The Future of UK and Canada Collaboration in Advanced Therapies

www.ktn-uk.org
Dr Sandeep Sandhu, KTM International & Development
sandeep.sandhu@ktn-uk.org
The Future of UK and Canada
Collaboration in Advanced
Therapies
Hosted by
Knowledge Transfer Network (KTN)

Dissemination Webinar &
Virtual Networking
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PLEASE NOTE – THE WEBINAR IS BEING RECORDED
The recording will be made available via the KTN website

AGENDA
16.00: Welcome and introduction – Sandeep Sandhu (KTN)
16.05: Overview of Innovate UK and Global Programme – Brendan Vickers (Innovate UK)
16:15: UK Policy Overview – Ian McKay (Innovate UK)
16.25: UK Advanced Therapies Landscape – Andreea Iftimia-Mander (Innovate UK)
16:35: Introduction – Biomanufacturing in Canada – Kasondra White (Innovation, Science and Economic Development Canada)
16:40: Advanced Therapies and Biomanufacturing in Canada and Opportunities for Canada – UK Engagement
– Kelley Parato (National Research Council of Canada)
16.55: Panel session; Key findings, next steps and support for collaboration
Chair: Sarah Goulding (Innovate UK)
Panellists: Ian McCubbin (UK Vaccine Taskforce Manufacturing Lead), Peter Coleman (Cobra Biologics), Jacqueline
Barry (Cell and Gene Therapy Catapult), Michael May (CCRM), Lucy Foley (Centre for Process Innovation)
17.45: Questions from the audience – Karen Wilkinson & Marcel Kuiper (KTN)
18.00: Wrap-up & Networking – Sandeep Sandhu (KTN)
The networking platform is open during the following times:
• 21st January 18:00-19:00 (GMT)
• 22nd January 16:00-19:00 (GMT)

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communities to drive innovation that
changes lives.

Finding valuable
partners
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building
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Knowledge
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strategy
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trends and market
drivers
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and markets
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support
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raising profile
Navigating the
innovation support
landscape
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strategy and
approach
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stakeholders
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innovation resources
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The Future. Faster.

• UK national innovation network set up 15 years
ago by the former Innovate UK with the purpose
of building a more effective and connected
UK innovation ecosystem.
• The logic was that: better connecting
innovation stakeholders would stimulate and
accelerate business-led collaboration for
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KTN Global Alliance
https://ktn-uk.org/global-alliance/
KTN Health
https://ktn-uk.org/health/
Canada Advanced Therapies Mission- GEM
report
https://ktn-uk.org/wp-content/uploads/2020/12/2020-Canada-
Advanced-Therapies-Expert-Mission-Report.pdf
KTN Newsletters
https://ktn-uk.org/health/

Overview of Innovate UK & Global Programme –
Brendan Vickers, Innovate UK

UK Policy Overview –
Ian McKay Innovate UK

Advanced Therapies Landscape –
Andreea Iftimia-Mander, Innovate UK

Introduction – Biomanufacturing in Canada –
Kasondra White, Innovation, Science & Economic
Development

Advanced Therapies and Biomanufacturing in Canada
and Opportunities for Canada – UK Engagement
–
Kelley Parato, National Research Council of Canada

Panel Session –
Sarah Goulding, Innovate UK

Q & A –
Karen Wilkinson & Marcel Kuiper, KTN

Innovate UK Advanced Therapies Global Expert Mission to Canada
KTN
Sandeep Sandhu
Karen Wilkinson
Marcel Kuiper
Innovate UK
Sarah Goulding
Andreea Iftimia-Mander
Nick Medcalf
Brendan Vickers
UK Research & Innovation
Sonny Rathod
Science & Innovation Network
Arthur Kong (Toronto)
Mark Keilthy (Vancouver)
The networking platform is open during the
following times:
21st January 18:00-19:00 (GMT)
22nd January 16:00-19:00 (GMT)

