BIOASSAYS 2015: SCIENTIFIC PROGRAM March 23 - 24, 2015 at the Sheraton Hotel in Silver Spring, Maryland. Irina Klimanskaya, Ocata Therapeutics, Inc., Marlborough, MA USA - Monday March 23, 2015 10:05 – 10:30 Bioassay Development for Human Stem Cell-derived Retinal Pigment Epithelium: Progress and Challenges

1. Welcome to Bioassays 2015: Scientific Approaches & Regulatory Strategies
On behalf of the Scientific Organizing Committee and CASSS, we are excited to welcome you to
Bioassays 2015: Scientific Approaches & Regulatory Strategies and look forward to your participation
and input March 23 - 24, 2015 at the Sheraton Hotel in Silver Spring, Maryland.
The CASSS Bioassays meeting has established itself as a premier conference and unique opportunity for
participants and opinion leaders to discuss and debate current regulatory and industry topics regarding
bioassays. Bioassays are a critical component of the analytical control strategies for biologics and other
complex molecules. The ability of an assay to characterize and demonstrate biological activity is
essential and developing such bioassays is becoming more difficult as biologic drugs are engineered to
be more complex and/or have multiple modes of action. Companies are continuously challenged with
developing assays that are biologically relevant for the analysis of multiple potential mechanisms.
Bioassays are also used for lot release, stability, comparability and characterization studies, which
requires that the assays be robust and, in most cases, suitable for a QC lab.
Bioassays 2015 is structured to encourage attendee interaction. Each session includes case study
presentations followed by a panel discussion allowing for lively dialogue between attendees from
academia, industry and regulatory agencies. As in previous years, we expect this format to result in
additional focus on the technical and regulatory details of the topic. Regulatory participation from the
US FDA, Health Canada and various European agencies has been strong each year. In addition, an
exhibitor showcase and poster reception at the end of Day One will give attendees the opportunity to
present additional topics and continue the day's discussion in an informal setting.
We would like to thank the speakers and the panel members who are giving generously of their time and
resources and to you for your attendance. We would also like to acknowledge the generosity of our
program partners for the continued support of the CASSS Bioassays meeting: AbbVie, Inc.; Biogen Idec
and MedImmune. We are grateful for the expert management from CASSS and the audio-visual
expertise of Michael Johnstone from MJ Audio-Visual Productions. Their experience and guidance in
the preparation of this meeting has been invaluable.
We are sure you will find Bioassays 2015 to be informative and productive, and that it will provide you
with current perspectives on bioassays.
Scientific Organizing Committee:
Thomas Arroll, Seattle Genetics, Inc., USA
Evangelos Bakopanos, Health Canada, Canada
Katrin Buss, Federal Institute for Drugs and Medical Devices, BfArM, Germany
Chana Fuchs, CDER, FDA, USA (Co-chair)
Denise Gavin, CBER, FDA, USA (Co-chair)
Hélène Gazzano-Santoro, Genentech, a Member of the Roche Group, USA
Stephen Hartman, AbbVie Inc., USA
Xu-Rong Jiang, MedImmune, A member of the AstraZeneca Group, USA
Helena Madden, Biogen Idec, USA (Co-chair)
Bruce Meiklejohn, Eli Lilly and Company, USA (Co-chair)
Thomas Anders Millward, Novartis Pharma AG, Switzerland
Noel Rieder, Amgen Inc., USA
Sally Seaver, Seaver Associates LLC, USA

2. The Scientific Organizing Committee gratefully acknowledges the following program
partners for their generous support of Bioassays 2015:
SUSTAINING PLATINUM PROGRAM PARTNERS
AbbVie, Inc.
Biogen Idec
MedImmune, A member of the
AstraZeneca Group
SUSTAINING SILVER PROGRAM PARTNER
Pfizer, Inc.
PROGRAM PARTNERS
Amgen Inc.
Genentech, A Member of the
Roche Group

3. EXHIBITOR PROGRAM PARTNERS
DiscoveRx Corporation
Eurofins Lancaster
Laboratories
PPD
Promega Corporation
Stegmann Systems GmbH

4. The Scientific Organizing Committee gratefully acknowledges the following media for
their promotional consideration of Bioassays 2015:
MEDIA PROGRAM PARTNERS
American Laboratory
American Pharmaceutical Review
BioProcess International
BioProcessing Journal
Genetic Engineering & Biotechnology News
International Pharmaceutical Quality
Pharmaceutical Outsourcing
RSC Advances
Technology Networks Limited

