Environment of Academic/Industry Relationships December 1, 2018 - - PowerPoint PPT Presentation

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Environment of Academic/Industry Relationships December 1, 2018 - - PowerPoint PPT Presentation

The Changing Environment of Academic/Industry Relationships December 1, 2018 Agenda Part I AMC Innovations and Commercialization Models a) Key Industry Trends AMCs, Industry b) Models of Collaboration between AMCs and the


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The Changing Environment of Academic/Industry Relationships December 1, 2018

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Agenda

Part I – AMC Innovations and Commercialization Models

a) Key Industry Trends – AMC’s, Industry b) Models of Collaboration between AMC’s and the Industry i. Incubator / Accelerators c) Industry Alliances and Partnerships – Examples / Case Studies d) Philanthropy – Social Impact of Investing

Part II – Strategic Focus – Risk s and Benefits – Case Studies

a) Translational Genomics b) Tool’s and Technology c) Women’s Health d) Data Science Program – Diabetes Data Science

Part III – Geographic Region / Precincts – Advances Shared Services Models

a) “ What if you could build a regional research infrastructure with out misaligned incentives?”

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Part I - AMC Innovation and Commercialization Models

  • A. Industry Trends – AMC’s Pharm

R&D, Data Driven Initiatives

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Traditional academic medical center research models are unsustainable in a time of fiscal uncertainty

Additional models of research development must be explored to remain competitive in the evolving academic research landscape

Sources: http://www.nsf.gov/news/news_summ.jsp?cntn_id=130156, http://fas.org/sgp/crs/misc/R43341.pdf, http://www.beckershospitalreview.com/strategic-planning/is-academic-medicine-as-we-know-it-doa.html, http://chronicle.com/article/Donations-Rose- Only-Slightly/139879/

Federal Research Funding at Risk Changes In Clinical Reimbursement Reductions in Philanthropy Margin Uncertainty Driven by Value-based Care Regulatory Uncertainty Technology Disruption

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Research funding organizations provide us an “insider’s” perspective on the key issues

Opportunities Challenges

Open Data, Open Science “Data science is driving the next wave of disruption” Keen to see their research support generate compounding returns, funders are particularly interested in promoting a culture of data sharing across investigators, and training researchers to use “big data”.

  • Tools, platforms, and expertise exist that can derive additional value from disparate data

relevant to oncology science

  • Federal open data initiatives – under-utilized resources to advance basic/applied science
  • Open science can enhance ‘traditional’ science (e.g. clinical trials) with patient engagement

A Technological Wall? “Support for oncology research has faced challenges primarily due to technological advances that require a highly specialized infrastructure.”

  • Lack of facilities, equipment, skilled technicians, data resources, etc. have

lessened funders’ ability to advance on-site innovative research protocols on

  • ncology issues
  • Has also led to difficulties in recruiting physician-scientists able to advance

these research areas Collaborate on Shared Priorities “PMI, health equity, and translational science are priorities” Public-private partnerships and joining forces with patient advocacy groups also offer potential appeal to help increase funding impact.

  • External partners can help identify gaps in science-to-practice
  • Emerging opportunities for joint funding, strategic partnership, & initiative co-promotion

Choosing to Focus “What will we give up to pursue the priorities?” Funders are deliberating which opportunities might have the largest impacts and how they should proportion resources across the research value chain.

  • Which diseases/conditions/public health issues deserve the most focus?
  • Which recent discoveries might be ripe for translational research?

Addressing the Patient/Community Voice “Patient engagement is the key to revitalizing research” External partners are at the forefront of new models for community engagement.

  • Digital as a catalyst for expanding funders’ voices for health equity and social determinants
  • Patient-centered organizations can help facilitate linkages to important/under-resourced

communities and constituents Remaining Flexible “How do we respond rapidly to emerging public health needs?” Emerging discoveries, public health needs and as-yet unknown future research trends drive the increasing value that funders place on flexibility.

  • Flexibility is a capability funders seek to develop for themselves and look for in

the research community

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Systemic healthcare trends are positioning health

  • rganizations to take advantage of data-driven

initiatives

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Scientific and technological advances, such as genomic medicine, are driving a deluge of new data and opportunities for academic institutions

A transformation in healthcare is underway

  • Increased accountability and value-based approaches
  • Treatment driven by the molecular underpinnings,

rather than the resulting symptoms Genomic medicine is at it’s tipping point

  • The $1000 genome is now a reality
  • Total human genomes sequenced increased from <

20 to >10K in the past 5 years Genomic medicine is a data-driven science

  • Sequencing a single human genome can produce up

to a terabyte of data

  • Robust analyses can involve hundreds or thousands
  • f genomes

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There is a fundamental need to collaborate and share data across organizations to realize unparalleled value

“We believe the next step must be to transition to a system whereby a body unconnected with the generation of data acts as the custodian of access to the data… [to make it] available from multiple companies, public-sector

  • rganizations, and funders.”
  • Perry Nisen, SVP of

Science and Innovation, GSK

…but there is a lack of sophisticated solutions to transform how data is being leveraged and shared… …and organizations are hoarding data and working in silos because of a lack of trusted solutions There is an unprecedented amount

  • f data being generated

by an increasing number of sources…

Analysis 93% of executives believe their organizations are losing revenue (14% annually) as a result of not leveraging information collected2

  • f the world’s data has been

created in the last 2 years alone1

90%

Sources: 1) IBM, Big Data, 2013 ; 2) Oracle; From Overload to Impact: An Industry Scorecard on Big Data Business Challenges

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PwC 9 Ado-trastuzumab emtansine (Kadcyla) Afatinib (Gilotrif) Aldesleukin (Proleukin) Alemtuzumab (Campath) Axitinib (Inlyta) Belimumab (Benlysta) Belinostat (Beleodaq) Bevacizumab (Avastin) Bortezomib (Velcade) Bosutinib (Bosulif) Brentuximab vedotin (Adcetris) Cabozantinib (Cometriq) Canakinumab (Ilaris) Carfilzomib (Kyprolis) Ceritinib (Zykadia) Cetuximab (Erbitux) Crizotinib (Xalkori) Dabrafenib (Tafinlar) Dasatinib (Sprycel) Denosumab (Xgeva) Erlotinib (Tarceva) Everolimus (Afinitor) Gefitinib (Iressa) Ibritumomab tiuxetan (Zevalin) Ibrutinib (Imbruvica) Idelalisib (Zydelig) Imatinib (Gleevec) Ipilimumab (Yervoy) Lapatinib (Tykerb) Nilotinib (Tasigna) Obinutuzumab (Gazyva) Ofatumumab (Arzerra, HuMax-CD20) Panitumumab (Vectibix) Pazopanib (Votrient) Pembrolizumab (Keytruda) Pertuzumab (Perjeta) Ponatinib (Iclusig) Ramucirumab (Cyramza) Regorafenib (Stivarga) Rituximab (Rituxan, Mabthera) Romidepsin (Istodax) Ruxolitinib (Jakafi) Siltuximab (Sylvant) Sipuleucel-T (Provenge) Sorafenib (Nexavar) Temsirolimus (Torisel) Tocilizumab (Actemra) Tofacitinib (Xeljanz) Tositumomab (Bexxar) Trametinib (Mekinist) Trastuzumab (Herceptin) Vandetanib (Caprelsa) Vemurafenib (Zelboraf) Vismodegib (Erivedge) Vorinostat (Zolinza) Ziv-aflibercept (Zaltrap)

