Steven Dubinett Michael Palazzolo Steven George June Lee Vish Krishnan

STEVEN DUBINETTMICHAEL PALAZZOLOSTEVEN GEORGE

JUNE LEEVISH KRISHNAN

University of California Center for Accelerated Innovation

National CAI Kick-Off MeetingOctober 29, 2013

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

University of California,

Los Angeles

UC-BRAID

UC Biomedical Research Acceleration Integration and Development (UC-BRAID)


Investigator

• IRB harmonization• Master contracting

• Integrated Research Data Repository

• Harmonizing biobanking

• 12 million patients

UCSF

UC Davis

UCLA

UC San Diego

UC Irvine

• 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


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

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)

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)

Introduction to Leadership

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

Local and National Announcements

Posted on Sept 30, 2013 Aired October 1, 2013CTSA Central NBC Channel 4

News

Governance

Center Director

Associate Director

Domain/Site Leaders

Cardiovascular

Therapeutics Devices

Lung & Sleep Disorders

BloodDiseases

Diagnostics

Domain Areas

Projects

Program Resources

Administrative& Budgetary

Support

Website & Data Management

Industry Relations & IP

CTSA Infrastructure

Evaluation & Tracking

Project Management

External Selection

Committee

Business Review Panel

Skills Development Program

External Advisory Board

Executive Committee

Site LeadersCampuses

June Lee, MDUCSF

Shaun Coughlin, MD,

PhDUCSF

Sotirios Tsimikas,

MDUCSD

Joseph Witztum,

MDUCSD

Laura Marcu, PhDUC Davis

Steven George, MD,

PhDUC Irvine

Tomas Ganz, MD, PhDUCLA

Domain Leaders & Skills Development Program

Shaun Coughlin, MD,

PhDCardiovascular Disease

Sotirios Tsimikas,

MDCardiovascular Disease

June Lee, MDLung and Sleep

Diseases

Tomas Ganz, MD, PhDBlood Disorders and Resources

Vish KrishnanSkills Development

ProgramUCSD

Dis

ease

s

Domain LeadersPlatforms

June Lee, MDCo-Leader,

Therapeutics

Shaun Coughlin, MD,

PhDCo-Leader,

Therapeutics

Sotirios Tsimikas, MDCo-Leader, Diagnostics

Joseph Witztum, MDCo-Leader, Diagnostics

Laura Marcu, PhDCo-Leader,

Devices and Tools

Steven George, MD,

PhDCo-Leader,

Devices and Tools

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, Pfizer

Founder, MindPiece Partners

Francis Duhay, MDVP Medical Affairs and CMD,

Edwards Lifesciences

Lawrence Souza, PhDFormer Senior VP, Amgen

Founder, Coastview Capital,

Business Review Panel• Five members

• VCR on each campus appoints one member

• Evaluate Center’s progress toward sustainability

Bill Ouchi, PhDUCLA Initial ChairAnderson School

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

Progress to Date

Progress to Date

http://uccai.ctsi.ucla.eduProgress to Date

http://uccai.ctsi.ucla.edu/


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

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)

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

Edwards Lifesciences CenterResearch Vision

Heart valve fluid dynamics Perfused human microtissues

(Professor Kheradvar) (Professors George, Lee, and Hughes)

• 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

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 (CARE Program)

• Business plan competition• 6 funded slots (3 doctoral positions per year)

• E-SURP (paid summer internships – UCI ugrads)• Summer Scientists (high school students)

Contents• Consortium Membership & Expertise• Leadership and Governance Structure• Center Resources• Processes: Technology Solicitation to

Exit• Skills Development Overview• Defining Success: Metrics & Deliverables

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

Technology Selection Overview

Pre-applicationReview

RFP Pre-application

FullApplication

External Selection Committee Review

NHLBI Review

UC BRAID Review Committee

Technologies Selected for

Entrance to Center

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/2yrsSolicit

Pre-Application

Review Pre-

Application

1 month

Develop and Submit Full Application

First Review of

Full Application

ESC Review of

Application

1 month 1 month 1 month2 months

Pre-application and Review


RFP Pre-application

FullApplication


NHLBI Review



Entrance to Center

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

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

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

Technology Selection: Full Application


RFP Pre-application

FullApplication


NHLBI Review



Entrance to Center

UC BRAID Review Committee• Selected by Domain Experts/

Platform Leads• UC BRAID Executive Committee• Domain Leads, Site Leads, Platform

Leads• External experts from industry

Full Application• Review Criteria

– Unmet medical need– Development feasibility– Commercial attractiveness– Intellectual property status– Relevance to NHLBI mission– Metrics for success

oEvidence of target validation (therapeutic)oTime and cost of prototyping (device)oCombination of the above (diagnostic)

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

Technology Development Pipeline


RFP Pre-application

FullApplication


NHLBI Review



Entrance to Center

Referral

Referral

Consultation Award

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

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


Key Goals and ObjectivesOffer 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.

Skills Development CanvasEngage “Proto-innovators”

Activ

ities

Funded Prospective Post-Docs Fellows Grad Faculty Faculty Students

Commercialization Content Portal (Readings, Videos, Cases)

Advisory Services/Clinics

Courses/Workshops on Commercialization

Networking Events and Contests

Segments

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

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

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

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

Deliverables

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

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

Why Is Sustainability Hard?

University Research

Tech Transfer

Venture Capital Biotech Pharma

The technological bottleneck is the mechanistic understanding of a disease that can be used to validate therapeutic intervention points. This happens largely in the university.

However, the value transition point occurs at proof-of-concept in clinical trials in phase I or phase II. This currently takes place in Biotech and Pharma.

Sustainability May become Even Harder

• “However, although investment in pharmaceutical research and development has increased substantially...the lack of a corresponding increase in terms of new drugs begin approved indicates that therapeutic innovation has become more challenging.”– Nature Reviews. Drug Discovery 10, No. 6, pp.428-

438.

• “venture financing for biotech has been in decline...Some venture capitalists have stopped funding new biotech altogether.”– Wall Street Journal, March 16, 2012

Bill Ouchi’s Views on Problems That Need to Be Solved to Achieve Sustainability

• Tech transfer offices are undercapitalized• Tech transfer offices tend to be highly

politicized• Tech transfer offices frequently have insufficient

business expertise and management• Faculty need mentoring• Universities may need to step up to incubate

technology to the point of proof of concept on its most promising programs

Documents

Steven Dubinett Michael Palazzolo Steven George June Lee Vish Krishnan