Turning TypeOne to

The Plan for a World without T1DJDRF Translational Development: Partnering for Success

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

LESS UNTIL NONE

JDRF’s Vision for the Future

BurdenUNTIL NONE

DangerLESS

ComplexityLESS

InvasiveLESS

LESS

LESS UNTIL NONE

Delivery of Continuous Therapeutic Improvements

Side-EffectsComplicationsPatient Burden

Glycemic ControlSafety

Normal PhysiologyRestored

Easier, Safer, Less Worry, Better Life

Therapies Cure

Artificial Pancreas

A series of systems that will automate insulin dosing and additional hormone delivery

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

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

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

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

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

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

Partnering for Success

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

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

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

The Plan: Work the Pipeline – Translational Strategy and Partnering for Success

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

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

DELIVERY TO PATIENTS

How JDRF collaborates with industry: Project-based Funding

JDRF and The Helmsley Charitable Trust: Sensor Initiative

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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

Tandem: Novel Dual-Chamber Infusion Pump

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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

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

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

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.

Artificial Pancreas Encapsulation Smart Insulin Prevention Restoration Complications

Your Support

Capabilities

Plan

Vision