Service platforms for the development of RNA therapies

Objectives

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 (Réseau DePTAQ), 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.

DePTAQ Network is managed in a concerted manner by three institutions and supported by three following platforms

1) The Sidney-Altman Therapeutic RNA Hub at the IRCM is currently operational for IRCM‘s 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

In addition to current services involving expression validation, RNA sequencing and functional tests, The RNomics Platform 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.

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.

Pilot Project from Médicament Québec and DePTAQ

Targeted Reprogramming of RNA Splicing for Precision Therapeutics

Challenge

Aberrant RNA splicing plays a critical role in both common and rare diseases. In colorectal cancer, ~200 alternative splicing events impact tumor growth, metabolism, immune response, and drug sensitivity. Meanwhile, more than 20% of disease-causing missense mutations across 7,000+ rare diseases disrupt splicing, leading to non-functional proteins and worsened outcomes. Yet, these splicing defects remain an underexploited therapeutic frontier.

Our Solution: A Precision Splicing Rescue and Discovery Platform

DePTAQ has developed an innovative screening platform based on CRISPR/dCas13Rx, enabling:

• Splicing Reprogramming in cancer through the identification of splice-switching antisense oligonucleotides (SSOs) that inhibit regulatory proteins (RBPs), altering splice site usage to produce beneficial mRNA variants.
• Mutation-Directed Splicing Rescue, by identifying guide RNAs that restore normal splicing in genes affected by pathogenic missense mutations, then using these guides to design therapeutic ASOs.
• Systematic Mapping of Splicing Regulatory Elements, through localized CRISPR interference screens that reveal the enhancer/silencer motifs controlling splice site decisions.

This platform not only supports rapid therapeutic discovery but also creates a rich resource for
AI-powered prediction tools and regulatory code deciphering.

Applications and Advantages

• Colorectal Cancer: Identification and validation of SSOs targeting tumor-associated splice variants using long-read transcriptomics and phenotypic assays.
• Rare Genetic Diseases: Targeting the splicing consequences of >100,000 known disease-causing mutations in HGMD and ClinVar.
• RNA Therapeutic Modalities: Supports both ASO-based therapy and LNP-enabled delivery of custom mRNA variants.e
• Data-Driven Discovery: Large-scale functional data generation across disease models fuels the creation of a predictive knowledge base..

Why Partner with DePTAQ ?

• First-Mover Advantage in the untapped therapeutic space of splicing regulation and correction.
• Scalable and Versatile Pipeline for ASO discovery, disease model validation, and future
  clinical development.
• Translational Readiness with clear paths to preclinical validation and commercialization.
• Transformative Impact across oncology, rare diseases, neurodegeneration, and beyond.

Opportunity for collaboration

We invite partners to engage through:

• Funded research collaborations
• In-kind contributions
• Licensing of intellectual property
• Participation to the elaboration of SOPs, AI tools and ASO production pipelines

Contact and More Information

Pr. Benoit Chabot – benoit.chabot@usherbrooke.ca
Dr. Panagiotis Prinos, Plateform Director – panagiotis.prinos@mcgill.ca