20276 cgt miami main brochure v2.qxp layout 1 18/10/2017 ......rubius therapeutics robert smith,...

22-25 JANUARY 2018 | HYATT REGENCY, MIAMI

THE WORLD'S NUMBER 1 PARTNERING EVENT FOR ADVANCED THERAPIES

Also part of the Phacilitate Leaders Forum

1200+Attendees

400+Organisations

200+Speakers

30+ Countries

Find out more at www.cellandgenetherapyworld.com

MEET OUR VISIONARY SPEAKERS!

Platinum sponsor

Katherine A. High, MD,President and ChiefScientific Officer,Spark Therapeutics

Maria T. Millan, MD,Interim President & CEO,California Institute forRegenerative Medicine(CIRM)

Naomi Aronson, PhD,Executive Director ofClinical Evaluation,Innovation and Policy in theOffice of Clinical Affairs,Blue Cross and BlueShield Association

Avak Kahvejian, PhD,Partner, FlagshipPioneering; ChiefInnovation Officer,Rubius Therapeutics

Robert Smith,Senior Vice President andEarly Commercial Lead,Gene Therapy ResearchUnit,Pfizer, Inc

Kapil Bharti, PhD,Stadtman Investigator, Uniton Ocular Stem Cell &Translational Research(OSCTR),National Eye Institute,NIH

Dr Hans Keirstead,Chief Executive Officer,AIVITA BiomedicalCandidate for USCongress

Timothy Moore,Executive Vice President ofTechnical Operations, Kite Pharma

Dr Pascal TouchonSenior Vice President,Global Head StrategyBD&L; Executive BoardMemberNovartis Oncology

Dr Tim Anderson,Managing Director,Global PharmaceuticalsEquity Research,Sanford C. Bernstein

Dr Greg Russotti,Vice President, TechnicalOperations,Celgene CellularTherapeutics

Dr Anthony Ridgway,Acting Director, Centre forEvaluation ofRadiopharmaceuticals andBiotherapeutics, Health Canada

SUPER EARLYBIRD ENDS

OCTOBER 20TH.HURRY!

20276 CGT Miami Main Brochure V2.qxp_Layout 1 18/10/2017 16:20

“Phacilitate is a must have event on

my calendar”Robert Deans, CTO, BlueRock Therapeutics

“Dynamic,interesting and

highly interactiveevent that promotes

exchange andnetworking in ahighly specialized

field of genetherapy.”

Nathalie Clement, Associate Director, Powell GeneTherapy Center, University of Florida

www.cellandgenetherapyworld.com


WELCOME TO THE WORLD'S LARGEST &MOST INFLUENTIAL MEETING FOR CELL

& GENE THERAPY!

A great way to expand network with global experts in cell and gene therapy who are facing similar challenges.

Steven Goodman, Director, Strategy and Engagement, GSK

PhacilitateLeaders Forum

Miami Phacilitate brings together

industry leaders to explore trends,opportunities, disruptions andmajor shifts on the horizon. Thisyear more than 1,200 decisionmakers and technical experts willunite to collectively shape,experience, and challenge thefuture of advanced therapies.

3 events for the price of 1

The only meeting that connects theENTIRE ecosystem. Our partnershipwith World Stem Cells Summit andco-location with Immuno-OncologyFrontiers World means that you cannow meet biopharma leaders as wellas KOLs from academia, science and

the clinic.

the largestshowcase of

ground-breakingsolutions!

Meet 150+ of the mostinnovative solution and

technology providers who willhelp you to overcome yourbusiness challenges!

“

“


DECISION MAKERS &

MUCH MORE!

Meet the technical guru's who willhelp streamline and advance yourbusiness processes. Brainstormwith 'super users' and join tailored

training sessions.

Setting a brand new standard fornetworking. From themed cocktailreceptions on the banks of the Miami

Canals, our Japan Partnering Lunch or ouralready-legendary Phacilitate Miami YachtParty, Phacilitate Leaders Forum 2018 willsupport your partnering activities like

never before.

Independent research from AMRInternational scored the event 4.5/5 - join'the world's leading partnering event'

“Rated Excellent”

Stay ahead of the game. Understandsuccessful business and

commercialization models. Identify futuretechnologies and trends. Capitalize on

opportunities!

Meet more decision makers in 4 days than you can in 4months! 1,200+ attendees, 200+ speakers, 100+ exhibitors –meet your next business partners, suppliers, and competitors.

PREMIUM PARTNERING IN THE WINTER SUN

Experience hands-on methods for finding creative solutions tobusiness problems. Take a journey through 4 dedicated

exhibition features - The Bioprocessing Hub, The Supply ChainHub , the Immuno-Oncology Hub and The Translation Academy.

HANDS-ON TECHNOLOGY DEMONSTRATIONS

FUN, EFFECTIVENETWORKING GENUINE MUST ATTEND PRICELESS STRATEGIC &

TACTICAL BUSINESS INSIGHT


JOIN AN INTERNATIONAL COMMUNITY OF INNOVATORS


Dr Evren Alici, CEO, Vycellix;Director, Cell & Gene Therapy Centre,Karolinska Institutet

Biren Amin, Managing Director &Senior Equity Research Analyst,Biotechnology, Jefferies LLC

Dr Tim Anderson, ManagingDirector, Global PharmaceuticalsEquity Research, Sanford C.Bernstein

Naomi Aronson, PhD, ExecutiveDirector of Clinical Evaluation,Innovation and Policy in the Office ofClinical Affairs,Blue Cross andBlue Shield Association

Dr Mark L. Bagarazzi, CMO,Inovio Pharmaceuticals

Stefano Baila, PhD,Director ofOperations & Business Development,Holding F.I.S.

Jakub Baran, CEO & COO,Famicord Group, Polski BankKomórek Macierzystych S.A.

Shirley Bartido, PhD, MBA,Director, Regulatory Affairs,Cellectis

Dr Nina Bauer, CommercialDevelopment, Autologous Therapies,Lonza, Inc

Dr John Beadle, CEO, PsiOxus

Dr Joanne Bedwell-Garner,Development Director, MacrophagePharma

Kapil Bharti, PhD, StadtmanInvestigator, Unit on Ocular Stem Cell& Translational Research (OSCTR),National Eye Institute, NIH

Diane Blumenthal,Head ofTechnical Operations, SparkTherapeutics

Greg Bonfiglio, Founder &Managing Partner, Proteus, LLC

Dr Christopher Bravery, Director,Advanced Biologicals Ltd

Lee Buckler, President & CEO,RepliCel Life Sciences, Inc

Catherine Cancian, MSc, M.Eng,MBA, Vice President,Pharmaceutical Operations,GenSight Biologics

Dr Stephen Cary, CEO, Omniox

Massimiliano Cesarini, GlobalSales Manager,Comecer Group

Doug Chambers, Head of Viral AndGene Therapy Operations,MilliporeSigma

Kenny Choi, Head of ProcessDevelopment & Tech Transfer,Mustang Bio

Laura Connelly-Smith,MBBCh,DM, Assistant MedicalDirector Apheresis and CellularTherapy, SCCA; Assistant Professor,Division of Hematology,UW;Assistant Member, FHCRC, SeattleCancer Care Alliance

David Courtman, PhD, Director,Biotherapeutics Core Facilities,Ottawa Hospital ResearchInstitute

Sybil Danby, Director of BusinessDevelopment, ParagonBioservices, Inc

Olivier Danos, PhD, Chief ScientificOfficer,REGENXBIO Inc.

