Abstract

Biotech investors and drug companies are slowly warming up to a new drug delivery strategy that uses exosomes, lipid vesicles naturally secreted by cells, to get drugs into places they normally can’t go. Oxford, England-based Evox Therapeutics is the latest start-up to raise significant funds—$45.4 million in its series B financing round—to move its experimental exosome therapies closer to the clinic. Now, Evox is disclosing the first details about its surprisingly diverse drug pipeline. Its most advanced program is for Niemann-Pick disease type C, a rare genetic condition in which a faulty protein causes an accumulation of cholesterol and lipids in the brain, liver, spleen, and other tissues. The buildup is particularly dangerous in the brain, where it leads to disability and early death. Evox hopes to treat the disease by packaging functional copies of the protein in exosomes, which in animal studies helped shuttle the protein into many organs,

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