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The RNA Zipcode Discovery Pipeline: Emerging tools for therapeutic targeting at subcellular resolution

2017 Disruptive Innovation in Genomics Competition For Phase 1 Projects Advancing to Phase 2
Genome Centre(s):
Génome Québec
Project Leader(s):
Eric Lécuyer (Institut de recherche clinique de Montréal (IRCM)), Mathieu Blanchette (McGill University), Jérôme Waldispühl (McGill University)
Project Description: 

The human body is made up of trillions of cells, each of which has its own role to play in making our bodies function properly. To carry out their specified role, cells needs to organize their inner components very precisely. If this organization is defective, we become susceptible to different diseases such as neuromuscular disorders or cancer.

The cell directs its components to the right place using an intricate addressing system, similar to the way postal codes get our mail to the right location. Dr. Eric Lécuyer and his team are working to increase our understanding of how this system works, focusing on RNA. In the first phase of this competition, the team developed methods to identify the “zipcodes” that are present in different kinds of RNA molecules, which help transport the RNA to specific places in the cell. Now the team plans to use these zipcodes as tools to target drugs to specific destinations inside our cells. The project will result in a proprietary prototype zipcode discovery platform, a collection of patentable RNA zipcodes that enhance the targeting of nucleic acid-based drugs, of RNA-based vaccines and of RNA editing systems, such as CRISPR.

By improving the delivery of therapeutic molecules at subcellular resolution, this project will speed up the development of drugs for currently incurable diseases, such as Myotonic Dystrophy type 1. The commercial value of the technologies will be captured through the creation, together with partner AmorChem, of a Montreal-based spin-off company.