Agenda

2019 Agenda Now Available

The Cell & Gene Meeting on the Mesa is a three-day conference combining discussions between senior executives and top decision-makers in the industry. The program also includes 80+ presentations by the field’s most promising companies and extensive one-on-one partnering capabilities.

The agenda below will be updated continuously so please check back for new speaker announcements and added programming!

6:45am – 9:15am | REGISTRATION AND BREAKFAST
Sponsored by SCM Lifescience

7:15am – 8:45am | CONCURRENT WORKSHOPS

DOING BUSINESS IN JAPAN WORKSHOP

7:15am – 8:45am
In Partnership with the Forum for Innovative Regenerative Medicine (FIRM)

DOING BUSINESS IN JAPAN WORKSHOP
An Evolving Market – Innovations for Industrialization Platforms and the Latest Rise of Commercialization

Chair:
Masayuki Nomura, Ph.D., General Manager, Business Development and Licensing Department, Healthcare R&D Center, Corporate R&D, Asahi Kasei Corporation

7:15am – 7:20am | Welcome Remarks
Speaker:
Tetsuya Tanaka, Director, Bio-Industry Division, Ministry of Economy, Trade and Industry, The Government of Japan

7:20am – 7:35am | Evolving Market and Industrialization Platform for Innovative Regenerative Medicine in Japan
Speaker:
Yoshitsugu Shitaka, Ph.D., President, Astellas Institute for Regenerative Medicine (AIRM)

7:35am – 8:05am | Update on Regulatory Landscape of Regenerative Medicine
Speaker:
Yoshiaki Maruyama, Ph.D., Review Director, Office of Cellular and Tissue-based Products, Pharmaceuticals and Medical Devices Agency (PMDA)

8:05am – 8:25am | Japan’s First Gene Therapeutic Drug
Speaker:
Ei Yamada, Ph.D., President and CEO, AnGes, Inc.

8:25am – 8:45am | CAR-T Therapy Development in Japan
Speaker:
Yoshie Tsurumaki, Japan Head, SCM CAR-T, CAR-T Supply Chain Management Group, Cell and Gene Franchise, Oncology Division, Novartis Pharma K.K.

GENE-BASED MEDICINE DEVELOPMENT WORKSHOP

7:15am – 8:45am
Sponsored by Aldevron

GENE-BASED MEDICINE DEVELOPMENT WORKSHOP

7:15am – 8:00am | Session 1: It’s Never Too Soon – Considerations for Gene Therapy Manufacturing in the Early Stages of Development
The success of gene therapy has resulted in significant benefits for patients and tremendous growth in the number of programs in development. Many programs have been granted accelerated designations (RMAT, etc.) and have the potential to reach patients far faster than the traditional drug development timeline. Given the accelerated development and the complexity of gene therapy manufacturing, strategies to produce the therapy and manage the supply chain must be considered very early in the development process. This session will discuss how programs with limited time and resources can address manufacturing challenges and review some lessons learned from those in the field.

Chair:
Gregory MacMichael, Ph.D., President, CMC BioServices
Speakers:
Michelle Berg, President, GMP Nucleic Acids, Aldevron
Jeffrey Castelli, Ph.D., Chief Portfolio Officer and Head of Gene Therapy, Amicus Therapeutics
Maritza McIntyre, Ph.D., President, Advanced Therapies Partners
Susan Nichols, CEO, Falcon Therapeutics
Fraser Wright, Ph.D., Professor of Pediatrics, Center for Definitive and Curative Medicine (CDCM), Stanford Medicine

8:00am – 8:45am | Session 2: Customized Solutions for Genetic Medicines – Navigating the GMP Supplier Landscape
For most translational research units, bringing a genetic medicine to the clinic requires the support of several outside partners. Researchers need to procure custom GMP raw materials (plasmid DNA, guide RNA, Cas9 Nuclease) as well as outsourced GMP services (cell therapy manufacturing). All of these services carry a cost and significant timelines. From the end-user perspective, it is necessary to balance these costs and timelines in order to enter the clinic and begin treating patients on time. From the supplier perspective, it is important to innovate and provide clients with flexible products and services. The purpose of this panel is to bring these two perspectives together to inform the audience of best practices in outsourcing to avoid delays and best provide support to translational clients.

