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2019 Biopharmaceuticals Week

2019 PDA Cell and Gene Therapy Conference

May 06 - May 07, 2019
Long Beach, CA

Concept through Commercialization - Patient-Focused Manufacturing

  • Conference
  • The Americas
Program Highlights

The 2019 PDA Cell and Gene Therapy Conference is right around the corner! Stay tuned to this page for any updates as we get closer to the event. For questions, please feel free to contact us at your convenience.

Overview

PDA will host the 2019 PDA Cell and Gene Therapy Conference, May 6-7, as part of its inaugural 2019 PDA Biopharmaceuticals Week. Building on the success of PDA’s recent Cell and Gene Therapy Conference in October 2018, this event will explore the most recent advances that have taken place over the ensuing months. The Program Planning Committee is building an agenda to address some of the key issues currently facing the industry: product realization, including the “nuts and bolts” of getting product to the clinical stage and how to target the right regulatory body; product characterization testing; electronic batch records; tech transfer and comparability; knowledge management; facility design; and vein to vein; among others.

Agenda

  • Day 1
  • Day 2
  • Monday, May 6

    7:00 a.m. – 6:30 p.m.
    Registration Open

    7:00 a.m. – 8:30 a.m.
    Continental Breakfast

    8:30 a.m. – 8:45 a.m.
    Welcome and Opening Remarks from Conference Co–Chair
    Michael Blackton, MBA,
    Vice President, Quality, CMC, Adaptimmune, LLC

    8:45 a.m. – 10:30a.m.
    P1: The Patient Perspective: How Innovative Therapies are Transforming Lives
    Moderator: Michael Blackton, MBA,
    Vice President, Quality, CMC, Adaptimmune, LLC

    Innovative cell and gene therapies have drastically changed how we address the technical challenges in developing and manufacturing these medicines. These products have also drastically changed the patient experience by providing a new set of options for the patient that were, not very long ago, simply pipe dreams. This session will explore how these innovative therapies impact patient’s lives and therefore help provide the audience with the necessary context as we follow the path toward commercialization

    8:45 a.m. – 9:30 a.m.
    Journey to T-Cell Therapy, The Whitehead Family
    Tom G. Whitehead, Co-Founder, Emily Whitehead Foundation

    9:30 a.m. – 10:00 a.m.
    Supplying Cell and Gene Therapies: It’s Going to Take More Than Cutting-Edge Science Alone for Us to Serve Our Patients
    Thomas S. Leitch, Vice President, VectorManufacturing, bluebird bio

    10:00 a.m. – 10:30 a.m.
    Questions and Answers/Discussion

    10:30 a.m. – 11:00 a.m.
    Refreshment Break and Poster Presentations

    Poster Presentations

    The following posters will be presented during refreshment breaks on Monday and Tuesday
    1. Analytical Characterization of a Complex Product: Lentiviral Vectors
      Julia Deuel, MS, Senior Scientist, bluebird bio
    2. Expand Outside the Hood: Cell Passaging Outside Biosafety Cabinet with Fully Closed and Aseptic in Cell Expansion System
      Charles P. Meadows, MBA, Product Manager, Sartorius Stedim Biotech
    3. Electronic Solutions for Manufacturing Challenges
      Crystal M. Booth, Regional Manager, PSC Biotech
    4. Particulate Matter in Single-Use Systems: Measurement and Risk Reduction
      Klaus R. Wormuth, PhD, Lead Scientist, Sartorius Stedim Biotech
    5. Validation of a Fully Automated Mycoplasma Detection Method Intended to Improve the Ease-of-Use and Time-to-Result for Nucleic Acid Tests
      William Barry, PhD, Scientist, BioFire Defense
    6. Leveraging High-Dimensional ‘-Omics’ Technologies for Comprehensive Profiling of CAR T Cells to Resolve Drug Product Complexity
      Eric S. Alonzo, PhD, Scientist, bluebird bio
    7. Advanced LC-MS Approach for Characterization of HCPs and Delivery Vehicle (Viral) Proteins in Gene Therapy Products
      Dongdong Wang, PhD, Director, BioAnalytix

    11:00 a.m. – 12:30 p.m.
    P2: The A, B, C’s of Cell and Gene Therapy Facility Design
    Moderator:
    Irving Ford, MSc, Head, CAR-T QC Laborato, Celgene Biotechnology Company

    The facility design for cell and gene therapy products must take into consideration the many aspects of the manufacturing process. Most processes involve manual aseptic manipulations performed within a biological safety cabinet and/or a combination of manual aseptic manipulations and automation. Additionally, the facility design must incorporate flexibility in the manufacturing areas as well as the need for future expansion. This session will explore approaches to consider when designing and/or modifying a facility to ensure a compliant yet seamless end to end manufacturing process for cell and gene therapy products during evolving and non-harmonized regulatory landscapes.

