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2017 PDA Cell and Gene Therapy Conference

The Journey of Cell & Gene Therapy - Bringing Science to Reality
Dec 05 - Dec 06, 2017 |
Dec 06, 2017 |
Manchester Grand Hyatt San Diego | San Diego, CA
  • Conference
  • Western Hemisphere Events
Exhibition: December 5-6
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As a follow-up to the successful Cell & Gene Therapy Workshop in April 2017, this Conference will expand upon the latest and most comprehensive information on a broad range of topics related to the field. This Conference is the ideal forum for sharing best practices and learning how industry is applying novel approaches for development and commercialization of pharmaceutical and biopharmaceutical products.

Plenary sessions focus on current and future applications for these emerging therapeutic entities, including the emerging fields of immunotherapy - gene and cell-based therapies. Emphasis is placed on science and technologies needed to bring these innovative products to market and ultimately to the patient. Additional plenary sessions further explore current and future trends in process development and manufacturing, including topics on next generation processing and facilities, application of big data for process design and optimization and accelerating the industry response to healthcare needs. 

Don’t miss out on this important learning opportunity. Make plans now to attend the 2017 PDA Cell & Gene Therapy Conference.

Learning Objectives

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

  • Determine manufacturing and quality requirements for immunotherapies, gene and cell therapy products
  • Describe continuous manufacturing applications and flexible facility designs of the future
  • Demonstrate how big data can be applied to design and optimize manufacturing processes
  • Define strategies to accelerate new products to the market
  • Interpret the latest trends in microbiological and adventitious agent control strategies
  • Identify advanced analytical approaches that can be applied for quality control and real time release
  • Explain delivery system design and manufacturing logistics for patient-centered therapies and precision medicine
  • Summarize best practices for identifying and introducing new technologies

Who Should Attend

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

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

Contact

Brooke Schneider, CMP
Manager, Programs
Tel: (301) 656-5900 ext. 111
Email: schneider@pda.org

Registration Customer Care
Tel: +1 (301) 656-5900 ext. 115
Email: registration@pda.org

Exhibition Inquiries

David Hall
Vice President, Sales 
Tel: +1 (240) 688- 4405
Email: hall@pda.org

Tuesday, December 5

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

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

8:00 a.m. – 8:15 a.m.
Welcome and Opening Remarks from Conference Co–Chair 
Michael N. Blackton, MBA,
Vice President, Quality, CMC, Adaptimmune LLC

8:15 a.m. – 9:45 a.m.
P1: Gene Therapy: Bridging Healthcare Innovation from Academia to Industry
Moderator: Michael N. Blackton, MBA,
Vice President, Quality, CMC, Adaptimmune LLC

Session Description: Gene therapy is just beginning to deliver on the promise of understanding and treating a variety of medical conditions with amazing results. As clinical trials continue and more knowledge is gained, options for potential treatments increase. This session will address the current progress toward comprehensive treatment options and opportunities in the gene therapy to help patients.

8:15 a.m. – 8:45 a.m.
Gene Therapy to Deliver Therapeutic Proteins and Edit Genes
James Wilson, MD, PhD
, Director of the Gene Therapy, University of Pennsylvania

8:45 a.m. – 9:15 a.m.
Developing Gene Therapies for Rare Diseases, a History of Synergy Between Academia and Industry
Federico Mingozzi, PhD,
Chief Scientific Officer, Spark Therapeutics

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

9:30 a.m. – 6:00 p.m. 
Exhibit Hall Open

9:45 a.m. – 10:30 a.m.
Refreshment Break in Exhibit Area

10:30 a.m. – 12:00 p.m.
P2: Navigating the Regulatory Environment, Understanding the Challenges, and Sharing Solutions
Moderator: Jean Stanton, MS,
Director, J&J Regulatory Compliance, Johnson & Johnson

Session Description: As with any drug, cell and gene therapies must meet the approval standard of being safe and effective. However, there are unique features and challenges for sponsors of these products. Regulatory filing strategies, considered routine for conventional biologics, demand a closer look when developing these biologically complex products. This session will present regulator and sponsor perspectives on the unique regulatory challenges sponsors face and how they can be managed.

