Astellas gene therapy repairs damage in neurodegenerative disease models

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Gene therapies that replace a single missing or faulty gene have been successful in treating some disorders, such as spinal muscular atrophy, the target of Novartis’ Zolgensma. But most neurodegenerative disorders are caused by multiple genetic abnormalities, making them difficult to address with gene therapy targeted at single mutations.

Scientists at the University of Cambridge have chosen a different path. Instead of going after the dysfunctional genes, they have developed a gene therapy that expresses two therapeutic proteins that boost nerve development and function. The therapy was the target of Astellas’ acquisition of U.K. gene therapy biotech Quethera in 2018.