    11:00 a.m. – 11:30 a.m.
    Flexible Gene Therapy Facilities: Current Best Practices
    John E. Dougherty, Lead Process Engineer, DPS Group

    11:30 a.m. – 12:00 p.m.
    CGMP Considerations for the Design and Operation of Cell Therapy Facilities
    Francesca A. McBride, Director Regulatory Compliance, Jacobs

    12:00 p.m. – 12:30 p.m.
    Questions and Answers/Discussion

    12:30 p.m. – 2:00 p.m.
    Networking Lunch

    2:00 p.m. – 3:30 p.m.
    P3: Tech Transfer and Comparability for Cell and Gene Therapies: Case Studies in Complexity
    Moderator: Michael Kuczewski, Associate Director, Oncology CMC, bluebird bio

    Accelerated clinical development timelines and regulatory pathways, highly complex products, and a still-maturing manufacturing infrastructure can lead to significant challenges for C&GT CMC organizations. Multiple site transfers, limited batch sizes, high inherent process variability. These are just a few of the hurdles to be cleared on the sprint from first-in-human to commercial licensure. We will explore case studies that reveal strategies for technology transfer and comparability that help to navigate these unique challenges while maintaining the speed and flexibility that is critical to this rapidly maturing field

    2:00 p.m. – 2:30 p.m.
    Launching Clinical Trials in Europe: A QP’s Perspective
    Stephanie M. Verbrugghe, QP, CPGP, CEO & Founder, Farbridge Pharma Consulting, LLC

    2:30 p.m. – 3:00 p.m.
    Regulatory Perspective on Tech Transfer and Comparability for Cell and Gene Therapies
    Steven S. Oh, PhD, Deputy Director, Division of Cellular and Gene Therapies, CBER, FDA

    3:00 p.m. – 3:30 p.m.
    Questions and Answers/Discussion

    3:30 p.m. – 4:00 p.m.
    Refreshment Break and Poster Presentations

    4:00 p.m. – 5:30 p.m.
    P4: Navigating the Product Characterization Process
    Moderator:
    Eden S. Fucci, Vice President of Biologics Manufacturing, Torque Therapeutics

    Cell and gene therapies present unique challenges related to product characterization and potency testing relative to traditional biologics. The link between product attributes/potency, starting material, methods, and clinical relevance can be poorly understood or may have little predictive value in many cases, while regulatory expectations are ambiguous. These case studies will explore recent challenges faced by companies during product characterization and development of potency methods.

    4:00 p.m. – 4:30 p.m.
    Phase Appropriate Lifecycle Management of Analytical Methods to Support Gene Therapy Products
    Brendan G. Keenan, MS, PhD, Associate Director Quality Control Sciences and Technology, bluebird bio

    4:30 p.m. – 5:00 p.m.
    Industry Perspective on Navigating the Product Characterization Process
    Tam Soden, PhD, Senior Director and Head of Analytical Development, Kite, a Gilead Company

    5:00 p.m. – 5:30 p.m.
    Questions and Answers/Discussion

    5:30 p.m. – 6:30 p.m.
    Networking Reception





  • Tuesday, May 7

    7:00 a.m. – 5:45 p.m.
    Registration Open

    7:00 a.m. – 8:45 a.m.
    Continental Breakfast

    7:15 a.m. – 8:30 a.m.
    Cell and Gene Therapy Interest Group Breakfast Session
    Moderator: Michael Blackton, MBA, Vice President, Quality, CMC, Adaptimmune, LLC

    One of the key challenges in cell and gene therapies is the need to thoroughly understand advanced products and processes. Analytics provide the key to understanding critical attributes of advanced products and processes as they advance toward commercialization. Given this, robust strategies for the development, qualification, and validation of innovative analytical techniques is an absolute requirement for success. In this session we will explore novel approaches to incorporation of analytical strategies supporting product and process understanding.   

