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Shares of Audentes rose more than 100% following news that the Japanese drugmaker would acquire it. The company’s lead candidate is a gene therapy for a rare muscular weakness disorder, currently in a Phase I/II study for which it presented data in October.NEXT ARTICLE
Gene therapy is the use of DNA as a pharmaceutical agent to treat disease. It derives its name from the idea that DNA can be used to supplement or alter genes within an individual's cells as a therapy to treat disease. The most common form of gene th...
In a clinical trial or interventional study, participants receive specific interventions according to the research plan or protocol created by the investigators. These interventions may be medical products, such as drugs or devices; procedures; or change...