JDRF Research and Partnerships to Improve the Lives of People with Type 1 Diabetes and Keep our Promise of a Cure Boston, ...
Mission and Research Strategy <ul><li>To find a cure for type 1 diabetes and its complications </li></ul><ul><li>Proactive...
Commitment to a Cure <ul><li>Placing the individual at the center with the end product in mind </li></ul><ul><ul><li>Provi...
JDRF Research Strategy <ul><li>1 ° Prevention  </li></ul><ul><li>Vaccines </li></ul><ul><li>2 °  Prevention </li></ul><ul>...
<ul><li>Primary Prevention:   Vaccines </li></ul><ul><ul><li>Infectious </li></ul></ul><ul><ul><li>Microbiota-based </li><...
Preventing through Earlier Diagnostics Interventions based on  earlier diagnostics % Beta Cells Time ( Years ) At-Risk Sub...
PREVENT TREAT CURE <ul><li>Complications </li></ul><ul><ul><li>Glucose-independent </li></ul></ul><ul><ul><ul><li>Genetics...
Preventing and Treating Complications Discover, develop, deliver therapies to prevent, arrest, and reverse diabetic compli...
PREVENT TREAT CURE <ul><li>Glucose Control </li></ul><ul><ul><li>Drugs </li></ul></ul><ul><ul><ul><li>Insulins, Amylin, Le...
Treating by Improving Glucose Control <ul><ul><li>Drugs  </li></ul></ul><ul><ul><ul><li>Glucose control </li></ul></ul></u...
JDRF Forms Partnership with Animas to Develop First-Generation Automated System for Managing Type 1 Diabetes and with Bect...
PREVENT TREAT CURE <ul><li>Encapsulated Alternative Cell Source </li></ul><ul><ul><li>Encapsulation </li></ul></ul><ul><ul...
Curing by Implanting Encapsulated Beta cells Immune Cells Complement 0 2 Nutrients GLUCOSE INSULIN C-Peptide Human Islet  ...
Curing by Arresting the Autoimmune Attack and… <ul><li>Discover, develop, and deliver therapies that arrest the autoimmune...
… Regenerating Native Beta Cells <ul><li>Discover, develop, and deliver therapies that restore glucose-responsive, insulin...
Exploratory research Target ID/Validation Lead identification Lead optimization Clinical development Generate new beta cel...
Multiple Immune Therapy Clinical Trials <ul><ul><li>Phase 3 new onset trials using anti-CD3 (TolerX, Macrogenics) </li></u...
TolerX Anti-CD3 Immunotherapy in Recent Onset Diabetes Increased Preservation of Functional Beta Cell Mass for 4 Years Dia...
Summary <ul><li>JDRF’s mission is to find a cure for type 1 diabetes and keeping individuals healthy while pursuing this m...
Appendix
Beta Cell Therapies  Current Industry Partners
Immune  Therapies  Current Industry Partners (Continued)
Current Industry Partners (Continued) Glucose Control  Complications Therapies
JDRF Venture Capital Industry People  with Type 1 Diabetes Providers Academia Funders FDA Payers JDRF JDRF’s Partners
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  • Discovery, Development, Delivery of Integrated Therapies at all Stages.