10:30 a.m. – 11:00 a.m.
Process and Technology Development of Advanced Therapies, an Industry Perspective
Michael Paglia, MS,
Senior Director CMC Operations, Oncorus

11:00 a.m. – 11:30 a. m.
Understanding the Regulatory Challenges and Expectations, a Product Reviewer's Perspective
Zenobia F. Taraporewala, PhD,
CMC (Product) Reviewer, Gene Therapies Branch, CBER, FDA

11:30 a.m. – 12:00 p.m.
Questions and Answers/Discussion

12:00 p.m. – 1:15 p.m.
Lunch in Exhibit Area

1:15 p.m. – 2:45 p.m.
P3: Development of a Process Control Strategy
Moderator: Diane I. Blumenthal,
Head, Technical Operations, Spark Therapeutics

Session Description: The maturing of cell and gene therapy products provides an opportunity to serve patients with options for treatment where none have previously existed. Like all emerging technologies in the biopharmaceutical arena, this presents the need to challenge well-established paradigms and to develop standards that fit the unique nature of this technology. Clinical acceleration can lead to key elements of process understanding lagging behind at a time when a control strategy must be defined. This session will provide an inside look at ways to redefine the approach for the development of a process control strategy that will ensure consistency of process performance and deliver product that meets quality requirements. Examples of challenges and how to overcome them will be presented.

1:15 p.m. – 1:45 p.m.
Developing Control Strategy for Gene and Cell Therapies – Journey of Unique Challenges
Julia O'Neill, MS,
CMC Statistician, Tunnell Consulting, Inc.

1:45 p.m. – 2:15 p.m.
Application of Risk Assessments to the Establishment of a Control Strategy for Cell Therapy Products
Darius Pillsbury,
Head of Quality Lifecycle Management, Adaptimmune LLC

2:15 p.m. – 2:45 p.m.
Questions and Answers/Discussion

2:45 p.m. – 3:30 p.m.
Refreshment Break in Exhibit Area

3:30 p.m. – 5:00 p.m.
P4: Leveraging Big Data to Speed Cell and Gene Therapy Product Development
Moderator: Brian J. Hawkins, PhD,
Senior Application Scientist, BioLife Solutions

Session Description: Rapid advances in 'omics' technologies allow scientists and clinicians to accurately assess biological variables at an unprecedented scale. The large data sets generated by these technologies can speed the development of cell and gene therapy products from the benchtop to the clinic. This session will focus on the application of 'big data' as it applies to both manufacturing and patient selection.

3:30 p.m. – 4:00 p.m.
Genomics and Gene Editing Tools in Commercializing Pluripotent Stem Cell Therapies
Robert Deans, PhD,
Chief Technology Officer, BlueRock Therapeutics

4:00 p.m. – 4:30 p.m.
Novel Feature Selection Strategies for Enhanced Predictive Modeling and Deep Learning in the Biosciences
Tom Chittenden, PhD,
VP Statistical Sciences/Founding Director, Advanced AI Research Laboratory, WuXi NextCODE

4:30 p.m. – 5:00 p.m.
Questions and Answers/Discussion

5:00 p.m. – 6:00 p.m.
Networking Reception in Exhibit Area

Wednesday, December 6

7:15 a.m. – 4:00 p.m.
Registration Open

7:15 a.m. – 8:15 a.m.
Continental Breakfast

8:15 a.m. – 9:45 a.m.
P5: Process Validation and Process Comparability for Cell and Gene Therapy Products
Moderator: Vijay Chiruvolu, PhD,
Vice President, Process Sciences & Engineering, Kite Pharma

Session Description: Although there is no specific regulatory guidance for comparability and process validation of cell and gene therapy products, concepts from existing guidance for biopharmaceuticals can be applied. Because of the complexity of cell and gene therapy products and difficulties in completely characterizing them, the concept of "process is the product" becomes important. This session will focus on some of the challenges and solutions related to conducting comparability studies and process validation of cell and gene therapy products.

8:15 a.m. – 8:45 a.m.
Navigating the Challenges of Designing Aseptic Process Simulations for Cell and Gene Therapy Products
Marsha Steed (Hardiman),
Senior Consultant, Valsource, LLC

8:45 a.m. – 9:15 a.m.
Comparability Studies for Autologous Cell Therapy
Yoko Momonoi,
Senior Fellow, Novartis Pharma AG

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

 

9:30 a.m. – 3:45 p.m.
Exhibit Hall Open

9:45 a.m. – 10:30 a.m.
Refreshment Break in Exhibit Area

10:30 a.m. – 12:00 p.m.
P6: Facility, Process Design, and Containment
Moderator: Maik W. Jornitz, MS,
CEO, G–Con Manufacturing, Inc.

Session Description: Cell and gene therapies have unique process and facility design needs, most importantly robust containment infrastructures as the aseptic processing requirements are rigorous. In addition, since these processes grow over time, it is necessary to scale processes and processing spaces without interrupting the existing space, which has to be contemplated within the design of infrastructure. Questions prevail regarding whether process centers need to be centralized or decentralized, requiring the facilities to be flexible, and potentially relocatable. The presentations within this session will use case examples to address the points outlined above.

10:30 a.m. – 11:00 a.m.
Design Elements of Autologous Cellular Immunotherapy Manufacturing Plant
Ernest A. Bognar,
Vice President Operations, Gradalis Inc.