Dr Anthony Davies, President,Dark Horse Consulting

Dr John E. Davies, Professor,University of Toronto; President,Tissue RegenerationTherapeutics, Inc

Dr Colleen Delaney, Founder andChief Medical Officer, NohlaTherapeutics

Dr Xavier de Mollerat du Jeu,Director R&D, Life Science Group,Thermo Fisher Scientific

Jerrod Denham, Principal & SeniorConsultant,Dark HorseConsulting, Inc

Boro Dropulic, PhD, MBA, ChiefScientific Officer & General Manager,Lentigen Technology, Inc AMiltenyi Company

Matthew Durdy, Chief BusinessOfficer, Cell and Gene TherapyCatapult

Dr Steve Elliman, CSO,OrbsenTherapeutics, Ltd

Simon Ellison, Cell and GeneTherapy Service Director,WorldCourier

Dr Nicole Faust, Chief ScientificOfficer, CEVEC Pharmaceuticals

Thomas Fellner, Head, CommercialDevelopment, Cell Therapy, LonzaWalkersville

Ed Field, President, brtxDISC™Division,BioRestorativeTherapies

Timothy C. Fong, PhD, MBA, VicePresident, Science & Technology,AllCells

Professor Miguel Forte, ChiefMedical Officer, BoneTherapeutics; Managing Director,mC4Tx (modified Cell forTherapeutics); ChiefCommercialisation Officer and Chairof Commercialisation Committee,ISCT

Marlin Frechette,Director,Regulatory Affairs/Quality Systems,Management Representative, IrvineScientific

Dr Francesco Galimi,GlobalProduct General Manager, EarlyDevelopment, Amgen, Inc

Dr Jason Gersting, Partner,Knobbe Martens

Anna Gilbert,Director, QualityAssurance, Nohla Therapeutics,Inc

Aniz Girach, MD, Chief MedicalOfficer, NightstaRx

Richard Grant, Global VicePresident, Cell Therapy, Invetech

Dr Christine Guenther, CEO &Medical Director, apcethBiopharma GmbH

Jon C. Gunther, PhD, AssociateDirector, Technical R&D, JunoTherapeutics

Heidi M. Hagen, Chief StrategyOfficer,Vineti

Patrick J. Hanley, PhD, LaboratoryFacility Director, Cellular Therapyand Stem Cell Processing, AssistantProfessor of Pediatrics, Children'sNational Health System, TheGeorge Washington University

Brian Hanrahan, Manager, CellTherapy, Invetech

Dorit Harati, Vice President, QualityAssurance, Manufacturing &Logistics,Gamida Cell

Dr Joshua M. Hare,Director,

Interdisciplinary Stem Cell Institute(ISCI), Louis Lemberg Professor ofMedicine,University of MiamiMiller School of Medicine

Dr Don Healey, Senior VicePresident, Operations, KBIBiopharma

Katherine A. High, MD, Presidentand Chief Scientific Officer, SparkTherapeutics

Cade Hildreth, BS, MS, President &CEO,BioInformant.com

Amy Hines, Senior Manager,Apheresis & Collection CenterNetwork Development, Be TheMatch Biotherapies

Dr Bent Jakobsen, CSO,Immunocore

Haiyan Jiang, PhD, Vice President,Preclinical Science, Editas Medicine

Karl K. Johe, PhD, Founder & ChiefScientific Officer,Neuralstem, Inc

Doug Jolly, PhD, Executive VicePresident Research & PharmaceuticalDevelopment, Tocagen, Inc

Louis Juliano, Senior Vice President,Global Sales & BusinessDevelopment,HemaCareCorporation

Dr Bo Kara, Head ProcessDevelopment, Cell & Gene TherapyPlatform CMC, GlaxoSmithKline

Mark A. Kay, MD, PhD, DennisFarrey Family Professor, Departmentsof Pediatrics and Genetics; Vice Chairfor Basic Research (Pediatrics),Stanford University

Avak Kahvejian, PhD, Partner,Flagship Pioneering; Chief InnovationOfficer,Rubius Therapeutics

Dr Hans Keirstead, Chief ExecutiveOfficer,AIVITA BiomedicalCandidate for US Congress

Dr Sven Kili, Vice President & Headof Cell & Gene Therapy Development,Rare Disease Unit,GlaxoSmithKline



JOIN AN INTERNATIONAL COMMUNITY OF INNOVATORS

David Kirn, MD, co-Founder,Chairman & CEO, 4D MolecularTherapeutics (provisionallyconfirmed)

Dr Karine Kleinhaus, DivisionalVice President, North America,Pluristem Therapeutics

Dr Wolfgang Knirsch, CEO,Vita34 AG

Thomas R. Kreil, PhD, AssociateProfessor of Virology, Head ofPathogen Safety, Shire

Dr Klaus Kühlcke, ManagingDirector, EUFETS GmbH

Dr Joanne Kurtzberg, Director,Carolinas Cord Blood Bank At DukeUniversity; President,Cord BloodAssociation

Dr Brian Lee, President and Co-Founder, PBS Biotech

Alain Lamproye, Chief ExecutiveOfficer,Yposkesi

Benjamin Le Quéré, PhD, BusinessDirector, Bioprocess Solutions, Saint-Gobain

Dr Yijia Li, Director, ShunxiRegenerative Medicine, YunnanProvince Stem Cell Bank

Dr Sheng Lin-Gibson, Leader,Biomaterials Group and Director,Regenerative Medicine Programs,NIST

Dr Ainslie Little,Director ofIntellectual Property,BlueRockTherapeutics

John Lunger, Vice President,Manufacturing & Supply Chain,Adaptimmune

Chris Mason, MD, PhD, CSO,AvroBio

Todd McAllister, PhD, ExecutiveDirector, Amnion Foundation

Dr Michael May, President & CEO,Centre for Commercialization ofRegenerative Medicine (CCRM)

Dr Mark Metzke, Associate,Knobbe Martens

Francois-Thomas Michaud, PhD,CEO, Feldan Therapeutics

Maria T. Millan, MD, InterimPresident & CEO, CaliforniaInstitute for RegenerativeMedicine (CIRM)

Dr Jeffrey Miller, Deputy Directorof the Masonic Cancer Center,University of Minnesota

Timothy J. Miller, PhD, President &CEO,Abeona Therapeutic

stefan Miltenyi, Founder &President, Miltenyi Biotec

Timothy Moore, Executive VicePresident of Technical Operations,Kite Pharma

Dr Shashi Murthy, Professor,Northeastern University

Dr Gopalan Narayanan, VicePresident, Disruptive Biologics,Voisin Consulting Life Sciences(VCLS)

Jessie H.-T. Ni, PhD, Chief ScientificOfficer, Irvine Scientific, JXTGEgroup

Scott Ohanesian, Senior VicePresident of Commercial Operations,Clinical Trial Logistics, QuickSTAT, AQuick Company

Peter Olagunju, Senior Director,Technical Operations, bluebird bio

Dr Biju Parekkadan, AssociateProfessor, Bioengineering, RutgersUniversity; co-Founder, SentienBiotechnologies, Inc

Akshay Peer, PhD, Vice President ofSales & Account Management,TrakCel

Robert Piperno,QA Director, Cell &Gene Therapies,GlaxoSmithKline

Dr Suyash Prasad, Chief MedicalOfficer & Senior Vice President,Audentes Therapeutics

Robert A. Preti, PhD, ChiefExecutive Officer & President, PCT

Dr Joseph Rabinowitz, SeniorDirector of AAV Capsid Development,Bamboo Therapeutics, Inc., awholly owned subsidiary ofPfizer Inc.

Dr Laszlo Radvanyi, Senior VicePresident Head of ImmunoOncologyTranslational Innovation Platform,EMD Serono

Dr Jim Richardson, Deputy Chief,Preclinical Translational Programs,Foundation Fighting Blindness

Dr Anthony Ridgway, ActingDirector, Centre for Evaluation ofRadiopharmaceuticals andBiotherapeutics,Health Canada(provisionally confirmed)

Rodney Rietze, PhD, TechnicalResearch & Development, Cell &Gene Therapy Unit, NovartisPharmaceutical Corporation

Donna Rill, Vice President ofManufacturing, Cell Medica

Dr Isabelle Riviere,Director,Michael G. Harris Cell Therapy & CellEngineering Facility,MemorialSloan Kettering Cancer Center

Dr Greg Russotti, Vice President,Technical Operations,CelgeneCellular Therapeutics

Dr R. Jude Samulski, VicePresident, Gene Therapy, BambooTherapeutics, Inc., a whollyowned subsidiary of Pfizer Inc.

Ziv Sandalon, PhD, AssociateDirector of Innovation & Translation,ABL, Inc.

