Phacilitate Cell & Gene Therapy Main Announcement

FORUM 2015

26-28 January, Grand Hyatt Washington, DC

Co-located with

11th Annual

FORUM 201526-28 January 2015, Grand Hyatt Washington, DC

Learn from – and meet – an unparalleled assembly of the global Cell & GeneTherapy community’s premier thought-leaders and decision-makers, including:

www.bioleaders-forum.com

Book by 14Nov for $200discount!

…see inside for the fullpanel and agenda!

Perry Karsen, Chief ExecutiveOfficer, Celgene CellularTherapies

Dr Kenneth LeClair,Executive Director,Novartis Cell &Gene Therapy Unit

Dr James Trager,Vice President,Research & ProductDevelopment,Dendreon

Dr Mahendra Rao,Consultant, NYSCF & VicePresident StrategicAffairs, Q Therapeutics

Jeb Keiper, Vice President,Oncology BusinessDevelopment,GlaxoSmithKlineWorldwide BusinessDevelopment

Albert Seymour,Global Head ofResearch andNonclinicalDevelopment, ShirePharmaceuticals

Dr C. Randal Mills.President & ChiefExecutive Officer,California Institutefor RegenerativeMedicine (CIRM)

Dr Keith Wonnacott,Chief of CellularTherapies Unit,OCTGT, CBER, US Food and DrugAdministration

Dr Devyn Smith, Head of Strategy,PharmatherapeuticsResearch &Development,Pfizer

John McDonald,Vice President,BusinessDevelopment,Biogen Idec

Dr Hansjoerg Duerr,Global HeadHaematology, Bayer Healthcare

Gold Sponsors: Silver Sponsors:

Bronze Sponsors: Roundtable Sponsor: Main Endorsing organisations:

The world’s biggest and fastest growing cell & gene therapybusiness meeting

The first and still theleading event specificallydriving productcommercialization

Unparalleled learning andnetworking opportunitieswith the decision-makerswho matter

14886 C & GT brochure V2_Layout 1 15/10/2014 12:44

The world’s biggest andfastest growing cell & genetherapy business meeting

n Overall attendance expectedbuilding on last year’s record – 550+

n Biggest ever panel – 150+ speakers

n Profile of attendees – 76% atDirector level and above

The first and still the leadingevent specifically drivingproduct commercialization

n Unmatched big pharmaparticipation – the only event thatall of the major pharma and bigbiotech players attend

n Investors, regulators, KOLs andother key stakeholders

n A who’s who of the world’s leadingCell & Gene Therapy biotechs

Number 1 for thoughtleadership, lead generationand brand promotion

n Expanded agenda, newinteractive conference formats(roundtables, drop in clinics, techroom demonstrations, etc)

n New Biotech Showcase

n New partnering tool plus provisionof 1-2-1 meeting area within theexpo

Key organizations already confirmed for the Cell & Gene Therapy Forum 2015:

The single best cell and gene therapy conference David Ichikawa, Vice President, Business Development, Sangamo BioSciences, Inc

FORUM 2015


Amgen, AstraZeneca/MedImmune, BayerHealthCare, Biogen Idec, Bristol-MyersSquibb, Celgene, EMD Serono,GlaxoSmithKline, Johnson &Johnson/Janssen R&D, Merck, Novartis,Pfizer, Roche/Genentech, Sanofi, Shire

California Institute for RegenerativeMedicine (CIRM), Centre forCommercialisation of RegenerativeMedicine (CCRM), City of Hope, EUCommittee for Advanced Therapeutics(CAT), Harvard Stem Cell Institute, LandmarkCapital, Leerink Partners, Memorial SloanKettering Cancer Centre, NIH, OregonHealth Science University Hospital, Paul-Ehrlich Institute, The University of Texas MDAnderson Cancer Center, University ofPennsylvania, US FDA

Adaptimmune, Argos Therapeutics, AsteriasBiotherapeutics, Athersys, bluebird bio,Capricor, Cell Medica, CelladonCorporation, CRISPR Therapeutics,Dendreon, Mesoblast, NorthwestBiotherapeutics, Organogenesis, OxfordBiomedica, Pluristem Therapeutics,Promethera Biosciences, Q Therapeutics,Replicel, Sangamo BioSciences, Tigenix,Tocagen, TxCell, UniQure

Why Cell & Gene Therapy Forum 2015 is the only eventyou absolutely must attend next year:

Brand new, highly sophisticated and effective 1-2-1 Partnering Tool

Investors, regulators, KOLs: Pharma/Big Biotech: Leading biotechs:

2015 sees the launch of our brand new app – meaning thatyou can start benefiting from your attendance at thePhacilitate Cell & Gene Therapy Forum before the eventeven begins!

n Book meetings with fellow attendees

n Find new contacts based on your profile and interests

n Build your conference agenda

n Download exclusive content

n Build your own library to access content easily before, duringand after the event

n Take part in industry polls

14886 C & GT brochure_Layout 1 13/10/2014 13:28

The Phacilitate meeting is one of those rare meetings where participants are very open in theircommunication and at the same time have their focus on business. It is rare in that over 80% ofconversations were relevant to our business objectives. Definitely coming back in 2015!Kurt Gielen, Acquisition & Business Development Manager, Chemelot Campus B.V.

The speaker panel for the Phacilitate Cell & Gene Therapy Forum 2015 is unparalleled in terms of its seniority and influence,delivering all the strategic insight and contacts you need to drive your own business model forward.

We will continue to add speakers to the panel over the coming weeks. You can stay abreast of the latest developments atany point by visiting the event website http://www.bioleaders-forum.com

Perry Karsen, Chief Executive Officer, Celgene Cellular Therapies

Dr Kenneth LeClair, Executive Director, Novartis Cell & Gene Therapy Unit

Dr James Trager, Vice President, Research & ProductDevelopment, Dendreon

Dr C. Randal Mills., President & Chief Executive Officer, California Institute for RegenerativeMedicine (CIRM)

Jan Thirkettle, Head of Advanced TherapyDelivery, GlaxoSmithKline

Dr Mahendra Rao, Consultant, NYSCF & Vice PresidentStrategic Affairs,Q Therapeutics

Dr Keith Wonnacott, Chief of Cellular Therapies Unit,Office of Cellular Tissue and GeneTherapies (OCTGT), US Food and Drug Administration(FDA)

Jeb Keiper, Vice President, Oncology BusinessDevelopment, GlaxoSmithKline WorldwideBusiness Development

Albert Seymour, Global Head of Research andNonclinical Development, Shire Pharmaceuticals

Dr Martina Schusler-Lenz, Vice-Chair, Committee forAdvanced Therapies (CAT) EMA &Clinical Assessor, Division ofMedical Biotechnology, Paul-Ehrlich Institute

Dr Devyn Smith,Head of Strategy,Pharmatherapeutics Research &Development,Pfizer

John McDonald, Vice President, BusinessDevelopment, Biogen Idec

Dr Jim Faulkner, Vice President, CMC & Supply, GlaxoSmithKline Rare Diseases

Dr Knut Niss,Senior Technical Project Leader, Novartis

Dr Hansjoerg Duerr, Global Head Haematology,Bayer Healthcare

Dr Jane Lebkowski, Vice President Research &Development, Asterias Biotherapeutics

James Noble, Chief Executive Officer,Adaptimmune

Dr Donna Skerrett, Chief Medical Officer,Mesoblast

Edward Lanphier, President & Chief Executive Officer, Sangamo BioSciences Inc

Dr Greg Russotti, Vice President TechnicalOperations, Celgene Cellular Therapeutics

Dr John Harrington,Executive Vice President & ChiefScientific Officer, Athersys Inc

Frederick Miesowicz, PhD, Chief Operating Officer & VicePresident Manufacturing, Argos Therapeutics Inc

Mitchell H.Finer, Chief Scientific Officer, Bluebird Bio

Dr Sven Kili, Senior Director, Global MedicalAffairs, Biosurgery, Sanofi Biosurgery

Boris Peaker,Managing Director, BiotechnologyStocks,Cowen and Company

Howard Liang, PhD,Managing Director BiotechnologyEquity Research,Leerink Partners LLC