    7:15 a.m. – 7:45 a.m
    Replication Competent Retrovirus Testing for Gene Therapy Vectors: Current Methodology and Regulatory Expectations
    Leyla S.  Diaz, PhD, Principal Scientist, MilliporeSigma

    7:45 a.m. – 8:15 a.m.
    Potency Assay Development and Qualification for B-Thalassemia and Sickle Cell Disease Autologous Gene Therapy Drug Products
    Ilya A. Shestopalov, Associate Director, Cell Analytics, bluebird bio

    8:15 a.m. – 8:30 a.m.
    Questions and Answers/Discussion

    8:45 a.m. – 10:15 a.m.
    P5: Application of Quality Risk Management Principals in Cell and Gene Therapy
    Moderator: Brian J. Hawkins, PhD, Chief Technology Officer, Pluristyx, Inc.

    The use of Quality Risk Management (QRM) principals and risk-based approaches are a regulatory expectation. This concept is highlighted in the draft revision of Annex 1. Risk-based approaches are needed in all aspects of cell and gene therapy low bioburden and sterile manufacturing. This session will review specific case studies of implementation of risk-based approaches and QRM principals in the design, control, monitoring, and operation of cell and gene therapy processes.

    8:45 a.m. – 9:15 a.m.
    Executing a Successful Microbial HACCP: Avoid Doing a Check the Box Risk Assessment and Calling it Done
    Marsha Steed, Director Global QC Microbiology and Contamination Control, bluebird bio

    9:15 a.m. – 9:45 a.m.
    CMO Case Study: Using the IREM (Intervention Risk Evaluation Model) to Build a Risk-Based Approach to Operator Qualification and APS
    Dr. Mike Long, MBB, Senior Director Consulting Services, ValSource LLC

    9:45 a.m. – 10:15 a.m.
    Questions and Answers/Discussion

    10:00 a.m. – 4:15 p.m.
    Exhibit Area Open

    10:15 a.m. – 11:00 a.m.
    Refreshment Break and Poster Presentations in Exhibit Area

    11:00 a.m. – 12:30 p.m.
    P6: Raw Material Selection and Control for Cell and Gene Therapy Manufacturing
    Moderator: Kimberly A. Carnes, Director, Quality Systems, REGENXBIO 

    Raw material selection and control presents unique challenges in cell and gene therapy manufacturing over traditional biopharmaceutical manufacturing. In this session we will explore some of these challenges and offer practical approaches for managing these hurdles using risk assessments and robust control strategies.

    11:00 a.m. – 11:30 a.m.
    The Role of Virus Filtration in Achieving Pathogen Safety of Cell and Gene Therapy Products
    Sebastian B. Teitz, PhD, Scientific Coordinator, ASAHI Kasei Bioprocess

    11:30 a.m. – 12:00 p.m.
    Building an Agile Raw Material Supply Chain for Gene Therapy Manufacturing
    Stefanie E. Brady, Director of Supply Chain, REGENXBIO 

    12:00 p.m. – 12:30 p.m.
    Questions and Answers/Discussion

    12:30 p.m. – 2:00 p.m.
    Networking Lunch

    2:00 p.m. – 3:30 p.m.
    P7: Needle to Needle Visibility and Supply Chain Challenges
    Moderator: Lori Daane, Director of Scientific Affairs, bioMérieux

    Cell therapy manufacturing begins with a collection of cells or tissue from the patient and ends with the administration of the final product to the patient. Between collection and final administration of a product, there is a complex manufacturing process. This complexity brings unique supply chain challenges and need for enhanced visibility of the entire cell therapy process. This session will address the need for heightened chain of custody and identity though use of electronic record keeping for every step of the process, prescriptive workflows and standardized operating procedures, and a robust distribution that may include digital real-time tracking and improved containers to maximize product effectiveness.

    2:00 p.m. – 2:30 p.m.
    Low-Temperature Storage and Preservation of Gene- and Cell-Based Drug Products
    Matthew R. Gehrmann, Senior Scientist, West Pharmaceutical Services. Inc.

