Turning TypeOne to The Plan for a World without T1D JDRF 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

LESSUNTILNONE JDRF’s Vision for the Future

Burden UNTILNONE Danger LESS Complexity LESS Invasive LESS LESS LESS UNTIL NONE

Delivery of Continuous Therapeutic Improvements Side-Effects Complications Patient Burden Glycemic Control Safety Normal Physiology Restored Easier, Safer, Less Worry, Better Life TherapiesCure

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) APP Strategy: Iterative Increases in Automation 8

Artificial Pancreas Smart 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 Insulin Encapsulation Implantable, beta cell replacement therapies restoring insulin independence without the need for intensive immune suppression

Artificial Pancreas Smart Insulin Encapsulation Encapsulation 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 Near TermMid Term Long Term Complications Diabetic Eye Disease Predictive Diagnostics Artificial Pancreas Low Glucose Suspend Treat-to-Range Artificial Pancreas Treat-to-Target Multihormone Encapsulation Implantable Islets Smart Insulin Control Prevention Secondary Prevention Prevention Primary: Vaccine Complications Comprehensive Prevention Therapy Restoration New Beta Cell Growth Protected 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 rides b.It’s the home of a leading T1D research institution c.It’s a TV series from which JDRF receives royalties d.Slows development of potential cures for T1D

Question #3 What is one reason partnering is essential to JDRF’s strategy? a.Partnering is more fun b.Partnering get you more friends c.Companies will eventually deliver products to patients d.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 CuresLaunching 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 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

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

Glucagon in a bi-hormonal pump 24 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 InsulinPrevention Restoration Complications

Your Support Capabilities Plan Vision