foreword
AAV vector has become one of the popular vectors in the field of gene therapy due to its high safety, good tissue targeting, continuous and stable expression of foreign genes, and low immunogenicity. In recent years, AAV gene therapy has shown good application prospects in the field of rare diseases, including age-related macular degeneration (AMD), hemophilia, spinal muscular atrophy, frontotemporal dementia, glycogen storage disease, Huntington’s disease A variety of indications have made certain progress in clinical.
As a research hotspot in the field of gene therapy, the field of AAV gene therapy has attracted a large number of pharmaceutical companies to enter the market, including Spark, Regenxbio, uniQure, BioMarin, Xinzhi Medicine, Zhishanweixin, etc. In addition to the patent summary analysis of the AAV gene therapy drugs that have been marketed, this research report also organizes and analyzes the key patents of the Top 3 targets (F9, F8, GBA1) in the current statistical analysis of the AAV treatment field, especially the target nod. The patent layout of the pharmaceutical company’s drugs.
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