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.

Progress with GSK in advancing VaxiRNA technology earns Alnylam $3.2M milestone payment

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has earned a development milestone payment from GlaxoSmithKline, totaling $3.2 million. The milestone payment is related to progress in the companies' collaboration on the use of VaxiRNA technology for the production of GSK influenza vaccine.

30 Jul 2012

uniQure receives positive recommendation from EMA CHMP for Glybera

uniQure announced today that the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion that recommends marketing authorization of Glybera (alipogene tiparvovec) as a treatment for lipoprotein lipase deficiency (LPLD) under exceptional circumstances.

21 Jul 2012

Sanquin, Baxter enter manufacturing services agreement for plasma therapies

Baxter International Inc. today announced that it has entered into a manufacturing services agreement with Stichting Sanquin Bloedvoorziening (Sanquin Blood Supply Foundation) in the Netherlands that will provide Baxter up to 1.6 million liters of incremental plasma fractionation capacity annually to support global growth of plasma-derived treatments.

From Baxter International Inc. 20 Jul 2012

Emmaus receives EC Orphan Medicinal Product designation for Levoglutamide

Emmaus Medical, Inc., a specialty pharmaceutical and regenerative medicine technology company, and subsidiary of Emmaus Life Sciences, Inc., today announced that the European Commission (EC) has granted Orphan Medicinal Product designation for the company's investigational drug Levoglutamide (L-glutamine) for the treatment of sickle cell disease.

18 Jul 2012

Premier research institutions, Biogen Idec partner to sequence genomes of patients with ALS

Biogen Idec today announced that it has entered into a research collaboration with premier academic and research institutions to sequence the genomes of up to 1,000 patients with amyotrophic lateral sclerosis (ALS) in an effort to gain a deeper understanding about the fundamental genetic causes of ALS.

17 Jul 2012

Positive results from Alnylam’s ALN-TTR02 Phase I trial on TTR-mediated amyloidosis

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today the achievement of positive clinical results from its Phase I trial with ALN-TTR02, an RNAi therapeutic targeting the transthyretin (TTR) gene for the treatment of TTR-mediated amyloidosis.

16 Jul 2012

New paper documents discovery of novel lipids, including MC3

Alnylam Pharmaceuticals, a leading RNAi therapeutics company, and collaborators announced today the publication of a scientific paper documenting the discovery of novel lipids used in second generation lipid nanoparticles (LNPs) for systemic delivery of RNAi therapeutics.

14 Jul 2012

SRI receives NIAID grant to develop HIV vaccine

The Scripps Research Institute has been awarded a grant expected to total more than $77 million from the National Institutes of Health's (NIH) National Institute of Allergy and Infectious Diseases (NIAID).

12 Jul 2012

PROLOR to present data on long-acting clotting factors for hemophilia at WFH World Congress

PROLOR Biotech, Inc., announced that the company will present data today on its long-acting clotting factors for the treatment of hemophilia at the World Federation of Hemophilia (WFH) World Congress.

11 Jul 2012

Roundup: Tough to get on Calif.'s new adult day care

Community-Based Adult Services began March 1 as part of a legal settlement between the state and advocates that sued over Gov. Jerry Brown's plan to eliminate Adult Day Health Care entirely.

11 Jul 2012

Inspiration receives FDA clinical hold order for IB1001 phase III studies on hemophilia B

Ipsen announced today that its partner Inspiration Biopharmaceuticals Inc. (Inspiration) was notified by the Food and Drug Administration (FDA) that both clinical trials evaluating the safety and efficacy of IB1001, an investigational intravenous recombinant factor IX (rFIX) therapy for the treatment and prevention of bleeding episodes in people with hemophilia B, were placed on clinical hold.

10 Jul 2012

Positive interim results from Inspiration's OBI-1 Phase 2/3 study for acquired hemophilia A

Inspiration Biopharmaceuticals, Inc. today reported data from its ongoing Phase 2/3 study evaluating the efficacy of OBI-1, an investigational recombinant porcine factor VIII (FVIII), for the treatment of serious bleeds in people with acquired hemophilia A. According to the interim analysis, seven out of seven trial participants receiving OBI-1 experienced control and subsequent resolution of their bleeds.

9 Jul 2012

Bayer commences enrollment in BAY94-9027 Phase II/III trial for hemophilia A

Bayer HealthCare today announced the company has started to enroll patients in an international Phase II/III trial to evaluate its investigational compound BAY94-9027 for the treatment of hemophilia A.

8 Jul 2012

Study sheds light on the assembly of ribosome

Scientists from the Florida campus of The Scripps Research Institute have identified a series of intricate biochemical steps that lead to the successful production of proteins, the basic working units of any cell.

6 Jul 2012

Novo Nordisk, BRI collaborate to speed-up translational research of autoimmune diseases

Benaroya Research Institute at Virginia Mason (BRI), a non-profit biomedical research institute in Seattle, Wash., and Novo Nordisk, a global health care company headquartered in Denmark, today announced a three-year collaborative agreement to potentially speed-up translational research of the diagnosis and treatment of rheumatoid arthritis, inflammatory bowel disease and lupus.

29 Jun 2012

Powerful new technique can manipulate the building-block molecules of organic chemistry

Scientists at The Scripps Research Institute have developed a powerful new technique for manipulating the building-block molecules of organic chemistry. The technique enables chemists to add new functional molecules to previously hard-to-reach positions on existing compounds-making it easier for them to generate new drugs and other organic chemicals.

28 Jun 2012

China SFDA approves China Biologic's Human Coagulation Factor VIII

China Biologic Products, Inc., a leading fully integrated plasma-based biopharmaceutical company in China, today announced that its indirectly owned subsidiary, Shandong Taibang Biological Products Co., Ltd. has received a manufacturing approval certificate from the China State Food and Drug Administration for Human Coagulation Factor VIII ("FVIII").

25 Jun 2012

FDA approves Baxter's GAMMAGARD LIQUID for multifocal motor neuropathy

Baxter International Inc. today announced that the U.S. Food and Drug Administration (FDA) has approved GAMMAGARD LIQUID 10% [Immune Globulin Infusion (Human)] as a treatment for multifocal motor neuropathy (MMN). This is the first immunoglobulin treatment approved for MMN patients in the United States, and it was approved for use with MMN patients in Europe in 2011.

From Baxter International Inc. 25 Jun 2012

FDA grants Orphan Drug Designation to Medgenics’ INFRADURE Biopump

Medgenics, Inc., the developer of Biopump, a novel technology for the sustained production and delivery of therapeutic proteins in patients using their own tissue, today announced that its INFRADURE Biopump has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development (OOPD) for the treatment of hepatitis D.

21 Jun 2012

FDA provides Orphan Drug Designation to Alnylam’s ALN-TTR02

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that the U.S. Food & Drug Administration (FDA) has provided Orphan Drug Designation to ALN-TTR02 as a therapeutic for the treatment of familial amyloidotic polyneuropathy (FAP), one of the predominant clinical manifestations of transthyretin (TTR)-mediated amyloidosis (ATTR).

20 Jun 2012