27-28 June 2017 SH Valencia Palace

Valencia | Spain

PDA Europe Conference, Exhibition

Advanced TherapyMedicinal Products

2 pda.org/EU-ATMPs2017

This year’s Conference on Advanced Therapy Medicinal Products (ATMPs) will be held in Valencia, Spain from 27-28 June 2017.

After many years of pioneering activities in cell and gene therapy with some ups and many downs, the field accel-erated all of a sudden with a number of major industry and investor activities.

A strong driver for these deals has been the development of CAR-T technology, enabling T-cells to be directed to kill any cells that express a particular antigen. This has proven to achieve long-lasting clinical effects in certain hematologic malignancies, while more development is needed to successfully apply CAR-T cell therapy to solid tumor settings. The attractiveness of the T-cell approach is clearly the potential of having complete and durable responses after a single treatment, with long-living T-cells providing immune surveillance including across the blood brain barrier. It is in this turmoil of industry and investment activity that makes discussing the many chal-lenges involved in the development, testing, production and registration of ATMPs as important and vital as ever.

We warmly invite your to join this amazing group for an event that has established itself to a unique and highly professional discussion forum!

We look forward to welcoming you to beautiful Valencia!

OVERVIEW

Dirk Groenewegen, PhD, Cells4Therapy, Conference Co-Chair

Margarida Menezes-Ferreira, PhD, Infarmed, Conference Co-Chair

S C I E N T I F I C P R O G R A M P L A N N I N G C O M M I T T E E

1 Dirk Groenewegen, Cells4Therapy, Chair

2 Margarida Menezes-Ferreira, Infarmed, Co-Chair

3 Manuel Carrondo, ibet

4 Fabio D’Agostino, University of Newcastle

5 Wilfried Dalemans, Tigenix

6 Giovanni Migliaccio, Eatris

7 Wenzel Novak, Optima Pharma

8 Valerie Pimpaneau, Voisin Consulting

9 Sol Ruiz, AEMPS

10 Jessica Kopf, Fraunhofer IPA

11 Margit Jeschke, Novartis

12 Georg Roessling, PDA Europe

13 Falk Klar, PDA Europe

1 2 3

4 5 6

7 8 9

10 11

12 13

S C H E D U L E A T A G L A N C E

26 June 9:00 – 17:30 Practical Application of GMP for Development of ATMPs

Training Course

27 June 9:00 – 17:30 Advanced Therapy Medicinal Products Conference, Exhibition

27 June 19:30 – 22:30 Networking Reception

28 June 9:00 – 16:30 Advanced Therapy Medicinal Products Conference, Exhibition

For latest information, please visit: pda.org/EU-ATMPs2017

4 pda.org/EU-ATMPs2017

CONFERENCE AGENDA

18 May 2017

Tuesday, 27 June 2017

9:00 Welcome and Introduction Georg Roessling, PDA Europe

Dirk Groenewegen, Chair, Cells4Therapy

Margarida Menezes-Ferreira, Co-Chair, Infarmed

9:05 KeynoteOverview of ATMPs Development

Jonathan Appleby, GSK Rare Diseases Cell and Gene Therapy

Session 1 GMP - Current Regulatory Status Moderator: Wilfried Dalemans, Tigenix

9:45 The GMP Guide Specific to ATMPs Rocio Salvador-Roldan, EU Commission

EU Assessor’s View Margarida Menezes-Ferreira, Infarmed

EU Inspector’s View Annie Rietveld, formerly PIC/S, Dutch Inspectorate

11:15 Coffee Break, Poster Session & Exhibition

Industry Perspective Speaker, tbc

GMP Experience from a CMO Perspective Marc Barthold, apceth Biopharma

12:45 Panel Discussion: GMP for ATMPs Moderator: Fabio D’Agostino, University of Newcastle

Panelists:• Inspectors• Assessors• Pharma Industry • Academia

13:30 Lunch Break, Poster Session & Exhibition

Session 2 ATMPs Development Moderator: Georg Roessling, PDA Europe

14:30 Developing a Manufacturing Control Strategy for ATMPs Christopher Bravery, Advanced Biologicals

GMP-conform Automatization for Manufacturing of CAR-expressing Effector Cells and Antigen-specific T-Cells

Ulrike Köhl, University of Hannover

15:30 Coffee Break, Poster Session & Exhibition

5pda.org/EU-ATMPs2017

CONFERENCE AGENDA

Potency Assays for MSC Products Marcel Hoefnagel, CBG-Meb

Alternative Methods for Sterility Testing Validation Requirements

Luís Meirinhos-Soares, Infarmed

Q&A, Discussion

17:30 Closing of Day 1 & Networking Reception

Networking ReceptionJoin us and your colleagues to enjoy drinks and conversation in a casual atmosphere.

