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Marc A. Gauthier

Better "Biologics" – Better Therapies

Intérêts de recherche


Projet de recherche en biotechnologie (doctorat et postdoctorat)

Recherche et développement pour la production d'anticorps monoclonaux (PDF)

Tremendous advances in medicine attest to how research in chemistry and biology irrevocably alters our quality of life. The mounting costs of healthcare hamper the capacity of healthcare providers in Quebec and Canada to effectively treat disease, thus providing impetus for developing less expensive, more effective, and even personalized medicine. There is growing consensus within the pharmaceutical sector that issues of drug cost and efficacy can be addressed using biomolecules such as proteins or nucleic acids (i.e., so-called “biologics”) as drugs, and as platforms for drug discovery and development. This is, in part, because their chemical and structural complexity allows e.g., enzymes to efficiently catalyze specific reactions, monoclonal antibodies to strongly bind to target molecules, and nucleic acids to selectively alter protein expression levels. In addition, tools for sequencing DNA, screening huge libraries of proteins, and mass-producing optimized lead molecules are now all realities. Presently, ~25% of all new drugs approved by the American Food and Drug Administration are peptides or proteins, and the global markets for therapeutic monoclonal antibodies ($58 billion by 2016) and enzymes ($3.9 billion by 2017) are enormous. Furthermore, the once abandoned field of gene therapy has become a hotbed, with 11 different companies raising at least $618 million from venture capitalists and the public markets since the beginning of 2013 (Forbes.com). However, as classes of molecules, proteins and nucleic acids suffer from inherent shortcomings that can limit their efficiency as therapeutics. More specifically, proteins, even large ones, fold into compact structures that are rapidly eliminated from the body by renal filtration within one to two days following systemic administration. Furthermore, many proteins are derived from non-human organisms and have the potential to induce an immune response, which can range from mild allergy to anaphylactic shock. Nucleic acids, on their side, do not readily cross cell membranes and are thus very difficult to deliver intact into cells. Finally, both proteins and nucleic acids are sensitive molecules that are susceptible to hydrolysis and can be digested by enzymes.

The core activities of the Gauthier group are designed to meet these challenges of using biologics as therapeutics:

(see Projets en cours section)

1) Develop new types of dynamic covalent bonds that respond to local micro-environments in the body, which would allow us to release drugs from protective carriers specifically in diseased tissue.

2) Develop new approaches to protect therapeutic proteins (from the body) without compromising their drug-like activity.

3) Develop new platform technologies for drug discovery, which would allow us to engineer desirable properties directly into new drugs.

4) Explore new opportunities for synthesizing and actuating therapeutic bioconjugates using emerging sources of non-ionizing radiation.





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