5. Bioassays 2015: Scientific Approaches & Regulatory Strategies
Scientific Program Summary
Monday, March 23, 2015
07:30 – 17:00 Registration in the Cypress Foyer
07:30 – 08:30 Continental Breakfast in the Magnolia Ballroom
08:30 – 08:45 CASSS Welcome and Introductory Comments in the Cypress Ballroom
Helena Madden, Biogen Idec, Cambridge, MA USA
Bioassays 2015 Welcome and Introductory Comments in the Cypress
Ballroom
Helena Madden, Biogen Idec, Cambridge, MA USA
Potency Tests for Cell and Gene Therapy Products: Overcoming the Challenges
Workshop Session One in the Cypress Ballroom
Session Chairs: Denise Gavin, CBER, FDA and Sally Seaver, Seaver Associates LLC
08:45 – 08:50 Introduction
08:50 – 09:15 Bioassays in the Lifecycle of Cell & Gene Therapy Products: A Regulatory
Perspective
Xiaobin (Victor) Lu, CBER, FDA, Silver Spring, MD USA
09:15 – 09:40 Developing a Potency Assay Matrix for a Viral Vector Gene Therapy
Product
Barbara Thorne, Celladon Corporation, San Diego, CA USA
09:40 – 10:05 Potency Assay Development for Cell Therapy Products
Erik Rutjens, Novartis Pharma AG, Basel, Switzerland
10:05 – 10:30 Bioassay Development for Human Stem Cell-derived Retinal Pigment
Epithelium: Progress and Challenges
Irina Klimanskaya, Ocata Therapeutics, Inc., Marlborough, MA USA
10:30 – 11:00 AM Break – Visit the Exhibits and Posters in the Magnolia Ballroom
11:00 – 12:15 PANEL DISCUSSION – Questions and Answers
Irina Klimanskaya, Ocata Therapeutics, Inc., USA
Xiaobin (Victor) Lu, CBER, FDA, USA
Anthony Ridgway, Health Canada, Canada
Erik Rutjens, Novartis Pharma AG, Switzerland
Barbara Thorne, Celladon Corporation, USA

6. Monday, March 23 continued…
12:15 – 13:30 Hosted Lunch in the Magnolia Ballroom
Advances in Bioassay Technologies and Platforms
Workshop Session Two in the Cypress Ballroom
Session Chairs: Thomas Arroll, Seattle Genetics, Inc., Stephen Hartman, AbbVie, Inc. and Xu-Rong
Jiang, MedImmune, A member of the AstraZeneca Group
13:30 – 13:35 Introduction
13:35 – 14:00 Development and Validation of a To-Be-Marketed Cancer Immunotherapy
Antibody
Max Tejada, Genentech, a Member of the Roche Group, South San Francisco, CA
USA
14:00 – 14:25 An ADCP Reporter Bioassay for Assessing Fc Effector Function
Shihua Lin, MedImmune, A member of the AstraZeneca Group, Gaithersburg,
MD USA
14:25 - 14:50 In-vitro Bioassay Using mRNA Transcription (Dual RT-qPCR) as Assay
Response
Michael Sadick, Catalent Pharma Solutions, Kansas City, MO USA
14:50 - 15:15 Expectations and Limitations of Bioassays: An EU Regulator’s View
Christian Mayer, AGES – Austrian Agency for Health & Food Safety, Vienna,
Austria
15:15 - 15:45 PM Break – Visit the Exhibits and Posters in the Magnolia Ballroom
15:45 - 17:00 PANEL DISCUSSION – Questions and Answers
Evangelos Bakopanos, Health Canada, Canada
Shihua Lin, MedImmune, A member of the AstraZeneca Group, USA
Christian Mayer, AGES – Austrian Agency for Health & Food Safety, Austria
Michael Sadick, Catalent Pharma Solutions, USA
Jennifer Swisher, CDER, FDA, USA
Max Tejada, Genentech, a Member of the Roche Group, USA
17:00 – 17:15 Break

7. Monday, March 23 continued…
Exhibitor Partner Scientific Showcase in the Cypress Ballroom
Session Chairs:
17:15 – 17:30 Introduction
17:30 – 17:45
Eurofins Lancaster Laboratories
17:45 – 18:00
Promega Corporation
18:00 – 18:15
DiscoveRx Corporation
18:15 – 18:30
Stegmann Systems GmbH
18:30 – 18:45
PPD
18:45 – 19:00 Discussion – Questions and Answers
19:00 – 21:00 Exhibitor and Poster Reception in the Magnolia Ballroom
21:00 Adjourn Day One