Ryan Scanlon, Business Head, ViralTherapeutics, Lonza

Dr Joshua Schimmer, SeniorManaging Director, Evercore ISI

Dr Alaina Schlinker, Manager, CellTherapy Application Support,Fresenius Kabi

Albert B. Seymour, PhD, ChiefScientific Officer, HomologyMedicines, Inc

Dr John Sharpe, Chief TechnologyOfficer,Cytonome

Dr Beth Shaz, Chief Medical &Scientific Officer,New York BloodCenter

Elizabeth J. Shpall, MD, Professor,Howard and Lee Smith Chair inCancer Research; Director, CellTherapy Laboratory and Cord BloodBank; Deputy-Chair, DepartmentStem Cell Transplantation andCellular Therapy,University ofTexas MD Anderson CancerCenter

Dr Euan Slorach, Senior Scientist,Caribou Biosciences

Dr Alan K. Smith, Executive VicePresident, Technical Operations,Bellicum Pharmaceuticals

Robert Smith, Senior Vice Presidentand Early Commercial Lead, GeneTherapy Research Unit, Pfizer, Inc

Richard O. Snyder, PhD, ChiefScientific Officer, Brammer Bio

Rizwana F. Sproule, PhD, VicePresident, Regulatory Affairs,KitePharma, Inc

Dr David F. Stroncek, Chief, CellProcessing Section, Department ofTransfusion Medicine, NIH ClinicalCenter

Nicolas Theys, PhD, ChiefOperating Officer, NovadipBiosciences

Dr Jan Thirkettle, ChiefDevelopment Officer, FreelineTherapeutics

Dr Mark Throsby, CSO,Merus

Dr Pascal Touchon, Senior VicePresident, Global Head StrategyBD&L; Executive Board Member,Novartis Oncology

Luk H. Vandenberghe, PhD,Director, Grousbeck Gene TherapyCenter, Mass Eye and Ear, AssistantProfessor,Harvard Medical School

Dr David Venables, CEO,Synpromics

Frances Verter, PhD, Founder &Director, Parent's Guide to CordBlood Foundation &CellTrials.org

Adam Villa, Director, ClinicalProduct Logistics, CRISPRTherapeutics

Deborah Wild, Vice President ofQuality & Regulatory Affairs,Paragon Bioservices, Inc

Mark Zimmerman, PhD, VicePresident, Strategy and BusinessDevelopment, Interim Vice Presidentof Operations, ViaCyte

Sanjin Zvonic, PhD, Senior Director,Business Leader, Clinical &Commercial Manufacturing, PCT

Claudia Zylberberg, PhD, CEO,Akron Biotech


PARTNERING TABLES

TRANSLATION ACADEMY WITH CCRM

IO HUB

724718

617615

716710

611609

706704 702

700

601603605

708

707 705 703 711

618616

614

519517

515

610

511

501

411

508

409

504502

401403

405

418416

414

319317

315

410408

311309

406

305

303

318316

314

219

217215

210308

211209

109

206

107

815814

715717

722720

714712

808

709

THE BIOPROCESS HUB

PHACILITATE SALES OFFICE


EXPERIENCE THE WORLD'S LARGEST SHOWCASE OF CELL & GENE THERAPY PIONEERS

206 Brooks Life Science

209 BioLife Solutions

210 Akron Biotech

211 Comprehensive CellSolutions

215 Wilson Wolf

303 Paragon Bioservices

305 Saint-Gobain Life Sciences

308 Key Biologics

309 MedCision

311 Eufets

314 The Cell and Gene TherapyCatapult

315 World Courier

319 Waisman Biomanufacturing

401 Sony Biotechnology

403 Quick· SpecializedHealthcare Logistics

405 Cobra Biologics

406 PCI Clinical Services

408 Fresenius Kabi

409 CellGenix

410 Lonza Biologics

411 PCT

414 TrakCel

416 G-CON Manufacturing

418 Sartorius Stedim

501 Thermo Fisher Scientific

502 KBI BioPharma

504 GE Healthcare

508 Irvine Scientific

511 Brammer Bio

515 Miltenyi Biotec

517 Diamond Pharma Services

519 ABL

601 PendoTECH

603 WuXi AppTec

605 Biocair

609 PeproTech

610 PBS Biotech

611 Bio-Techne

614 Vineti

615 ChemoMetec A/S

616 BioSpherix

617 Yposkesi

618 Terumo BCT

700 Be The MatchBioTherapies®

702 Hamilton Storage

706 Aldevron

707 CEVEC PharmaceuticalsGmbH

709 Myriad RBM

710 MAK-SYSTEM

711 Corning Inc

712 OmniSeq

714 VGXI Inc

715 Becton Dickinson

716 Blood Centers of America

718 Comecer S.p.A

720 MaxCyte

The Translation Academy: In partnering with CCRM, explore

industry-academic convergence, learnabout promising innovation andunderstand the pathways to

commercialsuccess, focusing on thethemes of:• Financing

• Manufacturing• Regulatory

• Reimbursement

The Supply Chain Hub: A vein-to-vein experiential journey throughpatient registration to transit, storage and

point of care administration

The I-O Hub:Gain expert insights from solution providersand technology pioneers who are key to the

progress of pre-clinical and clinicaldevelopment in I-O.

The Bioprocess Hub:Everything that you need to know aboutthe cutting edge solutions availableacross harvesting, cell expansion,purification, sorting and QA/QC


Full Day Comparability Workshop

Christopher Bravery9:00 – 17:00, Monday 22 January

$895Change is inevitable and necessary both in development and overthe post-approval product lifecycle. Whenever changes are made itis necessary to confirm they do not adversely impact the quality andtherefore safety and efficacy of the product; this requires databeyond meeting current specifications. With any biological productthis is challenging, for cell, gene and tissue products that cannot befully characterised the challenges are greater still. Concerns aboutcomparability undertaken during development are common issues atapproval with significant comparability concerns raised during 12 ofthe 17 ATMP submissions to the EMA. Such questions often delaymarket approval or contribute to failure to get approval. This courseexplains what comparability is and how to develop a successfulcomparability protocol.

Workshop Learning Overview:• What is comparability?• Why is meeting existing specifications not comparability?• How do I apply the principles of comparability to highly variableproducts?

• Case studies: Common mistakes with comparability and theirconsequences.

• Interactive exercise: Spot the weaknesses and proposeimprovements to a worked comparability study

Half Day Quality-by-Design

Anthony Davies & Jerrod Denham9:00 – 12:30, Monday 22 January

$495Quality by Design (QbD) is a science- and risk-based approach topharmaceutical development. Applied to manufacturing processes, itis intended to ensure a rational and cost-effective approach toproduct quality. Both the EMA and FDA have systematicallyassessedQbD elements of regulatory filings and, especially since theFDA’s 2011 Process ValidationGuidance and the ICH’s Q8 through Q11, it has become an intrinsicpart of regulatory guidance.

However, understanding and adoption of the principles of QbD hasremained relatively limited in the field of cell and gene therapy. Westrongly believe that its methodologies are critical to the successfuldevelopment of ATMPs and that they should be incorporated asearly as possible into the science of manufacturing. This workshopwill both demystify the fundamentals of QbD and provide concreteexamples of its application to this field. You will leave with a crystal-clear understanding of P Diagrams, RAMM matrices, the QTPP andmany more tools which you can immediately use to improve yourproducts and processes.

Half Day Intellectual Property 101

Mark Metzke & Jason Gersting13:30 – 17:00, Monday 22 January

$495This session is geared towards providing an overview of intellectualproperty, including trademarks, design patents and utility patents,geared especially for those new to the process. The course willinclude a general overview of different types of IP that a companymay employ to develop a robust portfolio to build and protect theirbrand, as well as their contemplated commercial products. Thesession will emphasize utility patents (their specifications andclaims), different types of applications (provisionals, non-provisionals, continuations, divisionals, and continuations-in-part),and what patents do and do not allow patentees to do (enforcementversus the ability to practice patents). The session will cover the nutsand bolts of the patenting process from conception of an invention,to filing/claiming strategies, to prosecution, patenting, andconsiderations for continued filings, with an aim to touch ondomestic and international strategies at a high level. Timepermitting, an interactive session will be held where filing strategiesare developed for hypothetical inventive entities.