Dr Hans Preusting, Chief Business Officer, UniQure

Dr Nessan Bermingham,Venture Partner, Atlas Ventures,Founder and Chief ExecutiveOfficer,Intellia Therapeutics

Prof John Rasko, Director, Department of Cell andMolecular Therapies,Royal Prince Albert Hospital

Dr Richard Maziarz, Researcher, Oregon Health Science UniversityHospital

Neil PalmerPresident and Principal Consultant,PDCI Market Access

Brock Reeve, Executive Director, Harvard Stem Cell Institute

Dr Ian Harris, Senior Director, Cell Therapy,Janssen Research & DevelopmentLLC

Dr James Wilson, Professor of Internal Medicine &Pathology and LaboratoryMedicine, Director of the GeneProgram, University ofPennsylvania, Research Director,Center for Orphan DiseaseResearch and Therapy

Katherine Tsokas, JD, Senior Director, Global RegulatoryAffairs, Janssen Research &Development, LLC

Dr Samuel Barone, Chief Medical Officer, Avalanche Biotechnologies

Dr Wilfred Dalemans, Chief Technical Officer, Tigenix

Dr Theresa Chen,Pharmacology/ToxicologyReviewer, Office of Cellular Tissueand Gene Therapies (OCTGT),Center for Biologics Evaluation andResearch (CBER), US Food and Drug Administration(FDA)

Dr Denise Gavin, Team Lead, Gene Therapy Branch,Office of Cellular Tissue and GeneTherapies (OCTGT), US Food and Drug Administration(FDA)

Dr Isabelle Riviére, Director Cell Therapy & CellEngineering Facility, Memorial Sloan Kettering CancerCentre

Donald Powers, Principal Scientist, Janssen Cell Therapy

Dr Anton Simeonov,Acting Deputy Scientific Director,National Centre for AdvancingTranslational Sciences (NCATS)

Neil Littman,Business Development Officer,California Institute for RegenerativeMedicine (CIRM)

Dr Ricardo Macarron,Vice President of Target andPathway Validation,GlaxoSmithKline

Dr Huseyin Mehmet,Vice President, Head of Cell &Molecular Biology,Proteostasis Therapeutics Inc

Colonel Daniel Wattendorf,Program Manager, DefenceScience Office,DARPA

Dr Carl G. Simon, Jr, Biologist and Project Leader of 3DTissue Scaffolds,National Institute of Standards andTechnology (NIST)

Dr Michele Cleary,Executive Director, Target andPathway Biology,Merck

Dr Artur Isaev, Chief Executive Officer,Human Stem Cells Institute

Professor Asterios Tsiftsoglou,AUTH Lab of Pharmacology,School of Pharmaceutical Sciences& EU CAT Member

Dr Eileen Dolan, Professor of Medicine, Chair,Committee on ClinicalPharmacology andPharmacogenomics, University ofChicago, Associate Director forEducation,Comprehensive Cancer Center

Carter Cliff,Business Development,Cellular Dynamics International Inc

Nick Timmons, Director, Product and ProcessDevelopment, Centre for Commercialisation ofRegenerative Medicine

Dr Traci Heath Mondoro, Chief, Translational Blood Sciencesand Resources, NHLBI

David Hall, Chief Executive Officer, Replicel

Dr Christopher Bravery, Principal Consultant, Advbiols

Dr Larry Couture, Senior Vice President and FoundingDirector, Centre for AppliedTechnology Development, Beckman Research Institute of theCity of Hope

Dr Thomas Vogt, Vice President Discovery & SystemsBiology, CHDI Management/ CHDIFoundation

Dr Kim Warren, Head of Custom DevelopmentServices for Cell Therapy, Lonza Walkersville Inc

Richard Grant, Director of Cell Therapy, Invetech Pty Ltd

Dr Rodger Novak, Chief Executive Officer, CRISPR Therapeutics

Dr John Maslowski,Vice President Scientific Affairs,Fibrocell

Dave Backer, Senior Director & GeneralManager, SAFC Carlsbad

Faraz Ali, Vice President, CommercialPlanning & External Affairs, Bluebird Bio

William Podd, Founder, President & ChiefExecutive Officer, Landmark Capital/LandmarkAngels

Dr Ohad Karnielli, Vice President, Technology &Manufacturing, Pluristem Therapeutics Inc

Eric Halioua, Co-Founder & Chief ExecutiveOfficer, Promethera Biosciences

Dr Andrew Hamer, Vice President, Medical Affairs, Capricor

Dr Joyce Frey-Vasconcells,Regulatory Expert, Frey-Vasconcells Consulting, LLC

Peter Nolan,Senior Vice President, CommercialDevelopment,Oxford Biomedica

Dr Geoffrey Nichol, Executive Vice President, Research& Development, Sangamo BioSciences

Dr Zorina Pitkin, Vice President, Quality Systems, Organogenesis

Dr Aby Mathew, Senior Vice President & ScientificOfficer, Biolife Solutions Inc

Dr Manish Singh, Chairman & CEO, Lion Biotechnologies, Inc

Lee Buckler, Managing Director, Cell Therapy Group, a Division ofCTG Consulting, Co

Professor Miguel Forte, Senior Vice President, Clinical &Regulatory,TxCell

Dr Helen Tayton-Martin, COO, Adaptimmune

Simon Ellison, Senior Business DevelopmentManager, Cell Therapy Catapult

Dr Kai Pinkernell, Global Head of Clinical Business, Miltenyi Biotec GmbH

Dr Gabor Rubanyi, Chief Scientific Officer, Taxus Cardium PharmaceuticalsGroup

Anne-Virginie Eggimann, Vice President, Regulatory Science, bluebird bio, Inc

Ed Field,Treasurer, ARM

Dr Barbara Thorne, Senior Director, ProcessDevelopment, Celladon Corporation

Dr Philip D. Gregory, Senior Vice President, Research &Chief Scientific Officer, Sangamo BioSciences

Dr Douglas Jolly, Co-Founder & Executive VicePresident Research &Pharmaceutical Development, Tocagen

Kurt Gielen, Senior Business DevelopmentManager, BioMedical Materials,Brightlands Chemelot Campus

Dr Stefanos Theoharis,Chief Business Officer,Apceth GmbH & Co. KG

Dr Robert W. Mays, Head of Neuroscience, Athersys, Inc

Dr Eytan Abraham, Head of Cell Therapy Research &Technology, Lonza Walkersville Inc

Margarita Gutova, MD,Associate Research Professor,Department of Neurosciences,City of Hope National MedicalCenter & Beckman ResearchInstitute

Who’s speaking?


DAY 1 - Monday, January 26th 2015

Followed by your choice of 4 highly interactive breakout sessions:

Focus session 1 Focus session 2 Focus session 3 Focus session 4

9.00 Chair’s introductionDr John Maslowski, Vice President Scientific Affairs, Fibrocell

9.05 Top 10 events in cell & gene therapy of 2014 - celebrating recent progress in the fieldwhilst putting it into context• Revealing the results of the 2014 ‘top 10 events ‘ survey: A countdown of the year’smost significant events in cell therapy/regenerative medicine and in gene therapy,as nominated and voted for by the global C&GT community

• 2013 top 10 revisited – how have they advanced in 2014? Ed Field, Treasurer, ARMLee Buckler, Managing Director, Cell Therapy Group, a Division of CTG Consulting, Co

9.20 Delivering a snapshot of recently approved C&GT products and progresswith Phase III clinical candidates on a global basisLee Buckler, Managing Director, Cell Therapy Group, a Division of CTG Consulting, Co

9.30 Reviewing FDA Breakthrough Therapy Designations awarded to cell orgene therapy products in 2014Dr Joyce Frey-Vasconcells, Regulatory Expert, Frey-Vasconcells Consulting, LLC

9.40 What are the next steps for the cell & gene therapy world’s hottesttechnology areas and indications? • The ongoing emergence of CAR-T cells and cellular immunotherapy in general • Where next for the adult stem cell therapy field? What lies beyond MSCs?• Gene editing – the future of gene therapy?• Pluripotent stem cell based therapeutics: next steps for clinical development andcommercialization

Dr Philip D Gregory, Senior Vice President, Research & Chief Scientific Officer,Sangamo BioSciencesDr Jane Lebkowski, Vice President Research & Development, AsteriasBiotherapeuticsDr Kenneth LeClair, Executive Director, Novartis Cell & Gene Therapy Unit

10.10 Thought leader roundtable: What is holding cell & gene therapy back (if anything)?• Identifying, ranking and removing remaining major bottlenecks and obstacles toprogress (Money? Lack of clinical success? Scale-up and Cost of Goods issues?Potency after stability? Regulatory disharmony? Other?)