11:00 a.m. – 11:30 a.m.
Trends in the Development, Optimization, and Commercialization of Immunotherapy Processing Facilities
George Wiker,
Executive Director, AES Clean Technology, Inc.

11:30 a.m. – 12:00 p.m.
Questions and Answers/Discussion

12:00 p.m. – 1:30 p.m.
Lunch in Exhibit Area

1:30 p.m. – 3:00 p.m.
P7: Quality Systems and Compliance
Moderator:
Donald Startt, Associate Director, Quality, REGENXBIO Inc.

Session Description: Compliance and quality systems play a critical role in the development and manufacture of medicines. The area of cell and gene therapy is relatively new when compared to small molecules or the more established biologics such as monoclonal antibodies. The regulatory pathways, manufacturing processes, and analytics are not as well defined and as such quality systems must be adaptable to a rapidly changing/unknown landscape. Additionally, many of the companies and organizations developing these products are often small or academic-based. They may not have fully developed quality systems and may leverage numerous external services such as manufacturing and testing. The purpose of this session is to hear different perspectives on quality systems and what it means to be compliant in this new and dynamic area.

1:30 p.m. – 2:00 p.m.
Implementing Quality Systems with an Externalized Model for Manufacturing and Testing
Kimberly A. Carnes,
Associate Director, Quality Systems, REGENXBIO Inc.

2:00 p.m. – 2:30 p.m.
Phase Appropriate Quality System Requirement for Cellular Therapy Manufacturing
Michael O. Skidmore,
Consultant, Pharmaceutical Quality Consulting, Inc.

2:30 p.m. – 3:00 p.m.
Questions and Answers/Discussion

3:00 p.m. – 3:45 p.m.
Refreshment Break in Exhibit Area

3:45 p.m. – 5:15 p.m.
P8: Next-Generation Approaches in Gene and Cell Therapies
Moderator: Michael N. Blackton, MBA, 
Vice President, Quality, CMC, Adaptimmune LLC

Session Description: The knowledge gained within the field over the past several decades provides much hope for the future of gene and cell therapies. Unfortunately, the journey of an idea from concept to the real world of healthcare is often long and arduous as less than 10 percent of discoveries in the field of medicine make it to the marketplace. The exciting possibility of treating many genetic and infectious disorders is now a reality with the success of clinical trials, novel engineering, and the recent discoveries of tools such as miRNAs and CRISPR/Cas9. Technical hurdles, such as the challenges around how DNA is incorporated into a gene, have been reduced through a new generation of vectors, and stem cell companies are increasingly excited about therapies that are safe and efficacious in treating diseases that are currently untreatable. This session examines the future of gene and cell therapy and explores approaches to overcoming obstacles for progressing discoveries into new devices, drugs, or therapies.

3:45 p.m. – 4:15 p.m.
CRISPRs and Gene Editing: Laboratory Use and Clinical Future
Industry Representative Invited

4:15 p.m. – 4:45 p.m.
Accelerating the Translation of Scientific Discoveries into Health Benefits for Patients
Don Healey, PhD, 
SVP Operations and Site Head, KBI Biopharma

4:45 p.m. – 5:15 p.m.
Questions and Answers/Discussion

5:15 p.m.
Closing Remarks from Conference Co-Chair
Vijay Chiruvolu, PhD, Vice President, Process Sciences & Engineering, Kite Pharma


Conference Registration | December 5-6, 2017

Member

Before September 25, 2017
$1,895

September 25, 2017 – Oct. 20, 2017
$2,095

After Oct. 20, 2017
$2,295

Non-member

Before September 25, 2017
$2,174

September 25, 2017 – Oct. 20, 2017
$2,374

After Oct. 20, 2017
$2,574


Government/Health Authority/Academic

Member

Before September 25, 2017
$700

September 25, 2017 – Oct. 20, 2017
$700

After Oct. 20, 2017
$700

Non-member*

Before September 25, 2017
$800

September 25, 2017 – Oct. 20, 2017
$800

After Oct. 20, 2017
$800


Student

Member

Before September 25, 2017
$280

September 25, 2017 – Oct. 20, 2017
$280

After Oct. 20, 2017
$280

Non-member*

Before September 25, 2017
$310

September 25, 2017 – Oct. 20, 2017
$310

After Oct. 20, 2017
$310


All prices in US dollars.

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

Please note: In order to receive the prevailing registration rate, your registration(s) must be received by PDA by 5:00 p.m. ET on or before the date noted.

Please read PDA's confirmation, substitution, refund and photo release policies for this Conference.

Group Registration Discount

Register 4 people from the same organization as a group (at the same time) for the Conference and receive the 5th registration free. Other discounts cannot be applied.