8. Tuesday, March 24, 2015
08:00 – 17:00 Registration in the Cypress Foyer
07:30 – 08:30 Continental Breakfast in the Magnolia Ballroom
Bioassays to Support Commercialization of Drug Products
Workshop Session Three in the Cypress Ballroom
Session Chairs: Hélène Gazzano-Santoro, Genentech, a Member of the Roche Group, Bruce Meiklejohn,
Eli Lilly and Company and Thomas Millward, Novartis Pharma AG
08:30 – 08:35 Introduction
08:35 – 09:00 Health Canada’s Lot Release Program & Potency Testing of Biotherapeutic
Products
Evangelos Bakopanos, Health Canada, Ottawa, ON Canada
09:00 – 09:25 Lifecycle of an Enzyme Activity Assay: Development and Validation to Post-
marketing Commitments
Loc Vo, BioMarin Pharmaceutical Inc., Novato, CA USA
09:25 – 09:50 Bioassay Strategies for Assessment of Co-stimulation Inhibitors in Immuno-
oncology: Considerations from Development to Commercialization
Jeffrey Glenn, Bristol-Myers Squibb Company, Princeton, NJ USA
09:50 – 10:15 Bioassay Evolution and Lessons Learned
Thomas Luntz, Catalent Pharma Solutions, Morrisville, NC USA
10:15 - 10:45 AM Break – Visit the Exhibits and Posters in the Magnolia Ballroom
10:45 – 12:00 PANEL DISCUSSION – Questions and Answers
Evangelos Bakopanos, Health Canada, Canada
Jeffrey Glenn, Bristol-Myers Squibb Company, USA
Thomas Lunz, Catalent Pharma Solutions, USA
Emily Shacter, ThinkFDA, USA
Craig Thelwell, National Institute for Biological Standards and Control (NIBSC),
United Kingdom
Loc Vo, BioMarin Pharmaceutical Inc., USA
12:00 – 13:15 Hosted Lunch in the Magnolia Ballroom

9. Tuesday, March 24 continued…
Bioassay Challenges during Product Globalization
Workshop Session Four in the Cypress Ballroom
Session Chairs: Katrin Buss, Federal Institute for Drugs and Medical Devices, BfArM, Helena Madden,
Biogen Idec and Noel Rieder, Amgen Inc.
13:15 – 13:20 Introduction
13:20 – 13:45 Global Company, Global Products, Local Laboratories
Camille Dycke, F. Hoffmann-La Roche Ltd., Basel, Switzerland
13:45 – 14:10 Collaborative Study for Proposed 1st International Standard for PEGylated
G-CSF (PEG-G-CSF)
Meenu Wadhwa, National Institute for Biological Standards and Control
(NIBSC), South Mimms, United Kingdom
14:10 – 14:35 Bioassay Strategy to Support the Transfer of a Monoclonal Antibody for
Manufacturing and Filing in China
Justin Jia, WuXi AppTec, Shanghai, China
14:35 – 15:00 Strategies to Assist Global Regulatory Acceptance of Assurance of Potency
Anthony Mire-Sluis, Amgen Inc., Thousand Oaks, CA USA
15:00 – 15:30 PM Break – Visit the Exhibits and Posters in the Magnolia Ballroom
15:30 – 16:45 PANEL DISCUSSION – Questions and Answers
Katrin Buss, Federal Institute for Drugs and Medical Devices, BfArM, Germany
Camille Dycke, F. Hoffmann-La Roche Ltd., Switzerland
Chana Fuchs, CDER, FDA, USA
Justin Jia, WuXi AppTec, China
Anthony Mire-Sluis, Amgen Inc., USA
Meenu Wadhwa, National Institute for Biological Standards and Control
(NIBSC), United Kingdom
16:45 – 17:00 Bioassays Workshop Recap
Closing Remarks and Invitation to Bioassays 2016
Bruce Meiklejohn, Eli Lilly and Company, Indianapolis, IN USA
17:00 Adjournment

