turning typeone to the plan for a world without t1d jdrf translational development: partnering for...
TRANSCRIPT
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Turning TypeOne to
The Plan for a World without T1DJDRF Translational Development: Partnering for Success
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JDRF’s Vision for the Future
JDRF’s strategic areas of research
JDRF’s translational strategy
JDRF’s efforts in partnering
Q&A
What We’ll Cover
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LESS UNTIL NONE
JDRF’s Vision for the Future
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BurdenUNTIL NONE
DangerLESS
ComplexityLESS
InvasiveLESS
LESS
LESS UNTIL NONE
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Delivery of Continuous Therapeutic Improvements
Side-EffectsComplicationsPatient Burden
Glycemic ControlSafety
Normal PhysiologyRestored
Easier, Safer, Less Worry, Better Life
Therapies Cure
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Artificial Pancreas
A series of systems that will automate insulin dosing and additional hormone delivery
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Treat-to-Target (multi-hormone,
automated)
Low Glucose Suspend
Predictive Low Glucose Suspend
Treat-to-Range Treat-to-Target
(automated)
Treat-to-Target (w/meal bolus)
3 4 521 1 6APP Strategy: Iterative Increases in Automation
8
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Artificial Pancreas Artificial PancreasSmart Insulin
A series of systems that will automate insulin dosing and additional hormone delivery
Single dose of insulin that delivers insulin only where and
when needed
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Artificial Pancreas Smart Insulin
Single dose of insulin that delivers insulin only where and
when needed
Smart InsulinEncapsulation
Implantable, beta cell replacement therapies restoring insulin
independence without the need for intensive immune suppression
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Artificial Pancreas Smart Insulin EncapsulationEncapsulation
Implantable, beta cell replacement therapies restoring insulin
independence without the need for intensive immune suppression
Prevention
Slow or stop the progression of T1D before insulin dependence and
ultimately eliminate the possibility of ever developing T1D
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Artificial Pancreas Smart Insulin Encapsulation
Prevention
Prevention
Slow or stop the progression of T1D before insulin dependence and
ultimately eliminate the possibility of ever developing T1D
Restoration
Beta cell growth protected from immune attack restoring insulin
independence without immune suppression
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Continuous Therapeutic Improvements N
ear T
erm
Mid
Ter
mLo
ng T
erm
ComplicationsDiabetic Eye Disease
Predictive Diagnostics
Artificial Pancreas
Low Glucose Suspend
Treat-to-Range
Artificial PancreasTreat-to-Target
Multihormone
EncapsulationImplantable Islets
Smart InsulinControl
Prevention
Secondary Prevention
PreventionPrimary: Vaccine
ComplicationsComprehensivePrevention Therapy
Restoration
New Beta Cell GrowthProtected from Immune Attack
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Partnering for Success
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Question #1
What is translational research?
a. Research that catalyzes innovative discoveries to move from bench to the clinic
b. Research which enables scientific discoveries to mature into potential products
c. Research that progresses through the developmental pipeline
d. All of the above
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Question #2
What is the “Valley of Death”?
a. It’s the location of a JDRF fundraising bike ridesb. It’s the home of a leading T1D research
institutionc. It’s a TV series from which JDRF receives
royaltiesd. Slows development of potential cures for T1D
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Question #3
What is one reason partnering is essential to JDRF’s strategy?
a. Partnering is more funb. Partnering get you more friendsc. Companies will eventually deliver products to
patientsd. None of the above
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The Plan: Work the Pipeline – Translational Strategy and Partnering for Success
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The “Valley of Death” is a Key Part of the Problem
“The valley of death has gotten dramatically more
fatal over the last few years.”
-NewsWeek
Basic Research Translational Research Commercialization
“Valley of Death”
Slows Cures
• Identifying causes of disease
• Identifying potential therapeutic targets
Understanding disease
• Human trials
• Drug launches
• Post-launch studies
• Identifying therapies
• Preclinical research
Developing Cures Launching Products
“The valley of death has gotten dramatically more fatal over the last few years.”NewsWeek
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JDRF Strategies to Overcome Pipeline Hurdles
Enhance Academic and T1D Foundation Collaborations Eliminate redundancy and improve efficiencies
1
Novel Models for Industry Partnerships Focused on translational research
2
Engage Strategically Across the Pipeline Stronger integration of Research and Advocacy efforts and build
additional healthcare assessment capacity 3
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DELIVERY TO PATIENTS
How JDRF collaborates with industry: Project-based Funding
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JDRF and The Helmsley Charitable Trust: Sensor Initiative
22
JDRF-HCT Sensor Initiative aims to advance CGM accuracy and reliability towards the next generation artificial pancreas systems. • Accurate• Reliable• Redundant• Form factor• Safe • Enabling artificial pancreas systems
Project Goals
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Tandem: Novel Dual-Chamber Infusion Pump
23
Create a “dual-chamber” pump device that can simultaneously deliver insulin and other drug therapies.
Project Goals
Dual Chamber Infusion Pump for
insulin and other
hormones
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Glucagon in a bi-hormonal pump
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Develop a soluble and stable glucagon drug for use in multi-hormonal artificial pancreas systems
Project Goals
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How JDRF collaborates with industry: JDRF-Pharma-Academic Partnering
25
Joint-research collaboration programs to fund academic research in partnership with industry.
CENTER FOR THERAPEUTIC INNOVATION
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How JDRF collaborates with industry: Venture Creation, Seed Funding
Advance T1D breakthroughs across
the valley of death through a novel, entrepreneurial
partnership focused on cures.
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Artificial Pancreas Encapsulation Smart Insulin Prevention Restoration Complications
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Your Support
Capabilities
Plan
Vision