Key Benefits:• High level overview of different types of IP• Distilled overview of utility patents and the patenting process• Overview of patent application types including considerations onwhen to file

• Interactive session to reinforce the course

Half Day Authoring the Common Technical Document

Christopher Bravery11:15 – 15:30, Thursday 25 January

$495The ICH Common Technical Document was developed tostandardize the way information is shared with regulators, and isaccepted or mandatory by agencies following ICH (e.g. EU, US,Japan, Canada etc). The CTD is used for all types of medicinalproduct but the purpose of each section may not always be clear forATMP. This workshop aims to clarify:• What the purpose of each section is• Whether the section is descriptive or data-driven• What sorts of data are expected• Tips and considerations for style, use of figures and tables, etc.

Pricing Explained

Each course is available to book separately, or booktwo half day courses for $895 and save $95! Simply register for your conference pass and add yourchoice of courses to your basket.



Meet the Trainers

Dr Anthony Davies, President, Dark Horse ConsultingAfter training as a biochemist, chemical engineerand molecular biologist, Anthony Davies hasworked in the cell and gene therapy field for some20 years. Anthony is now Executive Chairman ofDark Horse Consulting, a boutique consultingpractice which he founded in 2014, where hefocuses on strategic CMC and productdevelopment issues in cell and gene therapy.


Christopher Bravery,Director, Advanced Biologicals LtdChristopher founded Consulting on AdvancedBiologicals Ltd at the end of 2009 in order to focushis activities within the Regenerative Medicinesector. Christopher has spent most of his careerworking in regenerative medicine; starting with aPhD in xenotranplantation immunology. Christopherwas involved with national implementation of thenew Advanced Therapies Regulation and alsoinvolved through his participation in the CHMP’s cellproducts working party (CPWP) in implementationat the EMA level including drafting guidelines.

Jerrod Denham, Principal & SeniorConsultant, Dark Horse Consulting, IncAfter training at UCLA in Molecular Biology andPhysiological Sciences, Jerrod Denham hasworked in the cell therapy industry for 18 yearswith increasing roles of responsibility. Jerrod isnow a Principal and Senior Consultant at DarkHorse Consulting, where he focuses on upstreamand downstream process engineering for cell andgene therapy products.

Mark Metzke, Associate, Knobbe MartensMark A. Metzke has been an intellectual propertyattorney at Knobbe Martens since 2011. He has aclient practice with a focus on patent prosecutionand strategic portfolio management in thepharmaceutical and biotechnological fields. Markearned his Ph.D. from at the University ofCalifornia, Irvine, researching synthetic genedelivery vectors and biomaterials. He earned hisJ.D. from UCLA Law, where he focused onintellectual property law and was the Editor-in-Chief of the Journal of Law and Technology.

Jason Gersting, Partner, Knobbe MartensJason Gersting is a partner in the Orange Countyoffice. He focuses on patent and trademarkprosecution and strategic intellectual propertyportfolio management in the biotechnology andlife science industries. Dr. Gersting earned hisPh.D. and J.D. from the University of Florida,where his research focused on role of fetalneuroendocrine signaling and prostaglandins inthe initiation of labor and delivery at theconclusion of pregnancy. He also earned hismaster’s degree by developing methods to analyzedifferential expression of calcium channels inresponse to disease or injury.


AGENDA

7.30 Registration & Buffet Breakfast in the Exhibition Area

9.00 Chair’s introductionChris Mason, MD, PhD, CSO,AvroBio

9.05 2017’s Top 10 Events: Celebrating recent progress in the field whilst putting it into contextEd Field, President, brtxDISC™ Division, BioRestorative Therapies

9.20 Keynote Address - Reviewing the development stories and market access plans for 2017’s most high-profile product candidates Katherine A. High, MD, President and Chief Scientific Officer, Spark Therapeutics

9.40 Practical HTA and market access insights and solutions for current and future approved cell & gene therapy products Panellists:Naomi Aronson, PhD, Executive Director of Clinical Evaluation, Innovation and Policy in the Office of Clinical Affairs, Blue Cross and Blue Shield AssociationDr Sven Kili, Vice President & Head of Cell & Gene Therapy Development, Rare Disease Unit, GlaxoSmithKlineDr Jim Richardson,Deputy Chief, Preclinical Translational Programs, Foundation Fighting Blindness

10.30 How to drive the cost-effective execution of cell & gene therapy commercialization at the operational level? Moderator:Matthew Durdy, Chief Business Officer,Cell and Gene Therapy CatapultPanellists: Rodney Rietze, PhD, Technical Research & Development, Cell & Gene Therapy Unit,Novartis Pharmaceutical CorporationRichard Grant,Global Vice President,Cell Therapy, Invetech

11.00 Morning Coffee in the Exhibition Area

Opening plenary session

2017: The Year Everything Changed


DAY 1: TUESDAY, JANUARY 23

4.30 Registration for Phacilitate Cell & Gene Therapy World 2018 opens

6.00 Plenary Super SessionChanging modern medicine (Shared with I-O Frontiers 2018)Chair: Dr Anthony Davies, President,Dark Horse Consulting

A series of Fireside Chats with:Dr Pascal Touchon, Senior Vice President, Global Head Strategy BD&L; Executive Board Member,Novartis OncologyDr Tim Anderson, Managing Director, Global Pharmaceuticals Equity Research, Sanford C. BernsteinDr Hans Keirstead, Chief Executive Officer, AIVITA BiomedicalCandidate for US CongressStefan Miltenyi, Founder & President,Miltenyi Biotec

7.30 Gala Welcome Drinks Reception on The Terrace

Sponsored by

For full conference agenda www.cellandgenetherapyworld.com/agenda

PRE-CONFERENCE DAY:MONDAY, JANUARY 22


AGENDA


4 parallel breakout tracks for the rest of the day:

Defining best practices forCost of Goods control

Cell-based Therapy Track

Gene Therapy Track

Evaluating I-O Responders vs non-responders: Paving the way to higher

patient response rates

Emerging Science Track: O

ncology

Supply Chain Track see page 17

11.30Bioprocess R&D, scale up/out,commercial facility planning andregulatory lessons learned by celltherapy industry leaders• Bioprocess automation – identifying

current and future key priority areas fortechnology development

• Bioprocess acceleration (eg. of celltransduction) – where can one feasibly savetime, therefore reducing cost and risk,currently?

• Creating bioprocess-related efficienciesaround quantities of consumables andmaterials required

Chair: Professor Miguel Forte, ChiefMedical Officer, Bone Therapeutics;Managing Director,mC4Tx (modified Cellfor Therapeutics); Chief CommercialisationOfficer and Chair of CommercialisationCommittee, ISCTSpeakers: Rodney Rietze, PhD, Technical Research& Development, Cell & Gene Therapy Unit,Novartis Pharmaceutical CorporationTimothy Moore, Executive Vice Presidentof Technical Operations,Kite PharmaHeidi M. Hagen, Chief Strategy Officer,VinetiDr Brian Lee, President and Co-Founder,PBS BiotechBenjamin Le Quéré, PhD, BusinessDirector, Bioprocess Solutions, Saint-Gobain

How to address questions and challengesaround gene expression longevity/control,

immunogenicity and re-dosing in in vivo gene therapy?

11.30Comparing and contrasting differentAAV serotypes and novel capsidmodification-based approaches • Is immunogenicity a real problem?• How are recent breakthroughs ingene expression monitoring enablingthe space?

• How are the sector’s R&D leadersapproaching the re-administrationconundrum?

• What to the FDA would a morestandardized scenario look like forAAV gene therapy clinical application?

