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AssigneeApplied Molecular Transport Inc.
InventorsRandall J. Mrsny, Tahir Mahmood
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The field of the present invention relates, in part, to a strategy for novel pharmaceutical applications. More specifically, the present invention relates to a genetically detoxified form of Vibrio cholera exotoxin (cholix) and the use of cholix-derived polypeptide sequences to enhance intestinal delivery of biologically-active therapeutics. Importantly, the systems and methods described herein provide for the following: the ability to deliver macromolecule doses without injections; the ability to deliver cargo, such as (but not limited to) siRNA or antisense molecules into intracellular compartments where their activity is required; and the delivery of nanoparticles and dendrimer-based carriers across biological membranes, which otherwise would have been impeded due to the barrier properties of most such membranes.
An isolated delivery construct comprising a transporter domain and a cargo, said construct capable of delivering said cargo into targeted intracellular compartments.
An isolated delivery construct comprising a transporter domain and a macromolecule, said construct capable of delivering said macromolecule to a biological compartment of a subject without injections.
A polynucleotide that encodes a delivery construct, said delivery construct comprising a transporter domain and a cargo, said construct capable of delivering said cargo into targeted intracellular compartments.
Dependencies are inferred from the claim text.