Gene Therapies for Rare and Genetic Diseases

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Published: 25 May 2018

This edition of the Genetic Technology TOE provides insights across recent developments in gene therapies for rare and genetic disorders. The TOE also provides a clinical trial analysis for ex vivo gene therapy. 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: Gene therapy, rare disease, genetic disease, spinal muscular atrophy, AVXS-101, autologous ex vivo gene therapy, metachromatic leukodystrophy, Wiskott Aldrich’s syndrome, adenosine deaminase severe combined immunodeficiency, X-linked chronic granulomatous disease, beta-thalassemia, IMR-687, Niemann-Pick Disease Type C (NPC), sickle cell disease, AVROBIO, Fabry's disease, Gaucher's disease, Pompe's disease, cystinosis, plasmid lentiviral vectors, AVR-RD-01, AVR-RD-02, AVR-RD-03, AVR-RD-04

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