Melanoma Kidney Cancer

An Explosion of Therapeutic Options

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PwC 10 Ado-trastuzumab emtansine (Kadcyla) Afatinib (Gilotrif) Aldesleukin (Proleukin) Alemtuzumab (Campath) Axitinib (Inlyta) Belimumab (Benlysta) Belinostat (Beleodaq) Bevacizumab (Avastin) Bortezomib (Velcade) Bosutinib (Bosulif) Brentuximab vedotin (Adcetris) Cabozantinib (Cometriq) Canakinumab (Ilaris) Carfilzomib (Kyprolis) Ceritinib (Zykadia) Cetuximab (Erbitux) Crizotinib (Xalkori) Dabrafenib (Tafinlar) Dasatinib (Sprycel) Denosumab (Xgeva) Erlotinib (Tarceva) Everolimus (Afinitor) Gefitinib (Iressa) Ibritumomab tiuxetan (Zevalin) Ibrutinib (Imbruvica) Idelalisib (Zydelig) Imatinib (Gleevec) Ipilimumab (Yervoy) Lapatinib (Tykerb) Nilotinib (Tasigna) Obinutuzumab (Gazyva) Ofatumumab (Arzerra, HuMax-CD20) Panitumumab (Vectibix) Pazopanib (Votrient) Pembrolizumab (Keytruda) Pertuzumab (Perjeta) Ponatinib (Iclusig) Ramucirumab (Cyramza) Regorafenib (Stivarga) Rituximab (Rituxan, Mabthera) Romidepsin (Istodax) Ruxolitinib (Jakafi) Siltuximab (Sylvant) Sipuleucel-T (Provenge) Sorafenib (Nexavar) Temsirolimus (Torisel) Tocilizumab (Actemra) Tofacitinib (Xeljanz) Tositumomab (Bexxar) Trametinib (Mekinist) Trastuzumab (Herceptin) Vandetanib (Caprelsa) Vemurafenib (Zelboraf) Vismodegib (Erivedge) Vorinostat (Zolinza) Ziv-aflibercept (Zaltrap)

Lung Cancer Prostate Cancer Bone Cancer Lymphoma Leukemia Kidney Cancer Gastric Cancer Breast Cancer Thyroid Cancer Brain Cancer Head and Neck Cancer Myeloma Colorectal Cancer Pancreatic Cancer Melanoma Soft Tissue Sarcoma

An Explosion of Therapeutic Options

  • Multiple targets per therapy
  • Multiple therapies per target

2000 2005 2010 2015

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A Rapidly Shifting Landscape

50,000 100,000 150,000

1920 1960 2000 500 1000 1500 2000 2500 3000 3500 1954 1974 1994 2014 200,000 400,000 600,000 800,000 1,000,000 1,200,000 1,400,000 1,600,000

2/22/08 11/18/10 8/14/13

Oncology Publications Documented Mutations Clinical Trials

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3,140 Pages of Guidelines

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  • B. Models of Collaboration between

AMC’s and the Industry Incubator / Accelerators

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Partnership opportunities within the healthcare ecosystem

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Strategic Partners

Turning an AMC’s intellectual assets into capital

Research Data Software and Applications Faculty IP

Technology Transfer: Fee-for- service/ Licensing Business Development: Partnerships and knowledge sharing Incubators and VC funding: Integrated cultivation of innovation

Clinical Data Networks and Affiliates Centers and Institutes

Economic Development: Driving local/regional change

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AMCs can support commercialization by focusing

  • n four increasingly complex capabilities

Technology Transfer Economic Development Facilitate the development and commercialization of intellectual property to generate revenue from licenses Assist in attracting new businesses and creating new jobs in the region that may also generate down-stream returns for the AMC Business Development Develop new collaborations and initiatives to generate new services or value-streams (revenue or in-kind) Accelerators and VCs Provide funding, advice, resources, and/or space, to help small businesses grow to maximize returns and impact

Traditional Game changing

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The technology transfer office plays a central role in fostering innovation at academic medical centers

  • Benefits (examples)

 Alternative revenue stream for the institution  Improve brand value and ability to attract and retain top faculty, student and staff  Bridge to industry  Structured process to maximize ROI and minimize risk

  • Challenging decisions (examples)

 Objectives of the tech transfer office  Balancing budget with ROI  Establishing productivity targets  No / no-go on invention disclosures  License vs. start-up (e.g. internal investment)  Finding the right licensee  License terms

  • If it’s not measured, it cannot be managed

Academic Medical Center (Care, research & education) Inventor population (Research) Technology Transfer Office Industry Large licensees & sponsors Small licensees & start-ups

Internal External

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Challenges with managing a successful tech transfer office

  • Most TTOs cannot finance promising utility patents unless a licensee is in place, due

to lack of funds

  • Most TTOs are unable to advance promising technologies using internal funding to

create more valuable licenses

  • TTOs are often understaffed
  • Licensing staff often lack sufficient commercialization experience
  • Royalty revenues are not effectively tracked and collected
  • Licensing professionals are overburdened with non-core activities
  • High turnover; incentives not aligned
  • Licensing professionals receive insufficient support internally
  • Tech transfer processes are often disorganized
  • Tech transfer processes are often inconsistent and could be streamlined

TTO funding and support constraints TTOs are not working efficiently

  • Inventors lack visibility into tech transfer processes, including workflow, timelines

and licensing requirements, and are reluctant to collaborate with the TTO

  • Inventors feel they are not involved in key decisions regarding “their inventions”
  • Ineffective marketing of inventions and networking with industry on a broad scale
  • TTOs do a poor job networking with sector-specific licensees and rarely attend sector-

specific conferences

TTOs are perceived as “black holes” by faculty

Examples:

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Taking a comprehensive approach to monitoring technology transfer

Innovation Scorecard

Input Resources ($) Workforce

  • Research

expenditures

  • Gap funding
  • TTO

expenditures

  • People engaged
  • TTO staff
  • Faculty
  • Expertise
  • Tenure

Activities In/Outreach Services

  • Core TTO

services provided by the TTO

  • Non-core

services provided by the TTO

  • Beneficieries
  • Marketing
  • Networking
  • Events
  • Strategic

initiatives

  • Annual

reporting

Output Productivity Yield

  • Relative to staff
  • Relative to

faculty

  • Relative to key

services

  • Relative to

expenses

  • Patents filed
  • Revenue
  • Licenses
  • Start-ups
  • Cost of output

Impact Market Institutional

  • HHI score
  • Income

excluding top license/ Faculty

  • Faculty

engagement

  • Turnaround

time

  • Royalty sharing
  • FDA approvals
  • Licensed

technologies that became available

  • Job creation

from start-ups

  • Gross revenue

Four Pillars Eight Dimensions

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Benefits of proactive business development

Drive new revenue Generate philanthropy Engage industry partners Deliver novel care to patients Facilitate job creation Fuel economic development Promote clinician innovation Enhance AMC brand Accelerate research growth

Benefits fits

Recruit top faculty and scientists

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Targeting Larger Commercial Relationships

Degree of Benefit/Impact Across Partnership Types

Defining Objectives

External Licensing Co-Development Organic Growth

Impact to Operations and Structure

Limited restructuring or infrastructure development needed Some investment in equipment and expertise may be needed to accommodate

  • pportunities

Significant investment in operational and

  • rganizational structures

Transaction Volume

Highest transaction volume Greater transaction volume Limited transaction volume

Risk

Low financial risk Medium financial risk from investment in collaboration Medium to high financial risk from start-up investment

Capabilities Advancement

Limited advancement; Following industry trends Reaffirms Institute as a true competitor in a fast-paced field Will push Institute to the forefront of scientific advancement, technology and innovation

Control over Long- Term Success

Limited control; MWRI licenses products produced by others More control; MWRI contributes to co- development process Highest control; MWRI has full ownership of development process

Competitive Positioning

Following industry trends Reaffirms MWRI/MTRC as a true competitor in the women’s health field Will push MWRI/MTRC to the forefront of scientific advancement, technology and innovation

Short term Sustainability

Keep the doors open, but needs to be replenished constantly Potential for flexibility in negotiations Ramping up the agreement/relationship may take time