Advanced Therapies
GEM Canada 2020/21
Brendan Vickers
Partnership Manager – North America

Benefiting everyone
through knowledge,
talent and ideas.
2
UK Research and Innovation
brings together the 7 Research
Councils, Innovate UK and
Research England.
An organisation with combined
funding for UK R&D of over
£7bn per year.
Arts and
Humanities
Research
Council
Biotechnology
and Biological
Sciences
Research
Council
Economic
and Social
Research
Council
Engineering
and Physical
Sciences
Research
Council
Medical
Research
Council
Natural
Environment
Research
Council
Science and
Technology
Facilities
Council
Innovate UK
Research
England

Innovate UK drives productivity and
economic growth by supporting
businesses to develop new ideas.
We connect businesses to the people
that can help them, and fund businesses
and research collaborations in all
economic sectors, value chains and
UK regions to accelerate innovation,
increasingly with global partners.
3
The UK’s innovation agency

• UK is good at starting businesses but not so good when it comes to their growth
• OECD Entrepreneurship at a Glance 2017 - UK comes first globally for start-ups, but not
in the top ten for growing businesses into established, medium-sized companies with a
lasting impact on our economy
• The Scaleup Institute Scaleup Review 2018 highlights that being able to access UK and
international markets is the second most important factor for businesses looking to
grow and scale
• 95% of the world’s R&D takes place outside of the UK
• In exploring global markets, businesses often lack confidence and face the challenges of
finding international partners, customers and having the right connections
• Businesses look for trusted support and ability to open opportunities
Innovate UK supporting global R&D and innovation

• Aim is to help innovative UK businesses to grow and scale globally – even before they
have a market-ready product or service to sell
• Range of different types of support to facilitate partnership building and development
• Financial and non financial tools

Global Expert
Missions
Led by KTN
Group of 6-8 experts scoping opportunities for UK businesses in
targeted countries and specific technology and sector areas.
Three stages:
• Scoping visit – fact-finding and benchmarking UK capability
• Mission reports inc Advanced Therapies in Canada 2020
published at:
https://ktn-uk.org/news/global-expert-mission-reports/
• Dissemination

The Mission Team – Toronto & Vancouver March
2020

Global Expert
Missions
Global Business
Innovation
Programmes
Delivered by Innovate UK EDGE
(formerly EEN)
Cohort of c.15 innovative high growth
businesses exploring opportunities
and building collaborations and
partnerships in specific countries and
technology and sector areas.
Programme over 9-12 months with 3
stages –
• Get ready
• Innovation visit overseas
• Exploit the opportunity

Global Expert
Missions
Global
Incubator
Programme
Global Business
Innovation
Programmes
Cohort of c.6-8
innovative high
growth businesses
building long-term
relationships and
foundations for
future market
growth.
Pilot programmes
in Canada, USA,
Singapore and India
in specific
technology and
sector areas over
12-18 months

Global Expert
Missions
Global
Incubator
Programme
Global Business
Innovation
Programmes
Bilateral and
multilateral
R&D&I
funding
programmes

Global Expert
Missions
Global Business
Innovation
Programmes
Global
Incubator
Programme
Bilateral and
multilateral
R&D&I
funding
programmes
Group of 6-8 experts scoping
opportunities for UK
businesses in specific
countries and technology and
sector areas. KTN led.
Three stages –
• Scoping visit
• Dissemination report
• Dissemination workshop
Cohort of c.15 innovative
high growth businesses
exploring opportunities and
building collaborations and
partnerships in specific
countries and technology
and sector areas. EEN led.
Programme over 9-12
months with 3 stages.
Cohort of c.6-8 innovative high
growth businesses building long-
term relationships and foundations
for future market growth.
Piloted in Canada, USA, Singapore
and India in specific technology and
sector areas.
Programme over 12-18 months with
4 phases.
Collaborative R&D&I grant
funding programmes to support
projects up to 36 months in
specific technology and sector
areas.
Programmes can be bilateral or
multilateral.
Funds include H2020, ODA,
GCRF, Newton, UKRI FIC
Global Business
Innovation
Programmes