6 pda.org/EU-ATMPs2017

CONFERENCE AGENDA

18 May 2017

Wednesday, 28 June 2017

9:00 Welcome Dirk Groenewegen, Chair, Cells4Therapy

9:05 KeynoteGene & Cell Therapy: Differences, Hurdles & Potential

Felix Hermann, apceth Biopharma

PARALLEL TRACKS

Session 3 TRACK A TRACK B

Cell Therapy Gene Therapy

Moderator: Fabio D’Agostino, University of Newcastle Moderator: Margarida Menezes-Ferreira, Infarmed

10:00 Purification and Concentration of Cells for Cell Therapy

Manuel Carrondo, ibet

CAR-T Manufacturing: Delivering on the Promise of a Transformational Therapy

Greg Russotti, Celgene Cellular Therapeutics

Definition of Critical Quality Attributes of CTL019, a Genetically Modified T-Cell Product

Margit Jeschke, Novartis

Lentiviral Vector Production Strategies

James Miskin, Oxford Biomedical

11:00 Coffee Break, Poster Session & Exhibition

11:30 Title tbc

Brad King, Cook Regentec

Vector Manufacturing Production usingDisposable Fixed-bed Bioreactors

Margherita Neri, MolMed

Control Strategy and Lifecycle Managementfor ATMPs

Michael Blackton, Adaptimmune

The Critical Roles of Closed Systems, Automation and Integration in Manufacturing Genetically Modified Cellular Products

Ian Johnston, Miltenyi Biotec

Q&A, Discussion Q&A, Discussion

13:00 Lunch Break, Poster Session & Exhibition

Session 4 New Horizons: GMP & Business Moderator: Dirk Groenewegen, Cells4Therapy

14:00 Meeting the Demands of 21st Century Regenerative Medicine: Building a Cellular Therapy GMP Facility and Team from Scratch

Lee Chapman, Celixir

The Need for Biopreservation: Best Practices to Translate ATMPs from the Laboratory to the Clinic

Brian Hawkins, BioLife Solutions

CONFERENCE AGENDA

7pda.org/EU-ATMPs2017

Closing Panel DiscussionCan we handle the current challenges for getting ATMPs to the patient, or do we need a reality check?

Moderator: Wilfried Dalemans, Tigenix

16:30 Closing Remarks & Farewell

Training & Education Program

PDA Education offers courses that are developed and

taught by experts. They are uniquely targeted to professionals involved in the development and manufacturing of quality pharmaceutical and biopharmaceutical products.

Facts that Make a DifferenceUp-to date training courses and workshops taught by internatio-nally renowned experts

Customized in-house training courses and workshops available

Wide range of training courses with hands-on experience to drive expertise, awareness, and innovation

europe.pda.org

26 June 2017Practical Application of Phase-Appropriate GMP & Quality Principles to Clinical Development of ATMPsOne-Day Training Course

PDA Education Program

10 pda.org/eu/TCATMPs2017

ONE-DAY TRAINING COURSE

OverviewThere has been a surge in clinical development of Advanced Therapy Medicinal Products (ATMPs), also known as Cellular and Gene Therapy (CGT) Products in the USA. Many start up companies, hospitals, universities, and now even large biopharmaceu-tical companies, are planning or have already entered into manufacturing these genetically engineered virus and human cell products for clinical studies. While the ground rules for good manufacturing practices (GMPs) and quality of recombinant pro-tein and monoclonal antibody manufacturing and control are well established, for ATMPs these are still under development. ATMP manufacturing and control presents unique GMP and Quality challenges, such as the heightened concern about the safety and consistency of the starting material, the safety and quality of the raw materials added during processing, the need to protect against adventitious agent contamination during the entire manufacturing cycle, the limitations of the analytical methods available to characterize these virus and cell products, and the reality that the administered clinical medicinal prod-uct is a complex living organism.Patients in these clinical development programs need to be protected by common sense GMPs and Quality Principles. The available regulatory authority guidance documents (both EMA and FDA) for ATMPs will be thoroughly examined. In addition, the core principles presented in PDA’s 2016 Technical Report 56 ‘Application of Phase-Appropriate Quality System and cGMP to the Development of Therapeutic Protein Bulk Drug Substance (API)’ will be adapted to ATMPs.

