We enable UK life science companies to develop their drug discovery projects. And through networks of expert labs and CROs our Virtual R&D team can access and provide: > industrially rigorous advice in drug discovery > clinical and commercial insight > expertise in delivery and project management If you are an SME with a drug discovery project, or a CRO with expertise to provide, attend this event and find out how we can help you.

1. Accelerating innovative drug discovery
Connecting the UK drug discovery community
Transforming ideas into better medicines
for patients, faster

2. National facility connecting the UK community
to accelerate innovative drug discovery
Medicines Discovery Catapult
WHO WHY HOW WHAT WHERE WORK WITH US
• Independent not-for-profit
• Part of the UK’s Catapult network
• Helping to deliver the UK’s Industrial Strategy
• Funded by Innovate UK, part of UK Research and Innovation,
reporting to the Department for Business, Energy & Industrial
Strategy

3. • Target specific industry-led challenges that limit today’s discovery process
• Industrialise, and drive the adoption of new scientific tools and modern techniques for discovering medicines
• Deploy scarce expertise and know-how to connect a fragmented community and leverage current UK infrastructure
• Support the UK life sciences industry, SMEs and innovators to deliver growth for the UK bio-economy
• Maintain the UK’s heritage position as a global leader in drug discovery
The enabling products and services that we diffuse into the sector will help UK SMEs fill their pipelines
with targeted medicines tested in patients, faster
WHO WHY HOW WHAT WHERE WORK WITH US
Our aims
Our vision

4. Providing hard-to-access
scientific capabilities
Gateway to UK resources
& expertise
By providing access to technologies and expertise not readily accessible we will support UK SMEs to
drive the development and widespread use of new approaches for the discovery of new medicines
WHO WHY HOW WHAT WHERE WORK WITH US
We are focussed on enabling UK drug discovery

5. We will not
• Compete with pharmaceutical or service companies
• Perform independent medicines discovery
• Replicate readily available UK infrastructure
We will prioritise and exemplify our efforts
• Outside of areas already covered
• In areas where UK science is perceived as ‘strong’
• In areas where SME needs are not met by others
• On development and promotion of cross-cutting discovery approaches
WHO WHY HOW WHAT WHERE WORK WITH US
We are focussed on enabling UK drug discovery

6. DISCOVERY SCIENCE
& TECHNOLOGY
INFORMATICS SAMPLES & DATA VIRTUAL R&D SYNDICATES
WHO WHY HOW WHAT WHERE WORK WITH US
Our unique capabilities
Designed to address the needs of our UK drug discovery community and change the shape of R&D:
BETTER DATA FASTER
ACCESS TO UK RESOURCES & EXPERTISE

7. Establishing patient-relevant testing models and
new technologies to advance drug discovery
Discovery Science & Technology
WHO WHY HOW WHAT WHERE WORK WITH US

8. WHO WHY HOW WHAT WHERE WORK WITH US
Discovery Science & Technology
We work collaboratively with all sectors of the UK Medicines Discovery Industry, with the primary aim of
developing and growing SMEs

9. • Neuroinflammation; Fibrosis
• Primary, iPS-derived cells
• 3D, Co-culture, Organ-on-a-chip
• Compound-Target engagement studies
• Target validation
• Microscopy; Mass-spectrometry
• Analysis of novel drug delivery moieties
• Pre-clinical imaging (PET, CT)
• Ex vivo imaging - Mass Spec Imaging;
Microscopy
• Biomarker identification; translation
• Cell models/tissues/clinical samples
• Nanostring; Mass-Spectrometry
WHO WHY HOW WHAT WHERE WORK WITH US
Discovery Science & Technology

10. BBB Spheroid
Pancreatic
Organoids on
chip
Vascularised
Cardiac
microtissue
Hepatocyte
Spheroids
Colorectal
Spheroids
Neurite
Dynamics
Kidney models
Disease relevant
iPSC
Cardiomyocytes
OOAC - Tissuse
OOAC – Second
platform
Scaffold
Supports
Drug Delivery
systems into
Cells
Strategic
Agreements
UK OOAC
network
Fibrosis
Neuro-
inflammation
Organ on a Chip (OOAC)
Connected Microphysiology
2D
3D spheroid
monolayer
DAPI
GFAP
NG2
DAPI
GFAP
CD31
50 um
Confocal microscopy images of a
blood brain barrier spheroid model
Complex cell models
WHO WHY HOW WHAT WHERE WORK WITH US
Replicating human physiology and pathophysiology
in vitro to enable better translation
Human Relevant
Predictive.

