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Hemoglobinopathies Companies

Hemoglobinopathies are a group of genetic disorders characterized by abnormalities in hemoglobin, the protein responsible for transporting oxygen in red blood cells. Conditions such as sickle cell disease and thalassemia are examples of hemoglobinopathies. Companies involved in hemoglobinopathies focus on developing diagnostics, treatments, and therapies to manage these genetic disorders.

Hemoglobinopathies Key CompaniesLatest Hemoglobinopathies Companies Update

April 2023: ECHO India, a non-profit organization dedicated to building India's healthcare capacity, has partnered with the Post Graduate Institute of Child Health (PGICH) to create a statewide initiative focusing on the prevention and control of beta thalassemia and other hemoglobinopathies. The program, which began today with a rigorous two-day Training of Trainers (ToT), will be conducted as part of a countrywide collaborative project.  The National Hub for ECHO India's initiative will be PGICH, a prominent paediatric facility created. This effort intends to train healthcare personnel across the country in the prevention and management of Beta Thalassemia and other Hemoglobinopathies, with a focus on distant and disadvantaged areas.


February 2023: Vertex Therapeutics celebrated the launch of the first CRISPR-based gene editing medication as it nears completion of a historic FDA filing. Vertex estimates that a network of around 50 authorized treatment sites in the United States and 25 in Europe will be sufficient for its exagamglogene autotemcel, or exa-cel, gene therapy candidate for sickle cell disease and beta thalassemia. These individuals require many hospitalizations per year for vaso-occlusive crises or are dependent on blood transfusions on a regular basis. In November 2022, Vertex and CRISPR Therapeutics began submitting exa-cel for sickle cell disease and beta thalassemia. Outside of the United States, regulatory process has been a little speedier, since the European Medicines Agency and authorities in the United Kingdom have already confirmed the files.List of Hemoglobinopathies Key companies in the market

  • Sanofi

  • Sangamo Therapeutics, Inc.

  • Global Blood Therapeutics

  • Neusoft Medical Systems Co., Ltd

  • Bluebird Bio, Inc.

  • Emmaus Life Sciences Inc.

  • Prolong Pharmaceuticals

  • Celgene Corporation

  • Alnylam Pharmaceuticals

  • Gamida Cell

  • Acceleron Pharma

  • Mast Therapeutic

  • HemaQuest Pharmaceuticals

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