Speakers:
Delfi Krishna, Ph.D., Director, Cell and Gene Therapy Platform, R&D Strategy, Portfolio and Operations, GSK
Keith Thompson, CEO, Cell and Gene Therapy Catapult
Chy-Anh Tran, Director, Operations and cGMP Facilities, Stanford Laboratory for Cell and Gene Medicine

DEVELOPING A ROBUST EVIDENCE PACKAGE WORKSHOP

7:15am – 8:45am
Sponsored by IQVIA

DEVELOPING A ROBUST EVIDENCE PACKAGE WORKSHOP

7:15am – 8:00am | Session 1: Getting Prepared – Knowing What is Expected as Part of a Robust Evidence Package
When you think of bringing a new therapy to market, payers, providers, and other stakeholders are requiring robust information on effectiveness and real product use to understand the true value for patients and the wider healthcare system. Understanding what stakeholders are looking for as part of their review process is crucial to successfully develop and communicate your value message.

Chair:
Adrian McKemey, SVP Consulting Services, IQVIA

8:00 – 8:45am | Session 2: Getting it Done – Developing a Robust Evidence Package
Having insights on expectations and requirements from main stakeholders is the initial step to develop an action plan to solve the challenges at hand. This session will show you how to get the data needed, initiatives to put in place, and how to perform the necessary analytics to demonstrate results that are understood by your target audience.

Chair:
George Smith, Senior Director, Cell and Gene Therapy Center, IQVIA

9:00am | GENERAL SESSION AND PARTNERING OPENS

9:00am – 9:15am

WELCOME REMARKS {BlueRock Therapeutics Ballroom}

Speaker:

Janet Lambert, CEO, Alliance for Regenerative Medicine (ARM)

9:15am – 10:15am

PLENARY SESSION: THE CELL AND GENE THERAPY SECTOR’S COMMERCIAL TRAILBLAZERS {BlueRock Therapeutics Ballroom}

Chair:

Matthew Patterson, Chairman and CEO, Audentes Therapeutics

Speakers:

Vijay Chiruvolu, SVP, Global Process Development – Cell Therapy, Kite a Gilead company
David Lennon, Ph.D., President, AveXis
Rachelle Jacques, CEO, Enzyvant Therapeutics
Ron Philip, Chief Commercial Officer, Spark Therapeutics
Jeff Walsh, Chief Strategy Officer, bluebird bio

10:15am – 10:45am | MORNING BREAK
Sponsored by PeproTech, POMS and PTC Therapeutics

10:45am – 12:00pm | CONCURRENT TRACKS

FEATURED FIRESIDE CHAT: BREXIT’S EFFECT ON CELL AND GENE THERAPY REGULATION {BlueRock Therapeutics Ballroom}
10:45am – 11:15am

Chair:
Jacqueline Barry, Ph.D., Chief Clinical Officer, Cell and Gene Therapy Catapult
Speaker:
Christiane Niederlaender, Ph.D., Director, AMBR-Consulting; Former Senior Quality Assessor for Biologics, Medicines and Healthcare Products Regulatory Agency (MHRA); Former Member, Committee for Advanced Therapies (CAT), European Medicines Agency (EMA)

COMPANY PRESENTATIONS {Cognate Bioservices Ballroom}

10:45am – 11:00am
11:00am – 11:15am

COMPANY PRESENTATIONS {BlueRock Therapeutics Ballroom}

11:15am – 11:30am
11:30am – 11:45pm
11:45am – 12:00pm

COMPANY PRESENTATIONS {Cognate Bioservices Ballroom}

11:15am – 11:30am
11:30am – 11:45am
11:45am – 12:00pm

12:00pm – 1:15pm | LUNCH
Sponsored by: Dark Horse Consulting

1:15pm – 3:30pm | CONCURRENT TRACKS

PANEL: MYTH VS. REALITY – IMPLEMENTING PAYMENT ARRANGEMENTS FOR CELL AND GENE THERAPIES {BlueRock Therapeutics Ballroom}
1:15pm – 2:15pm