11. Biomarkers for precision medicine
WHO WHY HOW WHAT WHERE WORK WITH US
Transcriptomics
• RNA
• DNA
• Protein
Droplet digital PCR
Mass spec imaging
Mass Spectroscopy
• Lipidomics
• Metabolomics
• (peptides/proteins)
In vivo imaging
Biomarker Discovery
Biomarker Assay
Development
Biomarker Validation
Biomarker
prevalence
Early disease
linkage
studies
Human tissue

12. Profiling of complex biology and biomarkers
WHO WHY HOW WHAT WHERE WORK WITH US
Multiplexing quantification of up to 800 DNA, RNA and Protein molecules within a sample
• DNA
• RNA – mRNA, miRNA, fusion transcripts
• Protein expression

13. Target engagement & validation
WHO WHY HOW WHAT WHERE WORK WITH US
Strategy
• Does my compound reach its desired site of action?
• Does my compound engage with its molecular target in cells?
• What are the downstream consequences of compound/target binding?
In translatable
human cell models
At single-cell
resolution

14. Target engagement & validation
WHO WHY HOW WHAT WHERE WORK WITH US
Compound-Target Interaction with
Purified Protein
Direct Measurement of
Compound-Target
Interaction in Cells
Target Conformational
Change in Cells
Proximal pathway events
following target engagement
Distal pathway events following
target engagement
Super-Resolution Microscopy
Fluorescence Polarisation & FCS
BRET/FRET
Protein Stability
Dimerisation
Localisation
Cellular Mass-Spectrometry +/- Target
Knockdown
Cellular Mass-Spectrometry +/- Target Knockdown
Transcriptomics +/- Target Knockdown
Microscopy
build
underway
Target
Engagement
Target
Validation
Establishing
assay methods to
interrogate
TE/TV at all
levels
Solid-state NMR
Mass-Spectrometry

15. Mass spec imaging capabilities
WHO WHY HOW WHAT WHERE WORK WITH US
DESI-MSimaging;CD1micetumourxenografts
• Smallmoleculeexpressiondifferences
• Noveltargeteddeliverymethod
Instrumentation
Q-TOF
TQ-XS
DESI
Image visualisation
software
Application /
exploitation

16. What canitdo?
Uniqueimagingsolutionsenablesquantificationof:
• Organ accumulation(PK,biodistribution)
• Targetexpressionorengagement(stratification)
• Biologicalactivity(PD,optimalbiologicaldose)
• Toxicitiesordrug-druginteractions(safety,drugcombinationstudy)
• Surrogacyforclinicalendpoint(efficacy)
What canwedo?
Imaging Expertiseinmulti-parametricimagingmodalities:
• PositronEmissionTomography(PET)
• ComputedTomography(CT)
• HighFrequencyUltrasound(HFUS)
• Gamma countingex-vivoanalysis
• Nearinfraredopticalimaging
Targeted Drug Delivery
WHO WHY HOW WHAT WHERE WORK WITH US
Using Imaging technologies to visualise biological
functions to accelerate disease understanding and drug
development

17. WHO WHY HOW WHAT WHERE WORK WITH US
Drug discovery generates a huge quantity of complex biological, chemical,
clinical and safety information
To make the best decisions on which assets to further optimise or progress, and to
create the best data assets, secure sharing and associated data analytics are critical
Informatics
Using data science to discover new diagnostics,
biomarkers and drugs
Using our informatics skills, extensive proprietary databases and algorithms
we can help validate and drive adoption of new data-driven approaches
NOVEL DRUG COMBINATIONS
In silico TARGET VALIDATION
USING REAL WORLD DATA
DRUG REPURPOSING
EFFICACY AND SAFETY
ANALYSIS
UNDERSTANDING
CHEMICAL SPACE
DRUGGABILITY
We have deep expertise in scientific data analysis, information extraction,
natural language processing and machine learning

