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.

Biogen Idec 2011 total revenues increase 7% to $5.0 billion

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

1 Feb 2012

Alnylam receives U.S. patent allowance for LNP platform

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that the United States Patent and Trademark Office (USPTO) has issued a Notice of Allowance for patent application number 12/813,448, covering the company's proprietary second generation lipid nanoparticle (LNP) platform, including the "MC3" lipid.

30 Jan 2012

FDA approves Baxter's TISSEEL for general hemostasis in surgery

Baxter International Inc. announced today that the U.S. Food and Drug Administration (FDA) has approved TISSEEL [Fibrin Sealant] to include general hemostasis in surgery when control of bleeding by standard surgical techniques is ineffective or impractical.

From Baxter International Inc. 30 Jan 2012

Novo Nordisk Type 1 Diabetes R&D Center to be established in Seattle

Novo Nordisk announced today that the company will establish a type 1 diabetes research and development center in Seattle, Wash., U.S. The new center will combine Novo Nordisk's history of innovation and leadership in diabetes treatment with the company's growing expertise in immunotherapy.

24 Jan 2012

Baxter to commence second GAMMAGARD LIQUID Phase III trial in Alzheimer's

Baxter International Inc. today provided an update on its clinical program evaluating the use of its GAMMAGARD LIQUID 10%, for the treatment of mild to moderate Alzheimer's disease.

From Baxter International Inc. 24 Jan 2012

FDA approves product label change for Biogen Idec's TYSABRI to treat MS

Today Biogen Idec and Elan Corporation, plc announced that the U.S. Food and Drug Administration (FDA) has approved a product label change for TYSABRI that will help enable individual benefit risk assessment for patients with multiple sclerosis.

21 Jan 2012

Alnylam sues Tekmira over LNP-formulated siRNA molecules

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has filed a patent infringement lawsuit against Tekmira Pharmaceuticals Corporation in the U.S. District Court of the District of Massachusetts.

18 Jan 2012

Study confirms safety and effectiveness of rAHF-PFM in managing hemophilia A

A Rush University Medical Center led international research team has announced that a treatment to prevent bleeding episodes in children with hemophilia A also is effective for adolescents and adults.

10 Jan 2012

Baxter submits GAMMAGARD LIQUID sBLA to FDA for treatment of MMN

Baxter International Inc. today announced that the company has submitted a supplemental biologics license application (sBLA) to the U.S. Food and Drug Administration (FDA) for approval of GAMMAGARD LIQUID 10% for the treatment of multifocal motor neuropathy.

From Baxter International Inc. 9 Jan 2012

Sangamo BioSciences starts Phase 2 clinical studies to develop functional cure for HIV/AIDS

Sangamo BioSciences, Inc. announced today the initiation of two new Phase 2 clinical studies (SB-728-1101 and SB-728-902, Cohort 5) in its program to develop a "functional cure" for HIV/AIDS.

9 Jan 2012

Alnylam files CTA with the U.K. MHRA to initiate ALN-TTR02 Phase I trial in ATTR

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has filed a Clinical Trial Application (CTA) with the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a Phase I clinical trial with ALN-TTR02, an RNAi therapeutic targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis.

7 Jan 2012

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

Ipsen, Oncodesign partner to discover and develop LRRK2 kinase inhibitors against PD

Oncodesign, a Drug Discovery company and Oncology pharmacology service provider, and Ipsen, a global specialty-driven pharmaceutical company, announced today that the two companies have entered into a research collaboration to discover and develop innovative LRRK2 kinase inhibitors as potential therapeutic agents against Parkinson's Disease and for potential additional uses in other therapeutic areas.

5 Jan 2012

Positive preliminary results from Alnylam's Phase I ALN-PCS trial on severe hypercholesterolemia

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today the achievement of positive preliminary results from its ongoing clinical trial of ALN-PCS, an RNAi therapeutic targeting PCSK9 for the treatment of severe hypercholesterolemia.

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

St. Michael's Hospital endocrinologist receives Novo Nordisk Diabetes Innovation Award

Dr. Andrew Advani, an endocrinologist at St. Michael's Hospital, is one of 10 researchers in North America who received a newly created Novo Nordisk Diabetes Innovation Award.

30 Dec 2011

AMT raises € 2.5 million through private placement

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, announced today that the Company has raised € 2.5 million in new equity by means of a private placement to three of its existing shareholders: Forbion Capital Partners, Gilde Healthcare Partners and Advent Venture Partners.

30 Dec 2011

Momenta, Baxter partner to develop and commercialize biosimilars

Baxter International Inc. and Momenta Pharmaceuticals, Inc. today announced that they have entered into a global collaboration to develop and commercialize follow-on biologic products, also known as biosimilars.

From Baxter International Inc. 23 Dec 2011

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