JDRF Therapeutic Ventures

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    ACCELERATING LIFE-CHANGING

    BREAKTHROUGHS TO CURE, TREAT

    AND PREVENT TYPE 1 DIABETES AND

    ITS COMPLICATIONS

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    A NEW STRATEGIC PLAN:

    EffectiveAugust 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

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    How We Get There

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    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

    ENCAPSULATIONRestoration 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 withcommercialization 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

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    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

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    MULTIPLYING THE IMPACT

    JDRF Effectively Leverages Donor Funds

    100.9 112.2 26.25.0

    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

    Million

    s

    Attracted Funding

    JDRF Funding

    2000

    2022

    2.5 2.4 4.1 Multiplier

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    CF FUND: In 1998, the CFF established a Therapeutics Development Program, apioneering 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 VertexPharmaceuticals 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 itsroyalty rights at a profit and reinvested the funds in its research program

    JOE ODONNELL: 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

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    THE VEHICLE

    Modeled After Cystic Fibrosis Foundation

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    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 forreinvestment 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.

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    THE VEHICLE

    JDRF Therapeutic Ventures, LLC

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    Leverage JDRFs 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

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    PARTICIPATION:

    Advisory board seats; participation in fund investment strategy

    COMMUNICATION:

    Quarterly progress reports; annual donor briefing; additional access toJDRF staff and researchers

    NAMING OPPORTUNITIES:

    Fund-specific opportunities for leadership gifts

    JDRF THERAPEUTIC VENTURES

    Donor Engagement & Recognition

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    THANK YOU

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