Practical Application of Phase- Appropriate GMP & Quality Principles to Clinical Development of ATMPs

John Geigert, PhD, RAC, President, BioPharmaceutical Quality Solutions

John Geigert has been a consultant for the past 15 years, specializing in CMC regulatory strategy for the biopharmaceutical industry. He has held senior leadership positions in industry as Vice President of Quality for both IDEC Pharmaceuticals Corporation and Immunex Corporation. He has been a major participant in regulatory approvals for six biopharmaceutical products now commercially available in the U.S. and in Europe, and has over 40 years of experience in the biologic and biopharmaceutical industry. John obtained his B.S.

in Chemistry from Washington State University, and his Ph.D. degree in Organic/Analytical Chemistry from Colorado State University. He has served as a member of the PDA the Board of Directors, and is currently the chair of the PDA Biopharmaceutical Advisory Board. He is US Regulatory Affairs Certified (RAC) by the Regulatory Affairs Certification Board (RACB).

Who Should Attend:

This course is designed for those involved or interested in the manufacturing, quality and GMP compliance of ATMPs during clinical stages of development, including Senior Managers, Directors and Managers/Supervisors, QA/QC, Regulatory, Manufacturing and Process Development personnel.

Learning Objectives:Upon completion of this course, the attendee will befamiliar with:

• Explain the importance and underlying GMP and Quali-ty principles for manufacturing, control and compliance of ATMPs during clinical stages of development

• Appreciate the GMP and Quality similarities and differ-ences between protein medicinal products and ATMPs

• Appropriately apply phase-appropriate GMPs and Qual-ity Principles to the clinical development of ATMPs

TRAINING COURSE AGENDA

11pda.org/eu/TCATMPs2017

Monday, 26 June 2017 9:00 – 17:30

9:00 Welcome & Introduction

9:10 Understanding the Basics• Painting the terminology landscape: ATMP, HCT/P, GMP, Quality System, etc.• Introduction to the risk-based, phase-appropriate GMP and Quality approach

10:30 Coffee Break

11:00 Major Differences, and the Regulatory Consequences• Viruses/cells are not biologic proteins• GMP and Quality consequences of the differences between gene / cell-based medicines

and protein-based medicines

12:30 Lunch Break

13:30 Regulatory Authority (EMA/FDA) Expectations• Regulatory authority risk-based considerations to ATMP GMPs and Quality• Regulatory authority guidances for a phase-appropriate approach to ATMP GMPs

and Quality

15:00 Coffee Break

15:30 Industry Practice in Applying Phase-Appropriate GMPs and Quality to ATMPs• Adapting PDA Technical Report 56 as a model for phase-appropriate ATMP GMPs

and Quality• Lesson learned from industry

17:30 End of Training Course

SH VALENCIA PALACEVALENCIA

13pda.org/EU-ATMPs2017

INFORMATION

V E N U ESH Valencia PalacePaseo Alameda, 3246023 Valencia, SpainTel: +34 96 337 50 37Fax: +34 96 337 55 32http://www.hotel-valencia-palace.com/en/

Housing at the selected hotel will be in high demand, so we strongly recommend making your reservations early.

CO NTAC T I N FO R M ATI O NRegistration Customer CareTel: +49 30 436 55 08-10registration-europe@pda.org

Conference InquiriesMelanie DeckerDirector Events & Exhibitionsdecker@pda.org

Conference Program InquiriesSylvia Beckerprograms-europe@pda.org

Education Program InquiriesElke von Laufenbergtraining-europe@pda.org

Exhibition/Sponsorship InquiriesNadjeschda Gomez-Stahlexpo-europe@pda.org

O RG A N I Z E RPDA Europe gGmbHAm Borsigturm 6013507 Berlin, GermanyTel: +49 30 4365508-0Fax: +49 30 4365508-66

TO E X H I B IT:Exhibition and Sponsorship Opportunities are available. PDA meetings and conferences are a great opportunity for your company to gain on-site exposure in front of high-ly-qualified, upper-level professionals in the pharmaceuti-cal and biopharmaceutical industry. Exhibit at PDA events and let your company’s products or services become a valuable tool or resource for our attendees.

