The Pharmaceutical Industry’s Focus on Rare Diseases

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Published: 23 Feb 2018

This edition of the Genetic Technology TOE depicts life sciences trends across rare diseases. Since rare diseases affect only a small portion of the worldwide population; drug discovery, development, and optimization are difficult for such diseases. While only some companies are focusing in this regard, some large pharmaceutical companies are making outstanding advances in drug research and development. This deliverable profiles innovations of four pharmaceutical companies working on new solutions for uncommon and underserved medical conditions. The corresponding clinical trials scenario for Gaucher's disease is also depicted. The Genetic Technology TechVision Opportunity Engine (TOE) presents the latest R&D developments and advances with an emphasis on opportunities to profit in the explosive field of genetic technology through technology transfer, joint ventures, and acquisitions. The complete gamut of genetic technology applications is covered including the latest developments in omics technologies; genetic, cellular, and alternative therapies; sequencing technologies; and genetically altered animals and plants. The Health & Wellness cluster tracks developments in a myriad of areas including genetic engineering, regenerative medicine, drug discovery and development, nanomedicine, nutrition, cosmetic procedures, pain and disease management and therapies, drug delivery, personalized medicine, and smart healthcare. Keywords: Rare disease, genetic disorder, olipudase, protein-based therapy, lysosomal storage disorders, acid sphingomyelinase deficiency, Pompe’s disease, Gaucher’s disease, Mucopolysaccharidosis I, MPS II, neurofibromatosis, hereditary xerocytosis, enzyme replacement therapy, Hunter’s syndrome, Fabry’s disease, hereditary angioedema, blinatumomab, B-cell ALL, bispecific T cell engager


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