10.35 Your choice of highly interactive optional sessions,each for limited numbers of participantsWorkshop (for a maximum of 100 participants)Manufacturing and process development: Regulatory experts at hand • During this interactive session, a panel from regulatory agencies and industry willbe addressing the regulatory questions you are facing in your processdevelopment and

• GMP production.• Access to expert advice• One-to-one time with agency and industry experts • Discuss your specific topic of interestJiwen Zhang, PhD, Regulatory Affairs Director, GE HealthcareORRoundtable discussion(Very informal discussion-based session for maximum of 12participants)Global expansion into other regions. Top 5 challenges and how toaddress themKurt Gielen, Senior Business Development Manager, BioMedical Materials,Brightlands Chemelot Campus

FURTHER SESSIONS WILL BE ADDED OVER THE COMING WEEKS – CHECKBACK SOON FOR THE LATEST NEWS!

11.30 Morning coffee in the Exhibition/1-2-1 Meeting Area

The Top 10 ‘events’ can relate to anything - a financing round or a clinical trial; aparticular deal or a scientific breakthrough. The only criterion is that the ‘event’ hashad (or will have) a significant impact on the cell & gene therapy sector.

YOU will get the chance to nominate your most significant events of 2014, and to thenvote to help decide the final order of the Top 10. Look out for notification when theinitial nominations survey goes live online towards the end of the year.

Summarizing key trends affecting the C&GT industry over thepast 12 months - lessons learned in the past year

Morning plenaryCell & Gene Therapy ‘State of the Union’ - analyzing 2014’s big stories;setting strategic priorities for 2015 and beyond

7.30 Registration & buffet breakfast in the Exhibition/1-2-1 Meeting AreaOR

7.45 An Interactive Workshop Marken and Global Care Clinical Trials: Bringing the ClinicalTrial to the Patient

Presented by

Cell therapy manufacture 1: Arewe ready to deliver commerciallyfeasible cellular immunotherapyproducts at scale? (Shared with the Immunotherapy Forum)

12.05 US FDA perspective Dr Keith Wonnacott, Chief ofCellular Therapies Unit, Office ofCellular Tissue and GeneTherapies (OCTGT), US Food andDrug Administration (FDA)

12.20 Presentation reserved

12.40 Industry perspectiveDr James Trager, Vice President,Research & ProductDevelopment, Dendreon

1.00 Automate sooner than later:The need for flexible, fullyautomated processingplatformsDr Kai Pinkernell, Global Head ofClinical Business, Miltenyi BiotecGmbH

Examining the evolution andfuture role of automated celltherapy bioprocessingtechnologies• Addressing issues of cost andtiming (how/when to automate?)

Logistics 1: Placing logistics at theheart of the cell & gene therapybusiness model• Maximizing quality control, minimizing‘Cost of Logistics’ for cell and genetherapy products

12.05 Chair’s introductionDr Aby Mathew, Senior VicePresident & Scientific Officer,Biolife Solutions Inc

12.25 Setting the scene: Why ‘Costof Logistics’ is a greaterobstacle than Cost of Goodsto the viablecommercialization of celltherapy products Simon Ellison, Senior BusinessDevelopment Manager, CellTherapy Catapult

12.45 Industry perspectiveEric Halioua, Co-Founder & ChiefExecutive Officer, PrometheraBiosciences

1.05 Questions for the speakers &panel discussion• Exploring the impact of short vs.long product shelf life on Costsof Logistics

Early-stage R&D strategy:Optimizing preclinical-Phase Itranslation for C&GTs

12.05 Chair’s introductionKatherine Tsokas, JD, SeniorDirector, Global Regulatory Affairs,Janssen Research & Development,LLC

12.10 Preclinical considerations forcell and gene therapyproducts: US FDA perspective• Considerations for preclinicalstudies to enable initiation ofearly-phase clinical trials

• Communications withCBER/OCTGT

• Regulatory ResourcesDr Theresa Chen,Pharmacology/ToxicologyReviewer, Office of Cellular Tissueand Gene Therapies (OCTGT),Center for Biologics Evaluation andResearch (CBER), US Food andDrug Administration (FDA)

Regulator and industry earlystage R&D insights…Clarification of whichpreclinical studies arerequirements – and which areoptimal - for translation toFirst-in-Man studies• Defining adequate controls

Gene therapy clinical update:Assessing the prospects of leadingproduct candidates across the keytherapeutic areas12.05 Chair’s introduction

Dr James Wilson, Professor ofInternal Medicine & Pathology andLaboratory Medicine, Director ofthe Gene Program, University ofPennsylvania, Research Director,Center for Orphan DiseaseResearch and Therapy

12.15 Commercialisation of genetherapy products – a fullyintegrated lentiviral vectorstrategyPeter Nolan, Senior Vice President,Commercial Development, Oxford Biomedica

12.35 Selective cancerimmunotherapeutics on anovel replicating retroviralvector (RRV) platform• Tumor specific infection andreplication of RRV in preclinicalmodels and clinical trial patients

• Dual action of direct tumor killingand anticancer immunity

Case studies: The latest safetyand efficacy data for leadinggene therapy productcandidates in the clinic for a range of therapeuticareas/indications – where are different serotypesdemonstrating the greatest utility?

Sponsored by:

Sponsored by:


1.20 Buffet lunch in theExhibition/1-2-1 MeetingArea ORLunch BriefingOptimal Session for a maximum of50 attendees)Neural stem cell-mediatedtherapy for brain tumorsMargarita Gutova, MD, AssociateResearch Professor, Departmentof Neurosciences, City of HopeNational Medical Center &Beckman Research Institute

2.30 Case studies & multiplestakeholder panel discussion• How are the leading industrialand academic playersprogressing in developingeconomically feasible,commercial scalemanufacturing models for CART-cell immunotherapy and otherex vivo genetically modified celltechnologies?

• Ensuring sufficiently robust supplyof starting materials (eg.lentiviral vectors)

• Clarification of evolvingclassification of raw versusstarting materials and theassociated regulatoryrepercussions

• Cell procurement• Cell characterization • Cost of Goods• Testing of cells – how much canyou actually test after they havebeen modified?

• Traceability• Point of Care bioprocessing orregional manufacturing centers– which model isoptimal/commercially realistic?Examining the challenges andbenefits in either approach

Speakers/panellists to include:Dr Larry Couture, Senior VicePresident and Founding Director,Centre for Applied TechnologyDevelopment, Beckman ResearchInstitute of the City of HopeDr Kenneth LeClair, ExecutiveDirector, Novartis Cell & GeneTherapy UnitDr Isabelle Riviére, Director CellTherapy & Cell EngineeringFacility, Memorial Sloan KetteringCancer CentreMitchell H. Finer, Chief ScientificOfficer, Bluebird BioJ. Joseph Melenhorst, PhD,Director, Product Development &Correlative Sciences Laboratories,Translational Research Program,Department of Pathology andLaboratory Medicine, University ofPennsylvania

3.40 Close of session, followed byafternoon tea in the Exhibition/1-2-1 Meeting Area

Sponsored by:

1.20 Buffet lunch in theExhibition/1-2-1 MeetingArea ORLunch BriefingOptimal Session for a maximum of50 attendees)Neural stem cell-mediatedtherapy for brain tumorsMargarita Gutova, MD, AssociateResearch Professor, Departmentof Neurosciences, City of HopeNational Medical Center &Beckman Research Institute

2.30 Case study 1Frederick Miesowicz, PhD, ChiefOperating Officer & VicePresident Manufacturing, ArgosTherapeutics Inc

2.50 Questions & discussion



3.20 Case study 2Dr Wilfred Dalemans, ChiefTechnical Officer, Tigenix(Provisionally confirmed)



Sponsored by:

12.25 Industry perspective Dr Geoffrey Nichol, Executive VicePresident, Research &Development, SangamoBioSciences

12.40 Early R&D for stem cell basedtherapies: Preparing for thePhase 1 clinical trial andbeyondDr Jane Lebkowski, Vice PresidentResearch & Development, AsteriasBiotherapeutics

1.00 …followed by questions for thespeakers & panel discussionWhat can be done in practice toalleviate the lack of insight currentanimal models offer for dosing?