Long term value creation

Limited value generation Highly valuable IP generation with grater likelihood of ownership/involvement Financial gains; Institute becomes an industry leader; highly innovative; great for bringing in new customers; follow-on IP generation

Licensing technologies developed by Magee researchers Development of technologies with industry partners Primary and secondary development within AMC 21

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Illustrative example to operationalize an effective business development platform

  • Productize the biorepository
  • Operational collaborations w/ startups
  • Research collaborations
  • Population health strategies
  • Value-based care/payments
  • Employer strategies
  • 1. Improve the delivery of healthcare
  • 2. Consistent with Vision, Missions, research strategies

and clinical priorities

  • 3. Adds to national recognition as a transformative

healthcare delivery system and AMC mission

  • 4. Provides for a new or enhanced revenue stream, cost

savings, or other measurable benefit

  • 5. The opportunity potentially fulfills a market need
  • 6. Risks/cost acceptable relative to potential benefits
  • Leverage existing relationships
  • Participate in conferences and events
  • Host symposiums
  • Review with IP Committee and Executive

Leadership Committee

  • Market opportunity?
  • Investment required, if any
  • Revenue/cost projections

Non-financial benefits?

  • Benefits to partners?
  • Strategic opportunity

(e.g. alignment with goals)

  • High Priority partners?
  • Risks/challenges?
  • Community/economic

development benefit?

  • Patient volumes
  • Biorepository/tissue bank
  • Agility in clinical trials
  • Scientific knowledge,

particularly in bioengineering, surgery,

  • ncology, orthopedics,
  • bstetrics and pediatrics
  • New, innovative programs

Determine market- facing assets Innovator Identify viable potential Opportunity areas Present ‘strawman’ to Board for approval Prioritize opportunities with the Board Develop business for prioritized opportunities CDA, Collaborative Discussion, MoU Approval from Board if requires $500k+ investment or significant operational impact Approach Resources Counsel with plan for any opportunity requiring investment (excluding innovation fund) Confidentiality agreement w/ partner Collaborative discussions with potential partner Memorandum of Understanding (if needed) Develop robust business plan Exit/Close Review with Industry Advisory Committee Execution plan that reflects role of partners Present to Board (submitted 3 days in advance) Approval from Board monthly Signed by Leader Follow-up as appropriate No partners identified Examples Examples Examples = Template document required = Proactive approach = Only certain situations Identify potential partner(s) Exit/close opportunities that do not meet objectives Pursue potential partners Examples Examples

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Detailed Prioritization Discussions Based on Defined Criteria to Inform Initial Partner Targets

Aligned with Mission/Goals? Defined, plausible partnership model? Well-defined revenue stream/ product? Complexities of execution and timing? Are there quantifiable add-on benefits? Potential model has favorable margins?

Prioritization Questions

Minimal restructuring needed? Leverages existing platforms/expertise? Synergies with other existing partners?

  • Not restricted by existing

relationships

  • Interested in innovating
  • Willingness to collaborate
  • Can bring other partners

Ability to Partner

  • Announced interest
  • Existing/complementary

expertise

  • Synergistic core

competency

Relevant Expertise

  • Disease-specific
  • Technology-specific
  • Sector: pharma, devices,

diagnostics, services, etc

Market Share in Related Fields

  • Revenue, Mkt Cap, fund

size, financials

  • Access to customers
  • Access to sponsors

Ability to Fund

Strong Relationships

‘Master List’ Development

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Regularly evaluate its evolving ecosystem

DFCI DFCI DFCI DFCI DFCI

International U.S. Boston Therapeutics Diagnostics Devices Technology

Partners Payers Legal Finance Marketing Operations Research Network Extension

KEY Leading Co Emerging Co Leading Co Emerging Co International Co

NOT EXHAUSTIVE

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Incubators at AMCs to Support Company Development

Value Generating Company Iterative Testing Business Modelling Visioning Innovation Boot camp Selection

http://scholar.harvard.edu/files/mbarnett/files/ostrovsky_hjdsi_2014.pdf

An innovation incubator at an AMC is a program that cultivates the rapid transformation of ideas into value-generating products or services that benefit patients, providers, and/or payers. A number of AMCs nationwide are collaborating with industry partners through health start-up incubators.

Clinician Innovators

AMC Administrative Support functions: Tech transfer, Legal, Development, Space, etc.

Examples in digital health

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QB3 is an incubator and technology commercialization institute for the quantitative biosciences spanning three University of California campuses. Companies in the network have created more than 280 jobs and raised over $370M) in venture financing

Key corporate relationships Pfizer:

  • Strategic partnership for collaborative R&D, including

some through the Pfizer Seed Fund, resulting in over 25 collaborations funded since 2008 and several licensing opportunities and publications Johnson & Johnson Innovation:

  • Janssen, a Belgium-based subsidiary of J&J, will

found Janssen Labs in a QB3 facility Bayer:

  • Bayer’s scientists will work with counterparts at QB3

and Mission Bay Capital with the goal of more efficient evaluation of technologies Roche:

  • Alliance for “Collaborative startups” to identify, fund

and support early stage life science startup companies in the San Francisco Bay Area Novartis:

  • Novartis will join QB3 and Mission Bay Capital to

invest in startups Perspectives: Top 10 mistakes universities make 1. Muddled motives

  • 2. Impatientness
  • 3. Firewalls separating academic and commercial

activities

  • 4. Physical barriers
  • 5. Excessive transaction costs
  • 6. Myth of the rainmaker
  • 7. Excessive emphasis on leading faculty
  • 8. Heuristic belief that we can pick winners
  • 9. Failure to align universities with economic

development

  • 10. Greed

Source: QB3 web site, (Various, including: About, Quick Facts, Incubators (953), QB3 Buildings, Research: Core Facilities, Research: Affiliated Centers), Mission Bay Capital, Pfizer, J&J, Roche and Bayer, Bayer, Novartis, Capital IQ) 26

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Example – Joslin Diabetes Center and Jenesis Biosciences collaboration to accelerate commercialization of research

The focus of the partnership is to provide an active framework that will seed new ideas, partner technologies for co-development with other companies, and bring selected Joslin technologies to a commercially ready stage for spin-out opportunities and joint ventures.

  • Connect research and ideas

from Joslin Diabetes Center to academic and industry partners

  • Provide support through

experienced members of the Advisory Board

  • Focus on next-generation

products and services targeted towards diabetes management

  • Use in vivo validation,

preclinical characterization, and strategic alliances to bring technology to clinical and market stages

  • Treatment of complications
  • Explore clinical technology in

the fields of telemedicine, imaging, and e-health Collaboration Acceleration of Development Advancement of Clinical Technology

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Several dimensions of Incubators need to be considered to determine the right model

Cultural Operational Structural

  • How does an incubator

exist from a legal and financial perspective?

  • As simple as 2 people

working on a project inside an AMC, or can be a for-profit subsidiary

  • Financial return and

strategic optionality are key factors to create value

  • Defining strategic,

scientific and care goals

  • Governance
  • Leaders and teams
  • Operational plan
  • Shared services with AMC:

Legal, marketing, HR, etc.

