Service platforms for the development of RNA therapies

This platform, headed by the IRCM, Sherbrooke University and McGill University, aims to offer essential biomanufacturing services in RNA therapies, thus improving production and development capacities in Quebec.

To foster the growth in ribonucleic acid (RNA) in Quebec, the Montreal Clinical Research Institute (IRCM), Sherbrooke University and McGill University have created the Network for the development and production of RNA therapies, a unique access point for the industry and academic researchers. It provides access to the leading expertise in the development and production of RNA therapies. This network will be managed in a concerted manner by the three institutions and will be supported by the three following platforms:

1) The Sidney-Altman Therapeutic RNA Hub of the IRCM is currently operational for IRCM laboratories. There are already five RNA therapy development projects in progress for different cardiometabolic and neurodegenerative diseases and cancer. An installation update of the IRCM RNA therapy development platform will pave the way for offering academic and industrial laboratories specialized and turnkey services, including design (biocomputing/artificial intelligence), production and preparation on a small/medium scale of DNA vectors, RNA synthesis on a small/medium scale, conjugation of antisense oligonucleotide (ASO) and encapsulation of lipid nanoparticles, quality control on a pre-clinical level, screening, and validation and optimization of therapeutic RNA molecules (ASO, messenger RNA, self-amplifying RNA, circular RNA). This will also include analyses of biodistribution, toxicity and kinetics to facilitate the passage from pre-clinical steps toward clinical studies.

Access to this platform will be possible at the beginning of 2026

2) The new therapeutic messenger RNA platform from McGill University’s Centre for RNA Sciences, which will be implemented in the Biochemistry Department area, will meet the needs of activities and research in McGill University’s Centre for RNA Sciences, research programs arising from the D2R program, which was financed by the Canada First Research Excellence Fund, from network partner institutions and from industrial clients in the province. Messenger RNA modalities are key to the main therapeutic approaches emerging in precision medicine. These include neo-antigenic vaccines against cancer, CART programming and self-amplifying RNA. The platform will provide services in bioproduction and the formulation of LNP nanoparticles on a pre-clinical level of various messenger RNA modalities, individually or collectively. Their quality control will be based on strict standardized criteria, as well as a functional base validation.

Access to this platform will be possible at the beginning of 2026

3) The Sherbrooke University RNomics Platform, which, in addition to current services involving expression validation, RNA sequencing and functional tests, also offers a screening service based on CRISPR/dCas13Rx for identifying ASOs capable of reprogramming the splicing of pre-messenger RNA, as well as determining the functions associated with splicing variants. These tools will facilitate the identification of new targets and the development of innovative therapeutic tools based on the use of RNA.

Access to this platform will be possible in the fall of 2024

In short, the Network for the development and production of RNA therapies will enable:

  • the implementation of three complementary platforms, ensuring everything from the design to the pre-clinical validation of RNA therapies;
  • the implementation of a request process for standardized and accessible services for academic and industrial players;
  • the creation of a functional interface between the University and industry, which will enable the co-development of RNA therapies and boost partnerships with pharmaceutical and biotechnological companies;
  • the consolidation of expertise in RNA therapies in Quebec, via the training of graduated and postdoctoral students specialized in the development and
  • the production of RNA therapies;
  • the creation of a dynamic ecosystem around RNA therapies in Quebec, contributing to its international profile.

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