darrin disley - cambridge rare disease summit 2015
TRANSCRIPT
HORIZON DISCOVERY
Is Cambridge an ideal location to implement precision medicine for sufferers of rare diseases?
Dr Darrin M Disley
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Technology is powering translational genomics research and precision medicine
Technology Now Provides
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The Cambridge Ecosystem: key precision medicine resources
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Case study Neurofibromatosis9/11/15, 6:15 AMNetwork
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CharitiesVolunteered
CliniciansInterviewed
TechnologiesPilot Studies
PharmaGaps
Patient Seminars
IssuesScientists MentorsAdvice
A rare disease, 1/3,000 worldwide, no treatment, tumor predisposition, NF1, 2 mutations common in cancer
InstitutionsSample
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
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How about rare diseases like Neurofibromatosis
42%
21%
051015202530354045
Sporadic NF1
5-‐year relative survival rate
MPN
STs
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 tumorsmonitor 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
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§ 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
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Our future vision: The future of human healthcare
The power to help patients NOW!
Your Horizon Contact:
t + 44 (0)1223 655580f + 44 (0)1223 655581e [email protected] www.horizondiscovery.comHorizon Discovery, 7100 Cambridge Research Park, Waterbeach, Cambridge, CB25 9TL, United Kingdom
Dr Darrin M DisleyCEO & [email protected]+44 (0)7786 133893
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