Chair:
Roger Longman, Founder and Chairman, Real Endpoints
Speakers:
John Coombs, Pharm.D., Patient Access Lead, U.S. CAR-T, Novartis
C. Bernie Good, M.D., Senior Medical Director, Center for Value-Based Pharmacy Initiatives, UPMC Health Plan

COMPANY PRESENTATIONS {Cognate Bioservices Ballroom}

1:15pm – 1:30pm
1:30pm – 1:45pm
1:45pm – 2:00pm
2:00pm – 2:15pm

COMPANY PRESENTATIONS {BlueRock Therapeutics Ballroom}

2:15pm – 2:30pm
2:30pm – 2:45pm
2:45pm – 3:00pm
3:00pm – 3:15pm
3:15pm – 3:30pm

COMPANY PRESENTATIONS {Cognate Bioservices Ballroom}

2:15pm – 2:30pm
2:30pm – 2:45pm
2:45pm – 3:00pm
3:00pm – 3:15pm
3:15pm – 3:30pm

3:30pm – 4:00pm | AFTERNOON BREAK
Sponsored by PeproTech, POMS and PTC Therapeutics

4:00pm – 6:00pm | CONCURRENT TRACKS

PANEL: CELL AND GENE THERAPY FOR NEUROLOGICAL PRODUCT INDICATIONS {BlueRock Therapeutics Ballroom}
4:00pm – 5:00pm

Chair:
Timothy Schroeder, Founder and CEO, CTI Clinical Trial & Consulting
Speakers:
Jon Garen, Chief Business Officer, uniQure
Sheila Mikhail, President and CEO, AskBio
Emile Nuwaysir, Ph.D., President and CEO, BlueRock Therapeutics
Andre Turenne, President and CEO, Voyager Therapeutics

COMPANY PRESENTATIONS {Cognate Bioservices Ballroom}

4:00pm – 4:15pm
4:15pm – 4:30pm
4:30pm – 4:45pm
4:45pm – 5:00pm

COMPANY PRESENTATIONS {BlueRock Therapeutics Ballroom}

5:00pm – 5:15pm
5:15pm – 5:30pm
5:30pm – 5:45pm
5:45pm – 6:00pm

COMPANY PRESENTATIONS {Cognate Bioservices Ballroom}

5:00pm – 5:15pm
5:15pm – 5:30pm
5:30pm – 5:45pm
5:45pm – 6:00pm

6:00pm | PROGRAM AND PARTNERING CLOSES

6:30pm – 9:30pm | NETWORKING BASH
Sponsored by CCRM

6:45am – 9:15am | REGISTRATION AND BREAKFAST
Sponsored by KBI Biopharma

7:15am – 8:45am | CONCURRENT WORKSHOPS

IDENTIFYING PITFALLS WITHIN GENE THERAPY DEVELOPMENT AND HOW TO OVERCOME THEM WORKSHOP

7:15am – 8:45am
Sponsored by Thermo Fisher Scientific

IDENTIFYING PITFALLS WITHIN GENE THERAPY DEVELOPMENT AND HOW TO OVERCOME THEM WORKSHOP
This session will cover recent advancements in gene therapy with insights from gene therapy developers and CDMOs on common pitfalls and how to avoid and overcome them. Join this interactive session to learn about their strategies and lessons learned. Topics discussed will range from insourcing vs. outsourcing, building reliable and scalable processes, and their vision for the future of gene therapy.

Chair:
Amy Butler, VP and General Manager Cell Biology, Thermo Fisher Scientific
Speakers:
Nicole O’Brien, Ph.D., Senior Director, Commercial Operations, Viral Vector Services, Brammer Bio
Robert Pietrusko, SVP, Regulatory and Q&A, Voyager Therapeutics

