Stimuli-responsive traceless engineered platform for intracellular payload delivery

Abstract

Disclosed are conjugates and pharmaceutical compositions containing these conjugates for intracellular delivery of payloads. The conjugates contain a gene editing mechanism or protein as a payload, a preferably stimuli-responsive, self-immolative chemical linker, and a cell membrane fusogenic molecule. One or more stimuli within the cell, such as a reducing and / or acidic environment, cleave the stimuli-responsive chemical moiety, activating the self-destruction of the chemical linker. In some embodiments, this process results in the release of the payload from the conjugate without including the components of the linker and the cell membrane fusogenic molecule that are covalently attached to the gene editing mechanism or protein payload.

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