Chair: Dr Gopalan Narayanan, VicePresident, Disruptive Biologics, VoisinConsulting Life Sciences (VCLS)Speakers: Dr R. Jude Samulski, Vice President, GeneTherapy, Bamboo Therapeutics, Inc., awholly owned subsidiary of Pfizer Inc.Olivier Danos, PhD, Chief ScientificOfficer,REGENXBIO Inc.Luk H. Vandenberghe, PhD, Director,Grousbeck Gene Therapy Center, Mass Eyeand Ear, Assistant Professor, HarvardMedical SchoolAniz Girach, MD, Chief Medical Officer,NightstaRxDavid Kirn, MD, co-Founder, Chairman &CEO, 4D Molecular Therapeutics(provisionally confirmed)Dr David Venables, CEO, Synpromics

11.30Chairs Introduction

11.35Predictive Biomarkers in CancerImmunotherapyJeff Evelhoch, VP, Head of TranslationalBiomarkers,Merck ResearchLaboratories

11.50An Immune Report Card to Enrich forImmunotherapy Clinical TrialsMark Gardner, CEO,OmniSeq, Inc

12.05MRI tools for detection of clinical celltherapy products post-transferProfessor Eric Ahrens, Professor ofRadiology,University of California SanDiego

12.25Panel: How do we better understandresponse patterns to identify andmonitor clinical end points in immuno-oncology? Panellists: Speakers of the session


13.00Close of session - Buffet Lunch in the Exhibition Area

OR

Lunch Workshop Sponsored byThe Influence of the Apheresis Product on CAR T-cell ManufacturingSpeaker: Laura Connelly-Smith, MBBCh,DM, Assistant Medical Director Apheresis and Cellular Therapy, SCCA; Assistant Professor, Division of Hematology, UW; Assistant Member, FHCRC, Seattle Cancer Care Alliance

OR

Roundtable discussion -Discussion of the major AAV manufacturing platforms

(Highly interactive small group discussion for a maximum of 12 participants)Moderator: Sybil Danby, Director of Business Development, Paragon Bioservices, Inc


AGENDA


Securing sufficient manufacturingcapacity for a commercializing sector –what are the most viable short- and long-

term solutions?Part 1 - Outsourcing


Gene Therapy Track

Back to Basics: The road map torationalising combination trial designs for

clinical success


ncology


14.00How are CMO consolidation trends andbusiness/ collaboration models set toevolve (eg. the ‘one-stop shop’)? • What will be the impact for cell &gene therapy developers? On Cost ofGoods?

• When is/isn’t the right time toapproach a CMO?

Sanjin Zvonic, PhD, Senior Director,Business Leader, Clinical & CommercialManufacturing, PCTMatthew Durdy, Chief Business Officer,Cell and Gene Therapy CatapultThomas Fellner,Head, CommercialDevelopment, Cell Therapy, LonzaWalkersvilleDr Alan K. Smith, Executive VicePresident, Technical Operations, BellicumPharmaceuticals

Non-viral vectors: What are the pros andcons in clinical application compared to viral

platforms?

14.00What are the advantages/disadvantagesand common issues with the variousplatforms? (Eg. RNA, plasmid DNA,Sleeping Beauty) • What technologies can potentially be‘re-purposed’ for non-viral genetherapy applications?

Mark A. Kay, MD, PhD, Dennis FarreyFamily Professor, Departments of Pediatricsand Genetics; Vice Chair for Basic Research(Pediatrics), Stanford UniversityDr Mark L. Bagarazzi, CMO, InovioPharmaceuticalsFrancois-Thomas Michaud, PhD, CEO,Feldan Therapeutics

14.05Chairs Introduction

14.05Rational Combinations in anIr-Rational WorldAiman Shalabi, Chief Medical Officer,Venture Fund, Clinical Accelerator,CancerResearch Institute

14.20Addressing the Tidal Wave of CancerImmunotherapy Combinations ThroughInnovative Trial DesignsDr William Grossman, GlobalDevelopment Team Leader Tecentriq - GI &Cancer Immunotherapy Combinations -SolidTumors,Genentech

14.35Presentation reserved

14.50Debate: Rational Trial Design vsEmpirical Science• Which application provides mostcommercial benefit in the eyes ofindustry and regulators?

Speakers include:Dr William Grossman, GlobalDevelopment Team Leader Tecentriq - GI &Cancer Immunotherapy Combinations -SolidTumors,GenentechDr Roy Baynes, SVP & Head GlobalClinical Development, Chief Medical Officer,Merck Research Laboratories

15.30Afternoon Tea in the Exhibition Area

OR

Technology Showcase sponsored by Jessie H.-T. Ni, PhD, Chief Scientific Officer, Irvine Scientific, JXTGE group

Securing sufficient manufacturing capacityfor a commercializing sector – what are themost viable short- and long-term solutions?

Part 2 - In-house


Gene Therapy Track

Engaging with precision medicine: Adoptingthe use of immuneprofiling andbioinformatics to enhance

clinical development


ncology


16.00Just how realistic an option is ‘do ityourself’ bioprocessing for cell & genetherapy developers today? • To what extent have recenttechnological innovations (eg. closed,disposable bioprocessing solutions)opened the door to in-housemanufacture?

Gene editing platforms in therapeuticapplication

16.00What do we know so far about theefficiency/accuracy/safety of the variousgene editing platforms in both in vivoand ex vivo therapeutic applications? • How to remodel the existingpreclinical paradigm to enable thedevelopment of gene editingmedicine?

• Comparing the clinical trial protocolsof trailblazers

16.00Chairs IntroductionDr Masahide Yano, Research Scientist,Center for Drug Evaluation & Research,FDA (Provisionally confirmed)

16.05Genomics- a Silver BulletDr William Loging, Head, ProductionBioinformatics, Genetics & GenomicsSciences, Icahn School of Medicine,Mount Sinai



AGENDA


Securing sufficient manufacturing capacityfor a commercializing sector – what are themost viable short- and long-term solutions?

Part 2 - In-house


Gene Therapy Track

Engaging with precision medicine: Adoptingthe use of immuneprofiling andbioinformatics to enhance

clinical development


ncology


• How to address the key remaininglimitations (eg. scalability, ‘open’ inter-unit operation transitions andsampling, etc)?

• How do we link in-housemanufacturing sites?

Jon C. Gunther, PhD, Associate Director,Technical R&D, Juno TherapeuticsDorit Harati, Vice President, QualityAssurance, Manufacturing & Logistics,Gamida CellKenny Choi,Head of Process Development& Tech Transfer, Mustang BioMassimiliano Cesarini,Global SalesManager,Comecer Group

Gene editing platforms in therapeuticapplication

• How do we match genome editingtechnologies with viral and non-viralvector delivery platforms?

Albert B. Seymour, PhD, Chief ScientificOfficer, Homology Medicines, IncHaiyan Jiang, PhD, Vice President,Preclinical Science, Editas Medicine

16.20Presentation Dr Steven Anderson, Chief ScientificOfficer, Covance; Senior Vice President,Laboratory Corporation of America

16.35PresentationDr Scott Bornheimer, Applied researchmanager,BD Biosciences

16.50A Platform for the Discovery andValidation of Immune Gene ExpressionSignatures to Enhance CancerImmunotherapyDr. James J. Mulé, Associate CenterDirector, Translational Research, Director ofCell-Based Immunotherapies, MoffittCancer Center

17.05Panel: Transforming datasets intoactionable informationPanellists: speakers of the session

17.30Close of Day 1 – Drinks Reception in the Exhibition Area

DAY 2:WEDNESDAY, JANUARY 24

7.15Registration & Buffet Breakfast in the Exhibition Area

OR

7.30Breakfast Briefing sponsored byChallenges of cell therapy automation: a perspective on disposablesBenjamin Le Quéré, PhD, Business Director, Bioprocess Solutions, Saint-Gobain

OR

Breakfast Briefing sponsored by Exploring the value of proprietary platforms in progressing your therapyFacilitator: Brian Hanrahan, Manager, Cell Therapy, InvetechPanellists include:Rodney Rietze, PhD, Technical Research & Development, Cell & Gene Therapy Unit, Novartis Pharmaceutical CorporationTimothy Moore, Executive Vice President of Technical Operations, Kite PharmaKim Warren, PhD, Head of Operations, AvroBio Inc.