REAL WORLD EVIDENCE TO DRIVE ACCEPTANCE AND UPTAKE OF CELL AND GENE THERAPY: LESSONS AND BEST PRACTICES WORKSHOP

7:15am – 8:45am
Sponsored by: Evidera PPD

REAL WORLD EVIDENCE TO DRIVE ACCEPTANCE AND UPTAKE OF CELL AND GENE THERAPY: LESSONS AND BEST PRACTICES WORKSHOP
Cell and gene therapies offer transformative or curative potential, but you must prove it. Real world evidence (RWE) is a foundational tool to address uncertainty around asset value across the product lifecycle, from early development to expanded access. This is especially true in the case of therapies whose value proposition hinges on magnitude and duration of effect. Global regulatory agencies and payers are also weighing in and shifting RWE from “nice to know” to “got to have”, solidifying RWE as an essential to new cell and gene therapy strategy. Despite this momentum in RWE use, methods, stakeholder expectations, and “what good looks like” are still evolving. This workshop will discuss what RWE is, when and how to use it, and consider evolving lessons and best practices for cell and gene therapies.

Chair:
Eric Faulkner, VP, Real World Value and Strategy and Executive Director, Precision and Transformative Medicine Center of Excellence, Evidera
Speakers:
Jane Barlow, M.D., Senior Advisor, FoCUS Project, MIT Center for BioMedical Innovation/NEWDIGS
John Doyle, Dr.P.H., VP, Global Healthcare Innovation Lead, Pfizer

ALIGNING CLINICAL, MANUFACTURING, AND LOGISTICS PLATFORMS TO TREAT PATIENTS AT SCALE WORKSHOP

7:15am – 8:45am
Sponsored by World Courier

ALIGNING CLINICAL, MANUFACTURING, AND LOGISTICS PLATFORMS TO TREAT PATIENTS AT SCALE WORKSHOP
For advanced therapies to become a true industry, developers need to align the three platforms of manufacturing (closed and automated process), clinical (safe and efficacious) and logistics (seamless connection to patients). Without this alignment, companies risk creating a life changing therapy that can’t be made, or manufacturing a therapy that can’t be delivered. This workshop will look to understand the challenges in coordinating development across these platforms, providing lessons learned to help companies create therapies that can easily scale into globally viable commercial therapies.

7:15am – 7:25am | Welcome Remarks
Chair:
Simon Ellison, Cell and Gene Therapy Service Director, World Courier

7:25am – 7:35am | Presentation 1
Speaker:
Phil Vanek, Ph.D., General Manager, Cell and Gene Therapy Strategy, GE Healthcare

7:35am – 7:45am | Presentation 2
Speaker:
Dominic Clark, Ph.D., Global Head of Cell Therapy, HemaCare; Co-Chair, ISCT Process and Product Development Committee

7:45am – 7:55am | Presentation 3
Speaker:
Jacqueline Barry, Ph.D., Chief Clinical Officer, Cell and Gene Therapy Catapult

7:55am – 8:45am | Moderated Discussion with All Speakers

9:00am | GENERAL SESSION AND PARTNERING OPENS

9:00am – 9:15am

OVERVIEW OF THE ALLIANCE FOR REGENERATIVE MEDICINE’S INITIATIVES {BlueRock Therapeutics Ballroom}

Speaker:

Janet Lambert, CEO, Alliance for Regenerative Medicine (ARM)

9:15am – 10:15am | CONCURRENT TRACKS

PANEL: CLINICIANS WORKING WITH CAR-T THERAPIES {BlueRock Therapeutics Ballroom}
9:15am – 10:15am

Chair:
Gregory Hale, M.D., Senior Medical Director, Hematology and Oncology, Medpace
Speakers:
Prasad S. Adusumilli, M.D., Head, Solid Tumors Cell Therapy, Cellular Therapeutics Center; Vice Chair, Department of Surgery, Deputy Chief, Thoracic Surgery, Memorial Sloan Kettering Cancer Center
Dimitrios Tzachanis, M.D., Ph.D., Associate Clinical Professor of Medicine, UC San Diego Blood and Marrow Transplant Program
John A. Zaia, M.D., Aaron D. Miller and Edith Miller Chair in Gene Therapy; Director, Center for Gene Therapy, City of Hope

COMPANY PRESENTATIONS {Cognate Bioservices Ballroom}

9:15am – 9:30am
9:30am – 9:45am
9:45am – 10:00am
10:00am – 10:15am

10:15am – 10:45am | MORNING BREAK
Sponsored by PeproTech, POMS and PTC Therapeutics

