Thalassemia News and Research

Latest Thalassemia News and Research

Sangamo presents Phase 2 data from two ongoing clinical trials of SB-728-T for treatment of HIV/AIDS

Sangamo BioSciences, Inc., the leader in therapeutic genome editing, announced the presentation of Phase 2 data from two of the Company's ongoing clinical trials (SB-728-1101 Cohort 3* and SB-728-mR-1401) of SB-728-T, which is being developed for the functional control of HIV/AIDS.

11 Dec 2015

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

New research adds to a growing body of evidence that gene therapy, an experimental technique that involves correcting or replacing a person's mutated or malfunctioning genes, may improve health outcomes for patients with inherited bleeding and immune disorders as well as some forms of blood cancer.

7 Dec 2015

Boston Children's Hospital selects Vijay G. Sankaran to receive Rising Star Award

Saluting his spectacular track record to date of innovative research on red blood cell disorders and his future promise as a physician/scientist, Boston Children's Hospital has selected Vijay G. Sankaran, MD, PhD, of Dana-Farber/Boston Children's Cancer and Blood Disorders Center to receive the Rising Star Award at the hospital's third annual Global Pediatric Innovation Summit, Taking on Tomorrow (#PedInno15).

11 Nov 2015

New method may extend use of noninvasive prenatal testing to detect chromosomal abnormalities

Researchers at the University of California, San Diego School of Medicine developed a method to expand the types of chromosomal abnormalities that noninvasive prenatal testing (NIPT) can detect. The study, published November 9 by Proceedings of the National Academy of Sciences, uses a semiconductor sequencing platform to identify small chromosomal deletions or duplications, such as occur in Cri du Chat Syndrome and DiGeorge Syndrome, with a simple blood test from the expectant mother.

10 Nov 2015

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

Sangamo BioSciences, Inc., a leader in therapeutic genome editing, announced today that the company and its collaborator, Shire plc, have agreed to revise their January 2012 collaboration and license agreement to expedite the development of ZFP Therapeutics for hemophilia A and B and Huntington's disease.

2 Sep 2015

Gamida Cell reaches agreements with FDA and EMA regarding NiCord Phase III study design outline

Gamida Cell, a leader in cellular and immune therapies for the treatment of cancer and orphan genetic diseases, announced today that the company has reached agreements with the FDA and EMA regarding the Phase III study design outline of NiCord.

10 Aug 2015

Americord Registry, Smart Cells International partner to educate parents on benefits of cord blood banking

Today, Americord Registry announced a new partnership with Smart Cells International, the first cord blood company in the United Kingdom, as part of its initiatives during Cord Blood Awareness Month (July) to educate parents around the globe on the benefits of cord blood banking.

26 Jun 2015

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

Sangamo BioSciences, Inc. announced that data from clinical, preclinical and research-stage programs focused on the development of ZFP Therapeutics will be presented at the 18th Annual Meeting of the American Society of Gene and Cell Therapy

29 Apr 2015

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

Prolong Pharmaceuticals, LLC, a biopharmaceutical company dedicated to developing products for the treatment of anemias, cancers and their debilitating comorbidities, announced today that the U.S. Food and Drug Administration has granted Orphan Drug Designation for its flagship product SANGUINATE for the treatment of Sickle Cell Disease (SCD).

8 Apr 2015

New resource available to help older Chinese Americans better understand their healthcare needs

On the eve of National Minority Health Month, which helps raise awareness for disparities in health and care among minorities in the U.S., a new resource is available to help one such group, older Chinese Americans, better understand and drive their own well-being.

3 Apr 2015

Emmaus Life Sciences presents Phase 3 clinical trial of PGLG treatment for sickle cell disease

Emmaus Life Sciences, Inc., a biopharmaceutical company dedicated primarily to the discovery, development and commercialization of innovative treatments and therapies for rare and orphan diseases today announced that data from the Company’s Phase 3 clinical trial of its pharmaceutical grade L-glutamine (PGLG) treatment for sickle cell anemia and sickle beta-0 thalassemia, will be presented, during the 9th Annual Sickle Cell Disease Research and Educational Symposium and 38th National Sickle Cell Disease Scientific Meeting.

1 Apr 2015

MRI more accurate, safe and less painful than liver biopsy in measuring total body iron balance

Investigators at The Saban Research Institute of Children's Hospital Los Angeles have demonstrated that MR imaging of the liver is more accurate than liver biopsy in determining total body iron balance in patients with sickle cell disease and other disorders requiring blood transfusion therapy.

