Application

Protein polymers enabling engineering of human membraneless organelles.

Key Benefits

• Versatile applications across immunotherapy, cell therapy, and other therapeutic modalities.
• High biocompatibility with minimal reactivity with endogenous molecules.
• Deployable for organelle-level functionalization of human cells.

Market Summary

Proteins and RNA in cells can undergo natural phase separation—producing concentrated regions with specialized function called membraneless organelles (MLOs). Although these naturally occurring condensates have been characterized in basic research, attempts at synthesizing MLOs have had limited translational value—due to unknown effects of synthetic condensates on cell fitness, and limited knowledge of optimal scaffolding. Although MLOs have not yet been harnessed for disease intervention, they have broad potential in cell therapies and immunotherapies. The massive cell therapy biomanufacturing and cancer immunotherapy markets are hungry for novel therapeutic agents and cell functionalization methods—and MLOs represent an early and exciting research area for programming previously inaccessible subcellular functions.

Technical Summary

Emory and Georgia Tech researchers have created a robust platform of intrinsically disordered protein-polymers for generating intracellular protein-based biomaterials which function as heritable MLOs. The technology has been established in a range of human cell types, including immune cells, for diverse applications across disease areas. The technology enables the engineering and organelle-level functionalization of human cells with one, two or three distinctly addressable novel MLOs, and permits the replacement of select endogenous MLOs that are lost in disease states. This technology has demonstrated subcellular organelle-level functionality, interpretable sequence-structure information, minimal crosstalk with endogenous processes, sustained target engagement across time, and genetically encoded delivery in human cells.

Development Stage

The inventors have generated numerous compositions of diverse intracellular protein polymers and demonstrated methods for using them in human organelle engineering.