S PEC I A L R EQU I R E M E NT SIf you require special accommodations to fully par- ticipate, please attach a written description of your

needs with your registration form. Specific questions can be directed to registration-europe@pda.org.

Special offer: Discounted travel with Lufthansa Group Airlines

Lufthansa Group Partner Airlines offer a comprehensive global route network linking major cities around the world. We offer spe-cial prices and conditions to participants, visitors, exhibitors, in-vited guests as well as employees of the Contracting partner and their travel companions. To make a reservation, please click on www.lh.com/event-flight-booking and enter the access code DEZJPRC in the "Access to Your Special Lufthansa Offer" area. This will open an online booking platform that will automatically calculate the discount offered or provide you with an even better offer if another promotional fare is available.

NOTE: Pop-ups must be enabled otherwise the booking platform window will not open.

These promotional fares are also available through your IATA / ARC travel agent. Travel agents can obtain ticketing instructions by send-ing an email to lufthansa.mobility@dlh.de and providing the access code as a reference.

D I R EC TI O N S

© Google For directions click on the picture, scan the QR-code or go to https://goo.gl/maps/XoHLYg6CXqo

SH VALENCIA PALACEVALENCIA

The Universe of Pre-filled Syringes & Injection Devices

The Parenteral Drug Association presents:

2017 PDA Europe Conference, Exhibition

7-8 November 2017 Austria Center

Vienna | Austria

Register by 7 Oct 2017 and SAVE!

Accompanying Education Program on Monday, 6 November, and on Thursday and Friday, 9-10 November

1515

3 WAYS TO REGISTER

ONLINE: pda.org/EU-ATMPs2017FAX: +49 30 4365508-66EMAIL: registration-europe@pda.org

123

If this form is an update to a previously submitted form, please check here.

Mr. Ms. Dr. NonmemberI want to become a PDA Member. Please send me a subsription form

Job Title *

Mailing Address

Substituting for(Ch e ck only i f you are subs t i tut ing for a pre v iously e nrolle d colleag ue; a nonme mb e r subs t i tut ing for me mb e r mus t pay t h e me mb e r ship fe e.)

1 Your Contact Information

PDA MemberName (Last, First, MI) *

ID Number

Country Email

FaxBusiness Phone

Company* Department

City Postal Code

CONFIRMATION: Transmitting your filled-in registration form constitutes a binding application for the specific event. PDA Europe will send you a confirmation including payment details. A legally binding contract is concluded once PDA Europe has sent a written invoice by mail to you. A letter of confirmation will be sent to you within one week once payment has been received. You must have this written confirmation to be considered enrolled for this PDA event. PDA Europe reserves the right to deny access to anyone unable to provide written confirmation that all dues have been fully settled. SUBSTITUTIONS: If you are unable to attend, substitutions are welcome and can be made at any time, including on site at the prevailing rate. If you are registering as a substitute attendee, please indicate this on the registration form. Changes are free of charge until 2 weeks prior to the start of the event. After this two-weeks period, there will be a charge of € 100 excl. VAT per name change. REFUNDS: Refund requests must be sent to PDA Europe. If your written request is received on or before 27 May 2017 you will receive a full refund minus a 150 € excl. VAT handling fee. After that time, no refund or credit requests will be approved. If you are an unpaid registrant and do not attend the event, you are responsible for paying the registration fee. On-site registrants are not guaranteed to receive conference materials until all advanced registered attendees receive them. PDA Europe works PCI-Compliant. EVENT CANCELLATION: PDA reserves the right to modify the material or speakers/instructors without notice, or to cancel an event. If an event must be canceled, registrants will be notified by PDA as soon as possible and will receive a full refund. PDA will not be responsible for airfare penalties or other costs incurred due to cancellation. For more de-tails, contact PDA at registration-europe@pda.org or fax to +49 30 4365508-66. DOCUMENTATION: With your signature you give complete picture usage right to PDA and allow to film your exhibition space and intervention in the event, including the recording of your presentation for video purposes (with your slides, voice and image). This right extends also to the use of the resulting images in film documentation for webinars and similar items produced by PDA.