1.20 Buffet lunch in theExhibition/1-2-1 Meeting Area ORLunch BriefingOptimal Session for a maximum of50 attendees)Neural stem cell-mediatedtherapy for brain tumorsMargarita Gutova, MD, AssociateResearch Professor, Department ofNeurosciences, City of HopeNational Medical Center &Beckman Research Institute

2.30 Presentations & roundtablediscussion

Retrospectives: Lessons learned bycompanies and organizations thathave successfully negotiated thetranslation processes• How did they meet the variousexpectations and requirementsof investor, regulator andclinician in order to advancetheir product candidates tohuman trials?

Speakers & panellists to include:Anne-Virginie Eggimann, VicePresident, Regulatory Science,bluebird bio, Inc:Professor Miguel Forte, Senior VicePresident, Clinical & Regulatory,TxCellKatherine Tsokas, JD, SeniorDirector, Global Regulatory Affairs,Janssen Research & Development,LLCDr Stefanos Theoharis,Chief Business Officer,Apceth GmbH & Co. KG.


Sponsored by:

• Over 70 patients treated in 3 trialsfor High Grade Glioma

• Excellent safety profile, tumorshrinkage, and improved survivalversus relevant historical controls

• Planning registration-directedtrial in HGG and expansion intoother tumor types

Dr Douglas Jolly, Co-Founder &Executive Vice President Research& Pharmaceutical Development,Tocagen

12.50 Progress in gene therapy forheart disease• Coronary artery disease andcongestive heart failure areresponsible for the majority ofcardiovascular morbidity andmortality

• Several gene therapyapproaches have beenintroduced in the clinic for thesetwo indications, using nakedplasmid, adenovirus and AAVvectors via directintramyocardial injections andintracoronary delivery methods

• Progress in the most advancedclinical trials will be reviewed

Dr Gabor Rubanyi, Chief ScientificOfficer, Taxus CardiumPharmaceuticals Group

1.05 AVA-101 and challenges inthe clinical development of agene therapy for oculardisease• Ability to translate pre-clinicalanimal models

• Selection of appropriateendpoints

• Need to assess repeat dosing ordosing in fellow eye

• Varying international regulatoryrequirements

Dr Samuel B. Barone, ChiefMedical Officer, Avalanche Biotechnologies

1.20 Buffet lunch in theExhibition/1-2-1 Meeting AreaORLunch BriefingOptimal Session for a maximum of50 attendees)Neural stem cell-mediatedtherapy for brain tumorsMargarita Gutova, MD, AssociateResearch Professor, Department ofNeurosciences, City of HopeNational Medical Center &Beckman Research Institute


2.45 Case study - HaemophiliaDr Hansjoerg Duerr, Global HeadHaematology, Bayer Healthcare

3.00 Questions for the speakers &panel discussion


Sponsored by:

Afternoon plenaryGlobal regulatory update: Analyzing opportunities and challenges for industrypresented by evolving regulations worldwide• What are the most significant areas of increasing harmony/disharmony?

4.20 Chair’s introductionJiwen Zhang, PhD, Regulatory Affairs Director, GE Healthcare

4.25 European (CAT) perspective• What’s new in Europe which can potentially impact C&GTs? (Eg. AdaptiveLicensing pathway)

Dr Martina Schusler-Lenz, Vice-Chair, Committee for Advanced Therapies (CAT) EMA& Clinical Assessor, Division of Medical Biotechnology, Paul-Ehrlich Institute

4.45 Japanese perspectivePerspectives on novel Japanese regulatory pathways for cell & gene therapyproducts • What are the challenges in the new legislation for C&GTs from the industryperspective? And where are the pitfalls?

• Can therapeutic candidates developed and manufactured outside of Japan nowqualify to utilize the new regulatory framework?

• Does a manufacturer need to have fully market-ready bioprocess in order toreceive conditional approval in Japan, or is it possible to validate yourmanufacturing process at a later point?

David Hall, Chief Executive Officer, Replicel

5.05 Australasian perspective Regulation of clinical cell and gene therapy trials Down UnderProf John Rasko, Director, Department of Cell and Molecular Therapies, Royal PrinceAlbert Hospital

5.25 Questions for the speakers, followed by panel discussion with industry• International harmonization: From basic terminology to aspects such as potencyand dose specification - what are the key points of regulatory convergence anddivergence between the US, Europe and RoW?

• How does this change the strategic picture for cell & gene therapy companiesseeking to play in two or more continents?Panellist:Dr Keith Wonnacott, Chief of Cellular Therapies Unit, Office of Cellular Tissue andGene Therapies (OCTGT), US Food and Drug Administration (FDA)

6.00 Close of Day 1, followed by a Cocktail Reception in the Exhibition/1-2-1 Meeting Area

Keynote regulators’ perspectives: What are the latest and forthcoming key developments for cell & gene therapy/ATMPregulations, particularly with regard to pathways to market

and post-licensure requirements?

Would you like to sponsor this exclusive cocktail reception, or perhaps a subsequent Speakers’ Dinner celebrating our stellarpanel? Contact Michael Adeniya now! ([email protected])


Informative gathering of elite bioleadersJim Zacka, VP, Worthington Biochemical Corporation

FORUM 2015


DAY 2 - Tuesday, January 27th 2015

9.00 Chair’s introductionEdward Lanphier, President & Chief Executive Officer, Sangamo BioSciences Inc

9.05 VC perspectiveDr Nessan Bermingham, Venture Partner, Atlas Ventures, Founder and ChiefExecutive Officer, Intellia Therapeutics


9.25 US State stem cell funding organizationDr C. Randal Mills. President & Chief Executive Officer, California Institute forRegenerative Medicine (CIRM)


9.45 Philanthropists/family funds William Podd, Founder, President & Chief Executive Officer, LandmarkCapital/Landmark Angels


10.05 Cell & gene therapy funding/business opportunities in emerging regionsPresentation reserved


10.25 Wall Street analyst’s RoundtableTaking the pulse of the cell & gene therapy space from the institutional investors’perspective – what are their expectations for the short-, mid- and long-terms?Boris Peaker, Managing Director, Biotechnology Stocks, Cowen and CompanyHoward Liang, PhD, Managing Director Biotechnology Equity Research, LeerinkPartners LLC

10.50 What’s coming next? Showcasing the next wave of cell & gene therapytechnologiesWhat is coming over the horizon? What will follow MSCs and the latest batch ofcellular immunotherapies? What’s will drive further progress in the gene therapyspace? A timely look at the emerging technologies and modalities which willmaintain momentum in the cell & gene therapy sector


11.55 Presentation 1James Noble, Chief Executive Officer, AdaptimmuneJeb Keiper, Vice President, Oncology Business Development, GlaxoSmithKlineWorldwide Business Development

12.0 Presentation 2Edward Lanphier, President & Chief Executive Officer, Sangamo BioSciences IncJohn McDonald, Vice President, Business Development, Biogen Idec

12.05 Big Pharma and Big Biotech presentations & roundtable discussion• Many in the C&GT sector see 2014 as the year Big Pharma really came to the party– what does 2015 hold in store?