  • Funding sources/strategy
  • Indicators of progress,

success and value creation

  • Message to the market for

recruiting, press releases

  • Entrepreneurialism inside

clinical/academic setting

  • Providing rewards/return

to drivers of success (e.g. inventors, leaders)

  • Fail-fast environment,

while maintaining momentum

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Johns Hopkins University – Report of the committee

  • n the innovation ecosystem (May, 2014)

1. Physical space where start-ups can take root and entrepreneurs can interact

  • 2. Funding of translational activity through

three mechanisms:

  • An evergreen commercial seed grant to

promote advanced early concepts towards proof-of-concept and prototype development

  • An externally managed investment fund for

companies that emerge from Johns Hopkins discoveries and innovation

  • Grant program to fuel compelling student

and fellow technology development

  • 3. A set of resources and incentives

calibrated to provide the needed support for the university’s scholar-inventors (network of investors, experts, analysts, etc.) Bring innovation to market to benefit humanity Continue to recruit and retain the highest-caliber students, faculty, and staff Create sustainable revenue streams for the university Fulfill the civic duty to drive economic development in the surrounding community

Request: the university should make an investment in its innovation ecosystem that consists of three parts Goals

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  • Subcontract services
  • IDCs
  • Recruitment

Once of the Top AMCs View of the Entire Innovation Ecosystem to Support Economic Development

  • Sponsored research
  • Space rental
  • Core services

“Expanding the Pie” Ecosystem Market Solutions & New Revenue Academic Research Institutions Translational Institute Accelerator Venture Capital/Private Equity Funds $ $ $ Seed Fund

  • Sponsored research
  • Space rental
  • Equity
  • Royalty revenues
  • Philanthropy

IP & Knowhow IP & Knowhow IP & Knowhow Value Created from Collaboration: Organic/Industry-driven release of IP and knowhow into the market At-scale sophisticated market solutions Industry co-development deals which scale into market through established channels Community/Industry Partners 30

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Focusing on 8 strategic priorities

Set Growth-oriented Objectives

  • Start-ups:
  • 3-5 per year
  • Grow equity position
  • Alliances (5 currently):
  • Create 3
  • Grow 3
  • Continue 2

1

Streamline Work Processes

  • Develop IP strategy
  • Consistent SOPs/process, (w/

prior-art search & market assessments)

  • Decision-point checklists
  • Set defined timeline/

expectations with faculty

  • OGC-approved agreement

templates – some online

  • Pipeline Project to catalogue and

prioritize IP

  • Additional admin support
  • Install Wellspring database

2

Define and Expand the Ecosystem

  • Deepen relationships w/industry
  • Clusters to monitor and pursue

complex cancer ecosystem, by sector, including tech and big data

  • Coordinate ‘blitz days’ with

pharma to meet broad range of faculty

3

Prioritize Relationship Management and BD

  • Create CM clusters aligned to

strategic priorities with leads responsible for relationship management

  • Hire BD lead with robust industry

network

  • Identify 2-3 seasoned

Entrepreneurs In Residence to extend network and support business planning

4

Build POC & Venture Funds

  • Grow Breakthrough Innovation

(POC) Fund

  • 5-7 investments/yr
  • $100-200K each
  • DFCI Venture Fund
  • 3-5 investments/yr
  • $750K-1.5M each
  • Launch with $10-30M

($5M from MPM philanthropy, remainder from development) Utilize ISR, BDC for awards

5

Strengthen Faculty Relationships

  • Initiative to meet with each PI;

set cadence with each, specifically prolific PIs

  • Meet with each new PI ASAP to

introduce offerings and key contacts

  • Communicate clear timing

expectations with faculty

  • Message from leadership about

role of innovation

6

Improve Communications

  • Annual DFCI-wide report
  • Newsletter internally monthly

and externally quarterly noting activities, innovations, and celebrating successes

  • Coordinate activities with

Finance, OGC and G&C

  • Pipeline Project to create

platform for internal and external stakeholders to view extensive amount of science happening

7

Implement a Training Curriculum

  • Train Staff:
  • IP Strategy - prior art searches

and market assessments

  • Business development and

relationship mgmt.

  • Getting to ‘Yes’
  • Faculty training seminars:
  • ‘What is IP?’
  • Business Dvlpmt. Basics
  • Starting & Growing a Company
  • Commercial Activities in

Targeted Sectors

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CLOSE/RETURN

For inventions that do not fair well in the technology evaluation, or for which direct marketing proves unsuccessful, the decision is made to discontinue active marketing. The innovators then have the option of taking on protection and marketing themselves.

NEW VENTURE/STARTUP

Technologies that demonstrate potential to develop into multiple products, or that have broad and accessible markets are excellent candidates to build a company around. We are selective in launching new ventures/startups, and invest a great deal of resources into the most promising candidates.

The proposed commercialization process distinct steps

6. MONITORING

(15-25 Years)

Monitoring of our license agreements is important to ensure that licensees live up to their promise to develop products based on

  • ur technology, and

to ensure compliance with payment and other

  • bligations.

Auditing is an important part of this step.

  • 5. LICENSING

(4-6 Months)

Negotiation of the financial, diligence and legal terms of a license agreement is a complicated and labor intensive process. Negotiations typically take four to six months, and involve a great deal

  • f support from the

Office of General Counsel.

4. MARKETING

(Months)

Technologies that fair well in the initial evaluation undergo a comprehensive market analysis. Potential licensing partners are identified and engaged, and confidential information, data and prototypes are shared.

3. PATENTING

(4-10 Years)

Patent counsel is retained to draft a patent application for inventions that fair well in the evaluation step. The patent process may require significant time in the U.S. and may require even more time and money in foreign countries.

2. EVALUATION

(7-10 Weeks)

A detailed technology evaluation by a case manager is performed over an approximately two month period. Evaluation criteria include patentability, market potential, market readiness, and medical efficacy. This step culminates with a committee review.

1. DISCLOSURE

(2 Weeks)

Submission of an Invention Disclosure triggers the Commercialization

  • Process. Within two

weeks, the Invention Disclosure is processed, reviewed, and a preliminary assessment is performed.

*** Modeled after Cleveland Clinic Innovations process *** Draft checklist follows

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At the Right Time, Building the Company – Holistic View

Ambition Business Model Strategic Agenda Strategic Foundation CORPORATE STRATEGY Customers CUSTOMER OFFERING Products, Services & Solutions Channels Intermediaries Alliance Partners Brands PROCESS BUSINESS CAPABILITIES ORGANIZATION Processes Policies TECHNOLOGY Application Integration Infrastructure INFORMATION Reports & Analytics Semantics Data PEOPLE CAPABILITIES Competencies Workforce & Talent Reward Culture & Behaviours Networks & Interdependencies Governance Arrangements Physical Environment Roles & Accountabilities Suppliers Organization Structure Tax Structure & Arrangements CORPORATE STRUCTURE Legal & Regulatory Structure Capital Structure / Funds Flows Cash, Banking & Treasury Structure

ENTERPRISE PERFORMANCE MANAGEMENT METRICS

INTERACTION MODELS = Proposed approach is being vetted = Currently in development = Requires further exploration

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Startup preliminary business plan checklist (1/2)

1 Company Purpose - One sentence with a bold statement describing the business, it’s goal, it’s differentiation, it’s value No Yes A Fit on the back of a business card? B Easily understood, declarative, exciting but believable? C Serve as the lead-in to all that comes after in the pitch? 2 Problem Statement - What are the major customer and/or customer’s customers “pain points” of focus? No Yes A Say how they are doing things today and what is wrong? B Say why there is a gap that exists today and the severity of that gap? C Address which customers care and why they are interested in the product? 3 Solution Overview - What is the explicit value proposition for the customer, how will they be better off? No Yes A Describe specific customer use-cases? B Note who pays for the product/service? C Note the customer’s internal costs and barriers? 4 Why Now for the Opportunity - What is the summary of the market evolution creating the opportunity? No Yes A Describe the specific trends making it possible and attractive? B Outline key market dynamics with the entrenched large players in the market? C Summarize the expected timeframe for scaling the business? 5 Market Size - Profile the specific customer, and buyers, appealing to and the market size potential No Yes A Note the characteristics and driving forces of the customer and buyers? B Size the Total Addressable Market (TAM) in dollars, numbers of customers? C Identify market segments and what share of the market is potentially obtainable?