UK-Canada Bilateral R&D Funding
- MOU between UKRI and NRC Canada
signed March 2020
- The Biomanufacturing Innovations for Cell
& Gene Therapies competition opened for
UK bids in October 2020
- Funded by the NRC Global Challenge
Programme
- UK innovators working with NRC Lab teams
in Montreal
- 6 project proposals currently under
evaluation

Thank you.
brendan.vickers@innovateuk.ukri.org

Future of UK and Canada Collaboration in Advanced Therapies
UKAdvancedTherapies Policy background
21st January2021
Dr Ian McKay
Innovation Lead – Advanced Therapies
Ian.McKay@innovateuk.ukri.org

UK Advanced therapies
• Long-standing priority area

Coordination and cooperation
Government policy Research Funding Healthcare system

Support for Advanced Therapies
Infrastructure
• Cell and Gene Therapy Catapult + Manufacturing Centre
• EPSRC Centre for Innovative Manufacturing in Regenerative Medicine
Coordinated and targeted research funding
• Innovate UK - commercially led projects
• UK Regenerative Medicine Platform

The challenge of clinical adoption
“Investors need to see a clear pathway from
development to delivery in the NHS if they are
to have the confidence to invest in regenerative
medicine. It is not sufficient to rely on trail blazing
therapies to forge pathways to clinical delivery.
The NHS must shift from reacting to regenerative
medicine to a state of preparedness to deliver
new and innovative treatments”

Regenerative Medicine Expert Group
2015
Membership included:
NHS, NICE, Regulators, Industry, Researchers, Patient Groups
Key recommendations:
§ Advice for product developers – role for regulators
§ Clinical evaluation – ‘mock’ appraisal by NICE
§ Embed regenerative medicine in mainstream NHS

Industry led - Advanced Therapies Taskforce
Recommendations included support for:
• Expanded capacity for viral vector manufacture
• Support for manufacturing skills
• Creation of Advanced Therapy Treatment Centres
November 2016

Global investment in R&D comparisons

UK Research
and Innovation
Jan-Mar
2017
Apr-Jun
2017
Jul-Sep
2017
Oct-Dec
2017
Jan-Mar
2018
Apr-Jun
2018
Jul-Sep
2018
Wave 1
ISCF announced
Wave 2
ISCF announced
Industrial Strategy and Life Sciences Sector Deal
Oct-Dec
2018

AI and
data
economy
Ageing
society
Clean
growth
Future of
mobility
Putting the UK at
the forefront of the
artificial intelligence
and data revolution
Harnessing the power of
innovation to help meet
the needs of an ageing
society
Maximising the
advantages for UK
industry from the global
shift to clean growth
Becoming a world
leader in shaping the
future of mobility
Industrial Strategy Challenge Fund: Grand Challenges

UK Research
and Innovation
5 foundations of The Industrial Strategy
Ideas
The world’s most
innovative economy
Infrastructure
A major upgrade to
UK’s infrastructure
People
Good jobs and greater
earning power for all
Places
Prosperous
communities
across the UK
Business
Environment
The best place to start
and grow a business

UK Research
and Innovation
Medicines
Manufacturing
up to £188m
National
Satellite Test
Facility
up to £99m
Next Gen.
Affordable
Lightweight
Materials Mfg
(ATI projects) up
to £26m
Autonomous
Vehicles
(CCAV
projects)
up to £38m
Robotics and
AI in extreme
environments
Challenge
up to £93m
Faraday
Battery
Challenge
up to £246m
Wave 1 Challenges (c. £1bn)
12
Underpinning UKRI investments (Wave 1a) up to £283m

UK Research
and Innovation
Wave 2 Challenges (up to £729.5m)
Early diagnosis & precision
med (up to £196m)
Healthy ageing
(up to £98m)
Next generation
services (up to £20m)
Audience of the future
(up to £33m)
Quantum technology
(up to £20m)
Transforming construction
(up to £170m)
Transforming food
production (up to £90m)
Energy revolution
(up to £102.5m)
13