10:45am – 12:00pm | CONCURRENT TRACKS

FEATURED SPEAKER {BlueRock Therapeutics Ballroom}
10:45am – 11:15am

Speaker:
Peter Marks, M.D., Ph.D., Director, Center for Biologics Evaluation and Research, U.S. Food and Drug Administration

COMPANY PRESENTATIONS {Cognate Bioservices Ballroom}

10:45am – 11:00am
11:00am – 11:15am

PANEL: WHEN DOES INVESTING IN CELL AND GENE THERAPY MAKE BUSINESS SENSE? {BlueRock Therapeutics Ballroom}
11:15am – 12:15pm
The cell and gene therapy industry is undergoing a critical inflection point as is evident in large financing deals, exciting clinical data and landmark regulatory approvals. However assets also face challenges such as high development costs and significant manufacturing complexities. Through a presentation and panel discussion, this session will define drivers of cost of development and cost of goods and provide a framework for assessing investment potential of cell and gene therapy assets.

Chair:
Delfi Krishna, Ph.D., Director, Cell and Gene Therapy Platform, R&D Strategy, Portfolio and Operations, GSK
Speakers:
Mitchell Finer, Ph.D, Chief Scientific Officer, ElevateBio; President, ElevateBio Base Camp

COMPANY PRESENTATIONS {Cognate Bioservices Ballroom}

11:15am – 11:30am
11:30am – 11:45am
11:45am – 12:00pm
12:00pm – 12:15pm

12:15pm – 1:30pm | LUNCH
Sponsored by Homology Medicines

1:30pm – 2:45pm | CONCURRENT TRACKS

SPOTLIGHT SESSION: THE EVOLUTION OF CELL AND GENE THERAPY IN CHINA {BlueRock Therapeutics Ballroom}
1:30pm – 2:00pm

Chair:
Amy Butler, Ph.D., VP and GM Cell Biology, Thermo Fisher Scientific
Speakers:
BG Rhee, Ph.D., CEO, SCM Lifescience
Felix Hsu, SVP and Global Head of Advanced Therapies, WuXi AppTec
Yuling Li, CEO, Zhejiang Innoforce Pharmaceuticals Co. Ltd.

COMPANY PRESENTATIONS {Cognate Bioservices Ballroom}

1:30pm – 1:45pm
1:45pm – 2:00pm

COMPANY PRESENTATIONS {BlueRock Therapeutics Ballroom}

2:00pm – 2:15pm
2:15pm – 2:30pm
2:30pm – 2:45pm

COMPANY PRESENTATIONS {Cognate Bioservices Ballroom}

2:00pm – 2:15pm
2:15pm – 2:30pm
2:30pm – 2:45pm

2:45pm – 3:15pm | AFTERNOON BREAK
Sponsored by PeproTech, POMS and PTC Therapeutics

3:15pm – 5:30pm | CONCURRENT TRACKS

PANEL: THE IMPACT OF UNIVERSAL DONOR CELLS AND iPSCs ON THE CELL THERAPY INDUSTRY {BlueRock Therapeutics Ballroom}
3:15pm – 4:15pm

Chair:
Robert Preti, Ph.D., President and CEO, Hitachi Chemical Advanced Therapeutics Solutions; GM, Hitachi Chemical Regenerative Medicine Business Sector
Speakers:
Stewart Abbot, Ph.D., Chief Operating and Scientific Officer, Adicet Bio
Usman Azam, M.D., President and CEO, Tmunity Therapeutics
Tim Lu, M.D., Ph.D., Co-Founder and CEO, Senti Biosciences
Bastiano Sanna, Ph.D., CEO, Semma Therapeutics
Dan Shoemaker, Ph.D., Chief Scientific Officer, Fate Therapeutics

COMPANY PRESENTATIONS {Cognate Bioservices Ballroom}

3:15pm – 3:30pm
3:30pm – 3:45pm
3:45pm – 4:00pm
4:00pm – 4:15pm

COMPANY PRESENTATIONS {BlueRock Therapeutics Ballroom}

4:15pm – 4:30pm
4:30pm – 4:45pm
4:45pm – 5:00pm
5:00pm – 5:15pm
5:15pm – 5:30pm
5:30pm – 5:45pm
5:45pm – 6:00pm

