Thalassemia News and Research

Latest Thalassemia News and Research

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

Haploidentical bone marrow transplants for sickle cell disease: an interview with Dr. Javier Bolaños Meade

Bone marrow transplant (also called stem cell transplant) is a medical intervention that allows a physician to deliver very high doses of chemotherapy (if needed) but more importantly, deliver a new immune system to the patient to fight a disease (such as cancer).

3 Oct 2012

Enrollment complete in Gamida Cell’s NiCord Phase I/II trial for hematological malignancies

Gamida Cell, a leader in adult stem cell expansion technologies and products, announced today that it has completed enrollment for a Phase I/II clinical trial of NiCord, the company's second pipeline product.

20 Sep 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

Mechanical treatment shows promise in thalassemia

Vibration therapy may be an effective nonpharmacologic intervention to increase bone mass in patients with thalassemia, US researchers report

10 Jul 2012

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

Third Rock Ventures announces formation of Global Blood Therapeutics

Third Rock Ventures, LLC today announced the formation of Global Blood Therapeutics, Inc. and its $40.7 million Series A financing of the company.

15 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

Shire enters agreement to acquire FerroKin BioSciences

Shire plc, the global specialty biopharmaceutical company, announces that it has signed an agreement to acquire FerroKin BioSciences, Inc., for an upfront payment of $100 million, payable in cash at closing, plus potential post-closing milestone payments of up to $225 million, depending upon the achievement of certain clinical development, regulatory and net sales targets.

15 Mar 2012

HemaQuest closes $13 million extension of Series B financing

HemaQuest Pharmaceuticals, Inc., a biotechnology company focused on developing small molecule therapeutics to treat hemoglobin disorders, announced today that it has closed a $13 million extension of their Series B financing.

12 Mar 2012

Research assesses safety and efficacy of pre-operative transfusions, hydroxyurea therapy for SCD

Research assessing the safety and efficacy of hydroxyurea therapy in pediatric patients with sickle cell disease (SCD) and the use of pre-operative transfusions for patients with SCD who undergo low- and moderate-risk elective surgery will be presented today at the 53rd Annual Meeting of the American Society of Hematology.

12 Dec 2011

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

Novartis to showcase 160 presentations on breast cancer, hematological disease drugs at SABCS and ASH

Novartis Pharmaceuticals Corporation will showcase more than one hundred and sixty presentations on data from its robust oncology portfolio at two key medical congresses this month, demonstrating significant advances for patients with cancers and hematological diseases.

3 Dec 2011

Public health officials should address needs of people with blood disorders

Public health should focus not only on reducing the burden of common diseases but also address the needs of people with blood disorders , experts say in a supplement to December's American Journal of Preventive Medicine.

16 Nov 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

Thalassemia News and Research

Latest Thalassemia News and Research

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

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

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

Haploidentical bone marrow transplants for sickle cell disease: an interview with Dr. Javier Bolaños Meade

Enrollment complete in Gamida Cell’s NiCord Phase I/II trial for hematological malignancies

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

bluebird bio completes $60 million Series D financing

Mechanical treatment shows promise in thalassemia

Treating Sickle Cell Disease with L-Glutamine

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

Third Rock Ventures announces formation of Global Blood Therapeutics

Alnylam first quarter revenues decrease to $20.6 million

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

Shire enters agreement to acquire FerroKin BioSciences

HemaQuest closes $13 million extension of Series B financing

Research assesses safety and efficacy of pre-operative transfusions, hydroxyurea therapy for SCD

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

Novartis to showcase 160 presentations on breast cancer, hematological disease drugs at SABCS and ASH

Public health officials should address needs of people with blood disorders

FDA licenses NYBC's HEMACORD stem cell product

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

Newsletters you may be interested in