Future of UK and Canada Collaboration in Advanced Therapies
UK Advanced Therapies Landscape
21st January2021
Dr Andreea Iftimia-Mander
Innovation Lead – Medicines Manufacturing
andreea.iftimia@innovateuk.ukri.org

The UK is a premier site for ATMP development and manufacture
A strong ATMP sector
An extensive clinical
pipeline
Research excellence in
ATMPs across the UK
Policy-driven support
for ATMPs
World-leading skills,
talent and institutions
A strong investment
landscape
A compelling fiscal
environment for R&D
Significant
manufacturing
capacity
Innovating towards
adoption
*UK BioIndustry Association, Alliance for Regenerative Medicine, Leading Innovation: The UK's ATMP Landscape, July 2018. Available online via:
https://www.bioindustry.org/uploads/assets/uploaded/bb16e593-11ee-41e0-9407a44a7a084bb0.pdf

The Wider Medicines Manufacturing Ecosystem
Public funded
manufacturing
Infrastructure
Cell+Gene
Therapy Catapult
+ manufacturing
centre expansion
Medicines
Manufacturing
Innovation
centre
Vaccines
Manufacturing
Innovation
Centre
Innovation,
Digitalisation
and
Mechanisation
funding to
industry
Advanced
Therapy
Treatment
Centres
National
Biologics
Manufacturing
Centre
Nucleic Acid
Therapies
Accelerator
*MMIP, FTI Consulting, The fiscal benefits of establishing and retaining medicine manufacture in the UK, April 2017. Available online: https://www.bioindustry.org/resource-listing/briefing-paper--the-fiscal-
benefits-of-establishing-and-retaining-medicine-manufacture-in-the-uk.html
Innovation infrastructure investments
through UK Research and Innovation and
the Industrial Strategy Challenge Fund

ICSF Medicines Manufacturing Challenge
£300m to develop first-of-a-kind technologies for the manufacture of medicines
to accelerate patient access to new drugs and treatments
Medicines Manufacturing
Collaborative R&D
£31m
Advanced Therapies
Treatment Centres
£39m
Cell & Gene Therapy
Manufacturing Centre
£12m
Innovation Centre
£13m
Vaccines Development &
£160m
Digital Health Technology
Catalyst
£35m
Mechanising and
improving advanced
therapy manufacture
£1.3m
Viral vector production for
cell and gene therapies
£5.6m
Digitalisation of
medicines manufacturing
£4.3m

Vaccines Development and Manufacturing Centre

Medicine Manufacturing Innovation Centre
Advanced Manufacturing Innovation District Renfrewshire Scotland

26 OCT 2020
CPI has today announced the start of
construction at the Medicines
Manufacturing Innovation Centre in
Renfrewshire, Scotland.
https://www.uk-cpi.com/about/national-
centres/medicines-manufacturing-
innovation-centre

Collaborative R&D
£31m
Advanced Therapies
Treatment Centres
£39m
Cell & Gene Therapy
£12m
Innovation Centre
£13m
£160m
Catalyst
£35m
Digitalisation of
£4.3m
Mechanising and
improving advanced
therapy manufacture
£1.3m
£5.6m

CGT Catapult assets
- 1200m2 purpose-built centre
- Analytical characterisation
- Process development
- Viral vector
Development centre
- 7000m2 manufacturing centre designed
specifically for cell and gene therapies
- 12 segregated large clean room modules
- Secure supported collaboration model
- Centre of a cell and gene therapy cluster
Manufacturing centre

