Hijacking cellular mechanisms to treat complex diseases

Overview

Cells in the human body are equipped with various systems for producing and disposing of proteins. These include transcriptional and translational machinery, through which proteins are expressed, as well as the ubiquitin-proteasome system, which identifies, labels, and destroys unused or damaged proteins.

This whitepaper reviews two novel mechanisms in drug development, both of which harness the innate processes of the cell to modulate protein levels for therapeutic benefit. Armed with new insights into how these systems work, scientists have worked to design drug molecules that will harness these systems to treat or cure diseases of protein deficiency and/or overactivity. Nucleic acid therapies have the potential to circumvent defective genes and produce needed proteins. Meanwhile, targeted protein degradation offers a way to remove harmful proteins from the cell.

Key Objectivesï¼š

Overview of current drug discovery paradigm contrasted with novel drug modalities
Current landscape and biological mechanism of therapeutic nucleic acids
Targeted protein degradation, e.g. PROTACs platform
Perspectives on the business of bringing novel drug modalities to market

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