20 Mar 2015

New bone marrow transplant unit launched in Bangalore, India

A bone marrow transplant can mean the difference between life and death for people with blood cancers and related disorders. But many patients in India can't afford the high treatment costs, and for them a transplant is not an option. This is changing thanks to a newly launched bone marrow transplant unit at M.S. Ramaiah Medical College in Bangalore.

19 Mar 2015

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

Sangamo BioSciences, Inc. announced today that an Investigational New Drug (IND) application for the company's SB-BCLmR-HSPC genome editing approach, which is designed to provide a one-time lasting therapy for beta-thalassemia, has been accepted by the U.S. Food and Drug Administration and is now active.

4 Feb 2015

Arcturus to exhibit new mRNA therapeutics pipeline at two healthcare conferences

Arcturus Therapeutics Inc., developing the 'next wave' of RNA medicines, today announced that Joseph Payne, President and Chief Executive Officer, is scheduled to present a corporate overview, including the company's new messenger RNA (mRNA) therapeutics pipeline, at two upcoming healthcare conferences.

7 Jan 2015

Gamida Cell granted FDA and EMA orphan drug designation for NiCord

Gamida Cell, a leader in cell therapy technologies and products for transplantation and adaptive immune therapy, announced today that orphan drug designation has been granted by The US Department of Health and Human Services, The FDA Office of Orphan Products Development (OOPD) for the investigational medicinal product NiCord for the treatment of acute lymphoblastic leukemia (ALL), acute myeloid leukemia (AML), Hodgkin lymphoma and myelodysplastic syndrome (MDS).

6 Jan 2015

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

Emerging treatment approaches may reduce the burden of anemia associated with blood disorders by enhancing production of healthy red blood cells, according to data presented today at the 56th American Society of Hematology Annual Meeting and Exposition.

7 Dec 2014

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

In many countries, genome sequencing technology is now starting to become available in the clinic, where it helps to diagnose rare Mendelian diseases and contributes to personalized cancer therapy. The analysis of personal genomes also creates unprecedented opportunities for predictive health counseling, ancestry research, and many more applications that are just starting to emerge.

26 Nov 2014

BloodCenter's Erythroid Chimerism test available to monitor transplanted SCD patients

BloodCenter of Wisconsin's Diagnostic Laboratories today announced the availability of an innovative Erythroid Chimerism test to monitor erythroid lineage chimerism in patients with sickle cell disease (SCD) following allogeneic bone marrow transplantation.

28 Aug 2014

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

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today new pre-clinical results with its Development Candidate (DC) for ALN-CC5, a subcutaneously administered investigational RNAi therapeutic targeting complement component C5 in development for the treatment of complement-mediated diseases.

10 Jun 2014

Thalassemia News and Research

Latest Thalassemia News and Research

Sangamo presents Phase 2 data from two ongoing clinical trials of SB-728-T for treatment of HIV/AIDS

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

Boston Children's Hospital selects Vijay G. Sankaran to receive Rising Star Award

New method may extend use of noninvasive prenatal testing to detect chromosomal abnormalities

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

Gamida Cell reaches agreements with FDA and EMA regarding NiCord Phase III study design outline

Americord Registry, Smart Cells International partner to educate parents on benefits of cord blood banking

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

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

New resource available to help older Chinese Americans better understand their healthcare needs

Emmaus Life Sciences presents Phase 3 clinical trial of PGLG treatment for sickle cell disease

MRI more accurate, safe and less painful than liver biopsy in measuring total body iron balance

New bone marrow transplant unit launched in Bangalore, India

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

Arcturus to exhibit new mRNA therapeutics pipeline at two healthcare conferences

Gamida Cell granted FDA and EMA orphan drug designation for NiCord

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

BloodCenter's Erythroid Chimerism test available to monitor transplanted SCD patients

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

Expanding research and clinical options for children with cancer

Examining how pain could play a direct protective role in the gut

From Puberty to Menopause: Clue’s CEO, Audrey Tsang on the Power of Femtech

Thalassemia News and Research

Latest Thalassemia News and Research

Expanding research and clinical options for children with cancer

Examining how pain could play a direct protective role in the gut

From Puberty to Menopause: Clue’s CEO, Audrey Tsang on the Power of Femtech

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