    2:30 p.m. – 3:00 p.m.
    Is Cell Therapy Manufacturing Fundamentally All About Supply Chain Challenges?
    Patricia M. Seymour, MBA, CSCP, Managing Director, BPTG of BDO USA

    3:00 p.m. – 3:30 p.m.
    Questions and Answers/Discussion

    3:30 p.m. – 4:15 p.m.
    Refreshment Break and Poster Presentations in Exhibit Area

    4:15 p.m. – 5:45 p.m.
    P8: Navigating the Regulatory Pathway for Cell and Gene Therapy
    Moderator:
    David Smith, PhD, Associate Director, Hitachi Chemical Advanced Therapeutics Solutions

    In this session, we will have perspectives from regulators and industry on challenges and opportunities moving innovative cell and gene therapy Products through the regulatory lifecycle towards commercialization. We will explore how cell and gene therapies are viewed in comparison to more traditional biologics and what the future looks like to ensure the industry can drive to meet patient needs in a safe, effective, and efficient process.

    4:15 p.m. – 5:45 p.m.
    Panel Discussion
    EJ Brandreth, III, MBA,
    Senior Vice President, Quality, Inovio Pharmaceuticals
    Arifa S. Khan, PhD, Supervisory Microbiologist, CBER, FDA
    Dr. Mike Long, MBB, Senior Director Consulting Services, ValSource LLC
    Steven S. Oh, PhD,
    Deputy Director, Division of Cellular and Gene Therapies, CBER, FDA

    5:45 p.m.
    Closing Remarks from Conference Co-Chair
    Michael Blackton, MBA, Vice President, Quality, CMC, Adaptimmune, LLC









Additional Information

  • Learning Objectives

    Learning Objectives

    At the completion of this event, attendees will be able to:

    • Describe how these innovative therapies are transforming the biopharmaceuticals industry and directly impacting people’s lives.
    • Interpret regulatory and quality requirements for cell and gene therapy products with US and European regulatory insight.
    • Explain how facility and equipment design can be optimized for the unique challenges of cell and gene therapy.
    • Identify innovative approaches to technology transfer that address the unique aspects of cell and gene therapy manufacturing processes.
    • Summarize the importance and considerations for product characterization and potency testing given the uncertainty inherent with the application of disruptive technologies inherent to cell and gene therapy.
    • Discuss the unique considerations for developing effective supply chain strategies to meet global requirements for cell and gene therapy.
  • Who Should Attend

    Who Should Attend

    Department

    Manufacturing | Product Development | Quality | Research & Development | Regulatory Affairs | Engineering | Laboratory | Science | Information Technology | Validation | Training | Clinical

    Job Function 

    Scientist | Executive and Mid-Level Management | Project | Management | Technical Services | Supply Chain | Manufacturing | Application | Risk Management

  • Accreditation

    Accreditation

    Continuing Education for Pharmacists

    ACPE PDA is accredited by the Accreditation Council for Pharmacy Education (ACPE) as a provider of continuing pharmacy education. Participants may sign up to receive Continuing Pharmacy Education (CPE) credits. To do so, participants must sign in at the beginning of the program, submit the provided evaluation forms and mail the CPE credit request to the address stated on the form. Attendees must be present at the full event to receive Continuing Pharmacy Education credit, which will be awarded as follows:

    ACPE # 0116-0000-19-020-L04-P | 1.2 CEUs
    Type of Activity: Knowledge

    ALERT   ACPE and NABP developed the CPE Monitor that allows pharmacists to electronically track their continuing pharmacy education (CPE) credits. The CPE Monitor will reject any CPE credit requests submitted past 60 days from date of ACPE-accredited activity. Always submit CPE activity claims as soon as possible. For more information, please visit the ACPE or NABP website.

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Travel

PLEASE READ  PDA is not affiliated or contracted with any outside hotel contracting company. If someone other than PDA or the PDA chosen hotel contacts you suggesting that they represent any PDA event, they do not. It is PDA's recommendation that you book your hotel directly through the official PDA chosen hotel that is listed on our web site.

Hilton Long Beach

701 West Ocean Boulevard
Long Beach, CA
Phone: +1 (562)-983-3400

  • Accommodations

    The cut off date has now past, however reservations can still be made on a space-and rate-available basis only. To make a reservation, please contact the Hilton Long Beach directly at +1 (562)-983-3400. Check-in time is 3:00 p.m.; check-out time is 12:00 p.m.

