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.

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

Acetylon Pharmaceuticals, Inc., the leader in the development of selective histone deacetylase inhibitors for enhanced therapeutic outcomes, today announced that positive interim clinical data from the two proof-of-concept clinical trials with selective HDAC6 inhibitor, ricolinostat (ACY-1215), were presented at the 55th Annual Meeting of the American Society of Hematology in New Orleans, LA.

9 Dec 2013

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

Novartis will present updates on its broad cancer portfolio with more than 240 abstracts at the upcoming American Society of Hematology (ASH) annual meeting and CTRC-AACR San Antonio Breast Cancer Symposium (SABCS).

4 Dec 2013

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

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that it is advancing its Development Candidate for ALN-AS1, an RNAi therapeutic targeting aminolevulinate synthase-1 (ALAS-1) for the treatment of hepatic porphyrias including acute intermittent porphyria (AIP).

9 Oct 2013

Sangamo BioSciences signs definitive agreement to buy Ceregene

Sangamo BioSciences, Inc. announced that it signed a definitive agreement to acquire Ceregene, Inc., a privately held biotechnology company focused on developing adeno-associated virus gene therapies.

26 Aug 2013

FDA grants Orphan Drug Designation to Alnylam ALN-AT3 for treatment of hemophilia B

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that the U.S. Food & Drug Administration (FDA) has granted an Orphan Drug Designation to ALN-AT3 as a therapeutic for the treatment of hemophilia B.

17 Aug 2013

Alnylam releases positive top-line data from ALN-TTRsc Phase I trial for TTR-mediated amyloidosis

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today positive top-line results from its ongoing Phase I trial of ALN-TTRsc, a subcutaneously administered RNAi therapeutic targeting the transthyretin gene for the treatment of TTR-mediated amyloidosis.

12 Jul 2013

Alnylam Pharmaceuticals presents new pre-clinical data of ALN-AT3 at ISTH meeting

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has presented new pre-clinical data from its RNAi therapeutic program for the treatment of hemophilia and rare bleeding disorders.

2 Jul 2013

Alnylam presents new pre-clinical data of ALN-CC5 for treatment of complement-mediated diseases

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has presented new pre-clinical data with ALN-CC5, an RNAi therapeutic targeting complement component C5.

21 Jun 2013

Alnylam reports pre-clinical data from ALN-AS1 program for treatment of AIP

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has presented key pre-clinical proof-of-concept data from its RNAi therapeutic program targeting aminolevulinate synthase-1 (ALAS-1) for the treatment of porphyria including acute intermittent porphyria.

18 May 2013

Two studies shed light on molecular biology of three blood disorders

Two studies led by investigators at Weill Cornell Medical College shed light on the molecular biology of three blood disorders, leading to novel strategies to treat these diseases.

26 Mar 2013

Acceleron commences ACE-536 phase 2 study in beta-thalassemia

Acceleron Pharma, Inc., a biopharmaceutical company developing protein therapeutics for cancer and orphan diseases, today announced the initiation of a phase 2 study of its novel, investigational protein therapeutic, ACE-536, to treat patients with beta-thalassemia, a genetic hematologic disorder causing chronic and life-threatening anemia and serious complications affecting the spleen, liver and heart.

7 Mar 2013

Sangamo BioSciences fourth quarter revenues increase to $8.9 million

Sangamo BioSciences, Inc. today reported fourth quarter and full year 2012 financial results and accomplishments.

7 Feb 2013

Acceleron commences ACE-536 phase 2 study in myelodysplastic syndromes

Acceleron Pharma, Inc., a biopharmaceutical company developing protein therapeutics for cancer and orphan diseases, today announced the initiation of a phase 2 study of its investigational protein therapeutic, ACE-536, to treat anemia in patients with myelodysplastic syndromes (MDS).

30 Jan 2013

Acetylon announces results from selective HDAC 1/2 inhibitor preclinical study on SCD and bT

Acetylon Pharmaceuticals Inc., a leader in targeted epigenetic drug discovery and development for enhanced therapeutic outcomes, today announced results from a preclinical study of a selective histone deacetylase (HDAC) 1/2 inhibitor for the treatment of sickle cell disease (SCD) and beta-thalassemia (bT) at the 54th Annual Meeting of the American Society of Hematology (ASH), taking place in Atlanta, Georgia.

11 Dec 2012

Sickle cell disease trial completes phase 3 patient enrollment

Emmaus Medical, Inc., a specialty pharmaceutical and regenerative medicine company, today announced the completion of patient enrollment for its phase 3 clinical trial to study L-glutamine as a treatment for sickle cell disease.

5 Dec 2012

Novartis to highlight key data from extensive oncology portfolio at SABCS and ASH meeting

Novartis will highlight more than 140 presentations on key data from its extensive oncology portfolio at the leading year-end scientific meetings devoted to hematology and breast cancer, demonstrating continued innovation in research and development efforts to advance the care of patients with cancer and rare diseases.

30 Nov 2012

Acetylon's ective histone deacetylase inhibitors to be showcased at 54th ASH annual meeting

Acetylon Pharmaceuticals Inc., a leader in targeted epigenetic drug discovery and development for enhanced therapeutic outcomes, today announced that its selective histone deacetylase (HDAC) inhibitors will be featured in five posters and one oral presentation at the 54th Annual Meeting of the American Society of Hematology (ASH), taking place December 8-11, 2012, in Atlanta, Georgia.

21 Nov 2012

Sickle cell disease treatments: An interview with Dr Niihara, CEO of Emmaus Medical

Sickle cell disease is a devastating hereditary condition that affects millions of people, particularly those in West and Central Africa as well as people of African descent in the United States, Europe and South America.

23 Oct 2012

Data from Sangamo BioSciences’ SB-728-T Phase 1 clinical programs on HIV/AIDS

Sangamo BioSciences, Inc. announced that data from its Phase 1 clinical programs to develop SB-728-T, a novel therapeutic approach designed to generate a "functional cure" for HIV/AIDS, were presented at the 52nd Interscience Conference on Antimicrobial Agents and Chemotherapy (ICAAC).

11 Sep 2012

bluebird bio completes $60 million Series D financing

bluebird bio, a leader in the development of innovative gene therapies for severe genetic disorders, today announced the successful completion of a $60 million Series D financing. In this round, new investors Deerfield Partners, RA Capital, Ramius Capital Group, and two undisclosed blue chip public investment funds joined existing investors ARCH Venture Partners, Third Rock Ventures, TVM Capital, and Forbion Capital Partners. In addition, Shire plc joined the round as a strategic investor.

25 Jul 2012