Special Dietary Requirements

Please let us know if you have any special dietary requirement we may accommodate. Please contact us at registration@pda.org for help.

CONFIRMATION: A letter of confirmation will be sent to you once payment is received. You must have this written confirmation to be considered enrolled in a PDA event. Please allow one week for receipt of confirmation letter. If you have submitted a purchase order or requested an invoice please be advised that a credit card guarantee is needed. Please be advised that if your payment or written cancelation notice is not received by October 6, 2017 your credit card will be charged the prevailing rate.

SUBSTITUTIONS: If you are unable to attend, substitutions can be made at any time, including on site at the prevailing rate. If you are a non-member substituting for a member, you will be required to pay the difference non-member fee. If you are pre-registering as a substitute attendee, indicate this on the registration form.

REFUNDS: Refund requests must be in writing and faxed to +1 (301) 986-1093. (Emails and phone messages are not accepted). If your written request in received on or before October 6, 2017, you will receive a full refund minus a $200 processing fee. After that time, no refunds or credit requests will be approved. On-site registrants are not guaranteed to receive Conference materials until all advanced registered attendees receive them. PDA reserves the right to modify the material or speakers/instructors without notice, or cancel an event. If an event must be canceled, registrants will be notified by PDA in writing as soon as possible and will receive a full refund. PDA will not be responsible for airfare penalties or other costs incurred due to cancelation. For more details, contact PDA at info@pda.org or +1 (301) 656-5900.

PLEASE NOTE: Photo ID will be required in order to pick up badge materials on-site. This important security procedure will prevent anyone other than the registrant from picking up their badge and materials.

RECORDING/PHOTO RELEASE: By registering for these events, I authorize PDA to record and photograph me and to use the recordings/photographs in all formats and media for any purpose, including for education, marketing and trade purposes. I hereby release PDA from all claims arising out of the use of the recordings/photographs, including without limitation all claims for compensation, libel, invasion of privacy or violation of copyright ownership. Recordings are prohibited at all PDA Conferences.

Manchester Grand Hyatt San Diego
1 Market Place
San Diego, CA 92101
Phone: +1 (619) 232-1234

Rate: Single: $249.00, plus applicable state and local taxes.

Cut Off Date: Monday, November 6, 2017 (Rooms must be secured by this date in order to receive the PDA rate). Rates are guaranteed until the PDA block of rooms are sold out on a first come basis.

Hotel Accommodations

To make reservations at the Manchester Grand Hyatt San Diego, please call the Hotel at +1 (619) 232-1234 and reference PDA's Cell and Gene Therapy Conference to receive the PDA group rate. Check in time is 4:00 p.m., and check out time is 12:00 noon.

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 cancel 48 hours prior to the date of arrival by 3 p.m. to avoid cancellation charges.

Amenities

Manchester Grand Hyatt San Diego is ideally situated on San Diego Bay, and the city's popular Seaport Village. The hotel offers a spectacular waterfront resort-like setting, complete with shopping, dining, and entertainment venues that can be found steps away in the Gaslamp Quarter. All 1,628 guest rooms and suites provide complimentary high-speed Internet, Hyatt Grand Bed™, coffee makers, hair dryers, iron and an ironing board.

Travel

Area Airports
San Diego International Airport (SAN) is approximately 2.7 miles from the hotel and all major carriers service this airport.

Ground Transportation
Taxi fares from San Diego International Airport (SAN) are approximately $13 one way, not including tip, and will take about 10 minutes. There are several San Diego airport shuttle companies available at the Airport for an approximate cost of $8.00 per person.

Driving
Manchester Grand Hyatt San Diego hotel is located on 1 Market Place near Seaport Village.

Hotel Parking
$32.00 / Daily Self-Parking
$47.00 / Daily Valet Parking

High-impact, cost-effective sponsorship and exhibition packages are available for the 2017 PDA Cell and Gene Therapy Conference. Gain on-site exposure and connect with industry experts from manufacturing, product development, laboratory science, engineering, validation, and, technical services. Exhibit at or sponsor this Conference to align your company with industry leaders and world-class content.

Comprehensive sponsorship packages will provide your company the opportunity to strengthen brand image, increase visibility and reinforce its commitment to the visual inspection arena. Sponsorships are available for lanyards, tote bags, notepads, pens, refreshment breaks, lunch, the Networking Reception and more.

For more information about exhibit and sponsorship opportunities, please contact:
David Hall, Vice President, Sales
Tel: +1 (301) 760-7373 | Cell: +1 (240) 688-4405 | Fax: +1 (301) 986-0296 | Email: hall@pda.org

Closed
https://www.pda.org/global-event-calendar/events-misc-info/onsite-registration

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