Panellists:Perry Karsen, Chief Executive Officer, Celgene Cellular TherapiesJan Thirkettle, Head of Advanced Therapy Delivery, GlaxoSmithKlineAlbert Seymour, Global Head of Research and Nonclinical Development, ShirePharmaceuticalsJohn McDonald, Vice President, Business Development, Biogen IdecDr Devyn Smith, Head of Strategy, Pharmatherapeutics Research & Development, Pfizer

12.45 Presentations & panel discussionHow will industry-academia-public sector consortia and other collaborative modelssupport growth of regenerative medicine through 2015 and beyond?• Updates on the status and goals of key initiatives in North America and EuropePanellists:Dr Mahendra Rao, Consultant, NYSCF & Vice President Strategic Affairs,Q TherapeuticsBrock Reeve, Executive Director, Harvard Stem Cell Institute

1.20 Buffet lunch in the Exhibition/1-2-1 Meeting Area

Morning plenaryPart 1: Investment trends and keys to funding success for cell & genetherapy biotechs

Part 2: Licensing and partnering

Updates from traditional and alternative sources of cell & gene therapyR&D funding• Is the biotech IPO back to stay?• What has driven the significant growth in private investment in the space?• Reviewing the Q2 2014 biotech downturn; what is the status as of January 2015?Was it a flash in the pan, or something more significant?

• What specific technologies or areas are interesting them currently and why? Wheredo they see the greatest potential for significant ROI and/or improvements tohuman health?

• What are the latest trends in terms of the nature and structure of C&GT R&D fundingmodels?

Review and celebration of some of the major deals of 2014• Brief analysis of key drivers and deal structures from both sides • How and where was the necessary value created in the eyes of the pharmastakeholders?

7.30 Buffet breakfast in the exhibition areaOR

7.45 Breakfast Briefing (Optional session for a maximum of 50 attendees)Creating a cell therapy manufacturing system forcommercial productionWorkshop leader:Richard Grant, Director of Cell Therapy, Invetech Pty Ltd

Panellists:Dr Fred Miesowicz, Chief Operating Officer, Argos TherapeuticsDonald Powers, Principal Scientist, Janssen Cell TherapyNick Timmons, Director, Product and Process Development, Centre forCommercialisation of Regenerative Medicine

Sponsored by

Do you represent a biotech with a platform technology which may be of interestto our audience? Contact Michael Adeniya today ([email protected])to register your interest in presenting during this dedicated Cell & Gene TherapyForum Biotech Showcase morning plenary session.


Focus session 1 Focus session 2 Focus session 3 Focus session 4

Adoptive T cell immunotherapyclinical updates: • promise and limitations of CAR-T• TCRs and TILs waiting in the wings

2.25 Chair’s introductionSimon F. Lacey PhD, Director,Translational and CorrelativeStudies Laboratory, TranslationalResearch Program, PerelmanSchool of Medicine, University ofPennsylvania

2.35 Short presentations andpanel discussionDrawing on clinical case studiesspeakers will address issuesincluding:• Safety prediction andmonitoring

• Correlates of on- and off-targeteffects

• Efficacy prediction andmonitoring

Gene therapy manufacture: Howare the next wave of products inlate-phase development preparingfor commercial scale? 2.25 Chair’s introduction

Product supply for largemarket indications:MYDICAR® (AAV1/SERCA2a)for heart failure• Update on the path towards2000L commercial scalemanufacturing

Dr Barbara Thorne, Senior Director,Process Development, CelladonCorporation

Multiple stakeholder presentations…What are the remaining necessarytechnological and strategicdevelopments to enablecommercially feasible manufacturingof gene therapy products?

R&D strategy: Defining next steps forgenome editing technologies•How will the various approachesaddress technological and regulatorychallenges?

2.25 Chair’s introductionDr Thomas Vogt, Vice PresidentDiscovery & Systems Biology, CHDIManagement/ CHDI Foundation

2.30 Case study 1Dr Philip D Gregory, Senior VicePresident, Research & ChiefScientific Officer, SangamoBioSciences

Case studies: Updates on theleading technologies approachingthe clinic as well as those already in trials – how do they compare?• What will the developmentpathway to market for theseproducts look like?

Clinical/commercial strategyupdates: Preparing MSCs and otherstem cell therapeutics for market2.25 Chair’s introduction

Dr Kim Warren, Head of CustomDevelopment Services for CellTherapy, Lonza Walkersville Inc

2.30 Case study – CVDr Andrew Hamer, Vice President,Medical Affairs, Capricor

2.45 Case study – MusculoskeletaldiseasesDr Donna Skerrett, Chief MedicalOfficer, Mesoblast

Case studies: Examining the latestclinical, bioprocessing andcommercial business models forleading adult stem cell therapies inkey indications/therapeutic areas


Excellent presentations and discussions with lively andproductive debate. A very good overall summary of thepotential issues.Keren Winmill, Non-Executive Director, Biotec Services International

• Specificity – is one proteinenough?

• How broadly do thetechnologies scale?

• Application beyond malignantpatients

• How do TCRs and TILs comparewith CAR-T in terms ofcommercial promise andlimitations, particularly for solidtumors?

• Combination strategies – withcheckpoint inhibitors,vaccines…

• When/how to movecombinations up to earlier-stagecancer patients

• What has driven the interest ofbig pharma to thesetechnologies?

Speakers include:Dr Helen Tayton-Martin, COO,AdaptimmuneDavid D. Chang, MD, PhD,Executive Vice President R&D &Chief Medical Officer, Kite Pharma, IncMitchell H. Finer, Chief ScientificOfficer, bluebird bioDr Manish Singh, Chairman &CEO, Lion Biotechnologies, Inc


2.40 Preparing for file and launch.The challenges in deliveringand commercialising a genetherapy medicine.• What technical and regulatorychallenges need to be solvedfor an approvable file?

• What is ‘commercialisation’ of agene therapy product andwhat will it look like when donesuccessfully?

Dr Jim Faulkner, Vice President,CMC & Supply, GlaxoSmithKlineRare Diseases

2.55 How are the tools andservices sectors preparing tosupport large scale genetherapy manufacture?• Making commercial scalemanufacturing a reality,depending on therapeuticindication

• How tools and servicecompanies are reacting to thecurrent high interest in genetherapy

• The economics behindpotentially curative therapies

Dave Backer, Senior Director &General Manager, SAFC Carlsbad

3.15 FDA perspective• Update on guidelines andthinking around referencematerials for AAV gene therapyproducts

Dr Denise Gavin, Team Lead,Gene Therapy Branch, Office ofCellular Tissue and GeneTherapies (OCTGT), US Food andDrug Administration (FDA)

3.30 …and panel discussion:• How to ensure both quality andscalability of gene therapymanufacturing process tosupport the delivery of genetherapy products to largepatient populations?

• What will be the keys toadequately controlling Cost ofGoods at commercial scale?


2.50 Case study 2Dr Nessan Bermingham, VenturePartner, Atlas Ventures, Founderand Chief Executive Officer,Intellia Therapeutics

3.10 Case study 3Dr Rodger Novak, Chief ExecutiveOfficer, CRISPR Therapeutics

3.30 Questions for the speakers &panel discussion• Defining and addressing the keyregulatory questions

• How to define efficacy,potency, quality controls?


3.00 Case study –Immunomodulatory activityin autoimmune disease andischemic injuryDr John Harrington, Executive VicePresident & Chief Scientific Officer,Athersys Inc

3.15 Case studyPresentation reserved

3.35 Questions for the speakers &panel discussion• Can we reach a consensus onwhat the definition of an MSCshould be?

Panellists – speakers of the session,plus:Dr Christopher Bravery, PrincipalConsultant, AdvbiolsDr Ohad Karnielli, Vice President,Technology & Manufacturing,Pluristem Therapeutics Inc


4.40 Chair's introduction

4.45 How is the commercial manufacturing/product delivery model forGlybera continuing to evolve? • How are we addressing/have we addressed quality, stability and puritychallenges?

• How are we meeting the challenges of fulfilling long term follow-up requirementsfor gene therapy-treated subjects, as well as more conventional regulatory post-approval commitments?

Dr Hans Preusting, Chief Business Officer, UniQure

5.05 Short presentations & panel discussionUpdates from leading C&GT product candidates for rare disease indications • What Phase II/Phase III trial designs are they adopting and why?• What does the commercial business case look like?• How do they plan to address the commercial scale up challenge?• Exploring challenges in technology licensing for rare disease therapeuticcandidates – how can the sector work together to help de-risk R&D?

