Beta Thalassemia News and Research

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Beta-thalassemia (ß-thalassemia) is a form of thalassemia due to mutations in the HBB gene on chromosome 11 , inherited in an autosomal recessive fashion.

Treating Sickle Cell Disease with L-Glutamine

By focusing on how best to help patients, researchers can sometimes find solutions that are much simpler than anticipated. That has been the guiding approach to our efforts to develop a treatment for sickle cell disease.

3 Jul 2012

FDA, EMA grant orphan drug designation to bluebird bio's gene therapy product for adrenoleukodystrophy

bluebird bio, a leader in the development of innovative gene therapies for severe genetic disorders, announced today that both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have granted an orphan drug designation to its investigational gene therapy product for the treatment of adrenoleukodystrophy (ALD).

19 Jun 2012

Alnylam first quarter revenues decrease to $20.6 million

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, today reported its consolidated financial results for the first quarter 2012, and company highlights.

4 May 2012

New gene transfer technique can treat beta-thalassemia, sickle cell anemia

A team of researchers led by scientists at Weill Cornell Medical College has designed what appears to be a powerful gene therapy strategy that can treat both beta-thalassemia disease and sickle cell anemia. They have also developed a test to predict patient response before treatment.

28 Mar 2012

New gene editing technique can heal sickle cell patients with their own cells

Researchers at the Salk Institute for Biological Studies have developed a way to use patients' own cells to potentially cure sickle cell disease and many other disorders caused by mutations in a gene that helps produce blood hemoglobin.

8 Dec 2011

FDA licenses NYBC's HEMACORD stem cell product

The Food and Drug Administration has licensed HEMACORD for allogeneic hematopoietic (blood-forming) stem cell transplantation, the first such approval of a stem cell product in the world.

11 Nov 2011

Acceleron commences ACE-536 Phase 1 clinical trial in anemia

Acceleron Pharma, Inc., a biopharmaceutical company developing protein therapeutics for cancer and orphan diseases today announced that it has initiated a Phase 1 clinical study of ACE-536.

14 Sep 2011

EGT transfers clinical grade lentiviral vector to MSKCC

Errant Gene Therapeutics, LLC, a pioneering boutique drug development firm specializing in Rare Diseases, announced the transfer of its clinical grade lentiviral vector, TNS 9.55.3, to Memorial Sloan Kettering Cancer Center.

29 Jul 2011

genOway acquires worldwide exclusive license for RMCE technology from bluebird bio for genetically modified rodents

genOway and bluebird bio report today genOway acquisition of an exclusive worldwide license for the RMCE technology (developed at the Massachusetts Institute of Technology) in the field of genetically modified rodents.

12 Jul 2011

bluebird bio announces receipt of additional $30 million in financing

bluebird bio, an emerging leader in the development of innovative gene therapies for severe genetic disorders, today announced that the company has secured an additional $30 million in financing.

20 Apr 2011

Metallic copper surfaces kill bacteria on contact

Metallic copper surfaces kill microbes on contact, decimating their populations, according to a paper in the February 2011 issue of the journal Applied and Environmental Microbiology. They do so literally in minutes, by causing massive membrane damage after about a minute's exposure, says the study's corresponding author, Gregor Grass of the University of Nebraska, Lincoln. This is the first study to demonstrate this mechanism of bacteriocide.

17 Feb 2011

Researchers report gene therapy strategy that improves Beta Thalassemia in mice model

Researchers at Nationwide Children's Hospital report a gene therapy strategy that improves the condition of a mouse model of an inherited blood disorder, Beta Thalassemia.

11 Jan 2011

Hepcidin hormone may be potential therapeutic tool for anemic blood disorders

A hormone made by the body may be a potential therapeutic tool for the treatment of two anemic blood disorders beta-thalassemia and hemochromatosis. The new research was led by scientists at Weill Cornell Medical College and published in the Journal of Clinical Investigation and the journal Blood.

24 Nov 2010

bluebird bio publishes LentiGlobin gene therapy clinical trial data results

bluebird bio an emerging leader in the development of innovative gene therapies for severe genetic disorders, today announced publication in the journal Nature of its promising Phase 1/2 data highlighting positive results of LentiGlobin gene therapy treatment in a young adult with severe beta-thalassemia, a blood disorder that is one of the most frequent inherited diseases.

16 Sep 2010

HemaQuest closes additional $4M to Series B financing

HemaQuest Pharmaceuticals, a clinical stage biotechnology company developing small molecule therapeutics for sickle cell disease, beta thalassemia and EBV-related cancers, announced today the closing of an additional $4M to its Series B financing by new investor, Latterell Venture Partners. This investment brings the total Series B financing to $16M.

28 Jul 2010

NHLBI funds nine research grants to determine safety and efficacy of red blood cell transfusions

The National Heart, Lung, and Blood Institute, part of the National Institutes of Health, is funding nine research grants to determine if the safety and efficacy of red blood cell transfusions vary depending on how long the cells have been stored. One of the grants supports the first large, multi-center, randomized clinical trial to compare outcomes in heart surgery patients who receive transfusions of red blood cells that have been stored for shorter or longer amounts of time.

22 Jun 2010

HemaQuest Pharmaceuticals closes $12M Series B financing

HemaQuest Pharmaceuticals, a clinical stage biotechnology company developing compounds to treat sickle cell disease, beta thalassemia and EBV-related cancers, announced today the closing of a $12M Series B financing led by new investor, Aberdare Ventures. HemaQuest's investor syndicate includes Aberdare Ventures, De Novo Ventures, Forward Ventures and Lilly Ventures.

21 Apr 2010

Innovative drug design wins medical research prize

The development of a new way to treat iron overload disease has won the 2010 Sir Zelman Cowen Universities Fund Prize for Medical Research.

1 Apr 2010

Pathway Genomics' personal Genetic Health Report now available

Pathway Genomics Inc., a U.S. based genetic testing company, announced the immediate availability of its personal Genetic Health Report. This customized and innovative personal genetic health report contains information on more than 70 health conditions, including pharmacogenetics (prescription medication response), propensity for complex disease, and carrier status (pre-pregnancy health).

17 Mar 2010

Genetix Pharmaceuticals announces completion of $35M Series B financing

Genetix Pharmaceuticals, a leading gene therapy company developing breakthrough treatments for severe genetic disorders, announced today that it has completed a $35 million Series B financing with new investors Third Rock Ventures and Genzyme Ventures joining TVM Capital, Forbion and Easton Capital. Proceeds from the financing will be used to advance current clinical programs, strengthen platform capabilities and further expand the team.

12 Mar 2010