Collaborating companies
“CGT Catapult’s unique operational model allows
us to grow our manufacturing capacity, while
accessing a range of services provided by the
centre”. Jim Faulkner, SVP and Head of
Product Delivery
“We are looking forward to an important
collaboration with CGT Catapult scaling-up GMP
manufacturing strategies for commercial
production”. Gregg Sando, CEO
“We are delighted to establish this collaboration
for our next generation AAV gene therapy
platform for chronic systemic disease”. Jan
Thirkettle, Chief Development Officer
“With our own vector manufacturing capability at
the Catapult facility, we will extend vector supply
capacity beyond 2020”. James Noble, CEO
centre”. Jim Faulkner, SVP and Head of
Product Delivery
production”. Gregg Sando, CEO
platform for chronic systemic disease”. Jan
capacity beyond 2020”. James Noble, CEO
centre.” Jim Faulkner, SVP and Head of
Product Delivery
production.” Gregg Sando, CEO
platform for chronic systemic disease.” Jan
capacity beyond 2020.” James Noble, CEO
“Having advanced manufacturing facilities already
in place allows quick set up.” Garry Menzel,
CEO

Expansion phase
• Six new enlarged modules
• Enhanced throughput and batch capacity
• Modules constructed and commissioning well under way
• Qualification leverage
• Open for business and onboarding

Catapult Network – Fostering Innovation to Drive Economic Growth 2017
MEDICINES DISCOVERY CATAPULT
• Promoting and supporting innovative, fast-to-patient
drug discovery in the UK through collaborative projects
across the community.
• Working with biopharma companies, translational
researchers, technology experts, patient groups and research & finance sectors to help
transform great ideas into commercial products and services.
• Helping UK maintain position as a global leader in this key industry.
• Supporting disruptive medicines discovery programmes.
• Investing in new technologies and infrastructure to support SMEs and others to boost
the UK’s medicines pipeline.
• Developing new forms of financing to help stimulate collaborative activity and
innovation in UK drug discovery.

The adoption challenge:
Increasing patient demand
2018
Mainly clinical
trial ~200 patients
per year
2019
Licensed products
for larger patient
numbers
2021
Increased patient
delivery ~2500
patients per year
2023
Routine patient
delivery ~5000
patients per year
2028
Embedded
patient
delivery
~10000
patients per
year
Ø Currently 1 out of 8 advanced therapy clinical trials
in the world is taking place in the UK

Northern Alliance
Advanced Therapies
Treatment Centre
iMATCH – Manchester
Advanced Therapy
Centre Hub
MW-ATTC - Midlands &
Wales Advanced Therapy
Treatment Centre
Cell and Gene Therapy
Catapult
Northern Alliance
Advanced Therapies
Treatment Centre
iMATCH – Manchester
Advanced Therapy
Centre Hub
MW-ATTC - Midlands &
Wales Advanced Therapy
Treatment Centre
Cell and Gene Therapy
Catapult
Advanced Therapy Treatment Centre network
• The network increasing the ability of
the NHS to deliver disruptive
medicines
• Developing systems and processes to
increase the ability to deliver these
disruptive medicines to patients at
scale within ATTC NHS partners
• Disseminating the learnings and
systems from initial centres to enable
more widespread adoption of these
innovative therapies across the NHS
• 30 delivery partners – collaboration
between NHS, Industry and Academia
• 200-250 people working on the
programme since 2018
The network of Advanced Therapy
Treatment Centres developing and
delivering systems for cutting edge
cell and gene therapies.
London Advanced Therapy
Network
https://www.theattcnetwork.co.uk/

ATTC partnerships from supply to delivery
Objectives:
• Development of the clinical
delivery pathway
• Achieve utility at scale in
clinical pathway through use of
informatics
NHS providers
ATMP suppliers
Academia
Supporting industries

Collaborative R&D
£31m
Advanced Therapies
Treatment Centres
£39m
Cell & Gene Therapy
£12m
Innovation Centre
£13m
£160m
Catalyst
£35m
£5.6m
Digitalisation of
£4.3m
Mechanising and
improving advanced
therapy manufacture
£1.3m