  • Presentation from Dr. Hehenberger

    1. 1. JDRF Research and Partnerships to Improve the Lives of People with Type 1 Diabetes and Keep our Promise of a Cure Boston, April 6, 2010 Karin Hehenberger, MD, PhD Senior Vice President, Strategic Alliances
    2. 2. Mission and Research Strategy <ul><li>To find a cure for type 1 diabetes and its complications </li></ul><ul><li>Proactively accelerate the discovery, development, and delivery of drugs and devices to Cure, Better Treat or Prevent type 1 diabetes and its complications at any stage of the disease at the point-of-care </li></ul><ul><ul><ul><li>Arrest the immune attack on insulin-producing cells </li></ul></ul></ul><ul><ul><ul><li>Restore or replace beta cell function </li></ul></ul></ul><ul><ul><ul><li>Better control blood sugars </li></ul></ul></ul><ul><ul><ul><li>Protect people from (or reverse) complications. </li></ul></ul></ul>
    3. 3. Commitment to a Cure <ul><li>Placing the individual at the center with the end product in mind </li></ul><ul><ul><li>Providing solutions for the different stages of type 1 diabetes </li></ul></ul><ul><li>Core principles </li></ul><ul><ul><li>People with T1DM, Pipeline, Priorities, Partners </li></ul></ul><ul><li>Proactively managed pipeline through joint committees that govern projects </li></ul><ul><li>JDRF drives partnerships with academia, patients organizations, FDA, payers, providers, industry, venture capital and funders </li></ul>At-Risk Sub-Clinical Recent Onset Established (without Complications) Established (with Complications)
    4. 4. JDRF Research Strategy <ul><li>1 ° Prevention </li></ul><ul><li>Vaccines </li></ul><ul><li>2 ° Prevention </li></ul><ul><li>Diagnosis and staging </li></ul><ul><li>followed by antigen-specific immune therapy and beta cell therapies </li></ul><ul><li>Complications </li></ul>Glucose Control Beta Cell Survival & Glucose Control Complications Antigen-specific immune therapy and Beta Cell Regeneration Encapsulated Alternative Beta Cell Source PREVENT TREAT CURE
    5. 5. <ul><li>Primary Prevention: Vaccines </li></ul><ul><ul><li>Infectious </li></ul></ul><ul><ul><li>Microbiota-based </li></ul></ul><ul><ul><li>Immunoregulatory </li></ul></ul><ul><li>Secondary Prevention: Diagnosis and Staging </li></ul>PREVENT TREAT CURE Time ( Years ) % Beta Cells 100 10 <ul><li>Followed by </li></ul><ul><ul><li>Antigen-specific Immunotherapy, Anti-inflammatories + Beta Cell Therapy </li></ul></ul>
    6. 6. Preventing through Earlier Diagnostics Interventions based on earlier diagnostics % Beta Cells Time ( Years ) At-Risk Sub-Clinical Recent Onset Established (without Complications) Established (with Complications) Current diagnosis based on glucose Early diagnosis based on auto-antibodies
    7. 7. PREVENT TREAT CURE <ul><li>Complications </li></ul><ul><ul><li>Glucose-independent </li></ul></ul><ul><ul><ul><li>Genetics </li></ul></ul></ul><ul><ul><ul><li>Modifiable risk factors </li></ul></ul></ul><ul><ul><li>Therapeutics </li></ul></ul><ul><ul><ul><li>Biomarkers </li></ul></ul></ul><ul><ul><ul><li>Target validation </li></ul></ul></ul>
    8. 8. Preventing and Treating Complications Discover, develop, deliver therapies to prevent, arrest, and reverse diabetic complications
    9. 9. PREVENT TREAT CURE <ul><li>Glucose Control </li></ul><ul><ul><li>Drugs </li></ul></ul><ul><ul><ul><li>Insulins, Amylin, Leptin </li></ul></ul></ul><ul><ul><li>Devices </li></ul></ul><ul><ul><ul><li>Pumps and Sensors </li></ul></ul></ul><ul><li>Beta Cell Survival “Keep the beta cell happy” </li></ul><ul><ul><li>Glucose control </li></ul></ul><ul><ul><li>Reduce apoptosis </li></ul></ul><ul><ul><li>Limit inflammation </li></ul></ul><ul><ul><li>Immunotherapy </li></ul></ul>
    10. 10. Treating by Improving Glucose Control <ul><ul><li>Drugs </li></ul></ul><ul><ul><ul><li>Glucose control </li></ul></ul></ul><ul><ul><ul><li>Prevent hypoglycemia </li></ul></ul></ul><ul><ul><li>Devices </li></ul></ul><ul><ul><ul><li>Closed loop “artificial pancreas” </li></ul></ul></ul><ul><ul><ul><li>Improved delivery of insulin </li></ul></ul></ul>Induce improved glucose control in people with established diabetes and the newly diagnosed with:
    11. 11. JDRF Forms Partnership with Animas to Develop First-Generation Automated System for Managing Type 1 Diabetes and with Becton Dickinson to improve insulin delivery January 2010 <ul><li>Develop 1 st generation semi-automated system to manage glucose control in people with diabetes </li></ul><ul><ul><li>Insulin pump connected to CGM to prevent hypoglycemia and extreme hyperglycemia </li></ul></ul><ul><ul><li>Slow/stop insulin if blood sugar too low; delivery automatically resumes </li></ul></ul><ul><ul><li>Increase insulin if blood sugar too high; automatically returns to pre-set basal rate </li></ul></ul><ul><li>Improve insulin delivery through better cannulae for insulin pumps </li></ul><ul><ul><li>Faster insulin delivery which will improve control </li></ul></ul><ul><ul><li>Microneedles </li></ul></ul>
    12. 12. PREVENT TREAT CURE <ul><li>Encapsulated Alternative Cell Source </li></ul><ul><ul><li>Encapsulation </li></ul></ul><ul><ul><li>Alternative Beta Cell Source </li></ul></ul><ul><li>Antigen-specific Immune Therapy + </li></ul><ul><li>Beta Cell Regeneration </li></ul><ul><ul><li>Survival </li></ul></ul><ul><ul><li>Neogenesis </li></ul></ul><ul><ul><li>Proliferation </li></ul></ul><ul><ul><li>Reprogramming </li></ul></ul>
    13. 13. Curing by Implanting Encapsulated Beta cells Immune Cells Complement 0 2 Nutrients GLUCOSE INSULIN C-Peptide Human Islet PEG 25-50 
    14. 14. Curing by Arresting the Autoimmune Attack and… <ul><li>Discover, develop, and deliver therapies that arrest the autoimmune attack and induce immunoregulation to: </li></ul><ul><ul><li>Preserve function of restored beta cells in established diabetes </li></ul></ul><ul><ul><li>Preserve residual beta cell function in recent onset diabetes or at-risk of developing diabetes </li></ul></ul><ul><ul><li>Prevent the onset of autoimmunity in the at-risk population </li></ul></ul>BETA CELL MASS TIME NEWLY DIAGNOSED T1D DIABETES PRE-DIABETES GENETIC PREDISPOSITION
    15. 15. … Regenerating Native Beta Cells <ul><li>Discover, develop, and deliver therapies that restore glucose-responsive, insulin-producing cells </li></ul><ul><li>Proliferation </li></ul><ul><li>Reprogramming </li></ul><ul><li>Survival </li></ul><ul><li>Neogenesis </li></ul> cells  cells  cells
    16. 16. Exploratory research Target ID/Validation Lead identification Lead optimization Clinical development Generate new beta cells Beta Cell Survival Beta Cell Growth Reprogram JDRF has Formed Partnerships with NVS, JNJ and PFE in Regeneration and Pursuing Further Partners…..
    17. 17. Multiple Immune Therapy Clinical Trials <ul><ul><li>Phase 3 new onset trials using anti-CD3 (TolerX, Macrogenics) </li></ul></ul><ul><ul><li>Phase 3 new onset trials using GAD-Alum (Diamyd) </li></ul></ul><ul><ul><li>Phase 2 secondary prevention trials using GAD-Alum and oral and intranasal insulin </li></ul></ul><ul><ul><li>Phase 2 trials using Insulin (Bayhill/DNA) and anti-CD20 (DNA/Roche) </li></ul></ul><ul><ul><li>Over a dozen different immune therapies in clinical testing in new onset T1D in 2010, ongoing planning for multiple prevention trials </li></ul></ul><ul><ul><li>Early-stage vaccines </li></ul></ul>
    18. 18. TolerX Anti-CD3 Immunotherapy in Recent Onset Diabetes Increased Preservation of Functional Beta Cell Mass for 4 Years Diabetologia (2010) 53:614–623
    19. 19. Summary <ul><li>JDRF’s mission is to find a cure for type 1 diabetes and keeping individuals healthy while pursuing this mission </li></ul><ul><li>Focus on the individual at all stages of the disease with the end product in mind </li></ul><ul><li>Advances in early stage research driven by JDRF have lead to multiple partnerships with industry </li></ul><ul><li>Accelerated effort with focus on curing, treating and preventing the disease by partnering with biotech, pharma and other funders </li></ul>
    20. 20. Appendix
    21. 21. Beta Cell Therapies Current Industry Partners
    22. 22. Immune Therapies Current Industry Partners (Continued)
    23. 23. Current Industry Partners (Continued) Glucose Control Complications Therapies
    24. 24. JDRF Venture Capital Industry People with Type 1 Diabetes Providers Academia Funders FDA Payers JDRF JDRF’s Partners

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