Steven Dubinett Michael Palazzolo Steven George June Lee Vish Krishnan

1. University of California Center for Accelerated Innovation Steven Dubinett Michael Palazzolo Steven George June Lee Vish Krishnan National CAI Kick-Off MeetingOctober 29, 2013

2. Contents • Consortium Membership & Expertise • Leadership and Governance Structure • Center Resources • Technology Solicitation to Exit • Skills Development Overview • Defining Success: Metrics & Deliverables

3. UC Biomedical Research Acceleration Integration and Development (UC-BRAID) UC-BRAID University of California, Los Angeles

4. UC Biomedical Research Acceleration Integration and Development (UC-BRAID) UCSF • IRB harmonization • Master contracting • Integrated Research Data Repository • Harmonizing biobanking • 12 million patients UC Davis UCLA Investigator UC San Diego UC Irvine

5. UC Biomedical Research Acceleration Integration and Development (UC-BRAID) • UC CAI • UC ReX • Enables search of 12 million de-identified patient records from the 5 UC medical centers • Counts of eligible patients by gender, race, ethnicity • EngageUC • Global consent and biobanking • $2-million in supplemental funding from NCATS

6. Expertise • Rich research base: 7% of NHLBI’s FY2012 grant funding • Proximity to large biomedical industry clusters in San Diego, Irvine/Orange County and San Francisco • More than 70 industry experts in: • heart, lung and blood diseases • technology platforms • commercialization

7. Expertise • History of innovation: • breath biomarkers for asthma (D. Cooper) • a hemodynamic system for transfusion of blood products and administration of blood pressure medications (J. Rinehart) • hydrogel for cardiac tissue repair (K. Christman) • pulmonary vein ablation catheter for atrial fibrillation (M. Lesh) • biologic for idiopathic pulmonary fibrosis (D. Sheppard) • biopolymer implant for ventricular reconstruction in congestive heart failure patients (R. Lee)

8. Introduction to Leadership Michael Palazzolo, MD, PhD - Center Director • Director of Human Genome Center, Berkeley Lab • Associate Director, Drosophila Genome Project, Berkeley Lab • Senior Director of Biosystems, Amgen • led 270 scientists conducting high-throughput genomics research • Partner at Coastview Capital, a Los Angeles-based venture capital firm • Project manager • international Stand Up to Cancer collaboration • international, multiyear collaboration between academic laboratories at U Toronto (Mak) and UCLA (Slamon)

9. Contents • Consortium Membership & Expertise • Leadership and Governance Structure • Center Resources • Processes: Technology Solicitation to Exit • Skills Development Overview • Defining Success: Metrics & Deliverables

10. Local and National Announcements CTSA Central NBC Channel 4 News Posted on Sept 30, 2013 Aired October 1, 2013

11. Governance External Selection Committee Executive Committee External Advisory Board Business Review Panel Center Director Skills Development Program Associate Director Domain Areas Domain/Site Leaders Therapeutics Diagnostics Devices Projects Cardiovascular Lung & Sleep Disorders Blood Diseases Program Resources Industry Relations & IP CTSA Infrastructure Website & Data Management Administrative& Budgetary Support Project Management Evaluation & Tracking

12. Site Leaders Campuses JuneLee,MDUCSF ShaunCoughlin,MD,PhDUCSF SotiriosTsimikas,MDUCSD JosephWitztum,MDUCSD LauraMarcu,PhDUC Davis Steven George,MD,PhDUC Irvine TomasGanz,MD,PhDUCLA

13. Domain Leaders & Skills Development Program Shaun Coughlin,MD,PhDCardiovascular Disease SotiriosTsimikas,MDCardiovascular Disease June Lee,MDLung and Sleep Diseases Tomas Ganz,MD,PhDBlood Disorders and Resources Diseases Vish KrishnanSkills Development Program UCSD

14. Domain Leaders Platforms June Lee,MDCo-Leader, Therapeutics Shaun Coughlin,MD,PhDCo-Leader, Therapeutics SotiriosTsimikas,MDCo-Leader, Diagnostics Joseph Witztum,MDCo-Leader, Diagnostics Laura Marcu,PhDCo-Leader, Devices and Tools StevenGeorge,MD,PhDCo-Leader, Devices and Tools

15. External Advisory Board • Consists of no fewer than 5 members • Experienced business leaders • Includes NHLBI Program Officer • Advice about operations, project development Catherine Mackey, PhDFormer Senior VP, PfizerFounder, MindPiecePartners Francis Duhay, MDVP Medical Affairs and CMD, Edwards Lifesciences Lawrence Souza, PhDFormer Senior VP, AmgenFounder, Coastview Capital,

16. Business Review Panel • Five members • VCR on each campus appoints one member • Evaluate Center’s progress toward sustainability Bill Ouchi, PhDUCLA Initial Chair Anderson School

17. Progress to Date • Campus meetings • Face-to-face meetings • Site and domain leadership meetings • Website development • Administrative meetings • 100-day Implementation Plan