AGENDA

Followed by your choice of 5 parallel breakout tracks:

8.45 Chair’s introduction

8.50 Investor Keynote Panel: tips for successfully navigating today’s uncertain financing environmentPanellists:Biren Amin, Managing Director & Senior Equity Research Analyst, Biotechnology, Jefferies LLCDr Joshua Schimmer, Senior Managing Director, Evercore ISIGreg Bonfiglio, Founder & Managing Partner, Proteus, LLCDr Jim Richardson, Deputy Chief, Preclinical Translational Programs, Foundation Fighting Blindness

9.40 2017: the year of the partnership! How diverse key cell & gene therapy’s stakeholders are pulling together to build the foundations for a bright commercial futureSpeakers/panellists:Robert Smith, Senior Vice President and Early Commercial Lead, Gene Therapy Research Unit, Pfizer, IncMaria T. Millan, MD, Interim President & CEO, California Institute for Regenerative Medicine (CIRM)Dr Sheng Lin-Gibson, Leader, Biomaterials Group and Director, Regenerative Medicine Programs, NIST


Morning Plenary Session

Macro economic insights and innovative collaboration models to drive cell & gene therapy’s ongoing maturation

Tour of the Bioprocess Hub


Gene Therapy Track

Cardiomyocytes, pancreatic islet cells,MSCs… do the latest data support the

recent revival in regenerative medicine? Emerging Science Track: O

ncology


11.15This session will be staged in our brandnew Bioanalytics Hub in the exhibitionarea – an experiential journey to thecutting edge in comparability, potency,stability, sterility and purity testin

OR, running concurrently:

Panel: What is the desired future statefor contract manufacturing for celltherapy?• Lessons from CMOs in mature fieldsModerator: Sanjin Zvonic, PhD, SeniorDirector, Business Leader, Clinical &Commercial Manufacturing, PCT

How to address viral and non-viralvector manufacturing capacity/supplyshortfalls before they reach crisis point?

11.15Defining both the scale of thechallenge and the strategic solutionsavailable today and in the near future…for AAV…for lentiviral…for retroviral…for plasmid DNA• In-house or outsource? Weighing upthe key considerations of each whenseeking to guarantee sufficient/scalable and secure vector supply

• What will the optimal vectormanufacturing facility of the futurelook like?

Chair: Boro Dropulic, PhD, MBA, ChiefScientific Officer & General Manager,Lentigen Technology, Inc A MiltenyiCompanyRyan Scanlon, Business Head, ViralTherapeutics, LonzaDr Nicole Faust, Chief Scientific Officer,CEVEC PharmaceuticalsDr Klaus Kühlcke,Managing Director,EUFETS GmbH Ziv Sandalon, PhD, Associate Director ofInnovation & Translation,ABL, Inc.Dr Joseph Rabinowitz, Senior Directorof AAV Capsid Development,BambooTherapeutics, Inc., a wholly ownedsubsidiary of Pfizer Inc.Dr Xavier de Mollerat du Jeu, DirectorR&D, Life Science Group, Thermo FisherScientific

11.15Case studies & audience Q&A• Are we finally getting to the bottomof theunpredictability/inconsistency inefficacy data from clinicalapplication?

• What progress in betterunderstanding Mechanisms ofAction?

• How to build more efficacy/potencyinto the product profile?

Dr Joshua M. Hare, Director,Interdisciplinary Stem Cell Institute (ISCI),Louis Lemberg Professor of Medicine,University of Miami Miller School ofMedicineNicolas Theys, PhD, Chief OperatingOfficer, Novadip BiosciencesDr Biju Parekkadan, AssociateProfessor, Bioengineering, RutgersUniversity; co-Founder, SentienBiotechnologies, Inc

Cell-based therapy track : Tour of the Bioprocess Hub: An experiential journey which highlights the cutting edge solutions available across harvesting, cell expansion, purification, sorting and QA/QC


Cord Blood & Perinatal Stem Cells Track see page 16

13.00Buffet Lunch in the Exhibition Area

OR

Workshop sponsored by

OR

Japan Partnering Lunch



AGENDA


Supply Chain Track: Tour of the Supply Chain Hub:A vein-to-vein experiential journey through patient registration to transit, storage and point of care administration

How to rationalize QCfor commercial scale?


Gene Therapy Track

Genetically modified stem cells – hope or hype?


ncology


14.00Where can improvements be made atthe strategic level to the three keyelements: cost, speed andreproducibility of results? • What are the learnings we can takeas a sector from recent BLA filings interms of expectations andrequirements around quality,process controls, etc?

• What to do about extractables andleachables?

• How to accelerate release testingwithout compromising onrobustness?

• Driving regulatory guideline andstandards development

Dr Greg Russotti, Vice President,Technical Operations,Celgene CellularTherapeuticsStefano Baila, PhD, Director ofOperations & Business Development,Holding F.I.S.Dr Sheng Lin-Gibson, Leader,Biomaterials Group and Director,Regenerative Medicine Programs, NISTAnna Gilbert, Director, QualityAssurance,Nohla Therapeutics, IncShirley Bartido, PhD, MBA, Director,Regulatory Affairs, CellectisDr Christine Guenther, CEO & MedicalDirector, apceth Biopharma GmbH Doug Chambers, Head of Viral And GeneTherapy Operations,MilliporeSigma

Highlighting and addressing ‘critical path’issues in bringing vector manufacturingprocesses from Phase I/II to commercial

scale

14.00Maturation of processes, analytics, andspecifications as the manufacture ofvector products progresses towardslate stage clinical trials and commerciallaunch • Process characterization andscalability strategies

• Process control and validationstrategies

• Assay qualification and validationstrategies

• Vector product profiles andspecifications

Chair:Richard O. Snyder, PhD, Chief ScientificOfficer,Brammer BioSpeaker:Diane Blumenthal,Head of TechnicalOperations, Spark TherapeuticsDr Jan Thirkettle, Chief DevelopmentOfficer, Freeline Therapeutics Catherine Cancian, MSc, M.Eng, MBA,Vice President, PharmaceuticalOperations, GenSight BiologicsDoug Jolly, PhD, Executive VicePresident Research & PharmaceuticalDevelopment, Tocagen, Inc

14.00Do the latest preclinical and clinicaldata suggest an answer to the efficacyconundrum is within reach?• Has the time come for iPS celltherapies? (How to address theremaining issues? What are the leastrisky potential applications?)

• HSCs• MSCsKapil Bharti, PhD, StadtmanInvestigator, Unit on Ocular Stem cell &Translational Research (OSCTR),National Eye Institute, NIH Avak Kahvejian, PhD, Partner, FlagshipPioneering; Chief Innovation Officer,Rubius TherapeuticsDr Christine Guenther, CEO & MedicalDirector, apceth Biopharma GmbH



15.30Afternoon Tea in the Exhibition Area

Sense-checking product characterizationassay development – which tools

do we really need?


Gene Therapy Track

Tracking the migration of regenerativemedicine to new therapeutic areas and

indications


ncology


16.00Optimizing current assays and next-generation bioanalytics: what are thekey changes needed to legacypharmaceutical/biological qualitysystems currently employed in the celltherapy space? • Potency – what are the best assaysfor cell therapy? How relevant ispotency to clinical efficacy?

• What are the latest technologicaladvances (in the sensor field, forinstance)? And what are the criticaltechnologies or assays still missingfrom the toolbox?

• Defining next steps in drivingautomation: which assays should webring in-process? And how will weintegrate in-process and externalassays?

Comparing and contrasting current andemerging gene therapy productionplatforms and characterization tools

16.00• Which platform(s) will provide thefuture gold standard for robust,reproducible and cost effectiveproduction of viral vectors fromproducercell lines?

• Gene therapy productcharacterization and potency testing

Timothy J. Miller, PhD, President &CEO, Abeona TherapeuticsAlain Lamproye, Chief Executive Officer,YposkesiDr Joseph Rabinowitz, Senior Directorof AAV Capsid Development, BambooTherapeutics, Inc., a wholly ownedsubsidiary of Pfizer Inc.Deborah Wild, Vice President of Quality& Regulatory Affairs, ParagonBioservices, Inc

16.00Case studies & audience Q&A• What is the clinical justification todate for hopes pinned to diabetes andCNS applications, for instance?

