2017 PDA Europe Advanced Therapy Medicinal Products

PDA Europe Conference, Exhibition

Advanced Therapy Medicinal Products

27-28 June 2017 SH Valencia Palace Valencia | Spain

OVERVIEW 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 accelerated 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 challenges 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!

Dirk Groenewegen, PhD, Cells4Therapy, Conference Co-Chair

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

SCIENTIFIC PROGRAM PL ANNING COMMITTEE 1 Dirk Groenewegen, Cells4Therapy, Chair

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2 Margarida Menezes-Ferreira, Infarmed, Co-Chair 3 Manuel Carrondo, ibet 4 Fabio D’Agostino, University of Newcastle

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5 Wilfried Dalemans, Tigenix 6 Giovanni Migliaccio, Eatris 7 Wenzel Novak, Optima Pharma

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8 Valerie Pimpaneau, Voisin Consulting 9 Sol Ruiz, AEMPS 10 Jessica Kopf, Fraunhofer IPA

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11 Margit Jeschke, Novartis 12 Georg Roessling, PDA Europe 13 Falk Klar, PDA Europe

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pda.org/EU-ATMPs2017

S C H E D U L E AT A G L A N C E 26 June

9:00 – 17:30

Practical Application of PhaseAppropriate GMP & Quality Principles to Clinical 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

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

Conference, Exhibition

CONFERENCE AGENDA 30 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

Keynote Overview of ATMPs Development

Jonathan Appleby, GSK Rare Diseases Cell and Gene Therapy

Session 1 GMP - Current Regulatory Status

Moderator: Wilfried Dalemans, Tigenix

9:45

11:15

12:45

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

Coffee Break, Poster Session & Exhibition Industry Perspective

Annie Hubert, Alliance for Regenerative Medicine

GMP Experience from a CMO Perspective

Marc Barthold, apceth Biopharma

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 14:30

15:30

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Moderator: Georg Roessling, PDA Europe

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

Coffee Break, Poster Session & Exhibition

pda.org/EU-ATMPs2017

CONFERENCE AGENDA

Potency Assays for MSC Products

Marcel Hoefnagel, CBG-Meb

Alternative Methods for Sterility Testing Validation Requirements

LuĂ­s Meirinhos-Soares, Infarmed

Q&A, Discussion 17:30

Closing of Day 1 & Networking Reception

Networking Reception

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

pda.org/EU-ATMPs2017

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CONFERENCE AGENDA 30 May 2017

Wednesday, 28 June 2017 9:00

Welcome

Dirk Groenewegen, Chair, Cells4Therapy

9:05

Keynote Gene & 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

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Purification and Concentration of Cells for Cell Therapy

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

Manuel Carrondo, ibet

Greg Russotti, Celgene Cellular Therapeutics

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

Lentiviral Vector Production Strategies

Margit Jeschke, Novartis

James Miskin, Oxford Biomedical

11:00

Coffee Break, Poster Session & Exhibition

11:30

Treg Cell Therapy in Solid Organ Transplantation

Vector Manufacturing Production using Disposable Fixed-bed Bioreactors

Alessandra Petrelli, UniversitĂ degli Studi di Roma Tre

Margherita Neri, MolMed

Control Strategy and Lifecycle Management for ATMPs

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

Michael Blackton, Adaptimmune

Ian Johnston, Miltenyi Biotec

Q&A, Discussion

Q&A, Discussion

13:00

Lunch Break, Poster Session & Exhibition

Session 4

New Horizons: GMP & Business

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

pda.org/EU-ATMPs2017

Moderator: Dirk Groenewegen, Cells4Therapy

CONFERENCE AGENDA

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

16:30

Moderator: Wilfried Dalemans, Tigenix

Closing Remarks & Farewell

pda.org/EU-ATMPs2017

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Training & Education Program europe.pda.org

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 Difference Up-to date training courses and workshops taught by internationally renowned experts Wide range of training courses with hands-on experience to drive expertise, awareness, and innovation Customized in-house training courses and workshops available

PDA Education Program 26 June 2017 Practical Application of Phase-Appropriate GMP & Quality Principles to Clinical Development of ATMPs One-Day Training Course

ONE-DAY TRAINING COURSE

Practical Application of PhaseAppropriate GMP & Quality Principles to Clinical Development of ATMPs Overview There 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 biopharmaceutical 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 protein 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 product 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.