Panellists:Faraz Ali, Vice President, Commercial Planning & External Affairs, Bluebird BioDr Jim Faulkner, Vice President, CMC & Supply, GlaxoSmithKline Rare DiseasesDr James Wilson, Professor of Internal Medicine & Pathology and LaboratoryMedicine, Director of the Gene Program, University of Pennsylvania

6.00 Close of day 2

Would you like to sponsor an exclusive cocktail reception on the evening of day 2 of the event?

Contact Michael Adeniya now! ([email protected])

Afternoon plenaryEnsuring the next wave of cell & gene therapies for rare diseaseindications achieve commercial success• Robust clinical trial design and commercial scale up – recent lessons learned?


Good talks and networkingopportunityMike Adams, Director of Marketing, Client Services andProposal Management, Fisher BioServices

FORUM 2015


DAY 3 - Wednesday, January 28th 2015

9.00 Chair’s introductionFaraz Ali, Vice President, Commercial Planning & External Affairs, Bluebird Bio

9.05 Pricing and reimbursement perspectiveNeil Palmer, President and Principal Consultant, PDCI Market Access


9.30 Physician’s perspectiveDr Richard Maziarz, Researcher, Oregon Health Science University Hospital


9.55 Industry response – case studyHow can a cell therapy company address these challenges?• How can the industry become more efficient at meeting these challenges?• Ways for the cell therapy community to learn from Pharma mistakes• The importance of valueDr Sven Kili, Senior Director, Global Medical Affairs, Biosurgery Sanofi Biosurgery




10.45 Multiple stakeholder roundtable discussion • How to ensure the cell & gene therapy industry obtains fair value for what it bringsto the table? (How do you price a therapy that is curative of a chronic disease, forinstance?)

• How can the industry capitalize on EHRs and other emerging data sources toimprove the R&D and commercial prospects of their products?

• Unlocking the potential of cutting-edge informatics and analytics solutions totransform the cell & gene therapy development and commercial business models

Panellists - speakers of the session, plus:Dr John Maslowski, Vice President Scientific Affairs, Fibrocell


Morning plenaryIs your cell or gene therapy product candidate aligned with the needsand capabilities of the marketplace?• What is and isn’t viable in the eyes of today’s payers, physicians and patients?

Healthcare sector perspectives• Defining evolving trends and challenges for each stakeholder group which carryrepercussions for the cell & gene therapy industry

• Creative thinking around P&R models for premium-priced cell & gene therapies toease the burden of financial risk for practice managers and independent physicians

• What changes can the cell & gene therapy industry initiate to help make bothautologous and allogeneic products viable and affordable for all stakeholders?


7.45 Breakfast Briefing (Optional session for a maximum of 50 attendees)Strategy and innovation for cell therapymanufacturing – creating a roadmap to a commercial futureDr Knut Niss, Senior Technical Project Leader, NovartisDr Jurjen Velthuis, Vice President CMC, Kiadis Pharma

Sponsored by


Focus session 1 Focus session 2 Focus session 3

Cell therapy manufacture 2: Refining processscience to control Cost of Goods:

12.00 Chair’s introductionDr Ian Harris, Senior Director, Cell Therapy,Janssen Research & Development LLC

12.05 Allogeneic cell therapy manufacturing:What are the remaining technologygaps?Dr Greg Russotti, Vice President TechnicalOperations, Celgene Cellular Therapeutics

12.25 Industry perspectiveDr Knut Niss, Senior Technical Project Leader,Novartis

12.45 Reducing cell manufacturing CoGs whileimproving product quality - can oneplatform do it all? Dr Eytan Abraham, Head of Cell TherapyResearch & Technology, Lonza Walkersville Inc

1.05 Questions for the speakers & paneldiscussion• Exploring the latest guidelines and thinkingaround reference materials for cell therapyproducts – repercussions for the industry

Panellists – speakers of the session, plus:Dr John Harrington, Executive Vice President &Chief Scientific Officer, Athersys Inc

1.30 Close of the Phacilitate Cell & Gene TherapyForum 2015 – buffet lunch in the Exhibition/ 1-2-1 Meeting Area

Case studies: How to build CoGs into yourplanning from the start of bioprocessdevelopment – lessons learned

Logistics 2: Can next-generation storagetechnologies unlock regenerative medicinemarkets?

12.00 Chair’s introduction


12.25 Controlling cell delivery, frommanufacturing to the patient viashipment, storage, thawing and routs ofadministrationDr Ohad Karnielli, Vice President, Technology &Manufacturing, Pluristem Therapeutics Inc

12.45 Industry perspectives… Identifying best practices to keep regenerativemedicine products stable all the way to theclinical siteDr Zorina Pitkin, Vice President, Quality Systems,Organogenesis

1.15 …followed by questions for the speakers& roundtable discussionHow can the stem cell therapy industry bestprepare to meet labelling and other supplychain requirements at commercial scale?

1.30 Close of the Phacilitate Cell & Gene TherapyForum 2015 – buffet lunch in the Exhibition/1-2-1Meeting Area

Case studies: Comparing and contrastingdifferent emerging technologies/approachesto alleviating the challenges in stem cellfreezing and thawing• How does each example impact cost andquality?

Clinical strategy: What learnings do recentPhase II trials provide to help improveunderstanding of cell therapy MoA?

12.00 Chair’s introductionDr Andrew Hamer, Vice President, MedicalAffairs, Capricor

12.05 Case study 1Dr Robert W. Mays, Head of Neuroscience,Athersys, Inc.


12.45 Back and forth between bench andbedside: NHLBI and hematologic celltherapies• Two case studies of NHLBI projects that haveproduced phase II trials and ancillary studies

• The role of NHLBI in facilitating translationalstudies and early phase clinical trials

• Working between sister agencies—how tonavigate the FDA and the NHLBI

Dr Traci Heath Mondoro, Chief, TranslationalBlood Sciences and Resources, NHLBI

1.05 Questions for the speakers, followed byroundtable discussion• How does pharma assess the positive andnegative in 2014’s Phase II results?

• What is their view on best steps forward forrobust Phase II trial designs - and for R&D as awhole - for cell & gene therapy products in theindications in question? (Eg. What more canwe do at an earlier stage, such as in animalstudies?)

• Where are we seeing advancements inunderstanding of MoA to improve future trialdesigns? (To include a specific focus on theCNS/ophthalmology areas)

1.30 Close of the Phacilitate Cell & Gene TherapyForum 2015 – buffet lunch in the Exhibition/1-2-1Meeting Area

Case studies: In depth analysis of 2014’smajor cell therapy Phase II trials - whatcan we say we do know – and what don’twe know – about dosing, potency,delivery and Mechanism of Action?


9.00 Chair’s introductionDr Devyn Smith, Head of Strategy, Pharmatherapeutics Research & Development, Pfizer

9.10 Stem cells in drug discovery: a reality check• Preclinical models for efficacy and safety• Adoption of new technologies in exciting and stressful times• Lessons from the high throughput revolution of the 90sDr Ricardo Macarron, Vice President of Target and Pathway Validation,GlaxoSmithKline


9.35 Presentation 2Dr Huseyin Mehmet, Vice President, Head of Cell & Molecular Biology, ProteostasisTherapeutics Inc


10.00 Presentation reserved10.20 Questions & discussion

10.25 Presentations & panel discussionUpdates on leading cell banking initiatives and consortia• Current status: How are these cell banks being used and how are limitations beingaddressed?

