Sickle Cell Disease Drug Development Pipeline Review, 2017
Summary Sickle cell anemia is a genetic (inherited) blood disorder in which red blood cells, which carry oxygen around the body, develop abnormally. Signs and symptoms include anemia, delayed growth, vision problems, pain and frequent infections. There are a total of 62 products in development for this indication, by 45 companies and nine academic institutions.
Key companies operating in this pipeline space include Acceleron Pharma, Epidestiny, Errant Gene Therapeutics, Gamida Cell, Global Blood Therapeutics, Hillhurst Biopharmaceuticals, Novartis and Regenacy Pharmaceuticals. Hemoglobin and a range of histone deacetylases are the most common molecular targets being studied in sickle cell anemia.
Vaso-occlusive crisis associated with sickle cell disease is a painful episode that occurs in people who have sickle cell anemia. Symptoms include fever, pain, fatigue, abdominal pain and swelling, headaches, shortness of breath, dehydration and infection. There are a total of 10 products in development for this indication, by nine companies.
This report provides an overview of the Sickle Cell Disease pipeline landscape. The report provides comprehensive information on the therapeutics under development and key players involved in therapeutic development for sickle cell anemia and vaso-occlusive crisis associated with sickle cell disease, and features dormant and discontinued projects.
Scope - Which companies are the most active within each pipeline? - Which pharmaceutical approaches are the most prominent at each stage of the pipeline and within each indication? - To what extent do universities and institutions play a role within this pipeline, compared to pharmaceutical companies? - What are the most important R&D milestones and data publications to have happened in this disease area?
Reasons to buy - Understand the overall pipeline, with an at-a-glance overview of all products in therapeutic development for each indication - Assess the products in development in granular detail, with an up-to-date overview of each individual pipeline program in each indication, and a comprehensive picture of recent updates and milestones for each - Analyze the companies, institutions and universities currently operating in the pipeline, and the products being fielded by each of these - Understand the composition of the pipeline in terms of molecule type, molecular target, mechanism of action and route of administration
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