• Exploring new frontiers in aestheticmedicine

Mark Zimmerman, PhD, Vice President,Strategy and Business Development,Interim Vice President of Operations,ViaCyteDavid Courtman, PhD, Director,Biotherapeutics Core Facilities,OttawaHospital Research InstituteKarl K. Johe, PhD, Founder & ChiefScientific Officer, Neuralstem, IncLee Buckler, President & CEO,RepliCelLife Sciences, Inc



AGENDA


17.30Yacht Party

DAY 3: THURSDAY, JANUARY 25

7.30 Registration & Buffet Breakfast in the Exhibition Area

Morning Plenary Session

Finding the perfect balance between regulatory risk and opportunity: vital insights for a globally-minded sector


Sense-checking product characterizationassay development – which tools

do we really need?


Gene Therapy Track

Tracking the migration of regenerativemedicine to new therapeutic areas and

indications


ncology


Chair:Dr Christopher Bravery, Director,Advanced Biologicals LtdDr David F. Stroncek, Chief, CellProcessing Section, Department ofTransfusion Medicine,NIH ClinicalCenterSpeakers:Patrick J. Hanley, PhD, LaboratoryFacility Director, Cellular Therapy and StemCell Processing, Assistant Professor ofPediatrics, Children's National HealthSystem, The George WashingtonUniversityJon C. Gunther, PhD, Associate Director,Technical R&D, Juno TherapeuticsDr Shashi Murthy, Professor,Northeastern UniversitySpeaker from WuXi AppTec TBA

Comparing and contrasting current andemerging gene therapy productionplatforms and characterization tools


9.00 Chair’s introduction

Reviewing the pivotal trial design/regulatory review stories of 2017’s most high-profile product candidates in pivotal trials – what are the key regulatory insights andprecedents for the cell & gene therapy sector to take forward? (Eg, in terms of successfully navigating the Breakthrough Designation route to market approval?) Speakers to include:Rizwana F. Sproule, PhD, Vice President, Regulatory Affairs, Kite Pharma, Inc

9.40 21st Century Cures and the RMAT Designation, one year on - regulator and early user experiences: how has it been implemented?FDA Speaker TBA

10.15 Comparing and contrasting expedited development and conditional approval pathways, both within the US and worldwide Speakers to include:Dr Anthony Ridgway, Acting Director, Centre for Evaluation of Radiopharmaceuticals and Biotherapeutics, Health Canada(provisionally confirmed)



AGENDA




Emerging Science Track

How to truly enable decentralized manufacturing at commercialscale?

Pointing the way to future R&D success in rare indications

3 parallel breakout tracks:

11.15How comfortable are industry, regulators, clinicians andhealthcare providers alike with moving cell & gene therapybioprocessing closer to patients? Where do their chief remainingconcerns lie? • What would increasing point of care manufacturing mean forGMP oversight/levels, moving forward?

• How to (re-)design workflows around raw material acquisition,logistics and quality/release testing in the decentralizedscenario?

• How to deal with the thorny issue of demonstratingcomparability across multiple clinical sites?

• How far away is the fully sealed, ‘GMP in a box’ bedside solutionin reality?

Dr Bo Kara, Head Process Development, Cell & Gene TherapyPlatform CMC,GlaxoSmithKlinePeter Olagunju, Senior Director, Technical Operations, bluebirdbioDr Nina Bauer, Commercial Development, Autologous Therapies,Lonza, Inc

11.15Case studies• Preclinical strategy: critical insights for effective, rapidtranslation to First in Man

• Clinical trial design insights and data updates from globalleaders in AAV and non-viral vector-driven in vivo gene therapy

• How running Natural History of Disease studies isrevolutionising clinical trial designs in rare diseases

• Rare diseases: value of surrogate endpoints in clinical trials• Progress with targeted delivery and vector engineering: is thein vivo gene therapy field now ready to tackle ‘non-traditional’targets (eg . in the CNS space)?

Dr Suyash Prasad, Chief Medical Officer & Senior Vice President,Audentes TherapeuticsAniz Girach, MD, Chief Medical Officer,NightstaRxTimothy J. Miller, PhD, President & CEO,Abeona Therapeutics

13.00Buffet Lunch in the Exhibition Area

OR in the Translation Academy

Cell & Gene Therapy World in summary: Highlights and trends from this year’s eventSpeaker: Robert A. Preti, PhD, Chief Executive Officer & President, PCT


Emerging Science Track

How to truly enable decentralized manufacturing at commercialscale?

Pointing the way to future R&D success in rare indications

14.00Flexible, off the shelf vs. bespoke solutions? Unit operationsversus all-in-one? Can we define ‘best practices’ for the futuredevelopment of each?Dr Isabelle Riviere, Director, Michael G. Harris Cell Therapy & CellEngineering Facility,Memorial Sloan Kettering Cancer CenterDonna Rill, Vice President of Manufacturing,Cell MedicaKenny Choi, Head of Process Development & Tech Transfer,Mustang BioDr Don Healey, Senior Vice President, Operations,KBIBiopharmaDr John Sharpe, Chief Technology Officer, Cytonome

Session continues

15.30Close of Phacilitate Cell & Gene Therapy World 2018




AGENDA


CORD BLOOD & PERINATAL STEM CELLS

• Macro trends in investor, pharma and regulatory environments- Investor and pharma perspectives – how/where will we seek toengage in the cord blood and perinatal stem cell field in future?

- Global regulatory development updates: will recent evolution ofguidelines and legislation (eg, in China) translate to perinatal stem cellbanking and application opportunities?

• Analyzing evolving pubic and family cord blood and perinatal stemcell banking business models worldwide

- What do ongoing public-private convergence and M&A trends, andthe continuing development of hybrid business models, mean for therobustness of the banking sector globally?

- What is the relative health of the sector – and what are the emergingdominant business models – in North America, South America, Europeand Asia?

- The search for sustainability - weighing up the viability of emergingrevenue streams: how are banks, biotechs and tool/service providersinnovating to both realize and monetize the potential of cord blood &perinatal stem cells?

- Beyond the cord – how diversification into the wider biobanking arenais presenting new, sustainable business opportunities.

Business Models & Investment: WEDNESDAY, JANUARY 24TH

• Clinical updates: examining trial designs and the latest data forproduct candidates based on cord blood and perinatal stem cells

- Case studies: what is the latest evidence of therapeutic potential inspecific indications? (Cardiovascular diseases, autism, hypoxicischemic encephalopathy, etc.)

- What is the regulatory pathway for these candidates to marketapproval and ultimately, to patients?

- Perinatal stem cells vs. bone marrow-derived cells – what have beenthe outcomes from comparative studies to date?

- What are the future modalities and therapeutic areas/diseaseapplications to target? (Eg. Engineered perinatal cells)

• What progress in alleviating the cord blood & perinatal cell expansionbottleneck?

- Troubleshooting manufacture of perinatal cell and tissue-derived MSCs- How to enable closed transitions between cell selection, cellexpansion and cell harvesting unit operations?

- How to address the current shortfalls in testing guidance andavailable, fit-for-purpose assays?

- Closing roundtable discussion: How would we approach commercialscale up of a dedicated cord blood & tissue bioprocessing facility?(Where to start: small but scalable? Straight to full scale? Modular?)

Clinical Applications & Cell Expansion:THURSDAY, JANUARY 25TH

• Jakub Baran, CEO & COO, Famicord Group, PolskiBank Komórek Macierzystych S.A.