18. Core themes
WHO WHY HOW WHAT WHERE WORK WITH US
Collaborative Intelligence
• Mapping the UKs life science capability
Interoperable Data
• Inter-organization ELN data transfer
• Connected Data
• ‘Data on demand’ – better coverage of known facts
• Drug repositioning & reformulation – maximise asset use
• Synergistic drug combinations – chimeras, blends, cocktails
• Drug resistance – AMR, cancer, antivirals
• Leads for new target classes – not PPIs – channels, transporters, viruses…
Target Prioritization
• Variant Effect Prediction – ‘multiscale’ approach
• Target Validation - druggability & efficacy
e.g., small molecule protein kinase inhibitor
benchmark set. N=746
IT
Academic
Large
Pharma
Therapeutic
SME
CRO
Single Drug Multiple Drugs
SingleTargetMultipleTargets
Combination
Therapy
Classic Drug
Discovery,
Ehrlich’s ‘Magic
Bullet’
Drug Blending
Designed/Serendipitous
Polypharmacology
Drug
Target
Biochemic
al assay
Cell-
based
screen
Function
al assay
Animal
disease
model
Human
clinical
trial
ChEMBL MDC Clinicaltrials.gov

19. WHO WHY HOW WHAT WHERE WORK WITH US
The UK has millions of samples and billions of data points collected from
patients who have agreed their samples and data can be used for
research
However, small UK medical research companies struggle to access them
“Over 90% of SMEs said that access to high quality clinical samples was important,
however 80% found accessing samples unexpectedly difficult.”1
Samples & Data
Brokering easier access to consented patient
data and samples
We are working with key organisations supporting access to
consented tissues & data:
DIRECTORIES
RESEARCH
SERVICES
PROCUREMENT SERVICES

20. Access to samples is required by the sector
WHO WHY HOW WHAT WHERE WORK WITH US
Objectives
• Support UK SMEs in the supply of consented human tissue
• Set up collaborations with UK biobanks
• Provide access to expertise – Tissue Solutions
• A world leading provider of quality human tissue samples
• Benefit from connections and expertise
• Provide SMEs with required samples in a timely, cost
effective manner
• Provide vital cost recovery to biobanks to ensure that high
quality collections can be maintained
-100% 0% 100%
Access to biosamples for commercial
development is hugely important
It is easy to access high quality NHS biosamples
for commercial research
Access to the right health data such as registries
and activity is hugely important for innovative
companies
It is easy to access the right health data such as
registries and activity
The UK has a clear framework and process for the
commercialisation of NHS health data
Please could you indicate how much you agree / disagree with these
statements:
Neither agree nor disagree Somewhat agree Strongly agree
Somewhat disagree Strongly disagree

21. WHO WHY HOW WHAT WHERE WORK WITH US
As only patients know what it’s like to live with a disease, it’s crucial they
play a more central role in medical research
Talking to patients is often a better way to find out what medical products need to
developed, yet it doesn’t always happen
Discovery Syndicates
Spearheading patient involvement in early
drug discovery – a collective approach to R&D
Discovery Syndicates are a new, shared-resource model for disease focussed
consortia
Anchored by the leading medical research charity and centred
around patient needs
De-risk new promising assets
and drive commercialisation
Develop a patient centric
cohesive research agenda
Identify key barriers to
translation for a disease
Accelerate translation of
research to the clinic
Create new and more efficient
funding and investment models

22. WHO WHY HOW WHAT WHERE WORK WITH US
The Syndicate Programme
working together to:
• Set a patient-focussed agenda with
shared goals
• Build a portfolio of projects
• Leverage funding
• Share skills, knowledge and expertise
• Enable faster translation of new
treatments to the clinic

23. Making the delivery of medicines discovery
easier for UK innovators
Virtual R&D
WHO WHY HOW WHAT WHERE WORK WITH US
EXPERT NETWORK
DISCOVERY SERVICES
PROGRAMME MANAGEMENT

24. An academy of excellence to guide SME drug discovery
WHO WHY HOW WHAT WHERE WORK WITH US
Benefits and desired outcomes
• Leveraging existing expertise for
the benefit of UK SMEs
irrespective of location
• Critical advice and due diligence
for drug discovery projects
• Identifying new opportunities for
interaction and collaboration
• Creating a connected eco-system
of expertiseCentres of excellence
existing networks and
consultants:
Connecting expertise
in drug discovery
and clinical science.
Timely, flexible support
for business critical decisions
+
UK strength in
drug discovery