- How are they expanding cell lines?• How are we seeking to coordinate efforts on a multinational basis? Speakers/panellists include:Dr Christopher Bravery, Principal Consultant, AdvbiolsDr Artur Isaev, Chief Executive Officer, Human Stem Cells Institute


11.55 DARPA perspectiveColonel Daniel Wattendorf, Program Manager, Defence Science Office, DARPA

12.15 NIH perspective• What does the future hold for the CRM, particularly relating to stem cell tooldevelopment?

Dr Anton Simeonov, Acting Deputy Scientific Director, National Centre for AdvancingTranslational Sciences (NCATS), NIH

12.35 Review of CIRM’s latest stem cell tool funding round – future plansNeil Littman, Business Development Officer, California Institute for RegenerativeMedicine (CIRM)

12.55 Questions for the speakers & panel discussion1.20 Buffet lunch in the Exhibition/1-2-1 Meeting Area

2.25 Chair’s introductionDr Christopher Bravery, Principal Consultant, Advbiols

2.30 US perspectiveDr Joyce Frey-Vasconcells, Regulatory Expert, Frey-Vasconcells Consulting, LLC

2.50 European perspectiveInsight in safety and efficacy of stem cell based advanced therapy medicinal productsProf Asterios Tsiftsoglou, AUTH Lab of Pharmacology, School of PharmaceuticalSciences & CAT Member

3.10 Japanese perspectiveDavid Hall, Chief Executive Officer, Replicell

3.30 Questions for the speakers & regulator/industry roundtable discussion• How do recent regulatory changes in Japan translate to opportunity for the globalstem cell tool sector?

• What is next for cardio tox standards? How will regulators and industry move this fieldforward?

4.00 Close of session, followed by afternoon tea in the Exhibition/1-2-1 Meeting Area

4.40 Chair's introductionDr Mahendra Rao, Consultant, NYSCF & Vice President Strategic Affairs, Q Therapeutics

4.45 Presentations & panel discussion• Fine tuning economies of scale

- What role can cell/tissue banks play in assisting?• How are standardization initiatives progressing?• Update on cell differentiation initiatives• Developing tools that enhance the physiologic relevance of hiPSC models in relationto drug discovery

• Determining/measuring cell pluripotency- To what extent are cutting edge epigenetic mapping and deep sequencingtools and techniques improving assessment of pluripotency?

• Addressing licensing issues and challenges for iPSC-related technologyNeil Littman, Business Development Officer, California Institute for RegenerativeMedicine (CIRM)Professor John Rasko, Director, Department of Cell and Molecular Therapies, RoyalPrince Albert HospitalDr Carl G. Simon, Jr, Biologist and Project Leader of 3D Tissue Scaffolds, NationalInstitute of Standards and Technology (NIST)Dr Eileen Dolan, Professor of Medicine, Chair, Committee on Clinical Pharmacologyand Pharmacogenomics, University of Chicago, Associate Director for Education,Comprehensive Cancer Center

6.00 Close of day 2

9.00 Chair’s introductionDr Michele Cleary, Executive Director, Target and Pathway Biology, Merck

9.10 Establishing iPSC technology for Pharmacogenomic StudiesDr Eileen Dolan, Professor of Medicine, Chair, Committee on Clinical Pharmacologyand Pharmacogenomics, University of Chicago, Associate Director for Education,Comprehensive Cancer Center


9.40 Case study 2Reserved for Technology Spotlight Sponsor


10.05 Advent of Industry Scale hiPSC Models & Adoption in Drug Development• Off the shelf access to pure iPS human tissues at large scale and from desiredgenotypes is a significant advance.

• Data thus far present a strong case for use of human iPS models for late stagediscovery and predictive toxicology.

• Cost and concerns about functionality appear to limit application of the models atearlier stages.

• How do we accelerate use of hiPSC models from ‘cradle-to-grave’ in the discoveryprocess?

Carter Cliff, Business Development, Cellular Dynamics International Inc10.25 Questions & discussion

10.30 Case study 410.50 Questions & discussion

Reserved for Technology Spotlight Sponsor10.55 Case study 511.15 Questions & discussion

Reserved for Technology Spotlight Sponsor

11.20 Morning coffee in the Exhibition/1-2-1 Meeting Area12.00 Case study 6

Reserved for Technology Spotlight Sponsor12.20 Questions & discussion





1.15 Further questions for the speakers & panel discussion1.30 Close of Phacilitate Stem Cells as Discovery & Research Tools 2015 – buffet lunch in

the Exhibition/1-2-1 Meeting Area

Plenary Thought leader analysis of macro trends in the stem cell tools arena

Keynote Presentations: Assessing big pharma and biotech strategies forstem cell tool adoption and implementation – where do we see thegreatest value moving forward for drug discovery and development?• Does pharma still see tools as the ‘low hanging fruit’ in the stem cell space? Why thenhas uptake been comparatively slow to date across the sector, and what will it taketo change this trend?

• Are we learning lessons in terms of our adoption of potentially disruptive discoverytechnologies?

• How do we expect 3D screening and automation to impact discovery research,particularly in the targeted medicine and rare disease spaces?

• Licensing and partnering trends and deal analysis – what sort of collaborative modelsare pharma companies looking to adopt with stem cell tool providers today?

Plenary Regulatory updates from the US, Europe & Japan

Regulators’ perspectives from 3 continents• Driving standardization of iPS cells• What’s the latest view on the acceptability of stem cell-derived insights as predictorsof therapeutic safety and efficacy?

• Progressing standardization of raw/starting materials

Plenary Driving progress in the iPSC space

DAY 2 - Tuesday, January 27th 2015

DAY 3 - Wednesday, January 28th 2015

Plenary Next generation technologies showcase• Reviewing cutting edge applications for screening, toxicology and diagnostics

Case studies: Defining the utility/pros and cons of technologies inemerging technology areas, including:• Novel predictive models for phenotypic screening• Organ/tissue creation for toxicology screening (including 3D organ and tissue printing)• Robotics and automation


7.45 Breakfast Briefing Optional session for a maximum of 50 attendees)Creating a Cell Therapy Manufacturing System for Commercial ProductionWorkshop leader:Richard Grant, Director of Cell Therapy, Invetech Pty LtdPanellists:Dr Fred Miesowicz, Chief Operating Officer, Argos TherapeuticsDonald Powers, Principle Scientist, Janssen Cell TherapyNick Timmons, Director, Product and Process Development, Centre forCommercialisation of Regenerative Medicine

Sponsored by


7.45 Breakfast Briefing (Optional session for a maximum of 50 attendees)Strategy and innovation for cell therapymanufacturing – creating a roadmap to a commercial futureDr Knut Niss, Senior Technical Project Leader, NovartisDr Jurjen Velthuis, Vice President CMC, Kiadis Pharma

Sponsored by

SCADART attendees are automatically registered to also attend day 1 of the Cell & Gene Therapy and Immunotherapy Forums on Monday, January 26th


Definitely the best meeting wehave attending in the last yearwhere we could find potentialpharma/biotech clients.John R. Jaskowiak, Vice President, Angiocrine Bioscience, Inc.

FORUM 2015


Have you joined our Phacilitate LinkedIn group yet?This LinkedIn group is an international online community focused on providing pharma and biotech decision makers involved inthe Cell & Gene Therapy field with a platform for senior level knowledge exchange, benchmarking and networking, much asthe Phacilitate Washington’ Forum itself does. Search groups for ‘’Phacilitate’’

It was an excellent meeting,one of the best strategic/stateof the field meetings I’veattended.Gary Mansfield, Director of Custom BiologicalToxicity Programs, WuXi AppTec

Contact Michael Adeniya now to book yourown private meeting area for the [email protected]


Cell Therapy CatapultThe Cell Therapy Catapult was established as a centre ofexcellence in 2012 to create a world-leading cell therapy industryin the UK through innovation and collaboration.Supported by the UK Government, our mission is to drive thegrowth of the industry by helping cell therapy organisations acrossthe world translate early stage research into commercially viableand investable therapies.With one of the largest dedicated cell therapy teams anywhere,our scientists, business development, manufacturing andregulatory experts are collaborating with cell therapyorganisations and other interested parties from across the globe.Together we are also finding solutions to industry-wide challengesincluding business models, logistics and reimbursement.

GE HealthcareGE Healthcare Life Sciences delivers breakthroughs in drugdiscovery, biopharmaceutical manufacturing and the latest incellular technologies, so scientists and specialists around the worlddiscover new ways to predict, diagnose and treat disease. Formore information about GE Healthcare Life Sciences, visit ourWebsite at www.gelifesciences.com.

