Cell and Gene Therapy Challenge program: Our impact

Led by the Human Health Therapeutics Research Centre, the Disruptive Technology Solutions for Cell and Gene Therapy Challenge program, which ran from 2019 to 2026, is now closed.

It was created to advance the development and manufacturing of cell and gene therapies for chronic diseases and rare genetic disorders in Canada. It brought together researchers, clinicians and industry partners to reduce manufacturing costs and make cell and gene therapies more accessible for Canadians.

Key areas of focus were:

• CAR T-cell therapy
• Affordable gene therapies
• Improved engineering adeno-associated virus (AAV) for gene therapy
• Precision editing to create universal donor cells
• Precision targeting using antibodies for next generation CAR and non-viral gene therapies

CAR T-cell therapy

The program advanced the development of made‑in‑Canada CAR T‑cell therapies to improve access to innovative cancer treatments for Canadians. Working with The Ottawa Hospital, BC Cancer, BioCanRx and other partners, NRC researchers created the first Canadian CAR T‑cell therapy targeting the CD22 protein on leukemia and lymphoma cells, now in a Phase 1 clinical trial. The team also developed new nanobody‑based CAR T‑cell therapies for hard‑to‑treat solid tumours, such as gynecological and pancreatic cancers, and explored CAR‑NK (natural killer cell) approaches to create "off‑the‑shelf" products that could treat many patients more affordably. These collaborations strengthened Canada's capacity to manufacture, test and deliver next‑generation immunotherapies. The program also developed new producer cell lines with integrated helper genes for lentiviral particle generation, significantly reducing the cost of manufacturing GMP‑grade lentivirus used to deliver CAR genes into patient T cells.

Learn more about our work on CAR T-cell therapies.

Affordable gene therapies

The program advanced the development of more affordable and accessible gene therapies for Canadians living with chronic and rare diseases. NRC researchers worked with partners to improve how adeno‑associated virus (AAV) materials used in gene therapies are made. Together, they developed cleaner, scalable production methods and expanded good manufacturing practice facilities to produce these materials in Canada. The team also created new cell lines to boost AAV productivity and collaborated on projects to lower costs and improve monitoring for gene therapies targeting rare conditions such as lipoprotein lipase deficiency.

Improved engineering adeno-associated virus (AAV) for gene therapy

The program advanced the development of improved AAV vectors to make gene therapies safer, more effective and easier to produce. NRC researchers worked with partners to engineer better viral capsids, refine patient selection and identify biomarkers that support long‑term monitoring of clinical trial participants. This work helped improve the quality, safety and cost‑effectiveness of AAV‑based therapies, positioning Canada as a leader in advanced viral vector manufacturing and innovation.

Precision editing to create universal donor cells

The program advanced precision gene‑editing technologies to develop universal donor cells that could be used to treat many patients without immune rejection. NRC researchers collaborated with Kyoto University's Centre for Induced Pluripotent Stem Cell Research and Application (CiRA) and Concordia University to expand its Genome Foundry, establishing mammalian genome‑editing and automation workflows for high‑throughput cell engineering. This project helped create new tools and methods for safely and efficiently editing stem cells in the lab. Other projects focused on identifying safe harbour regions in the genome for inserting therapeutic genes and developing off‑the‑shelf stem‑cell‑derived immunotherapies that could be used for multiple patients.

Precision targeting using antibodies for next‑generation CAR and non‑viral gene therapies

The program advanced next‑generation CAR T‑cell therapies for solid tumour cancers and explored innovative delivery systems, such as lipid nanoparticles, to precisely target treatments to specific cells and tissues. NRC researchers are working with clinical and industry partners to translate these discoveries into clinical practice.

These efforts strengthened Canada's research capacity in emerging therapeutic technologies and supported the development of more effective and accessible treatments for the future.