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ACCELERATING LIFE-CHANGING
BREAKTHROUGHS TO CURE, TREAT
AND PREVENT TYPE 1 DIABETES AND
ITS COMPLICATIONS
CONFIDENTIAL
A NEW STRATEGIC PLAN:
Effective August 30, 2015
A RESTRUCTURED ORGANIZATION:
Improved efficiency and productivity with low...
ico
THE OPPORTUNITY
Changing the Course of T1D
PROGRAM THERAPEUTIC GOAL SHORT-TERM (5-7 YEAR) DELIVERABLES
ARTIFICIAL
PANC...
THE NEED
Changing the Course of T1D: $375-$625MM
PROGRAM SHORT-TERM (5-7 YEAR) DELIVERABLES INCREMENTAL FINANCIAL NEED
ART...
5
MULTIPLYING THE IMPACT
JDRF Effectively Leverages Donor Funds
100.9 112.2 26.2 5.0
247.3 267.8
107.2
1,642.0
0
200
400
6...
CF FUND: In 1998, the CFF established a Therapeutics Development Program, a
pioneering partnership with the biotech commun...
STRUCTURE: Single member LLC sponsoring a series of closed-end therapeutic funds;
maximum expense load of 10% over fund li...
 Leverage JDRF’s knowledge, network, staff and portfolio, knowledge to
identify most promising opportunities
 Create T1D...
PARTICIPATION:
Advisory board seats; participation in fund investment strategy
COMMUNICATION:
Quarterly progress reports; ...
THANK YOU
CONFIDENTIAL 10
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JDRF Therapeutic Ventures, LLC

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JDRF Therapeutic Ventures, LLC

  1. 1. ACCELERATING LIFE-CHANGING BREAKTHROUGHS TO CURE, TREAT AND PREVENT TYPE 1 DIABETES AND ITS COMPLICATIONS CONFIDENTIAL
  2. 2. 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 2 How We Get There CONFIDENTIAL
  3. 3. ico 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 low- glucose suspend and treat-to-range systems; develop, test and commercialize cell phone controlled, integrated seven- day 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; 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, thereby reducing patient burden, short-term risk, and long-term incidence of complications Establish clinical proof of concept of at least one new GRI technological approach; one or more companies in addition to Merck on path to near- 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; 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; Development and proof of principle of gene edited human stem cell derived islets resistant to immune attack 3CONFIDENTIAL
  4. 4. 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; develop, test and commercialize cell phone controlled, integrated seven-day micro-patch pump/CGM $50-$100 MM ENCAPSULATION Clinical proof of concept of stem cell-derived islet & islet progenitor transplants from multiple companies; 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; one or more companies in addition to Merck on path to near- to mid-term commercialization $25-$50 MM PREVENTION Development of diabetes prevention vaccines and therapies, including alteration in intestinal microbiome -induced immunoregulation; clinical proof of concept with commercialization in 7-10 years $100-$150 MM BETA-CELL RESTORATION Clinical validation of drug repurposing to reverse disease; Development and proof of principle of gene edited human stem cell derived islets resistant to immune attack $150-$250 MM 4CONFIDENTIAL
  5. 5. 5 MULTIPLYING THE IMPACT JDRF Effectively Leverages Donor Funds 100.9 112.2 26.2 5.0 247.3 267.8 107.2 1,642.0 0 200 400 600 800 1,000 1,200 1,400 1,600 1,800 Industry Partners Foundations and Non- Profit Partners Foreign Government Support US Government Support Millions Attracted Funding JDRF Funding 2000 – 2022 2.5 2.4 4.1 Multiplier
  6. 6. CF FUND: In 1998, the CFF established a Therapeutics Development Program, 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, the CFF invested $150 million in Vertex Pharmaceuticals to develop the drug Kalydeco, effective in patients whose CF is caused by a specific gene mutation. Following FDA approval of Kalydeco in 2012, 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. He currently chairs the Milestones II campaign and plans to raise additional $75 million by the end of 2015 6 THE VEHICLE Modeled After Cystic Fibrosis Foundation CONFIDENTIAL
  7. 7. STRUCTURE: Single member LLC sponsoring a series of closed-end therapeutic funds; maximum expense load of 10% over fund life. FUNDING: Tax-deductible $500,000 contribution units; returns accrue to fund for reinvestment or distribution to JDRF; Investments may include equity, royalty-based, low-interest loans with warrant coverage, and/or pilot grants with shared IP or commercial rights. OPERATIONS: Separate entity for liability purposes; initially staffed by Steven Griffen, M.D., JDRF VP, Translational Development & Jit Patel, JDRF VP, Research Business Development; facilities, administration and finance contracted from JDRF. GOVERNANCE: Five member JDRF Therapeutic Ventures board appointed by JDRF; fund-specific oversight by scientific and device/bio-pharma experts, early stage investors, and selected donors. 7 THE VEHICLE JDRF Therapeutic Ventures, LLC CONFIDENTIAL
  8. 8.  Leverage JDRF’s knowledge, network, staff and portfolio, 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, financial and strategic investors  Utilize and fund JDRF for regulatory, reimbursement, and adoption efforts  Re-invest returns in therapeutic funds JDRF THERAPEUTIC VENTURES Change the Course of T1D 8CONFIDENTIAL
  9. 9. PARTICIPATION: Advisory board seats; participation in fund investment strategy COMMUNICATION: Quarterly progress reports; annual donor briefing; additional access to JDRF staff and researchers NAMING OPPORTUNITIES: Fund-specific opportunities for leadership gifts JDRF THERAPEUTIC VENTURES Donor Engagement & Recognition 9CONFIDENTIAL
  10. 10. THANK YOU CONFIDENTIAL 10

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