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 be familiar with: •

Explain the importance and underlying GMP and Quality principles for manufacturing, control and compliance of ATMPs during clinical stages of development

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Appreciate the GMP and Quality similarities and differences between protein medicinal products and ATMPs

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Appropriately apply phase-appropriate GMPs and Quality Principles to the 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).

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pda.org/eu/TCATMPs2017

TRAINING COURSE AGENDA

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

pda.org/eu/TCATMPs2017

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SH VALENCIA PALACE VALENCIA

INFORMATION

VENUE SH Valencia Palace Paseo Alameda, 32 46023 Valencia, Spain Tel: +34 96 337 50 37 Fax: +34 96 337 55 32 http://www.hotel-valencia-palace.com/en/ Housing at the selected hotel will be in high demand, so we strongly recommend making your reservations early.

D I R EC T I O N S

SH VALENCIA PALACE VALENCIA

ORGANIZER PDA Europe gGmbH Am Borsigturm 60 13507 Berlin, Germany Tel: +49 30 4365508-0 Fax: +49 30 4365508-66 T O E X H I B I T: 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 highly-qualified, upper-level professionals in the pharmaceutical 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 E QU I R E M E N T S If you require special accommodations to fully participate, please attach a written description of your needs with your registration form. Specific questions can be directed to registration-europe@pda.org.

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

CO N TAC T I N FO R M AT I O N Registration Customer Care Tel: +49 30 436 55 08-10 registration-europe@pda.org Conference Inquiries Melanie Decker Director Events & Exhibitions decker@pda.org Conference Program Inquiries Sylvia Becker programs-europe@pda.org Education Program Inquiries Elke von Laufenberg training-europe@pda.org Exhibition/Sponsorship Inquiries Nadjeschda Gomez-Stahl expo-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 special prices and conditions to participants, visitors, exhibitors, invited 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 sending an email to lufthansa.mobility@dlh.de and providing the access code as a reference.

pda.org/EU-ATMPs2017

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The Parenteral Drug Association presents:

2017 PDA Europe Conference, Exhibition

The Universe of Pre-filled Syringes & Injection Devices Accompanying Education Program on Monday, 6 November, and on Thursday and Friday, 9-10 November

Register by 7 Oct 2017 and SAVE!

7-8 November 2017 Austria Center Vienna | Austria

Advanced Therapy Medicinal Products

For contact at PDA Europe registration-europe@pda.org

27-28 June 2017 | Valencia | Spain

3 WAYS TO REGISTER

1

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

1 ONLINE: pda.org/EU-ATMPs2017 2 FAX: +49 30 4365508-66 3 EMAIL: registration-europe@pda.org

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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 submitting 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).

2 Registration

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

One-Day Training Course (26 June) Practical Application of GMP for Development of ATMPs All Participants Conference (27-28 June) PDA Member Nonmember** Govern./Health Authority/Academic**

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**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).

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.

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.

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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 gGmbH IBAN: DE73 1007 0024 0922 8735 00 BIC (SWIFT-Code): DEUTDEDBBER Bank Address: Deutsche Bank, Welfenallee 3-7, D-13465 Berlin, Germany

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

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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.

Date

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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 details, 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.

Organizer: PDA Europe, Am Borsigturm 60, 13507 Berlin, Germany

pda.org/EU-ATMPs2017

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2017 PDA EUROPE CONFERENCES 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 30 May 2017

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

General Information PDA Europe gGmbH Am Borsigturm 60 13507 Berlin, Germany Tel: +49 30 4365508-0 Fax: +49 30 4365508-66 info-europe@pda.org

Connecting People, Science and Regulation ®

www.pda.org/pda-europe