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Startup preliminary business plan checklist (2/2)

6 Business Model - Describe the revenue and pricing model No Yes A Notes categories of revenue streams expected? B Identify revenue model? C Note expected average account size and lifetime customer value? 7 Competition - Describe competitive framework No Yes A Note top competitors? B Note market differentiation? C Note possible new market entrants? 8 Product Description - What is the product’s base and future functionality? No Yes A Describe the features, form, and overall design? B Outline current/potential IP? C Discuss what the Minimally Viable Product (MVP) looks like and timeframe? 9 Team - Outline the management team, advisors, key personnel requirements No Yes A Is there an existing team with near-term expected additions? B Is there a Board of Directors and a plan for potential additions? C Is there a Board of Advisors and/or industry experts involved? 10 Financials - Current and projected financials, capital requirements No Yes A Are P&L, balance sheet, cash flow statements available? B Is there a capitalization table? C Is there a funding “ask” and use plan for proceeds?

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  • C. Industry Alliances and Partnerships

– Examples / Case Studies

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Pharma companies struggle to recoup revenue at risk from lucrative patent expiries in the next few years

  • The patent cliff puts at risk an estimated $290 billion in sales revenue from 2012-

2018, with an expected $148 billion loss in sales

  • Drug development costs are spiraling with investments increasing from $500M

per drug in the 1990s, heading toward $10B per drug in 2013

  • A weak pipeline has decreased the number of new drugs approved, down from

39 new drugs in 2012 to 27 in 2013

53 33 43 44 40 30 35 38 25 24 16 14 17 17 10 20 30 40 50 60 2012 2013 2014E 2015F 2016F 2017F 2018F Total Sales at Risk Expected Sales Lost WW Sales in billions ($) Source: Evaluate Pharma “World Preview 2014, Outlook to 2020”, Kevin Lustig, “Strategic outsourcing of pharmaceutical R&D”, FDA.gov

Worldwide sales at Risk from Patent Cliff by Year (2012-2018F)

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Big Pharma is creating venture funds to support early R&D

“Corporate venture arms [have] fundamentally changed the landscape for startups and the entire field of early-stage drug development”

  • Silicon Valley Bank

Estimated ‘Fund Size’ for Biopharma’s Corporate Funds

Sources: FierceBiotech.com, Forbes.com, Burrill & Company 38

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AstraZeneca perspective – “Diligence Process: Five Dimensions for success”

Source: Chris Yochim, AstraZeneca - Global Head of External Relations; ‘Partnering for success: scientific, business & economic development’ (2014)

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AstraZeneca open innovation

Source: Chris Yochim, AstraZeneca - Global Head of External Relations; ‘Partnering for success: scientific, business & economic development’ (2014)

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Example – Johnson & Johnson sourcing external innovation through J & J Innovation Centers

Johnson & Johnson (J&J) Innovation Centers focus on accelerating early-stage innovation worldwide and forming collaborations between entrepreneurs and J & J’s global healthcare businesses.

Source: Johnson & Johnson presentation by Paul Stoffels, Chief Scientific Officer and Worldwide Chairman of Pharmaceuticals 41

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Example of a collaboration to maximize use of clinical and genomic data

Pre-competitive Consortium Discoveries Target and biomarker hypotheses Inventions and data to public Industry partners:

Priority access for 5 companies

Tailored and Novel therapy trials

Open access for researchers

At-large Community Program Collaborators:

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Innovation incubator process for Fortune 50 company – Taking ideas to programs to businesses

43

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Expediting discovery through big data, industry collaboration, and open-science frameworks—the way of the future for personalized health & medicine

Life Science organizations are rapidly embracing big data/open data initiatives to:  Capitalize on a leadership position in the industry  Foster greater scientific collaboration  Aggregate and mine richer data sets  Expedite discovery

“Neurodata Without Boarders” Project

"There is an urgent need and increased opportunities for advanced collaboration and coordination of access to, and analysis of, the rapidly expanding collections of biomedical data" - Dr. Francis Collins, NIH

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Consortium Examples: Large scale genomics initiatives and research consortia provide a unique

  • pportunity for new platforms

Background

  • Consortiums are particularly useful, when the nature of

disease is highly heterogeneous and specific with multiple subtypes

  • The value of data is much more than the sum of

individual databases when data is aggregated for researchers

  • A cloud based platform is the next step in efficient

distribution of data to speed research and discovery Consortium Examples:

  • The Cancer Genome Atlas/ Google / Cancer

Genomics Cloud: NIH funded cloud-based platform for TCGA

  • International Cancer Genome Consortium: Global

initiative to characterize 50 different tumor types and subtypes

  • Human Longevity Inc.: Privately funded initiative by J.

Craig Venter to utilize sequencing technologies to understand the cause of human disease and aging

  • Oncology Clinical and Translational Consortium: A

public-private partnership headed by GSK to drive development of anti-cancer compounds

  • eMERGE / MMRF / 1000 Genomes / Children’s

Oncology Group: Other genetics research consortiums

Oncology Clinical and Translational Consortium MedCloud Value Proposition

  • Reduces time and costs associated with

building an informatics infrastructure

  • Increased financial flexibility by

converting more fixed costs to variable costs

  • Improves efficiency through a decentralized

process with less reliance on institutional silos serving as points of coordination

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Illustrative example: Platform that integrates data and information and enables collaboration to catalyze breakthrough advances in health

Core Platform & Governance Precision Medicine

(Compound Rx and Dx)

Consumer Health

(Behavior Mod, Education, Prevention)

Network Democratization

(Access to Excellence)

Pragmatic Clinical Trials

(Large Realistic Studies)

Population Health

(Public Safety, Efficient Care Allocation)

Accumulation of critical health data

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Examples of large-scale industry/academia partnerships and collaborations

Select major publicly-disclosed collaborations Industry partner Academic institution Sector/strategy

Agilent UC Berkeley Synthetic Biology AstraZeneca University College London Stem Cells AstraZeneca University of Manchester Inflammatory Disease AstraZeneca University of Pennsylvania Alzheimer’s Bayer UCSF Master R&D Agreement J&J MIT Oncology J&J Sanford-Burnham Alzheimer’s Genentech UCSF Neurodegenerative Diseases Gilead Yale Oncology GSK MarS Innovation University Consortium GSK University of Manchester Inflammatory Disease GSK Harvard Stem Cells GSK Undisclosed (Global) 10 “Academic Superstars” Novartis Harvard Stem Cells Pfizer CTI: SF, NYC, Boston University Consortium Pfizer UCSF 20 PI-sponsored projects Pfizer Washington University Immunology Sanofi Harvard Cancer, Diabetes, Inflammation Sanofi Columbia University Diabetes Sanofi Salk Institute Gene Therapy Sanofi Stanford Stem Cells Sanofi UCSF Master R&D Agreement Takeda Sanford-Burnham Obesity UCB Harvard Neurology, Immunology Veridex Mass General Hospital Circulating Tumor Cells Zambion UCSF Drug Delivery

Best practices

  • Develop a portfolio approach: To move beyond

solely technology transfer commercial relationships, develop a portfolio approach to various services and programs you can offer that will support industry research and development goals

  • Identify research evangelists: find researchers

that are: (1) Established within a given field (2) Open to supporting the institution as a whole, (3) Willing to put in time, and (4) Have a desire to help younger researchers and the institution.

  • Business development expertise: hire

individuals that can uniquely bridge gap between science and business innovation to obtain researcher support and translate assets into industry opportunities.

  • Measure performance like a corporate

entity: develop a clear plan to measure progress and accountability for missing performance goals to meet industry expectations for successful long term partnerships.

  • Enable ease of investment into innovation:

Developing a means for corporate entities to co- invest or invest into another private entity, outside

  • f the academic institution.

