Through gene therapy, researchers aim to permanently reprogram cells in the body to express healthy proteins or stop expressing damaging ones, potentially curing genetic disorders such as Huntington’s Disease. The main challenge has been finding gene vehicles such as harmless adeno-associated viruses (AAVs) that can deliver genes to specific cells and tissues in patients at low doses and without unwanted side effects. Latus Bio has developed a platform for screening tens of millions of engineered AAVs to identify the viruses whose capsids, or protein shells, are best tailored to infect target cells. In preclinical work in non-human primates, Latus has proved that AAVs with specific capsid variants can reach precise locations in the brain, integrate their gene cargo into host DNA, and express high levels of new proteins. Now the company is working to move candidate gene therapy treatments for Huntington’s and CLN2 disease toward safety studies in humans.
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