise the alarm on the insufficient research money for blood disorders. "Blood cancers kill over 95,000 in the EU every year; the EU must be made aware that more research funds are needed", states Professor Robin Foa, President of the European Hematology Association (EHA) at their annual congress in London.

8 Jun 2011