Source: Chris Yochim, AstraZeneca - Global Head of External Relations; ‘Partnering for success: scientific, business & economic development’ (2014)

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  • D. Philanthropy – Social Impact of

Investing

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Philanthropic opportunities to fund innovative research

Philanthropists are recognizing the need and opportunity to support translational research with a credible commercial path to drive new products into the market. $240 billion of individual philanthropic giving, only 2-3% supports research today

Total Giving 1973-2013 Philanthropy Market Size

$ Billions

Sources: 1) Benefunder 2) Forbes: Annual Philanthropy Numbers On The Rise, June 2014

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Benefunder: An new model for philanthropic giving to support research and innovation

Benefunder Approach - Benefunder is a marketplace that allows donors to find, fund, and follow researchers and other university initiatives in a simple, efficient way. Access to Multiple Donor Channels including: 4 top wealth management firms, major private foundations, Fortune 50 corporate donors and over 300 family offices

Source: 1) Benefunder

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Part II – Strategic Focus – Risk s and Benefits – Case Studies

i. Translational Genomics

  • ii. Tool’s and Technology
  • iii. Women’s Health
  • iv. Data Science Program –

Diabetes Data Science

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Translational Genomics

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The promise of Precision Medicine—treating the right patient, with the right therapy, at the right time—is starting to take hold

Federal Investments & Initiatives Private Investments & Initiatives

Philanthrop y Venture Funding

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Precision Medicine has been fueled by biomarker R&D, advancements in high throughput technologies, and high performance computing

9 12 13 15 15 18 20 23 27 31 11 10

201 9 201 8 201 7 201 6 +14% 202 201 5

Global biomarkers market ($B)

Oncology Others 4,500 2,930 512 19 88 2 7 62

Metabolomics MicroRNA Transcriptions Proteomics

49

Genomics

Public Domain FDA Approved

Oncology biomarkers discovered by OMICS

  • Growing market

The global

  • ncology

biomarkers market (of which US has the largest share) has experienced substantial growth over the last couple of years, and is expected to reach 15 $B by 2020 at a CAGR of 11.6%

  • Opportunity in drug development

Genomics and proteomics have helped in discovery of many biomarkers but most of them fail during FDA testing

DRIVERS CHALLENGES

  • Favorable government funding for cancer research
  • Increased demand for personalized medicines in cancer

therapies

  • Adoption of high throughput technologies for the discovery of

biomarkers

  • Government’s Early Detection Research Network initiative for

development and testing of biomarkers for early detection of cancer

  • High capital investments for biomarker discovery and development,

and sample collection and storage

  • Characterizing cohorts for statistical validation of candidates
  • Lack of standardized criteria for validation of biomarker-based

assays

  • Acquiring and using big data analytics capabilities for

aggregation, collection, and the interpretation of the data to be implemented into the clinical trial

  • Limited knowledge of the analytical, diagnostic, and

regulatory requirements for a clinical assay 54

Sources: Markets and Markets, Research and Markets, Elsevier

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Since its inception, TGen has been on the bleeding- edge of ‘omics’ technology and Precision Medicine— and the market continues to grow

Companies Technologies Gene Expression Genotyping DNA Sequencing Proteomics Metabolomics

  • Affymetrix
  • Agilent
  • Illumina
  • Life Technologies

(ThermoFisher)

  • Roche
  • Illumina
  • Roche
  • PacBio
  • Life Technologies

(ThermoFisher)

  • Complete Genomics
  • Beckman Coulter
  • Sequenom
  • Illumina
  • Life Technologies

(ThermoFisher)

  • Life Technologies

(ThermoFisher)

  • Agilent
  • Bio-Rad
  • Bruker Daltonics
  • Waters
  • Life Technologies
  • (ThermoFisher)
  • Agilent
  • Bruker

Current Market Position

Trends

  • Microarrays
  • RT-PCR
  • RNA-seq
  • Primer extension

based platforms

  • Ligation-based
  • PCR-based
  • 2D Gels
  • GC / MS
  • Protein arrays
  • HPLC
  • GC / MS
  • NMR
  • FTIR
  • Capillary sequencing
  • Next generation
  • Third generation
  • Microarrays are being

displaced by sequencing messages

  • Increasing number of

applications include including mRNA, methylation, and clinical applications

  • The replacement of

GWAS with whole genome sequencing continues to impact the genotyping market

  • Large potential, and

fragmented market with technological challenges due to wide dynamic range of protein conc.

  • Emerging field with

technology limitations for comprehensive analysis

$6.2B CAGR 22% $3.4B CAGR 9% $4.5B CAGR 21% $10.2B CAGR 15% $0.6 B CAGR 30%

‘Omic’ Segments Many of these market leading companies are TGen’s strategic partners

55

Sources: Marketsandmarkets; BCC Research

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Personalized Hope Program Straw Model

Can Allow TGen and City of Hope to be the First to Fully Implement Quantitative Medicine into the Care Stream

Tackling Rare Diseases

Advanced genome/exome sequencing in the pediatric and rare disease populations Biomarkers for Monitoring Treatment Response & Cancer Recurrence Non-invasive cancer diagnostics that measure cancer progression at multiple points in time “Omic” Characterization of Tumors Identification of clinically meaningful genetic markers and use of liquid biopsy that informs accurate cancer diagnosis

Personalized Comfort

Enable an integrative care approach that treats disease while also supporting the patients quality

  • f life

Personalized Immune Evaluation Using immuno-genomics to unveil new insights into immune system function Early Detection in High Risk Individuals Genetic epidemiology and liquid biopsy combined with population health analysis tolls that identify and stratify high-risk patients Personalized Imaging Use of Radiomics that provide comprehensive tumor phenotype analysis Personalized Health Monitoring Characterizing both healthy and individual members of a disease community to identify nurtigenomic or microbiotic patterns Rapid Deployment of COH/TGen Therapies/Diagnostics into Clinical Care Internal infrastructure to support GMP- compliant drug manufacturing / development

Exercise 1 - Breakout Team 1.A

Aspire

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San Diego Cluster Case Study

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Cities, Regions and Clusters: 3 Major interdependent Instrumental Frameworks for local and regional development

Developing an efficient local or regional development model to ensure the success of innovation commercialization in an innovative and attractive place

The European Council, in March 2008, has recognised the important role of clusters in stimulating innovation and improving competitiveness, and urged to improve coordinated efforts to sustain them, …” “There is no question that cities are the engines of economic growth and centres of dynamic social and cultural activities and development…”.John

Naisbitt - Author and Futurist, USA

58

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PwC Accelerating Strong VC network Entrepreneurs Commercial partners and smart suppliers Cluster organizations:

  • BD for contract

negotiation, and partners search

  • Legal for IP and

contracts

  • Finance for

fundraising and P&L management

  • IT
  • RA for registration

and P&R

  • Talent search for

resources access

  • Access to experts

Innovation and the value chain challenge: The clustering approach

BioMedical and the role of cluster actors

Prototype Pilot Launch Stable Product Incubating Policy makers Public funding entities Commercial banks Strong VC network Cluster Organizations related services (BD, contract negotiation, Legal, Finance, IT, RA, talent search, etc) Prototyping units and certified labs New scientists and Entrepreneurs Scaling-up Strong VC network Entrepreneurs SMEs as potential acquisition targets, or for licensing in or out) Commercial partners (internal and external to the cluster) and smart suppliers Strong research (active principles and technology) partners (Universities, and RI, and SMEs) Cluster organizations as source of network and experts access Testing Policy makers Public funding entities Commercial banks Strong VC network TTOs Universities and RI Cluster Organizations related services (BD, contract negotiation, Legal, Finance, IT, RA, etc) Prototyping units and certified labs Entrepreneurs Proof of concept Plateau Declining Declining Strong VC network Entrepreneurs SMEs as potential acquisition targets, or for licensing in or out) Commercial partners (internal and external to the cluster) and smart suppliers Strong research (active principles and technology) partners (Universities, and RI, and SMEs) Cluster organizations as source of network and experts access

