Health Challenge: Disruptive Technology Solutions for Cell and Gene Therapy

 

Hosted via the NRC's Human Health Therapeutics Research Centre, the Disruptive Technology Solutions for Cell and Gene Therapy Program will design and develop disruptive technology solutions for precision-engineered cell and gene therapies for the treatment and potential cure of chronic diseases and rare genetic disorders affecting Canadians.

In doing so, the NRC will coordinate a national effort to increase the affordability and accessibility of these ground-breaking technologies—in collaboration with academic facilities, research centres and networks, clinicians, hospital centres and others—to enable a national ecosystem for health innovation in the area of cell and gene therapies.

Cross-sectoral collaborative approach to achieve program success

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  • AI 4 Design: accelerating discovery
  • Collaboration to inform policy
  • Collaboration for translation
  • NRC foundational investments: expanding capacity

interact with

  • biodevices for gene and cell therapy automation
  • capacity building
  • gene therapy vectors
  • precision editing
  • precision targeting

to deliver

  • safe, affordable and accessible cell and gene therapies

Contact us

If you are interested in collaborating with us, making investments in this area, or if you have any questions, please contact:

Kelley Parato
Program Director, Disruptive Technology Solutions for Cell and Gene Therapy
Telephone: 613-990-1512
Email: NRC.healthprogram-programmesanté.CNRC@nrc-cnrc.gc.ca

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Long description

Key elements for collaboration include:

  • biodevices
  • capacity building
  • precision editing
  • precision targeting
  • viral vectors

Areas of focus

The Disruptive Technology Solutions for Cell and Gene Therapy Program will unify the value chain for engineered cell and gene therapies across the continuum from discovery to commercialization.

Through collaborative partnerships with key stakeholders, it will advance a unique initiative to bring disruptive solutions to the design, development and delivery of cell and gene therapy to patients in Canada.

Learn more

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Collaborate with us

The Disruptive Technology Solutions for Cell and Gene Therapy Program invites stakeholders to express their interest in collaborating with NRC researchers on this program. Find out more about collaborating with us and how to apply to currently active opportunities; contribution funding is available to defray the research costs of eligible collaborators.

Express your interest

News and events

Stay up to date on the latest program announcements, events and workshops.

Program team

Meet the leading NRC experts who are contributing to the Disruptive Technology Solutions for Cell and Gene Therapy Program.

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Areas of focus

The Cell and Gene Therapy Health Challenge Program will unify the value chain for engineered cell and gene therapies across the continuum from discovery to commercialization. Through collaborative partnerships with key stakeholders, it will advance a unique initiative to bring disruptive solutions to the design, development and delivery of cell and gene therapy to patients in Canada.

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Research and discovery Biomanufacturing Clinical trials Commercialization

Foundational Investments

Synthetic biology genome foundry
University of Concordia

3D microfluidic devices
U of T – CRAFT

Canadian current Good Manufacturing Practice facilities

Cell product manufacturing

Viral vector manufacturing

Hospitals, research centres and health services

Centres of Excellence for Commercialization and Research

Canadian small and medium-sized enterprises and multi-national enterprises

 

Master Projects

By the end of its 7‑year mandate, the program aims to deliver significant outcomes cross 6 master project program areas:

  1. Glybera biobetter: a made-in-Canada biobetter formulation of Glybera, a gene therapy that will provide Canadians with affordable access to treatment for the monogenic disorder lipoprotein lipase deficiency (LPLD)
  2. CD19 CAR T: a personalized cancer treatment approach for previously incurable forms of leukemia
  3. Biodevices for automation: the design, qualification and validation of a number of device‑based tools to answer fundamental and practical questions related to the development, validation and safety assessment of new generations of cell and gene therapy. Ultimately, this will lead to the development of an integrated and automated microfluidic device for point‑of‑care manufacture of engineered or differentiated therapeutic cell products
  4. Precision editing: establishment of a national, precision gene-editing platform to optimize universal donor cell immunoediting, to rapidly enable AI-assisted iterative improvements in cellular therapies
  5. Precision targeting: leveraging the NRC's unique strengths in single-domain antibody generation and nanotechnology to develop novel, more efficacious and safer CARs, and in vivo non-viral genome editing strategies using targeted nano-delivery vehicles
  6. Viral vectors for gene therapy: step-change improvements in 2 critical viral-based gene therapy platforms: adeno-associated virus (AAV) and lentivirus to support the next generation of AAV gene therapies

Foundational investments

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Translational technologies (high technology readiness level)

  • Biomanufacturing platforms
  • Capacity-enabling projects

Disruptive technologies (low technology readiness level)

  • SynBio foundry
  • Living microsystems foundry
  • Translational research platforms
  • Biodevices for automation
  • Precision editing
  • Precision targeting
  • Viral vectors for gene therapy

To deliver

  • safe, affordable and accessible cell and gene therapies
 

The NRC has established key collaborations with a number of partners to provide foundational investments in technologies that will be essential for the Disruptive Technology Solutions for Cell and Gene Therapy Program's capacity to deliver on its aims. These include:

  • University of Toronto – Centre for Research and Applications in Fluidic Technologies (CRAFT)
    Creation of device-based in vitro models and analytical technologies to help understand cell programming and differentiation, and assess their efficacy.
  • Concordia University – Synthetic biology genome foundry
    Essential infrastructure for the development of high-throughput and high-precision gene editing of induced pluripotent stem cells (iPSC) for the rapid evaluation of optimal cell characteristics for a universal donor iPSC platform.