25. Drug discovery is complex, costly and resource intensive
WHO WHY HOW WHAT WHERE WORK WITH US
There is a need for independent,
agnostic, drug discovery
enablement
~40% of UK biotech SMEs have fewer than 5 employees
Limited band width can result in suboptimal decision making
The ‘eco-system’ is large but fragmented
Limiting knowledge transfer and access to know how

26. Drug discovery is complex, costly and resource intensive
WHO WHY HOW WHAT WHERE WORK WITH US
Complex web of service providers, enablers and funders
is challenging to navigate
Successful projects require:
• Knowledge; Decision making advice and expertise in
drug discovery
• Expertise; CROs, specialist academic institutes, etc
• Delivery; Experienced drug discovery project
management and due diligence
TI/TV
Hit
finding
Med.
Chem.
Pharmacology
Biomarkers
IND
enabling
Cell
models
Assay
generation
Comp.
Chem.
Library
design
Synth.
Chem.
Analyt.
Chem.
Process
Chem.
Formulation
In vivo
models
In vivo
efficacy
ADME
PK
PD
Dose to
man
Safety
GLP
tox
Idea
Drugcandidate

27. Virtual R&D capabilities and services
WHO WHY HOW WHAT WHERE WORK WITH US
Explore
Develop
Deliver
• Gap analysis, high level plan,
essential requirements, advice
• Detailed project plan, technical
report and due diligence
• Virtually embed with project
• Fully outsourced project
management
Virtual project
management
‘Discovery
Services’
CRO
relationships
‘Expert
Networks’
Access to
drug
discovery
and disease
expertise
MDC capabilities

28. How do we help?
Explore
• Gap analysis, high level plan,
essential requirements, advice
Develop
• Detailed project plan, technical
report and due diligence
Deliver
• Virtually embed with project
• Fully outsourced project
management
WHO WHY HOW WHAT WHERE WORK WITH US
• Project and data due diligence
• Quality assessment of the data and gap analysis
• Asset positioning, data needed, guidance for fundingTTO, VC, SME
Customers WHAT? HOW? DELIVERABLE
MDC / ExN
WHO?
• Draw up detailed project plans to agreed milestones
• Identify providers and define the studies
• Provide costs and timelines
TTO, VC, SME
MDC / DxS
• Integrated virtual project management
• Delivery capabilities; external and internal
• Milestones driven; agreed GO/NO GO for progressionSME
MDC / DxS

29. Project
Data
Package
“Status of
the Project”
MDC
Expertise to Progress your Drug Discovery Project?
WHO WHY HOW WHAT WHERE WORK WITH US
Explore
Develop
Deliver
• Gap analysis, high level
plan, essential
requirements, advice
• Detailed project plan,
technical
report and due diligence
• Virtually embed with
project
• Fully outsourced project
management
The Virtual Project Team

30. Clinical DevelopmentClinical
End to end services
Disease agnostic
Disease
specific
Niche
WHO WHY HOW WHAT WHERE WORK WITH US
Virtual R&D Project Delivery A Functional Network of CROs

31. WHO WHY HOW WHAT WHERE WORK WITH US

32. Application of drug
discovery expertise
to academic research
projects
Medicines Discovery
Catapult
Informaticians
identify specialist &
niche provision
Development of
expertise by
Medicines Discovery
Catapult internal
capability
Existing relationships
with expert labs.
Knowledge of the
service/ expertise
offered
WHO WHY HOW WHAT WHERE WORK WITH US
The Advantages
Reduced Project
Management time
and guidance
Access to new
markets and
customers
Increase breadth of
expertise through
collaborative
relationships
Benefit from
Medicines Discovery
Catapult
communications and
increased publicity
Customer Provider

33. WHO WHY HOW WHAT WHERE WORK WITH US
We work with our partners in a range of different models
Shared risk and reward
Joint grant applications for
collaborative R&D funding
Contracted fee-for-service projects
Signposting and connecting

34. md.catapult.org.uk
01625 238734 @meddisccat
Business Development
alexander.henzing@md.catapult.org.uk
Visit our website for our latest partnerships and
funding opportunities.
Get in touch for more information, expert guidance,
strategic consultancy and access to our technology.
Or to discuss a potential project or service.
WHO WHY HOW WHAT WHERE WORK WITH US
Contact us