Cluster Actors

Research Results

Phases

Cash flow over time Time Discovering Policy makers Public funding entities Commercial banks Strong VC network TTOs Universities and RI Cluster Organizations related services (BD, Legal, Finance, IT, etc) 59

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Pittsburgh Case Study

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Representation of a mature biocluster ecosystem

Research & Development Facilities Specialized Research Service Providers Laboratory, Clinical Testing Educational Institutions Research Organizations Teaching & Specialized Hospitals Support Services Laboratories Plasma extraction Medical waste management Diagnostic Medical Equipment Central sterilization Security services Laundry Services Transport Specialized Business Services Banking, Legal, Accounting Specialized Risk Capital VC Firms, Angel Networks Ancillary Industries Hospitality, Retail, Food & Beverage Medical Nursing Dental Pharmacy Other back office Training & internships Continued medical education (CME) Masters & Doctorate &

  • ther programs

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Community

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Magee Women’s Research Institute – Case Study

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Vision: Establish MWRI as a global leader in reproductive sciences research through the development of cures for and prevention of diseases affecting women and infants

  • Developmental and

Regenerative Biology

  • Pregnancy
  • Infectious Diseases
  • Gynecology
  • Reproductive Physiology,

Biology and Fertility

  • Women’s Cancer
  • Women’s Health

MWRI Focus Areas

  • Advance scientific knowledge in the fields

in reproductive biology and medicine

  • Translate discoveries into improved health

for women and infants

  • Train current and future reproductive

sciences scholars

  • Foster community investment and

involvement in women’s health MWRI Central Mission

MWRI Focus and Mission

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MWRI is a Recognized Leader in OB GYN research closely affiliated with University of Pittsburgh and UPMC

MWRI was established in 1992 as an independent research institute and the research arm of the Department of Obstetrics, Gynecology & Reproductive Sciences (OBGYNRS) of the University of Pittsburgh

  • Closely affiliated with University of Pittsburgh and UPMC- home to Pitt faculty whose work

ranges from reproductive biology and development and diverse aspects of women’s health spanning from embryonic development through to menopause and aging.

  • Strong clinical partnership with Magee-Womens Hospital of UPMC, top-10 in U.S. News &

World Report Top-Ranked Hospitals for Gynecology.

  • # 1 in NIH research project funding for OB GYN research programs
  • Researchers at MWRI are served by the University of Pittsburgh’s Office of Technology

Management (OTM). The OTM is responsible for the protection, management, and commercialization of intellectual property for the University of Pittsburgh, including all standard licensing agreements.

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Overall Market Potential

MWRI’s Current Research Strengths Align with the Overall Commercial Market Demand and Growth $32B (6% CAGR)

Expected overall global market for women’s health therapeutics by 2016– increased market attention due to disease patterns and treatment differentials

$8B

(30% CAGR)

Expected global prenatal and newborn genetic testing market by 2019 $5B (4% CAGR) Expected global infertility drug market by 2017

$5B

(6% CAGR)

Expected global gynecological medical devices market by 2018

MWRI Current Research Strengths (Sample)

1) Fertility and Reproductive Endocrinology

  • Male and Female Fertility
  • Germ stem cell transplants

2) Pregnancy and Newborn Medicine

  • Prenatal genetic testing

3) Urogynecology

  • Pelvic floor disorders

4) HIV and Infectious Diseases

  • HIV microbicides
  • Drug delivery systems

5) Womens Cancers

  • Personalized medicine

Potential Applications to other areas:

  • Gender-based biology
  • Genomic medicine
  • Geriatric chronic disorders

$5B

(16% CAGR)

Expected global breast imaging and diagnostic market by 2019

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MWRI Can Develop a New Platform to Transform and Accelerate Promising Discoveries to Reach Commercial Potential

Research Discoveries Application / Development Commercialization

Industry Partners

Deal Flow MWRI UPitt Other

Innovation (GAP) Funding Licensing/ Start-up / New Venture Development

New Platform to Transform Value with Access / Expertise:

  • Translational / Applied Research
  • Product Development / Validation
  • Project / Portfolio Management
  • Business Development / Commercialization
  • Financial Capital / Venture Philanthropy

Positive Impact:

  • Products
  • Newco’s
  • Jobs

Translational / Applied Research Expertise Scientific Core Facilities Specialized Research Services Venture Philanthropy Funds Commerciali- zation Expertise Business Development

Critical Resources for Successful Development

Reinvestment Reinvestment

Increased Value / Decreased Risk

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University of Virginia– Case Study

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(International Diabetes Federation, 2017)

Diabetes

68

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Genomics (Stephen Rich)

Genetic risk stratification Surveillance for autoantibodies Metabolic and immune correlates Prevention/Early Intervention trials

Control Technology (Boris Kovatchev)

Virtual Image of the Patient (VIP) Artificial Pancreas / Remote Monitoring Neural Networks and Deep Learning In silicon modeling and Systems Biology Diabetes Data Science Statewide screening program

Cure Immunology (Larry Lum)

Treg infusion / Imaging Tregs Beta cell regeneration/encapsulation Islet cell Transplantation Target organ imaging Immune Function Tests

Detect

The Virginia PrIMed Project – Strategic Investment

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Personalized VIP treatment: example of PrIMeD data infrastructure application

Virtual Image of the Patient (VIP) initialization and update supported by the HMP Database

Secure, HIPA compliant Database

VIP Initialization and update

Data: reassessment of self-treatment Frequency: triggered by elevated risk Methods: in silico treatment replay Action: physician feedback to patient

Physician

Risk Assessment Treatment Optimization

Data: CGM; SMBG Frequency: therapy-dependent Methods: Risk Analysis Action: Alert Physician Data: retrospective CGM and simple home test Frequency: once Methods: VIP mapping

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Part III – Geographic Region / Precincts – Advances Shared Services Models “ What if you could build a regional research infrastructure with out misaligned incentives?”

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Sophisticated translational research strategy based on core strengths will drive valuable research discovery

Key Outcomes

  • Improved patient prognosis and
  • utcomes through early and/or more

accurate diagnosis, disease prevention, and targeted treatment

  • Growing patient repository of

molecular profiles and outcomes data for discovery research

  • More rapid and efficient identification
  • f patients and alignment with clinical trial
  • pportunities
  • Improved understanding of disease

etiology and causal underpinnings

  • Accelerated translation of discovery to

clinical implementation (from bench to bedside)

  • Proactive commercialization strategies

for immediate, intermediate, and long-term revenue sources

Clinical Care Delivery Translational Research

Core Strengths

Reimbursement Billing and Collections Comprehensive Outreach Program Clinical Trial Operations Enablers Bioethics & Patient Consent Research Talent World Class Leadership and Scientific/Clinical Expertise Technology Cutting Edge Scientific and Medical Technologies Infrastructure Supporting Lab Testing, Biobanking, and Informatics Process Integration Across Research and Clinical Activities Collaboration Strategic Academic and Commercial Partnerships Basic Research Commercial Return 72

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Assess existing capabilities and develop a set of strategic imperatives based on foundational, competing and differentiated products and services. Consider the following…

Therapeutic expertise – embed genomic medicine into routine cancer care and screening Patient recruitment and enrolment – provide ease of access to clinical trials, allowing seamless recruitment and enrolment for both physician and patient Sample and phenotype collection – develop and integrate standardized processes for gathering and recording biological samples and phenotypic data DNA extraction and testing – expertise extracting DNA samples from tissues, blood and other media, whether in- house or through 3rd party collaborations Biorepository / bio-banking – ensure biorepositories that store patient tissues / specimens are of high quality, safety and consistency Data Management and Analytics – develop secure infrastructure to support data acquisition, integration and analysis across disparate sources Governance – establish appropriate oversight to meet regulatory requirements and ethical standards for clinical trials, data sharing and inter-facility collaborations