    Individual Cancellation

    Please be aware that obtaining and canceling hotel reservations is the responsibility of the meeting registrant. Individuals will be responsible for payment of their own cancellation fees. The cancellation policy is 72 hours prior to check-in to avoid cancellation fees.

  • Amenities

    Whether conducting business, keeping up with a fitness routine or looking to spend quality time with your family, our Long Beach, California hotel is here to help you make the most of your stay by offering the services you need, the amenities you expect, and the extras you deserve.

    Read more about the amenities offered at the Hilton Long Beach.

How to Get Here

By Air

The Hilton Long Beach is located less than an hour from three airports:

Long Beach Airport (LGB) is located 7 miles (about a 15 minute drive time) northeast of the Hilton Long Beach, an estimated $25 taxi ride.

Los Angeles International (LAX) is located 21 miles (about a 30 minute drive time) northwest of the Hilton Long Beach, an estimated $50 taxi ride.

John Wayne/Orange County Airport (SNA) is located 20 miles (about a 40 minute drive time) southeast of the Hilton Long Beach, an estimated $50 taxi ride.

By Car

Hilton Long Beach is located at 701 West Ocean Boulevard, Long Beach, CA 90831.

Take the 405 Freeway to the 710 Freeway South, the Long Beach Freeway. Once on the 710 South, exit Downtown/Broadway/Pine Avenue (exit is on the left side of the freeway). Make the next right on Magnolia and the next right on Ocean Blvd. Hilton is down two lights on the right hand side (the corner of Ocean and Golden Avenues).

PDA attendees will receive a discounted $15/day self-parking rate (garage and surface). Valet parking is also available at $28/day.

Other Options

More information coming soon.

Directions

Registration Fees

Early Registration
Registration Type Price by March 22
Conference
Member $1,995
Non-member $2,274
Young Professional Member $998
Gov’t/Health Auth. Member $700
Gov’t/Health Auth. Non-member* $800
Academic Member $700
Academic Non-member* $800
Student Member $280
Student Non-member* $310
Regular Registration
Registration Type Price after March 22
Conference
Member $2,595
Non-member $2,874
Young Professional Member $1,298
Gov’t/Health Auth. Member $700
Gov’t/Health Auth. Non-member* $800
Academic Member $700
Academic Non-member* $800
Student Member $280
Student Non-member* $310

* For this member type or discounted rate, online registration is not available and must be faxed in.

† You must be a member to receive this rate.

All rates in US dollars. To receive the prevailing rate, PDA must receive your registration(s) by 5:00 p.m. ET on or before the date noted.

Group Registration Discount

Register 3 people from the same organization as a group (at the same time) for the Conference or Workshop and receive the 4th registration free. Other discounts cannot be applied.

Special Dietary Requirements

If you require special accommodations and/or have special dietary requirements, please state them in the appropriate field when you register for the event(s).

GENERAL TERMS AND CONDITIONS: PDA will send you a confirmation letter within one week of payment being received. You must have this confirmation letter to be considered enrolled in a PDA event. If you have submitted a purchase order or requested an invoice, please be advised that a credit card guarantee is needed. PDA reserves the right to modify the material or speakers/trainers without notice or to cancel an event. If an event is cancelled, registrants will be notified by PDA as soon as possible and will receive a full refund. PDA will not be responsible for any costs incurred by registrants due to cancellation. Please note that the attendee list is shared with attendees and exhibitors and may be used to follow up on specific areas of interest after the event. Tape recordings are prohibited at all PDA events. For more information, contact [email protected] or +1 (301) 656-5900.

SUBSTITUTION: If a substitution request is sent 30 days before the event, no fee will be charged. After this period, a $200 fee will be charged. If you are a non-member substituting for a member, you will be required to pay the difference of the non-member fee.

REFUND: If a refund request is sent 30 days before the event, a full refund will be given minus a $200 fee. After this period, no refunds will be given. Refund requests must either be faxed to + (301) 986-1093 or emailed to [email protected]. Phone messages are not accepted.

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Contact

Program Inquiries
  • Brooke Schneider, CMP
    Manager, Programs
    Tel: +1 (301) 656-5900 ext. 111
Exhibition Inquiries
Training Course Inquiries
  • Stephanie Ko
    Sr. Manager, Lecture Education
    Tel: +1 (301) 656-5900 ext. 151
Registration Customer Care

Tel: +1 (301) 656-5900 ext. 115
[email protected]

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