Collaborate with us

The Health Challenge Program in Disruptive Technology Solutions for Cell and Gene Therapy is seeking collaborators with research interests, expertise, specialized capacity or technologies which have the potential to accelerate and transform innovation in cell and gene therapy approaches. We invite you to contact us and tell us about your area of expertise, and your interest in collaborating with the Program.

Current collaborative opportunities are open in the Master Project research theme areas outlined below (click on the options below for more information, and to submit an expression of interest).

Capacity-building projects

Master Project 1: Infrastructure Capacity Modification or Expansion to support GMP manufacture of virus-based gene therapy vectors at 50+L scale

While the NRC has significant capability for gene therapy vector manufacturing and bioprocess development and optimization in a pre-GMP environment, Canada is currently lacking in sufficient access to affordable GMP biomanufacturing for viral-based gene therapy vectors.

  • Lack of affordable capacity will limit Canadian ability to support clinical and commercial development of ground-breaking innovations domestically, thereby impeding access to Canadian-developed novel gene therapies for Canadians
  • The primary objective is to modify or expand an existing GMP-ready facility to increase capacity for suspension culture AAV production in Canada, in support of clinical trials of AAV-based gene therapies
  • The driving project is the development of an improved version of Glybera, the previously commercialized AAV gene therapy for rare disease (LPLD: lipoprotein lipase deficiency), which was priced at approx. $1.1 million USD. The objective is to develop and manufacture LPL-AAV gene therapy in Canada and offer it to Canadian patients through clinical trials and at significantly reduced cost, commencing in 2023

We are seeking collaborators to jointly develop capacity to bring online AAV GMP manufacturing at a clean room facility in Canada. Such a collaboration will leverage NRC expertise and internal support for process development, analytics and vector design and optimization, and technology transfer of an optimized manufacturing process for one or more AAV-based therapeutic products:

  • Potential collaborator facility with ability to establish 50L-200L GMP biomanufacturing capacity for novel AAV gene therapy vectors in suspension culture, in a GMP host facility
  • Preferably, collaborating facility/institution will already have a GMP environment to host incoming infrastructure, and ideally experience in viral vector manufacturing
  • The collaborating GMP facility should also be containment level 2, and be amenable to housing at least 50L but preferably 200L stirred tank bioreactors and supporting purification equipment and analytical instrumentation
  • The GMP facility should have adequate cleanroom spaces to accommodate the development process. Physical segregation between upstream (bioreactors) and downstream (purification) will be an asset
  • The facility would need to have, or establish, a separate unit for filling (grade A in grade B background, or grade A isolator in a grade D room)

In the short- to medium-term, this expanded GMP capacity for AAV production will support a critical manufacturing program for an NRC Challenge Program-supported clinical translation project in gene therapy for a rare disease indication (LPLD). In the longer term, the new capacity will become available to the Canadian academic and SME community for GMP scale-up of other AAV-based gene therapies.

Expressions of interest to collaborate in this initiative will be considered until September 30, 2019.

Please use the Expression of Interest Form to provide summary information of your existing capabilities and how you would envision building on them through collaboration to meet the goal of this project.

Contribution funding is available to support collaborators' investments. Potential collaborators with the required existing facilities and an interest in funding support will be invited to apply to the NRC's National Program Office. Collaborator proposals will be reviewed by peer reviewers external to the NRC.

Master Project 2: Establishment or Expansion of Cell Therapy Manufacturing Infrastructure to support iterative clinical development of made-in-Canada engineered cell therapies

The primary objective for this Master Project within the Challenge Program is to enable the expansion of domestic capacity to support accelerated development of Canadian innovations in CAR-T therapy and facilitate access of these ground-breaking therapies to Canadian patients.

We are currently seeking:

  • collaborative teams to propose a solution to leverage or build upon existing cell therapy GMP infrastructure with a view to developing or expanding capacity to rapidly conduct small iterative phase 1 clinical trials with novel CAR T therapeutic products
  • collaborators will preferably originate from multiple sites in Canada across multiple provinces

A successful outcome of this collaboration will be the expansion or coordination of access to cell therapy product manufacturing infrastructure at one or more linked sites, to support early clinical development of NRC or NRC/collaborator-developed assets, in addition to assets of the collaborators' existing networks. As such, existing expertise in manufacture of GMP-grade CAR-T products would be an asset to facilitate fast turnaround time in the establishment and use of this GMP infrastructure for clinical development of novel CAR-T products.

Expressions of interest to collaborate in this initiative will be considered until September 30, 2019.

Please use the Expression of Interest Form to provide summary information of your existing capabilities and how you would envision building on them through collaboration to meet the goal of this project.