• Greg Bonfiglio, Founder & Managing Partner,Proteus, LLC

• Dr John E. Davies, Professor, University of Toronto;President, Tissue Regeneration Therapeutics, Inc

• Dr Colleen Delaney, Founder and Chief MedicalOfficer, Nohla Therapeutics

• Dr Steve Elliman, CSO,Orbsen Therapeutics, Ltd

• Dorit Harati, Vice President, Quality Assurance,Manufacturing & Logistics, Gamida Cell

• Cade Hildreth, BS, MS, President & CEO,BioInformant.com

• Dr Karine Kleinhaus, Divisional Vice President,North America, Pluristem Therapeutics

• Dr Wolfgang Knirsch, CEO,Vita 34 AG

• Dr Joanne Kurtzberg, Director, Carolinas CordBlood Bank At Duke University; President,Cord BloodAssociation

• Dr Yijia Li, Director, Shunxi Regenerative Medicine,Yunnan Province Stem Cell Bank

• Todd McAllister, PhD, Executive Director,AmnionFoundation

• Dr Beth Shaz, Chief Medical & Scientific Officer, NewYork Blood Center

• Elizabeth J. Shpall, MD, Professor, Howard and LeeSmith Chair in Cancer Research; Director, Cell TherapyLaboratory and Cord Blood Bank; Deputy-Chair,Department Stem Cell Transplantation and CellularTherapy,University of Texas MD AndersonCancer Center

• Frances Verter, PhD, Founder & Director, Parent'sGuide to Cord Blood Foundation & CellTrials.org

SPEAKERS INCLUDE:



AGENDA


• Facing up to critical raw materials supply shortfalls – the singlelargest elephant in the commercial cell & gene therapy room?

- Where specifically do/will the greatest shortfalls current exist and justhow significant are they in the context of expected clinical andcommercial demand over the short- to mid-term? (Eg. plasmids, cellculture media)

- How to develop the innovation and infrastructure needed toovercome raw material supply shortages whilst minimizing Cost ofGoods? (Eg. capitalizing on novel/emerging cell sources for the celltherapy industry)

• Starting material optimization for autologous and allogeneic cell &gene therapies

- Viral safety considerations for cell- and gene-based therapies- How to manage and standardize global/multi-site apheresis?Addressing quality and consistency challenges

- Defining best practice in allogeneic cell therapy donor supply andmanagement

• How to maximize efficiency in raw and starting materials qualitycontrol/characterization?

- Optimizing quality of raw and starting materials – what exactlyconstitutes ‘good enough’?

- Identifying and overcoming key challenges in raw and startingmaterials management for decentralized/point of care manufacturing(eg. of a CAR T cell immunotherapy)?

Raw & Starting Materials: TUESDAY, JANUARY 23RD

• Supply chain data management and integration - Integrated approach to management of CGT product supply chain- How to identify the data that is really critical to collect, and how tointegrate and utilize it to the benefit of the overall supply chain?

- Scaling up to commercial: how to automate the data managementpiece to allow the move from hundreds to thousands of patients?

- Track and trace and supply chain security – how to satisfy disparateregulatory requirements and standards on a multinational basis?

- Generic ‘off the shelf’ vs. tailored cell therapy supply chainmanagement software – analysing the practical utility/applicability ofboth options

• Tour of the Vein-to-Vein Supply Chain HubThis session will be staged in our expanded Supply Chain hub – anexperiential, circular journey along the entirety of the autologous cell-basedtherapy supply chain: from starting material obtainment/managementthrough cold chain logistics, bioprocessing, quality control, fill-finish andmanagement/ delivery to the patient back at the point of care.- Delivering a strategic roadmap for supply chain development andvalidation for patient-specific cell & gene therapies

- How to identify/predict and minimize the potential variables in yoursupply chain to achieve true consistency in product quality anddelivery?

- Leveraging supply chain strategies and technology to scale fromclinical to commercial volumes

• Supply chain scale up strategies and enabling technologies tominimize CoGs

- Sector leaders’ tips for designing and implementing efficient, scalablecell & gene supply chains

- Next generation cold chain technologies (including automated/closedsolutions) – what real improvements are they delivering in practice?(Eg. cost, quality, configurability, flexibility and ease of integration)

Cold Chain & Data Integration/Management:WEDNESDAY, JANUARY 24TH

• Stefano Baila, PhD, Director of Operations &Business Development,Holding F.I.S.

• Shirley Bartido, PhD, MBA, Director, RegulatoryAffairs, Cellectis

• Simon Ellison, Cell and Gene Therapy ServiceDirector, World Courier

• Timothy C. Fong, PhD, MBA, Vice President, Science& Technology, AllCells

• Marlin Frechette, Director, RegulatoryAffairs/Quality Systems, Management Representative,Irvine Scientific

• Anna Gilbert, Director, Quality Assurance, NohlaTherapeutics, Inc

• Amy Hines, Senior Manager, Apheresis & CollectionCenter Network Development, Be The MatchBiotherapies

• Louis Juliano, Senior Vice President, Global Sales &Business Development, HemaCare Corporation

• Thomas R. Kreil, PhD, Associate Professor ofVirology, Head of Pathogen Safety, Shire

• John Lunger, Vice President, Manufacturing & SupplyChain, Adaptimmune

• Timothy Moore, Executive Vice President ofTechnical Operations,Kite Pharma

• Scott Ohanesian, Senior Vice President ofCommercial Operations, Clinical Trial Logistics,QuickSTAT, A Quick Company

• Peter Olagunju, Senior Director, TechnicalOperations, bluebird bio

• Akshay Peer, PhD, Vice President of Sales & AccountManagement, TrakCel

• Robert Piperno, QA Director, Cell & Gene Therapies,GlaxoSmithKline

• Dr Alan K. Smith, Executive Vice President, TechnicalOperations,Bellicum Pharmaceuticals

• Adam Villa, Director, Clinical Product Logistics,CRISPR Therapeutics

• Claudia Zylberberg, PhD, CEO, Akron Biotech

SPEAKERS INCLUDE:


SUPPLY CHAIN FOR ADVANCED THERAPIES



JAPANESEPARTNERING

LUNCHThis dedicated networkingsession delivers a uniqueopportunity to enable newpartnerships and discussionswith attendees from Japan

Expanding on the success of last year's cord blood track, this year will feature a

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After 2017’s successful launch, we and ourpartners are teaming up together to empower

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TranslationAcademy

Phacilitate and Cell and Gene TherapyInsights have teamed up to further supportcollaboration between academic and industrypioneers. The Translation Academy will

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Yacht PartyOur legendary yacht party returns biggerand better than before for 2018! Ticketsare limited and will be allocated on a firstcome, first served basis so don't miss out!

THE BRAND NEWBIOPROCESS HUB......will highlight the cutting edge solutionsavailableacross harvesting, cell expansion,purification, sorting and QA/QC. Also join

MaSTherCell for a virtual reality walk through ofaGMP facility from the future!

Co-location with CELL & GENE

THERAPY World WorldStem Cell Summit

The only I-O event co-located with the leading Celland Gene Therapy and translational stem cell globalmeeting. Providing access to the most commerciallyadvanced technical expertise specifically for cellularimmunotherapies. As a combined event we will have1,200+ attendees, 200+ speakers, 100+ exhibitors –it is the one meeting on the global calendar attendedby all of the critical decision-makers from bothinside and outside of the cell & gene therapy/

immuno-oncology industry.


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1. 21st Century Cures and the RMAT Designation; one year on, how has it been implemented?

6. Investor insights: How to safeguardagainst the threat of becoming

technologically obsolete in today’s ‘next-gen’ environment?

2. Exploring monetization models forcurative therapies: what are the viable

models in payers’ eyes today?

7. How to rationalize QC for commercial scale?

3. How to address questions and challenges around gene expression

longevity/control, immunogenicity and re-dosing in in vivo gene therapy?

8. How to identify the data that is reallycritical to collect, and how to integrateand utilize it to the benefit of the overall

supply chain?

4. Beyond AML: What is being done toovercome solid tumor resistance to

cellular immunotherapies?

9. What do ongoing public-privateconvergence and M&A trends, and the

continuing development of hybrid businessmodels, mean for the robustness of theCord Blood banking sector globally?

5. Optimizing quality of raw and starting materials – what exactlyconstitutes ‘good enough’?

10. How to truly enable decentralizedmanufacturing at commercial scale?


20276 cgt miami main brochure v2.qxp layout 1 18/10/2017 ......rubius therapeutics robert smith,...

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