UK Research
and Innovation 21
• 12 Advanced Therapies
Ø £10.2m grant funding + £3.6m co-funding
• 10 Medicine and therapeutic development
• 9 Pharmaceutical production technology
Medicines Manufacturing CR&D Projects

Prospering from Innovate UK’s Industrial Strategy boost for
UK medicines
• Cobra Biologics, an international CDMO of biologics and pharmaceuticals, was
awarded £2.6 million capital infrastructure investment grant at the end of 2017.
• The extended capacity and capability has allowed Cobra to increase process
development, analytics and GMP manufacturing, and importantly provided the
necessary infrastructure to offer commercial in-market supply of viral
vector and DNA products to Advanced Therapy Medicinal Product (ATMP)
developers. The expansion has doubled the footprint of Cobra’s UK site and has
led to the creation of over 30 additional jobs.
• Oxford BioMedica plc, a leading gene and cell therapy group, was awarded a £3 million
capital infrastructure investment grant by Innovate UK, under the Industrial Strategy
Challenge Fund, to support the UK’s efforts to produce viral vectors and ensure adequate
supply to meet future demand, at the end of 2017.
• The grant has supported investment in equipment for vector development, vector
manufacture, storage and analytical equipment, as well as other items that are key for the
operation of vector GMP facilities. In addition, a small part of the grant has been used to
support the planning for the transition of GMP suites from the use of adherent to
suspension cultures.

WORLD
LEADING
SKILLS
TALENT
INSTITUTIONS
Apprenticeships
Higher
Education
CPD skills
Clinical
training
World-leading skills, talent and institutions

EPSRC investment in Advanced Therapies
• Future Targeted Healthcare Manufacturing Hub
• £10M (2017 – 2023). UCL, Imperial College London, Warwick, Nottingham, Manchester,
Loughborough & industry partners.
• Researcher in Residence at the Cell and Gene Therapy Catapult (2018 – 2020).
• Investment in training and skills through our Centres for Doctoral Training (CDTs).
• Standard Mode routes through the Manufacturing the Future and Healthcare Technologies themes.

MRC Remit and Partners
• MRC: basic research to early
clinical trials
• Underpinning and aetiological
• Prevention
• Detection and diagnosis
• Treatment development &
evaluation
• Phase 1 & 2 trials
• Other funders/partners
• National Institute of Health Research
(NIHR)
• Other Research Councils
• Medical Charities
• Innovate UK
• Industry
Basic
research
Discovery Preclinical
Early
Clinical
Late
Clinical
HTA
Medical Charities
Innovate UK
NIHR
BBSRC &
EPSRC
MRC

UKRMP2 Hubs
• Pluripotent stem cells and engineered cell Hub
o Director Prof Roger Barker, Cambridge
§ Includes Sheffield, Loughborough and Babraham
• The engineered cell environment Hub
o Director Prof Stuart Forbes, Edinburgh
§ Includes Birmingham, Cambridge, KCL and UCL
• Acellular / smart materials – 3D architecture Hub
o Director Prof Molly Stevens, Imperial
§ Includes Nottingham, Edinburgh, Glasgow, Liverpool,
Manchester, Oxford, Southampton, QMUL and UCL
https://www.ukrmp.org.uk/hubs/

@InnovateUK Innovate UK
Innovate UK

1
January 21, 2021
VIDEOCONFERENCE
Program Director: Kelley Parato, PhD
Cell & Gene Therapy (CGT)
Challenge Program:
Advanced Therapies and
Biomanufacturing in Canada and
Opportunities for Canada – UK
Engagement

2
BUSINESS
INNOVATION
POLICY
SOLUTIONS
FOR
GOVERNMENT
ADVANCING
KNOWLEDGE
The NRC’s 3 core
roles within the
Canadian Science,
Technology, and
Innovation
ecosystem
• 1,093 R&D clients*
• $211M total revenues*
• $1,016.5M total expenditures* ($494.8M
RCs; $276.5M IRAP )
• $294.0M G&C expenditures
• 968 peer reviewed publications**
• 303 patents filed*
• 1,900 active patents*
* 2017-2018 / ** 2017

