Grants and Contributions

The UKRI will hold a call for proposals aiming to solicit collaborations between UK researchers and the National Research Council of Canada’s Cell and Gene Therapy (CGT) Challenge Program, on research projects aiming to innovate therapeutic viral vector biomanufacturing processes.

Procurement, installation and commissioning of equipment to augment the University of Toronto Living Micro-Systems Foundry's capabilities for research and development of microfluidic devices for miniaturization and automation of cell and gene therapies.

Procurement, installation and commissioning of equipment to augment the University of Toronto Living Micro-Systems Foundry's capabilities for research and development of microfluidic devices for miniaturization and automation of cell and gene therapies.

Procurement, installation and commissioning of equipment to augment the University of Toronto Living Micro-Systems Foundry's capabilities for research and development of microfluidic devices for miniaturization and automation of cell and gene therapies.

Potent and specific gene therapies used to treat patients have been limited by the lack of delivery vehicles to introduce them into specific cells.

Myotubular myopathy is a severe congenital disorder marked by muscle
weakness and hypotonia due to mutations in the MTM1 gene, which encodes
myotubularin. This protein is crucial for muscle cell maintenance and repair, and
its deficiency disrupts these processes, causing significant muscle dysfunction.
Current treatments are symptomatic and fail to address the genetic cause,
necessitating therapies that target the root of the disease. Gene therapy,
particularly with viral vectors, shows promise but has limitations such as immune
responses and high costs. Recent safety setbacks in AAV8-MTM1 gene therapy
trials underscore the need for alternative delivery methods.

Inferring the structural properties of a protein from its amino acid sequence is a challenging yet important problem in biology. Discovering these structures is important, since they determine a wide array of protein functions. However, experimental structure determination is costly and, as a result, atomic structures have only been determined for a tiny fraction of known proteins. The objective of this project is to develop an AI system capable of predicting protein-protein interaction and physicochemical properties of proteins solely from their amino acid sequences without any recourse to expensive, time-consuming and computationally prohibitive methods such as homology modelling, crystallography, nuclear magnetic resonance, molecular dynamics and macromolecular docking. The system, will allow for the design of proteins with specific properties by means of AI-based reverse engineering.

The goal of this project is to establish a method to synthesize Fusogenix DNA lipid nanoparticles using a single step manufacturing process. To achieve this, we will evaluate a number of methods to incorporate our fusogenic proteins into lipid nanoparticles using the Precision Nanosystems microfluidic platform. We expect that by pre-formulating one of the lipid components with fusogenic protein, we may be able to reduce the manufacturing to a single step compared to the current 2 step process. This will allow us to significantly improve our encapsulation efficiency and simplify our manufacturing process as we move to the clinic.

The specific objectives of this funding opportunity are to:

• Increase and advance the development of gene therapies for rare disease clinical trials in Canada;
• Generate the evidence required for first-in-human clinical trials, in part by working with Canada’s biomanufacturing capacity (National Research Council of Canada) and health technology regulator (Health Canada); and,
• Increase current and future capacity across the Canadian rare disease landscape (i.e., among researchers, knowledge users, patient(s)/caregiver(s)/family) to improve readiness of gene therapies for first-in-human clinical trials.

The specific objectives of this funding opportunity are to:

• Increase and advance the development of gene therapies for rare disease clinical trials in Canada;
• Generate the evidence required for first-in-human clinical trials, in part by working with Canada’s biomanufacturing capacity (National Research Council of Canada) and health technology regulator (Health Canada); and,
• Increase current and future capacity across the Canadian rare disease landscape (i.e., among researchers, knowledge users, patient(s)/caregiver(s)/family) to improve readiness of gene therapies for first-in-human clinical trials.