18. Progress to Date

19. Progress to Date

20. Progress to Date http://uccai.ctsi.ucla.edu

21. Contents • Consortium Membership & Expertise • Leadership and Governance Structure • Center Resources • Processes: Technology Solicitation to Exit • Skills Development Overview • Defining Success: Metrics & Deliverables

22. Center Resources by Development Stage • Discovery • Cardiovascular Research Institute (UCSF) • Lung Biology Center (UCSF) • Biomarker Laboratory (UCSD) • Drug Discovery Institute (UCSD) • Institute for Engineering in Medicine (UCSD) • Small Molecule Discovery Center (UCSF) • Pre-clinical and Clinical • Cardiovascular Physiology Core (UCSD) • National Primate research Center (UCD) • Large Animal Survival Science Service Facility (UCD) • Airway Clinical Research Center (UCSF) • Animal Care Program Diagnostic Laboratory Services (UCSD) • Applied Physiology-Human Performance Lab (UCI) • UC Medical System

23. Center Resources by Platform • Diagnostics • Translational Pathology Core Laboratory (UCLA) • Tissue Array Core Facility (UCLA) • West Coast Central Comprehensive Metabolomics Resource Core (UCD) • Crump Institute for Molecular Imaging (UCLA)’ • Therapeutics • GMP Facility (UCD) • Medicinal Peptide Synthesis Core (UCLA) • Center for Molecular and Genomic Imaging (UCD) • Metabolomics Central Service Core (UCD) • Molecular Screening Shared Resource (UCLA) • Devices • Edwards Lifesciences Center for Advanced Cardiovascular Technology (UCI)

24. Center Resources for Commercialization • Entrepreneurial • von Liebig Center for Entrepreneurism and Technology Advancement (UCSD) • Business of Science Center (UCLA) • Harold and Pauline Price Center for Entrepreneurial Studies (UCLA) • West Health • CONNECT • BIOCOM • Incubators • QB3 (UCSF) • Institute for Technology Advancement (UCLA) • Center for Innovative Therapeutics (UCSD) • Industry Partners • Bristol-Myers Squibb • Care Fusion • Edwards Lifesciences • Life Technologies • MedImmune Ventures • Pfizer Centers for Therapeutic Innovation • Quest Diagnostics

25. Edwards LifesciencesCenterResearch Vision • 6 core (22 affliated) faculty in 13,000 asf • 40 doctoral students and postdoctoral researchers • Research focus areas: • Valve replacement technology • Regenerative cardiovascular medicine • Non-invasive (wireless) cardiovascular monitoring • Novel stent or catheter-based therapies Heart valve fluid dynamics Perfused human microtissues (Professor Kheradvar) (Professors George, Lee, and Hughes)

26. Edwards Lifesciences CenterTraining Vision • Train future translational cardiovascular researchers at all levels (undergraduate, graduate, post-doctoral) • Training fellowships from endowment • Cardiology Fellow (3-yr dedicated research time) • NIH T32 training grant (CAREProgram) • Business plan competition • 6 funded slots (3 doctoral positions per year) • E-SURP (paid summer internships – UCI ugrads) • Summer Scientists (high school students)

27. Contents • Consortium Membership & Expertise • Leadership and Governance Structure • Center Resources • Processes: Technology Solicitation to Exit • Skills Development Overview • Defining Success: Metrics & Deliverables

28. Technology Selection Overview • Solicit 2-page pre-applications • Each campus reviews and selects the best proposals for full application • 1st Review: Highest ranked proposals from each campus submitted for review by panels assembled by Domain Leaders • 2rd Review (External Review): External Selection Committee scores proposals and sends to NHLBI • 3rd Review: NHLBI makes final selection

29. Technology Selection Overview RFP Pre-application Pre-applicationReview FullApplication UC BRAID Review Committee External Selection Committee Review Technologies Selected for Entrance to Center NHLBI Review

30. Technology Selection Timeline • Annual solicitation for 3 tracks • Therapeutics • Devices/ digital health • Diagnostics • Timeline from initial solicitation to External Selection Committee recommendation: 6 months • Awards of up to $200K/2yrs Review Pre-Application Solicit Pre-Application Develop and Submit Full Application First Review ofFull Application ESC Review of Application 2months 1 month 1 month 1 month 1 month

31. Pre-application and Review RFP Pre-application Pre-applicationReview FullApplication UC BRAID Review Committee External Selection Committee Review Technologies Selected for Entrance to Center NHLBI Review

32. Eligibility • Faculty in all series and ranks at UC Davis, UC Irvine, UCLA, UC San Diego, and UC San Francisco • Postdoctoral scholars are eligible to submit applications as Co-PI with a faculty PI • Projects with existing or imminent target validation and a clear clinical indication • Patents or patent applications are filed or potential for obtaining defensible intellectual property is strong

33. Solicitation Process • Broad solicitation • Focus on NHLBI priority areas (heart, lung, blood) • Centralized RFP for all 5 campuses • Webinar on submission process • Each campus is accountable for supporting the highest potential projects with product development related issues

