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ACCELERATING LIFE-CHANGING

BREAKTHROUGHS TO CURE, TREAT
AND PREVENT TYPE 1 DIABETES AND
ITS COMPLICATIONS

CONFIDENTIAL

How We Get There A NEW STRATEGIC PLAN: Effective August 30. 2015 A RESTRUCTURED ORGANIZATION: Improved efficiency and productivity with lower costs INCREASE IN LEADERSHIP GIVING: Dedicated infrastructure to support high-level giving LAUNCH JDRF THERAPEUTIC VENTURES: New venture philanthropy approach with donors as active partners SCALE LEVERAGE OF PUBLIC AND CORPORATE CAPITAL: Use JDRF Therapeutic Ventures to exploit a proven strength CONFIDENTIAL 2 .

short-term risk. one or more companies in addition to Merck on path to near. integrated sevenday micro-patch pump/CGM ENCAPSULATION Restoration of insulin independence with replenishable encapsulated beta cell product without immunosuppression Clinical proof of concept of stem cell-derived islet & islet progenitor transplants from multiple companies. test and commercialize cell phone controlled. thereby reducing patient burden. and long-term incidence of complications Establish clinical proof of concept of at least one new GRI technological approach. multiple companies within 5-10 years of product availability GLUCOSE RESPONSIVE INSULIN Change T1D treatment paradigm to once a day injection resulting in near-normal glucose levels with minimal glucose measurement. Development and proof of principle of gene edited human stem cell derived islets resistant to immune attack ico CONFIDENTIAL 3 . develop.THE OPPORTUNITY Changing the Course of T1D PROGRAM THERAPEUTIC GOAL SHORT-TERM (5-7 YEAR) DELIVERABLES ARTIFICIAL PANCREAS Fully-automated system combining continuous glucose monitor and pump with algorithms to maintain near-normal glucose levels with minimal user effort Patient access and reimbursement of predictive lowglucose suspend and treat-to-range systems. clinical proof of concept with commercialization in 7-10 years BETA-CELL RESTORATION Return of biological insulin production through restoration and regeneration of functional beta-cell mass combined with autoimmunity mediation Clinical validation of drug repurposing to reverse disease.to mid-term commercialization PREVENTION Eliminate T1D as a threat to the next generation through strategies and therapies to intercept and disrupt disease onset and progression Development of diabetes prevention vaccines and therapies. including alteration in intestinal microbiome induced immunoregulation.

THE NEED Changing the Course of T1D: $375-$625MM PROGRAM SHORT-TERM (5-7 YEAR) DELIVERABLES INCREMENTAL FINANCIAL NEED ARTIFICIAL PANCREAS Patient access and reimbursement of predictive low-glucose suspend and treat-to-range systems. including alteration in intestinal microbiome -induced immunoregulation. develop. Development and proof of principle of gene edited human stem cell derived islets resistant to immune attack $150-$250 MM $50-$100 MM CONFIDENTIAL 4 .to mid-term commercialization $25-$50 MM PREVENTION Development of diabetes prevention vaccines and therapies. multiple companies within 5-10 years of product availability $50-$75 MM GLUCOSE RESPONSIVE INSULIN Establish clinical proof of concept of at least one new GRI technological approach. test and commercialize cell phone controlled. integrated seven-day micro-patch pump/CGM ENCAPSULATION Clinical proof of concept of stem cell-derived islet & islet progenitor transplants from multiple companies. clinical proof of concept with commercialization in 7-10 years $100-$150 MM BETA-CELL RESTORATION Clinical validation of drug repurposing to reverse disease. one or more companies in addition to Merck on path to near.

2 Industry Partners Foundations and NonProfit Partners Foreign Government Support 2.800 1.200 1.600 1.400 Millions 1.5 2.4 4.2 5.MULTIPLYING THE IMPACT JDRF Effectively Leverages Donor Funds 2000 – 2022 1.9 112.000 1.642.3 267.2 107.8 0 100.1 26.0 US Government Support Multiplier 5 .0 800 Attracted Funding JDRF Funding 600 400 200 247.

a pioneering partnership with the biotech community intended to expedite CF research and the development of drugs to treat CF VERTEX/KALYDECO: In return for royalty rights. effective in patients whose CF is caused by a specific gene mutation. the CFF established a Therapeutics Development Program. the CFF invested $150 million in Vertex Pharmaceuticals to develop the drug Kalydeco. He currently chairs the Milestones II campaign and plans to raise additional $75 million by the end of 2015 CONFIDENTIAL 6 . Following FDA approval of Kalydeco in 2012.THE VEHICLE Modeled After Cystic Fibrosis Foundation CF FUND: In 1998. CFF sold its royalty rights at a profit and reinvested the funds in its research program JOE O’DONNELL: Chairman of the ‘Milestone to a Cure’ major gifts campaign and raised over $175 million for the Cystic Fibrosis Foundation.

royalty-based.THE VEHICLE JDRF Therapeutic Ventures. Research Business Development. maximum expense load of 10% over fund life. fund-specific oversight by scientific and device/bio-pharma experts. early stage investors. JDRF VP. Investments may include equity.D. LLC STRUCTURE: Single member LLC sponsoring a series of closed-end therapeutic funds. returns accrue to fund for reinvestment or distribution to JDRF. OPERATIONS: Separate entity for liability purposes. initially staffed by Steven Griffen. and selected donors. CONFIDENTIAL 7 . JDRF VP. low-interest loans with warrant coverage.. administration and finance contracted from JDRF. GOVERNANCE: Five member JDRF Therapeutic Ventures board appointed by JDRF.000 contribution units. FUNDING: Tax-deductible $500. M. Translational Development & Jit Patel. and/or pilot grants with shared IP or commercial rights. facilities.

JDRF THERAPEUTIC VENTURES Change the Course of T1D  Leverage JDRF’s knowledge. network. staff and portfolio. financial and strategic investors  Utilize and fund JDRF for regulatory. reimbursement. knowledge to identify most promising opportunities  Create T1D-focused venture philanthropy funds to accelerate development. commercialization and adoption of therapies  Leverage capital through co-investments with universities. and adoption efforts  Re-invest returns in therapeutic funds CONFIDENTIAL 8 .

participation in fund investment strategy COMMUNICATION: Quarterly progress reports. additional access to JDRF staff and researchers NAMING OPPORTUNITIES: Fund-specific opportunities for leadership gifts CONFIDENTIAL 9 .JDRF THERAPEUTIC VENTURES Donor Engagement & Recognition PARTICIPATION: Advisory board seats. annual donor briefing.

THANK YOU CONFIDENTIAL 10 .