Journeying within: exploring new frontiers in therapeutic delivery

- Quebec City, Quebec

An NRC mass spectrometer, which is used for analysis of molecule samples.

Dag Hammarskjöld once commented that "the longest journey is the journey inward." As a diplomat and statesman, Hammarskjöld was keenly aware of the barriers to human progress. The Nobel Peace Prize winner was iconic as Secretary General of a nascent United Nations, and he understood all too well that for humanity to reach the next level of its development, being able to transform ourselves would be more significant than being able to fly to the moon.

In today's world of biomedical research, this is a reality that remains all too real, even in a literal sense. Recent decades have seen the discovery of a slew of ground-breaking new therapeutic technologies—we've sequenced the human genome, learned how to modify the basic building blocks of life by editing DNA, developed ways to alter genes and modify viruses, and discovered how to engineer new cells and molecules. But unless we're able to transport these new technologies where they need to be—inside specific cells—we may never know just how effective they can be.

Researchers at the National Research Council of Canada (NRC) are working hard to discover new ways to deliver therapeutics into human cells. It may be hard to imagine, but even now, surrounded as we are by 21st century technology, humanity can get itself to the moon, but getting medicine inside of our own cells is still a work in progress.

A different kind of shuttle

A DNA sequencing sample.

When most of us hear the word 'shuttle' we probably think of the kind that takes people to space, and why wouldn't we? The space shuttle program captured the human imagination, pushing us to dream of new frontiers that we might one day explore. For the past few years, the Quebec-based company Feldan Therapeutics has been working on a different kind of shuttle, one that will hopefully help us to reach new frontiers in therapeutic delivery into diseased cells.

When the company first started in 2007, Feldan was focused primarily on the development of research tools and services. In 2015, the company shifted gears towards the development of what it calls the Feldan Shuttle – a protein-based technology platform for the direct delivery of proteins inside of cells. The Feldan Shuttle allows for the delivery of antibodies, peptides, active nucleases (used in gene editing), and transcription factors (used in gene modulation). More recently, Feldan has been exploring the use of its technology for the treatment of diseases such as cystic fibrosis and basal cell carcinoma.

"Back when we were focussed on recombinant proteins, we were able to do most of our work in an in vitro setting," says François-Thomas Michaud, Feldan's CEO. "However, when it came to the Shuttle, intended for use as a delivery vector in biological systems, we needed proofs of concept in a preclinical laboratory setting (in vivo). Partnering with the NRC has allowed us to successfully meet that objective, as they have tools and expertise complementary to ours in preclinical research and development."

The NRC's Human Health Therapeutics Research Centre works with partners in industry, academia and government to advance and accelerate the development of innovative biologic medicines in Canada. They're currently working with Feldan to explore the potential applications that the shuttle could have in enabling advanced therapeutics in skin and lung cells, through the pursuit of proof-of-concept studies.

François-Thomas Michaud, CEO of Feldan Therapeutics.

"We strongly believe that intracellular delivery of biologics will have a highly positive impact on patients' quality of life," says Michaud. "Key to achieving that vision is having access to the types of research expertise that the NRC provides. For example, their R&D certificate program allowed us to continue our collaboration with them in a cost-effective manner, which is vitally important for a small but growing enterprise such as ours. Thankfully the NRC provides a place to grow for new technologies and developing ventures… after all, support for innovation begins at home."

This exciting research is not without its challenges, but the eventual outcomes could be extremely worthwhile. After all, the better we understand ourselves, the more effectively we'll be able to navigate our changing world.