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.
Experimental gene therapy may improve health outcomes for patients with some forms of blood disorders

Experimental gene therapy may improve health outcomes for patients with some forms of blood disorders

Shire and Sangamo revise agreement to expedite development of ZFP Therapeutics for Huntington's disease, hemophilia A and B

Shire and Sangamo revise agreement to expedite development of ZFP Therapeutics for Huntington's disease, hemophilia A and B

Sangamo BioSciences to present data on ZFP Therapeutic platform at ASGCT meeting

Sangamo BioSciences to present data on ZFP Therapeutic platform at ASGCT meeting

Prolong Pharmaceuticals' SANGUINATE granted FDA Orphan Drug Designation for SCD treatment

Prolong Pharmaceuticals' SANGUINATE granted FDA Orphan Drug Designation for SCD treatment

FDA accepts Sangamo BioSciences' IND for SB-BCLmR-HSPC genome editing approach

FDA accepts Sangamo BioSciences' IND for SB-BCLmR-HSPC genome editing approach

Arcturus to exhibit new mRNA therapeutics pipeline at two healthcare conferences

Arcturus to exhibit new mRNA therapeutics pipeline at two healthcare conferences

Emerging strategies to boost healthy RBCs may reduce burden of anemia linked to blood disorders

Emerging strategies to boost healthy RBCs may reduce burden of anemia linked to blood disorders

Genom Austria project to explore impact of genome sequencing on science and society

Genom Austria project to explore impact of genome sequencing on science and society

Alnylam announces new pre-clinical results with DC for ALN-CC5 to treat complement-mediated diseases

Alnylam announces new pre-clinical results with DC for ALN-CC5 to treat complement-mediated diseases

CIRM grants $5.6 million to develop HIV/AIDS therapeutic using Sangamo's ZFN genome-editing technology

CIRM grants $5.6 million to develop HIV/AIDS therapeutic using Sangamo's ZFN genome-editing technology

Acceleron, Celgene to give oral presentations on beta-thalassemia, myelodysplastic syndromes at EHA meeting

Acceleron, Celgene to give oral presentations on beta-thalassemia, myelodysplastic syndromes at EHA meeting

Biogen Idec, Sangamo BioSciences partner to develop therapeutics for hemoglobinopathies

Biogen Idec, Sangamo BioSciences partner to develop therapeutics for hemoglobinopathies

New projects advance treatments for radiation syndrome, cardiac arrest-induced brain injury and rare blood disorder

New projects advance treatments for radiation syndrome, cardiac arrest-induced brain injury and rare blood disorder

Alnylam initiates Phase II study with ALN-TTRsc for treatment of ATTR

Alnylam initiates Phase II study with ALN-TTRsc for treatment of ATTR

Acetylon Pharmaceuticals presents positive preclinical data for selective HDAC1/2 inhibitors at ASH meeting

Acetylon Pharmaceuticals presents positive preclinical data for selective HDAC1/2 inhibitors at ASH meeting

Alnylam presents new pre-clinical data with ALN-AT3 for treatment of hemophilia and RBD

Alnylam presents new pre-clinical data with ALN-AT3 for treatment of hemophilia and RBD

Acetylon announces positive preclinical data of ricolinostat (ACY-1215) in models of lymphoma

Acetylon announces positive preclinical data of ricolinostat (ACY-1215) in models of lymphoma

Acetylon Pharmaceuticals presents positive interim data from two ricolinostat clinical trials at ASH 2013

Acetylon Pharmaceuticals presents positive interim data from two ricolinostat clinical trials at ASH 2013

Novartis to present updates on broad cancer portfolio at ASH and SABCS symposiums

Novartis to present updates on broad cancer portfolio at ASH and SABCS symposiums

Alnylam advances Development Candidate for ALN-AS1 for treatment of hepatic porphyrias

Alnylam advances Development Candidate for ALN-AS1 for treatment of hepatic porphyrias