Technology Enablers

Big data analytics Bioinformatics Gene sequencing Drug discovery Companion diagnostics and

  • thers

Applications

Oncology CNS Immunology Respiratory medicine Infectious and

  • thers

Post annotation and validation – provide expertise in genomic sequencing analysis and validation

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Medical schools spend, on average, $0.53 for each dollar of sponsored research received

Institutional investment in research has soared and AMCs are struggling to right-size their portfolios and re-design administrative functions

Cost levers can be evaluated to identify those investments that are most important to the research mission as well as what is affordable

Source: Academic Medicine; Investment in Medical Research, AAMC

Research

  • Equipment maintenance, service costs
  • Utility costs (and utility studies)
  • Direct vs. indirect costs and indirect cost recovery
  • Total professional effort (faculty)
  • Salary coverage, compensation models and incentive packages
  • Research staff salaries
  • Seed, bridge, and other internal funds
  • Funds flow (e.g., grants, inter-institutional)
  • Institutional sponsored research
  • Start-up packages
  • Research administration (pre- and post-award, compliance)
  • Space management (capacity, utilization)
  • Core facilities (e.g., biostatistics, vivarium) and degree of centralization
  • Academic, graduate support

Clinical Research

  • Clinical research administration (protocol development, setup, IRB approval,

patient recruitment/enrollment, CRO/vendor contracting, site selections, satellite locations, affiliates)

  • Core facility shared services (e.g., specimen storage)
  • IT systems and electronic data capture (EDC) (efficiency, integration)
  • Clinical trials billing, reporting (time & effort recognition)
  • Regulatory compliance
  • Clinical trial portfolio management
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AMCs have been facing challenges with core laboratories that support research—fast-paced technological advances require highly specialized infrastructure that is expensive

Illustrative List of Core Laboratories

Epigenetics Profiling Ultra High Throughput DNA Sequencing Expression Profiling Familial and Population Genetics Proteomics Metabolomics Bioinformatics / Computational Biology Transgenic & Knockout Animal Facility siRNA and Compound Screening Structural and Functional Imaging Microscopy Computational 3D Modeling

  • Core labs should be carefully

monitored for usage and capacity issues

  • Internal investment in core

laboratories should be aligned with strategic priorities

  • Aging or less used

technologies should be retired

  • Chargebacks for cores should

be optimized

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Integration across the manufacturing value chain will generate self- supporting revenue and provide a single platform for clinical and research laboratories

Reagent and Media Production Opportunities The Federal Drug Administration requires all reagents utilized in isolating and creating stem cells to be GMP certified reagents, which creates a market for two product lines including GMP and non-GMP reagents and media Equipment Manufacturing Opportunities There is an opportunity in the field to develop proprietary equipment to isolate sub- populations of stem cells, drive more efficient future production, and ensure GMP- compliant manufacturing standards are met Cell Therapy Product Opportunities As clinical applications of regenerative medicine continue to increase due to regulatory and scientific advances, a vertically integrated stem cell manufacturing company has the

  • pportunity to manufacture

clinically applicable stem cells and to partner with academic medical centers, outpatient clinics, inpatient hospitals, and potentially pharmaceutical companies Bio-Banking Opportunities Tissues for stem cell production can be stored in biobanks for future, long-term use. Specific types of tissues can generate thousands of doses of stem cell treatments, making the proper storage of tissues a critical intermediate process

Manufacturing and Banking

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  • Cell characterization

and testing

The opportunity exists today to own the entire manufacturing life cycle within regenerative medicine – from patient extraction to clinical delivery

  • Patient specific manufacturing avoids immune rejection challenges and does

not require costly immunosuppression

  • Reduced start-up costs for large-scale manufacturing given potential for on-site

isolation, expansion, and delivery

  • Potentially simpler regulatory environment

Challenges

  • Minimal economies of scale and lower patient throughput
  • Patient biopsy required for each treatment
  • Producing cells for many patients can provide scale-up or scale-out opportunities
  • QC can be applied to large lot sizes
  • Off the shelf availability can address emergency incidences
  • Patient biopsy not required for each treatment

Challenges

  • Immune rejection and risk of cell abnormalities create regulatory and clinical

challenges

  • Requires major manufacturing investment or partnerships up front

Stages Activities Stem Cell Harvest Transportation Sample Processing and Isolation Expansion and Storage QA Testing Transportation

  • Patient biopsy
  • Sample labeling
  • Cryopreservation and

hypothermic sample capabilities

  • Sample processing /

prep (centrifuging, filtration, etc..)

  • Pre-treatment

validation / QC

  • Pre-treatment data

entry

  • Cell culture and

expansion for autologous an allogeneic use

  • Cryopreservation of

stem cells Requirements

  • Operating room or
  • utpatient center access

for harvesting cells

  • Logistics fleet /

contracts

  • Cryopreservation and

hypothermic sample storage capabilities

  • Accessioning
  • cGMP facilities
  • cGMP laboratory and

SOP for cell expansion

  • Biorepository
  • cGMP quality

standards and testing capabilities Therapy Delivery

  • Cell washing, thawing

(or cryo-recovery), characterizing prior to delivery

  • Patient delivery
  • Patient monitoring
  • Operating room or
  • utpatient center access

for clinical delivery

  • Standardized protocol for

procedures Scalable in Allogeneic Therapies

Allogeneic and Autologous Therapies

Allogeneic Advantages Autologous Advantages

Potential institutional point-of- care manufacturing in autologous therapies

  • Transportation to

clinical delivery site

  • Cryopreservation and

hypothermic sample storage capabilities

  • Vial / sample

stabilization Manufacturing to Scale-up or Scale-out

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Manufacturing and Banking

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An increasingly important factor in research are big data analytics, AI and machine learning,

  • ffering potential increased efficiencies and

effectiveness 02 Translational research

  • Apply AI techniques on R&D activities to

transform basic science outcomes into new and innovative approaches for prevention diagnosis, and treatment of disease

  • Analyze and identify patterns in large and

complex datasets faster and more precisely

  • Search scientific literature for relevant

studies, and to combine different kinds of data

04 Genomics - gene editing and

sequencing

  • Use machine learning to identify patterns

within high volume genetic data sets

  • Translate these patterns to computer

models which can predict an individual’s probability of developing certain diseases

  • r help inform the design of potential

therapies

R&D Efficiencies & Effectiveness

Clinical trials design and recruitment

  • Apply scientific research techniques to

discover targets and viable biological pathways for new disease therapies

  • Analyze patient data to identify

biomarkers / validate surrogates for disease predisposition, sub-types, progression and therapy effectiveness

  • Match suitable patients to clinical studies

and design investigator-led trials that serve needs of a specific patient sub- population

03

Drug design and discovery

  • Analyze scientific knowledge and data to

reveal known and hidden connections that can help increase the likelihood of scientific breakthroughs

  • Leverage AI techniques for new drug

target identification and drug repurposing

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So institutions are exploring ways to consolidate costs and leverage technology to alleviate the burden

  • f time consuming administrative activities

Technology Enablement Research Administration Principal Investigator Research Compliance Non-compliant transaction reporting Respond to flagged transactions

Shared Services: Developing a standard for administrative activities around a common technology will facilitate the reduction of non- value add tasks across the research function. Example benefits of Shared Services:  Reduction of administrative burden for principal investigators; enabling them to ‘focus on the science’  Scaling of support staff to accommodate portfolio growth without increasing headcount  Creation of trusted analytics and reporting; reducing the need to reconcile systems  Identification of cost reduction opportunities

  • ver time

Core Laboratories: Aligning core labs to support priority research areas can help contain costs and increase capacity. Some core labs, such as next-generation sequencing and CLIA facilities, blur the line between research and clinical care and thus have potential to generate revenue.