COMPANY PRESENTATIONS {Cognate Bioservices Ballroom}

4:15pm – 4:30pm
4:30pm – 4:45pm
4:45pm – 5:00pm
5:00pm – 5:15pm
5:15pm – 5:30pm
5:30pm – 5:45pm
5:45pm – 6:00pm

6:00pm | PROGRAM AND PARTNERING CLOSES

7:30am – 9:30am | REGISTRATION AND BREAKFAST
Sponsored by Polyplus-transfection

9:00am | GENERAL SESSION AND PARTNERING OPENS

9:00am – 9:05am

WELCOME REMARKS {BlueRock Therapeutics Ballroom}

Speaker:

Janet Lambert, CEO, Alliance for Regenerative Medicine (ARM)

9:15am – 9:30am | CONCURRENT TRACKS

FEATURED SPEAKERS {BlueRock Therapeutics Ballroom}
9:05am – 9:30am

Speakers:
Emily Whitehead, Co-Founder, Emily Whitehead Foundation
Tom Whitehead, Co-Founder, Emily Whitehead Foundation

COMPANY PRESENTATIONS {Cognate Bioservices Ballroom}

9:15am – 9:30am

9:30am – 10:30am | CONCURRENT TRACKS

PANEL: A LOOK INTO THE CRYSTAL BALL – WHAT DOES THE FUTURE HOLD FOR GENE EDITING? {BlueRock Therapeutics Ballroom}
9:30am – 10:30am
This dynamic panel discussion featuring executives from leading gene editing companies will explore the major milestones and advances anticipated in the near term (12 month), midterm (5 year), and long term (10+ year) timeframes for these promising therapies. This session will also consider how the rapidly evolving climate surrounding these technologies will potentially impact the future of the sector’s success.

Chair:
Robert Smith, SVP, Global Gene Therapy Business, Pfizer
Speakers:
Jim Burns, Ph.D., President and CEO, Casebia Therapeutics
Cindy Collins, CEO, Editas Medicine
Michael Dombeck, SVP, Corporate Development, Precision BioSciences
Sandy Macrae, Ph.D., President and CEO, Sangamo Therapeutics
Arthur Tzianabos, Ph.D., President and CEO, Homology Medicines

COMPANY PRESENTATIONS {Cognate Bioservices Ballroom}

9:30am – 9:45am
9:45am – 10:00am
10:00am – 10:15am
10:15am – 10:30am

10:30am – 10:45am | MORNING BREAK
Sponsored by PeproTech, POMS and PTC Therapeutics

10:45am – 12:15pm | CONCURRENT TRACKS

SPOTLIGHT SESSION: STATE INNOVATION IN CELL AND GENE THERAPY MARKET ACCESS {BlueRock Therapeutics Ballroom}
10:45am – 11:15am

COMPANY PRESENTATIONS {Cognate Bioservices Ballroom}

PANEL: INNOVATION AROUND MANUFACTURING TECHNOLOGIES {BlueRock Therapeutics Ballroom}
11:15am – 12:15pm

Chair:
Kelvin Lee, Ph.D., Institute Director, National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL)
Speakers:
Nina Bauer, Ph.D., Chief Commercial Officer, FloDesign Sonics
Jian Irish, Ph.D., SVP, Global Head of Manufacturing, Kite Pharma, a Gilead Company
Jerry Keybl, Ph.D., Head of Cell and Gene Therapy Manufacturing, MilliporeSigma
Alberto Santagostino, Head, Cell and Gene Technologies Business Unit, Lonza Pharma and Biotech
Jason Slingsby, Ph.D., Chief Business Officer, Oxford Biomedica

COMPANY PRESENTATIONS {Cognate Bioservices Ballroom}

11:15am – 11:30am
11:30am – 11:45am
11:45am – 12:00pm
12:00pm – 12:15pm

12:15pm – 1:30pm | LUNCH
Sponsored by Paragon Bioservices

1:30pm | CONFERENCE CLOSES

By using this website you agree to accept our Privacy Policy and Terms & Conditions