Challenge Programs at NRC
3
• Budget allocation to NRC for new collaborative
programming
• Catalyze transformative, high-risk, high-reward
research with potential for game-changing
scientific discoveries and technological
breakthroughs
• Enable our researchers to work with domestic
and international innovators from post-
secondary institutions and businesses on multi-
party R&D programs addressing issues of
importance to Canada

4
Our Challenge
Deliver made-in-Canada disruptive solutions
to enable accessibility and affordability
of engineered cell and gene therapies of the
future.
Ø Disruptive Technology Solutions
(tools, platforms, enabling technologies)
Ø Collaborative Partnerships to build
Canadian capacity for development and
delivery

5
A Multi-Disciplinary Collaborative Program
• Pipeline of disruptive
made-in-Canada therapies
(universal donor cell,
virus-free gene editing)
• Technologies that reduce
cost & complexity of
manufacturing
• Innovation in
commercialization, health
policy & delivery models.
NRC Canadian & Global Ecosystem
Technology
Toolbox
Enabling
Expertise
CGT
• Antibody platforms, sdAb
• Nanomaterials
• iPSC
• Viral vector design
• Packaging cell lines
• Device fabrication
• Automation
• AI
• Viral vector scale-up
manufacturing
• Analytics, metrology &
standards
• Clinical targets
• Clinical datasets
• CAR safety switches
• RM cell biology
• Nanomaterials
• Gene editing strategies
• Mammalian genome
foundry
• GMP device
fabrication
• Translational models
• cGMP facilities
• Clinical trial expertise
$

Master Projects:
Collaborative Partnerships to Build Capacity
6
CAR-T – expanding accessibility through clinical trials
Gene therapy – Glybera reinvented
2019-2021 2022-2025 2023-2026
Demonstration of collaborative partnership models to support and leverage Canadian
value chain and enable clinical deployment of accessible and affordable made-in-Canada
gene (targeting genetic disease) and cell-gene (targeting cancer) therapies.
Public-public partnerships
Public-public and public-private partnerships
Pathway for Gene
Therapy
Pathway for
CAR T

Master Projects: Disruptive Technology Solutions
7
Disruptive technologies that will reduce the costs of biomanufacturing and enable new
generation of safe (precise), accessible (allogeneic, universal) and affordable (cell-free)
engineered cell and gene therapies.
Microfluidic scale cell
production
Universal Donor Cells
Targeting for safety and efficacy
Viral vector bioprocess
innovation

NRC-UKRI/IUK Collaboration
8
GOAL: to establish collaborations between NRC researchers and those within the UK
CGT research community to advance novel strategies/technologies to optimize viral
vector biomanufacturing processes for two important platforms that NRC is developing.
IMPACT: to drive down cost of goods and dramatically improve yield of therapeutic
products for use in clinical trials to advance novel CAR T and gene therapy strategies for
important unmet clinical needs.
• Project 1 – Collaborative process improvement for AAV-based gene therapy
• Project 2 – Deployment of process analytical technologies to perfusion-enabled
lentiviral vector manufacturing
NATIONAL RESEARCH COUNCIL CANADA

The Canadian Biomanufacturing
Ecosystem
9

10
Collaboration across the ATMP
continuum

Areas of Opportunity in ATMPs
11
• Viral vector process innovation at research scale, rapid
translation to GMP and large-scale
• Non-viral vector gene therapy/gene editing technologies
• Process optimization, automation, predictive manufacturing for
cell therapies
• Microfluidic scale biomanufacturing
• Cell product qualification/metrology/analytics

THANK YOU
Kelley Parato • Program Director
Challenge Program: Disruptive Technology Solutions for Cell and Gene
Therapy
Kelley.Parato@nrc-cnrc.gc.ca

The Future of UK and Canada Collaboration in Advanced Therapies

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The Future of UK and Canada Collaboration in Advanced Therapies