Contribution funding is available to support the research costs of eligible collaborators.

Disruptive technology development projects

The Health Challenge Program in Disruptive Technology Solutions for Cell and Gene Therapy comprises 5 early-stage areas of research, each of which are seeking complementary expertise and technologies.

The goals of these projects are:

Master Project 1: We are inviting interest in collaborations on developing AAV-based gene therapies for various monogenetic diseases that would become ready to benefit from expanded GMP AAV capacity in Canada and clinical trials in the medium-term (5 years)

Master Project 3: Development of microfluidic processes and biodevices for therapeutic cell product engineering and manufacturing, including automated workflows and integrated analytic approaches

Master Project 4: Development of high-throughput automation and precision editing platforms to accelerate safe allogeneic-based cell therapies

Master Project 5: Development of novel targeting strategies for CAR T products directed to solid tumours, and development of targeted in vivo gene editing strategies

The main objective of this Master Project will be to deploy next-generation cell and nanoparticle-gene therapeutics using novel antibody reagents against difficult solid tumour peptide or glycan antigens.

At this time, collaborations are sought for:

  • the development of solid tumour targeted CAR-T collaborations, in preclinical development of animal models for evaluation of glycan-based CAR therapeutics and development of novel gene-switches for enhanced control and safety of cell and gene therapy
  • nano-gene delivery systems, including the development of packaging CRISPR-Cas9 gene editing machinery in lipid-, carbon- and exosome-based nanoparticles, and the development of animal models of monogenetic diseases for the in vivo evaluation of targeted gene editing

Master Project 6: Development of an efficient system for genome editing via HDR, and tangential flow filtration membrane

  1. Development of an efficient system for genome editing by homology directed repair (HDR) in quiescent cells that could be used in the context of gene therapy with viral vectors such as adeno-associated virus (AAV)
  2. Possessing surface chemistry knowledge, develop a tangential flow filtration membrane (hollow fiber or cassette-based) containing specific ligand or charge on the lumen side to adsorb or bind to virus, produced via mammalian cell culture and used for gene therapy applications, as well as to allow for elution in smaller volumes

We welcome general expressions of interest to collaborate on these initiatives on an ongoing basis, using the Expression of Interest Form.

More specific opportunities for collaboration will be posted as they are defined. Contribution funding is available to support the research costs of eligible collaborators.

Program Overview

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NRC technology toolbox

  • Antibody platforms
  • Nanomaterials
  • Induced pluripotent stem cells
  • Viral vector design
  • Packaging cell lines

Canadian ecosystem technology toolbox

  • Clinical targets and disease models
  • Clinical datasets
  • Chimeric antigen receptor designs
  • Regenerative medicine cell biology
  • Nanomaterials
  • Gene editing strategies

NRC enabling expertise

  • Device fabrication
  • Automation
  • Artificial intelligence
  • Viral vector scale up manufacturing
  • Analytics, metrology & standards

Canadian ecosystem enabling expertise

  • Mammalian genome foundry
  • Good manufacturing practice device fabrication
  • Translational models
  • Current good manufacturing practice facilities
  • Clinical trial expertise

to deliver

Challenge Program

  • Pipeline of disruptive made-in-Canada therapies (universal donor cell, virus-free gene editing)
  • Technologies that reduce cost and complexity of manufacturing
  • Expanded and new good manufacturing practice and clinical capacity
  • Collaboration for innovative commercialization, health policy, and delivery models
 

News & Events

The latest information on news and events related to the NRC's Health Challenge Program in Disruptive Technology Solutions for Cell and Gene Therapy will be posted here as they become available.

Program Team

The NRC's Health Challenge Program in Disruptive Technology Solutions for Cell and Gene Therapy is made up of a highly skilled and multi-disciplinary team possessing a broad range of expertise in biotechnology, business and advisory services, and program management.

Kelley Parato
Program Director, Disruptive Technology Solutions for Cell and Gene Therapy
Kelley.Parato@nrc-cnrc.gc.ca

Danica Stanimirovic
Theme Lead, Universal cell editing
Danica.Stanimirovic@nrc-cnrc.gc.ca

Risini Weeratna
Theme Lead, Chimeric antigen receptor engineering
Risini.Weeratna@nrc-cnrc.gc.ca

Teodor Veres
Theme Lead, Biodevice design, fabrication and automation
Teodor.Veres@cnrc-nrc.gc.ca

Anna Jezierski
Theme Lead, Precision targeting and editing
Anna.Jezierski@nrc-cnrc.gc.ca

Rénald Gilbert,
Theme Lead, Gene therapy platforms and viral manufacturing
Renald.Gilbert@cnrc-nrc.gc.ca

Umar Iqbal
Theme Lead, Nanotechnology platforms and cell imaging
Umar.Iqbal@nrc-cnrc.gc.ca

Eileen Raymond
Business and Advisory Services
Eileen.Raymond@cnrc-nrc.gc.ca

Helen Loughrey
Program Challenge Officer
Helen.Loughrey@nrc-cnrc.gc.ca