Darrin Disley, CEO of Horizon Discovery, spoke in the 'Potential of the Cambridge cluster to address unmet therapeutic need' panel at the Cambridge Rare Disease Summit 2015.

1. HORIZON
DISCOVERY
Is
Cambridge
an
ideal
location
to
implement
precision
medicine
for
sufferers
of
rare
diseases?
Dr
Darrin
M
Disley

2. 2
Technology
is
powering
translational
genomics
research
and
precision
medicine
Technology
Now Provides

3. 3
The
Cambridge
Ecosystem:
key
precision
medicine
resources

4. 4
Case
study
Neurofibromatosis
9/11/15, 6:15 AMNetwork
Page 1 of 1file:///Users/lydiameyerturkson/Desktop/NF1/Network.webarchive
Charities
Volunteered
Clinicians
Interviewed
Technologies
Pilot
Studies
Pharma
Gaps
Patient
Seminars
Issues
Scientists
Mentors
Advice
A
rare
disease,
1/3,000
worldwide,
no
treatment,
tumor
predisposition,
NF1,
2
mutations
common
in
cancer
Institutions
Sample
HZD
VP
US
Business
Development
Lydia
Meyer-­‐Turkson
is
a
parent
of
an
NF
child:
spent
2
yrs
networking
to
understand
the
issues
and
frame
opportunities
to
action
Set
up
a
company
with
a
Mission:
To
empower
patients
and
create
new
biomarker
tests
to
monitor
early
signs
of
cancer
and
personalize
cancer
therapy

5. 5
How
about
rare
diseases
like
Neurofibromatosis
42%
21%
0
5
10
15
20
25
30
35
40
45
Sporadic NF1
5-­‐year
relative
survival
rate
MPNSTs
Patients
Genetic
NF1
testing
can
identify
95%
of
mutations
A
blood
test
to
differentiate
benign
from
cancerous
tumors
Current
cancer
treatments
fail
to
cure
50,000
NF1
patients
with
benign
tumors
monitor
2x
per
year
over
5-­‐25
yrs
• Non
invasive
tests
can
avoid
biopsies,
triage
patients
to
MRI
• Trigger
personalised
medicine
approaches
to
therapy
• Locus
Genetics
a
new
initiative
utilising
Cambridge/UK
resources
Radiation/chemotherapy
resistant
nerve
sheath
tumors
21
mth
survival

6. 6
§ Horizon
is
at
the
forefront
of
the
precision
medicine
revolution
§ The
pharmaceutical
landscape
is
changing
rapidly
§ Horizon’s translational
genomics
platform
make
the
addressing
of
diseases
that
affect
smaller
patient
populations
tractable
Horizon’s
mission
is
to
be
a
fully
integrated
life
science
company
that
provides
enabling
products,
services
and
research
programs
to
clients
engaged
at
every
stage
of
the
personalised
medicine
continuum
from
sequence
to
treatment
About
Horizon
Discovery
Group
plc

7. 7
Our
future
vision:
The
future
of
human
healthcare
The
power
to
help
patients
NOW!

8. Your
Horizon
Contact:
t +
44
(0)1223
655580
f +
44
(0)1223
655581
e info@horizondiscovery.com
w www.horizondiscovery.com
Horizon
Discovery,
7100
Cambridge
Research
Park,
Waterbeach,
Cambridge,
CB25
9TL,
United
Kingdom
Dr
Darrin
M
Disley
CEO
&
President
d.disley@horizondiscovery.com
+44
(0)7786
133893
8