34. Two-page Pre-application • Centralized online submission • Two-page pre-application contains: • Executive summary • Unmet Need/ Clinical impact • Research/development/ regulatory plan • Intellectual Property • Business strategy/Commercialization plan • Local review of two page pre-applications assess: • Scientific merit • Product development potential • Highest potential projects will be requested to submit full proposals

35. Technology Selection: Full Application RFP Pre-application Pre-applicationReview FullApplication UC BRAID Review Committee External Selection Committee Review Technologies Selected for Entrance to Center NHLBI Review

36. UC BRAID Review Committee • Selected by Domain Experts/Platform Leads • UC BRAID Executive Committee • Domain Leads, Site Leads, Platform Leads • External experts from industry

37. Full Application • Review Criteria • Unmet medical need • Development feasibility • Commercial attractiveness • Intellectual property status • Relevance to NHLBI mission • Metrics for success • Evidence of target validation (therapeutic) • Time and cost of prototyping (device) • Combination of the above (diagnostic)

38. Full Application: External Selection Committee • Prioritize applications received from Leadership Review • Same review criteria as Leadership Review • 1 month to review • Reviewers • Targeting total pool of 100 • Selected to review proposals based on domain/functional expertise • Must be external to institution(s) • Chair/Co-Chair to finalize recommendations to be submitted to NHLBI

39. Technology Development Pipeline RFP Pre-application ConsultationAward Pre-applicationReview FullApplication Referral UC BRAID Review Committee Referral External Selection Committee Review Technologies Selected for Entrance to Center NHLBI Review

40. Consultation Awards • Eligibility • Proposal not selected for Center but identified as high potential • Leadership Review or External Review recommends Consultation Award consideration • Amount and duration of awards vary • Most awards for 3-6 months • Recipients must agree to resubmit and target a specific RFP for resubmission

41. Consultation Awards • Awards will address the following gaps: • In vivo proof of principle • Hypothesis testing • IP assessment • Target product profile discussion • Regulatory assessment • Further development planning • Based on results of work done during award, awardee may be invited to submit full proposal for subsequent cycle

42. Contents • Consortium Membership & Expertise • Leadership and Governance Structure • Center Resources • Processes: Technology Solicitation to Exit • Skills Development Overview • Defining Success: Metrics & Deliverables

43. Key Goals and Objectives Offer actionable cross-campus skills development opportunities that: • Encourage PI’s to consider commercialization considerations when writing grant proposals • Impart core project management and business planning skills for life sciences projects. • Improve awareness of market, financial, and IP issues in the early stages of a project. • Guide researchers on the assessment of technologies on their readiness and risks • Mitigate various types of risks with appropriate project design and partnership business models.

44. Skills Development CanvasEngage “Proto-innovators” Segments Funded Prospective Post-Docs Fellows Grad Faculty Faculty Students Commercialization Content Portal (Readings, Videos, Cases)Advisory Services/ClinicsCourses/Workshops on CommercializationNetworking Events and Contests Activities

45. Education and Training • Technology Commercialization Primer • Gateway course for Center innovators • Teach innovators how to frame proposals for Center • Covers technology readiness, market research, risk mitigation, competitive analysis, company start up • Builds on existing courses • Lab to Market (UCSF), Idea to IPO (UCSD) • Catalog commercialization resources across Center • Business plan development • SBIR grant writing • IP, licensing, contract negotiation • Existing courses on campuses • Match innovators with educational and networking events • Annual Heart, Lung, Blood Technology Forum • Innovators present progress and lessons learned • Networking with industry participants

46. Mentoring and Advising • Highly experienced industry professionals, venture investors, entrepreneurs • “Mentor the Mentor” training • Modeled on UCD Mentoring Academy • Recognition for superior mentoring • Three levels of mentoring: • Generalist -- Help innovators prepare competitive proposals • Lead -- Guide innovators selected for the Center • Specialist -- Available to address specialized problems during technology development • Online Commercialization Clinics • Led by mentors • Held quarterly • Archived on Center website

47. Feedback • Compile data on technology pipeline and licensing activity • Provide feedback to Center on investment needed to enhance attractiveness of pipeline Timing • Develop and launch Technology Commercialization Primer in year 1 • Mentoring programs and technology commercialization clinics in years 1-2

48. Contents • Consortium Membership & Expertise • Leadership and Governance Structure • Center Resources • Processes: Technology Solicitation to Exit • Skills Development Overview • Defining Success: Metrics & Deliverables

49. Goals • Build an entrepreneurial ecosystem in the NHLBI domain – skills development • Identify the best technologies across all 5 UC campuses • Incubate 30-40 of the most promising technologies within the center towards a commercially rewarding exit • Sustainability

50. Why Did We Institute a Business Review Panel? • Each campus has the responsibility to manage its own exits • We agreed there isn’t a dataset that exists that would allow the selection of an optimal exit strategy for all five campuses • Best approach would be to run an experiment and use the data to guide us in the future • The data would best be evaluated by experts outside the Center reporting to the EC and not the Center