Discount for Exhibiting Companies Please mark here if your company is an exhibitor to this event and you will receive the conference ticket at the special price of 995 Euro per ticket. No further discounts are applicable with this option (as PDA Membership Discount or Group Ticket discount). This special rate does not include one-year PDA membership.

PDA Europe VAT I.D.: DE254459362

3 Payment Options

Billing Address:

Same as contact information address above. If not, please send your billing address to: registration-europe@pda.org

Date Mandatory Signature

By Credit Card

American Express MasterCard VISA

For your credit card information safety:Please send your details by fax only (+49 30 4365508-66) or register online.

By Bank Transfer Beneficiary: PDA Europe gGmbHIBAN: DE73 1007 0024 0922 8735 00BIC (SWIFT-Code): DEUTDEDBBERBank Address: Deutsche Bank, Welfenallee 3-7, D-13465 Berlin, Germany

By Purchase Order Purchase Order Number

Group Registration Discount Register 5 colleagues for the conference at the same time and receive the 5th registration free. For more information on group discounts please contact us at registration-europe@pda.org. Other discounts cannot be applied.

The fee includes course documentation as well as mid-session refreshments and lunch. Excellent networking opportunities with snacks and drinks will be given. The fee does not include the hotel accommodation. PDA Europe has secured a limited number of rooms at a special group rate.

Information about Visa Matters • All registrations which will involve visa matters will have to be submitted to PDA EU four weeks prior to the start of the event at the latest. For later registrations, PDA Europe will be unable to assist participants in any visa affairs.

• All costs incurring in connection with visa affairs shall be borne by registrants. (This applies in particular to costs for sub-mitting documents by courier.)

• Potential participants must be clients of UPS shipping agency and submit their UPS customer reference number to PDA EU (together with their registration).

Your Company VAT I.D.:

This number starts by your country code with two characters (example: PDA Europe’s country code starts with: DE | followed by the number)

2 Registration

All fees given in Euro and excluding VAT (21 %)

Conference (27-28 June)PDA Member 1495Nonmember** 1795Govern./Health Authority/Academic** 750

Poster Presenter please mark here (written approval required, conference fee applies)

One-Day Training Course (26 June)Practical Application of GMP for Development of ATMPsAll Participants 845

**Registration fee includes a one-year PDA membership if no further special discount is granted. If you do not wish to join PDA and receive the benefits of membership, please check here (same rate applies).

Advanced Therapy Medicinal Products27-28 June 2017 | Valencia | Spain

For contact at PDA Europeregistration-europe@pda.org

Organizer: PDA Europe, Am Borsigturm 60, 13507 Berlin, Germany pda.org/EU-ATMPs2017

This PDF-file provides an automatic fill-in function. Your signature, however, is needed in writing.

2 0 1 7 P D A E U R O P E C O N F E R E N C E S

Events with additional Education Program. More information – www.pda.org/pda-europe

26 – 27 April Current Trends in Aseptic Fill & Finish of Pre-filled Syringes Lindau, Germany

30 May – 1 June Virus & TSE Safety Forum Dubrovnik, Croatia

13 – 14 June 2nd PDA Europe Annual Meeting Berlin, Germany

27 – 28 June Advanced Therapy Medicinal Products Valencia, Spain

19 – 20 September Pharmaceutical Freeze Drying Technology Cologne, Germany

26 – 27 September Particles in Injectables Berlin, Germany

26 – 27 September 10th Workshop on Monoclonal Antibodies Berlin, Germany

10 – 11 October Pharmaceutical Cold & Supply Chain Logistics Prague, Czech Republic

7– 8 November The Universe of Pre-filled Syringes and Injection Devices Vienna, Austria

21 – 22 November Outsourcing & Contract Manufacturing Munich, Germany

Subject to change For latest info: pda.org/pda-europe Shortlist 18 May 2017

www.pda.org/pda-europe

General InformationPDA Europe gGmbHAm Borsigturm 60 13507 Berlin, GermanyTel: +49 30 4365508-0Fax: +49 30 4365508-66info-europe@pda.org Connecting People, Science and Regulation ®