LonzaLonza offers world class technology platforms in the areas of GMPcell culture and viral-based therapeutic manufacturing, custombiotherapeutic culture media, a large selection of primary andstem cells and a full line of custom bioassays. Our extensiveexperience in Cell and Gene Therapy process optimization andscale-up innovation helps clients to safely and effectivelyadvance their products through all phases of the commercialpipeline and maximize their return on investment.

Oxford Biomedica logoOxford BioMedica is a leading gene-based biopharmaceuticalcompany developing innovative medicines to improve the lives ofpatients with high unmet medical needs. We have establishedplatform technologies in gene delivery and immunotherapy,protected by an extensive intellectual property estate. TheCompany’s technology platform includes a highly efficient genedelivery system (LentiVector®), which has specific advantages fortargeting diseases of the central nervous system and the eye; andis also widely used in the ex-vivo cell therapy arena.Oxford BioMedica’s product portfolio is focused on high value,growing markets. These include gene therapy productsengineered to treat Parkinson’s disease (preclinical, Phase I/II),Motor Neurone Disease (preclinical) and ocular diseases(preclinical), especially retinal diseases (Phase I and Phase I/II).

Progenitor Cell TherapyPCT is an industry leader in development and manufacturing ofcell therapy products, and is the only contract development andmanufacturing organization (CDMO) to see Client's cell therapyproduct receive marketing approval from the FDA. With over 12years of exclusive cell therapy-focused experience, PCT hashelped over 100 Clients bridge the gap between discovery andpatient care through unparalleled strategic insight and efficienttransfer of cell-based therapies from laboratory into clinicalpractice.

Sangamo BiosciencesSangamo BioSciences, Inc. is developing novel zinc finger DNA-binding proteins (ZFPs), for therapeutic gene regulation andgenome editing and has ongoing Phase 2 clinical trials toevaluate safety and efficacy of a ZFP Therapeutic® for thetreatment of HIV/AIDS. Other therapeutic programs are focusedon monogenic diseases. Sangamo engineers sequence-specificZFP Nucleases (ZFNs) for gene modification and ZFP transcriptionfactors (ZFP TFs) for gene regulation. The company has anagreement with Shire to develop therapeutics for hemophilia,Huntingtons’ disease and other monogenic diseases and, in non-therapeutic applications of its technology, has strategicpartnerships with Dow AgroSciences and Sigma-AldrichCorporation.

Fisher BioservicesFisher BioServices, part of Thermo Fisher Scientific, is a professionalstem cell and cell therapy services provider. With locations aroundthe world, Fisher BioServices is committed to providingbiorepository storage, logistics infrastructure and clinical trialservices to the cell therapy industry. Our facilities and capabilitiesenable us to provide integrated solutions from collection site orCMO to repository or clinical site. Services include GTPbiorepository, laboratory services, cell and specimen collectionkits, product distribution, on-site inventory management and ultracold chain management. For more information please visit:www.fisherbioservices.com.

Life TechnologiesLife Technologies is a global biotechnology tools companydedicated to improving the human condition. With more than50,000 products used by more than 75,000 customers around theglobe, Life Technologies is advancing scientific research in areassuch as next-generation sequencing, drug discovery,bioproduction and cellular medicine.

MarkenMarken is the leading global clinical supply chain service providerdedicated 100% to the pharmaceutical and life sciencesindustries, supporting over 49,000 investigator sites in more than 150countries. With decades of experience in the logistics, transportand distribution of temperature sensitive life savingpharmaceuticals, clinical trial supplies and specimen collection;Marken integrates depot and logistics services into solutions thatextend the reach of clinical trials to even the most remotetreatment naïve geographies. Our team members and network offacilities bridge the distance between patients and the essentialresources of life science companies.

Pall Life SciencesPall Life Sciences provides process, pilot and laboratory filtration,separation, purification and fluid handling devices, systems andservices, with single-use systems available for all unit operationsfrom cell culture – including cell therapy applications - throughfinal formulation and filling. Based on Pall’s long history of providingquality equipment for the biopharmaceutical, vaccine and celltherapy industries, all products – whether standard or customizedto match users exact process needs – are backed up withextensive documentation and experience in extractables,leachables and particulate validation. Fully automated single-usesystems allow process control and data acquisition to meet orexceed the standards expected from traditional fixed equipment.New product highlights include microcarriers, pyrogen-free vialsand a range of pharmaceutical packaging.

SAFCSAFC is the custom manufacturing and services business unit ofSigma-Aldrich Corporation. As a trusted manufacturer for the lifescience and high technology industries, SAFC works closely withcustomers to resolve development challenges and accelerate theproduct pipeline. Its rich portfolio includes critical raw materials,contract manufacturing of viral vaccines and gene therapy drugproducts, and extensive biologics safety testing services.

apcethapceth is a pioneering clinical-stage biopharmaceuticalcompany expanding its pipeline of next-generation cell-basedtherapeutics.Our modular platform technology is based on genetically-modified MSCs and the lead program, Agenmestencel, is a first-in-man genetically-modified MSC, for the treatment for cancer. Inaddition, we are developing drug candidates for the treatment oflung diseases and inflammation.Based in Munich, we provide our know-how, expertise and GMP-certified facilities to industry and academic partners around theworld.

BioLife SolutionsBioLife Solutions develops, manufactures and marketsbiopreservation media and high performance thermal packagingproducts for cells, tissues, and organs. The Company's proprietaryHypoThermosol® and CryoStor® platform of solutions are highlyvalued in the biobanking, drug discovery, and regenerativemedicine markets. BioLife's products are serum-free and protein-free, fully defined, and are formulated to reduce preservation-induced cell damage and death. BioLife's enabling technologyprovides academic and clinical researchers significantimprovement in shelf life and post-preservation viability andfunction of cells, tissues, and organs. For more information pleasevisit www.biolifesolutions.com.

Invetech PtyInvetech is an innovative contract development companyworking with cell therapy and bioprocess companies to bring newproducts to market by combining our system definition tools,technology and automation expertise with our client's processknowledge. We develop appropriate scale-up strategies andinnovative platforms, including functionally closed systems, incollaboration with processors to meet their quality andtherapeutic goals.

Miltenyi BiotecMiltenyi Biotec provides innovative products and services thatadvance biomedical research and cellular therapy. Our cutting-edge tools support research at every level, from basic research totranslational research to clinical application. Used by scientists andclinicians around the world, our integrated technologies covertechniques of sample preparation, cell isolation, cell sorting, flowcytometry, cell culture, molecular analysis, and preclinicalimaging. Our more than 25 years of expertise spans research areasincluding immunology, stem cell biology, neuroscience, andcancer. Today, Miltenyi Biotec has more than 1,400 employees in25 countries – all dedicated to empowering discovery andimpacting lives.

Terumo BCTTerumo BCT, a global leader in blood component, therapeuticapheresis and cellular technologies, is the only company with theunique combination of apheresis collections, manual andautomated whole blood processing, and pathogen reductioncoupled with leading technologies in therapeutic apheresis andcell processing. We believe in the potential of blood to do evenmore for patients than it does today. This belief inspires ourinnovation and strengthens our collaboration with customers.Terumo BCT—Unlocking the Potential of Blood.

BrightlandsA unique location for R&D, scale up and manufacturing in Biomaterials, Tissue Engineering and Regenerative Medicine.Brightlands Chemelot Campus is a world-leading innovationlocation and home to a vibrant and fast-growing opencommunity of groundbreaking companies and knowledgeinstitutes.It offers state of the art R&D and manufacturing infrastructures, on-campus education and science-oriented business support,venture capital, and business development services.With its location, ecosystem and easily available expertise andknowledge, Brightlands Chemelot Campus is a unique location forinnovative start-ups and corporations, forward-thinking knowledgeinstitutes, daring entrepreneurs, brilliant researchers, talentedstudents, and visionary investors.

GOLD SPONSORS

SILVER SPONSORS

BRONZE SPONSORS

ROUNDTABLE SPONSOR


I found this year's Cell & Gene Therapyconference was probably the best conference Ihave ever been to on all levels – thepresentations, the networking and the venue.Dr Peter French, Chief Executive Officer, Benitec Biopharma Limited

FORUM 2015


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Phacilitate Cell & Gene Therapy Main Announcement

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