10. Potency Tests for Cell and Gene Therapy Products:
Overcoming the Challenges
Session Abstract
Session Chairs: Denise Gavin, CBER, FDA and Sally Seaver, Seaver Associates LLC
Cell and Gene therapies (CGT) are an evolving class of promising biological products. However, despite
over two decades of investigation there are still few cell and gene therapies (CGT) that are commercially
available or in late stage clinical development. Successful development of any biological therapeutic
requires full product characterization to ensure safety and efficacy. Animal or cell based bioassays are a
critical part of this product characterization. Usually several bioassay candidates are developed as
potential potency tests, and the most robust, quantitative and QC friendly method that can be validated is
selected to become the potency test for lot release and monitoring lot stability. The other bioassays are
reserved for product characterization and comparability assessment after process changes including
scale-up.
To date few CGT have had robust and meaningful potency tests. Some of the challenges faced by
developers of potency tests for CGT products include (1) extremely short product shelf life (hours to
days), (2) the need to express a GT vector in one cell type and to quantitate its biological activity in a
second cell type, (3) developing bioassays for all of the active ingredients, especially for multi-gene or
multi-cell products, (4) developing bioassays that measure relevant biological activities (5) defining and
banking a “reference standard” for these products, esp. if the product is autologous, and (6) determining
which bioassays need to be executed for routine lot release and stability assessments. In addition,
implementing a potency assay early in CGT product development is critical for understanding the
product attributes related to biological activity and clinical efficacy.
This session will provide an overview of regulatory expectations for measuring potency for CGT
products as well as several case studies describing how these challenges were overcome to develop
potency tests for lot release and stability and comparability assessments.
NOTES:

11. Advances in Bioassay Technologies and Platforms
Session Abstract
Session Chairs: Thomas Arroll, Seattle Genetics, Inc., Stephen Hartman, AbbVie, Inc. and Xu-Rong
Jiang, MedImmune
Bioassays are the only test methods that quantify a drug’s biological activity and thus play a crucial role
in the characterization of biologic therapies. The interplay of living cells, biologically active reagents,
various quantifiable readouts, multi-step procedures, specific instrumentation, and complex data analysis
makes the development of a robust, accurate, precise bioassay extremely challenging.
Products with multiple MoAs pose an even greater challenge. The increasing complexity of large
molecule modalities (such as ADCs, bispecifics/bifunctionals, and immunomodulators) and their
multiple biological activities also greatly increases the complexity of bioassay development. Many new
technologies, approaches, and platforms can facilitate bioassay development, as well as provide
additional information or performance enhancements, but they may also introduce new challenges.
This session will discuss some recent advances in technologies and platforms that have improved
bioassay method performance, facilitated/accelerated development and implementation, and revealed
new/additional information about the product. We will also address the challenges faced when
introducing a new technology/approach and consider the risks and benefits to making these changes. We
will learn about bioassay approaches used for cancer immunotherapy products and assessing effector
functions, as well as learn about alternative bioassay readouts. We will also discuss regulatory agency
experiences, opinions, and expectations regarding the use of new bioassay method technologies.
NOTES:

12. Bioassays to Support Commercialization of Drug Products
Session Abstract
Session Chairs: Hélène Gazzano-Santoro, Genentech, a Member of the Roche Group, Bruce Meiklejohn,
Eli Lilly and Company and Thomas Millward, Novartis Pharma AG
As a product progresses through clinical development towards commercialization, the requirements and
expectations of the bioassays used to release and characterize the product evolve. By the time the
product is approaching registration, the bioassay needs to be fully validated, and specifications need to
be defined and justified. But in addition, the relationship between the bioassay and the clinical mode of
action needs to be clearly established and reagents that are critical for the bioassay need to be under
control and their supply guaranteed. For some products more than one bioassay may be needed for
commercial release, and these bioassay(s) may need to be transferred to one or more QC testing labs.
This session will discuss these and other aspects relating to bioassays during product commercialization.
NOTES:

13. Bioassay Challenges during Product Globalization
Session Abstract
Session Chairs: Katrin Buss, Federal Institute for Drugs and Medical Devices, BfArM, Helena Madden,
Biogen Idec and Noel Rieder, Amgen Inc.
Global drug registration enables the biopharmaceutical industry to enter markets all over the world and
to provide life-changing medicines to patients everywhere. This requires a strategic approach to
analytics from early drug development throughout post-approval lifecycle management. Analytical
control strategies are influenced by country and region specific expectations and testing requirements
that result in added complexity. Scientists and regulators must work together to understand regional
requirements and best approaches to ensure successful licensure and assurance of product quality.
Considerations for globalization of bioassays include: instrument and software selection, critical reagent
availability, method robustness and reproducibility, and the requirement for registration and import
testing. These challenges are more daunting when considered in the face of likely language, time and
cultural differences. This session will explore and contrast requirements from several areas of the world.
We will also discuss strategies for selection, development, transfer, performance and maintenance of a
bioassay to support successful biopharmaceutical globalization.
NOTES: