Agenda

Initial 2018 Agenda Released!

The Cell & Gene Meeting on the Mesa is a three-day conference combining discussions between key opinion leaders, senior executives and top academic researchers. The program also includes 60+ presentations by the field’s most promising companies and 35 poster abstracts by leading scientists from around the globe.

See the initial agenda for the 2018 program below. Please check back soon for continuous updates!

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

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

DOING BUSINESS IN JAPAN WORKSHOP

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

DOING BUSINESS IN JAPAN WORKSHOP:
The Innovative Regulatory Pathways for Commercialization in Japan – Latest Progress and Outlook

Japan takes pride in its forward-looking regulatory system for regenerative medicine products that include cell and gene therapies, and has further upgraded its scheme by deploying the Sakigake Designation System since 2016. An introduction to the system was presented at last year’s Cell & Gene Meeting on the Mesa. This workshop will dive even further into the system this year, and will be augmented with firsthand experiences from three progressive clinical development companies from overseas.

Workshop Facilitator:
Kunihiko Suzuki, Vice Chairman, Forum for Innovative Regenerative Medicine (FIRM); Vice Chairman and Member of the Board, MEDINET Co.

7:15am – 7:20am | Welcome Remarks
Speaker:
Yuzo Toda, Chairman, Forum for Innovative Regenerative Medicine (FIRM)

7:20am – 8:35am | Presentations
Speakers:
Masahiro Uemura, Director, Bio-Industry Division, Commerce and Information Policy Bureau, Ministry of Economy, Trade and Industry (METI)
Kiyohito Nakai, Ph.D., Director, Medical Device Evaluation Division, Pharmaceutical Safety and Environmental Health Bureau, Ministry of Health, Labor and Welfare (MHLW)
Yoshiaki Maruyama, Ph.D., Review Director, Office of Cellular and Tissue-based Products, Pharmaceuticals and Medical Devices Agency (PMDA)
Gil Van Bokkelen, Ph.D., Chairman and CEO, Athersys
Douglas Losordo, M.D., EVP, Global Head of Research and Development and Chief Medical Officer, Caladrius Biosciences
David Lennon, Ph.D., President, AveXis

8:35am – 8:40am | Q&A

8:40am – 8:45am | Closing Remarks
Speaker:
Yoshitsugu Shitaka, Ph.D., Vice Chairman, Forum for Innovative Regenerative Medicine (FIRM); President, Astellas Institute for Regenerative Medicine (AIRM)

GENE-BASED MEDICINE DEVELOPMENT WORKSHOP

Learning Theater | 7:15am – 8:45am
Sponsored by Aldevron and MaxCyte

GENE-BASED MEDICINE DEVELOPMENT WORKSHOP
Assessing the challenges and implementing solutions to improve treatments

7:15am – 8:00am | Session 1: Gene Editing: Overcoming the Technical Challenges of CRISPR
Chair:
James Brown, Ph.D., VP, Corporate Development, Aldevron
Speakers:
Jim Burns, Ph.D., President and CEO, Casebia Therapeutics
Jennifer King, Ph.D., SVP, Business Development, Intellia Therapeutics
Bill Lundberg, M.D., Senior Advisor, CRISPR Therapeutics

8:00am – 8:45am | Session 2: CMC Solutions for Viral and Non-Viral Vector Gene Therapy
Chair:
Jessica Carmen, Ph.D., Director of Business Development, Cellular Therapy Partnerships, MaxCyte
Speakers:
Steven Howe, Ph.D., Director, Cell and Gene Therapy Process Research, GSK
Chris Mason, M.D., Ph.D., Chief Science Officer, AVROBIO
Kyriacos Mitrophanous, Ph.D., Chief Scientific Officer, Oxford BioMedica

OVERCOMING THE UNIQUE CHALLENGES OF CELL AND GENE THERAPY CLINICAL DEVELOPMENT: U.S. AND GLOBAL PERSPECTIVES

Ballroom 2 | 7:15am – 8:45am
Sponsored by IQVIA

OVERCOMING THE UNIQUE CHALLENGES OF CELL AND GENE THERAPY CLINICAL DEVELOPMENT: U.S. AND GLOBAL PERSPECTIVES

Streamlined, rigorous and effective clinical trials translate to greater and faster knowledge generation of a therapy’s risk-benefit profile. For cell and gene therapies, operational and regulatory complexities present a major hurdle to achieving this goal. This session will provide insight on the challenges developers of cell and gene therapies have encountered in conducting clinical trials including: manufacturing/supply chain, access to data, site selection, regulatory and start-up, patient recruitment, training, and other critical areas. Strategies that were successful in overcoming those challenges and the implications for strategic product development will be discussed. Perspectives from manufacturers and clinical trial sponsors, sites, and investigators based in the U.S., EU and APAC will be represented.

9:00am | GENERAL SESSION AND PARTNERING OPENS

9:00am – 9:15am

WELCOME REMARKS {Ballroom 1}

Speaker:

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

9:15am – 10:15am

PLENARY SESSION: CHARTING THE PATH – LESSONS FROM THE PIONEERS OF CELL & GENE THERAPY COMMERCIALIZATION {Ballroom 1}

Speakers:

David Lennon, Ph.D., President, AveXis

Ron Philip, SVP, Head of Global Commercial, Spark Therapeutics

Pascal Touchon, SVP and Global Head, Cell and Gene, Novartis Oncology

10:15am – 10:45am | MORNING BREAK
Sponsored by AD Biosciences, PeproTech and WiCell

10:45am – 12:00pm | CONCURRENT TRACKS

SPOTLIGHT SESSION: COMMERCIALIZING GENE THERAPIES FOR HEMOPHILIA {Ballroom 1}
10:45am – 11:15am

Speaker:
Jonathan Garen, Chief Business Officer, uniQure

COMPANY PRESENTATIONS {Ballroom 2}

10:45am – 11:15am

COMPANY PRESENTATIONS {Ballroom 1}

11:15am – 12:00pm

COMPANY PRESENTATIONS {Ballroom 2}

11:15am – 12:00pm

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

1:15pm – 3:30pm | CONCURRENT TRACKS

PANEL: NAVIGATING ACCEPTANCE, UPTAKE AND AFFORDABILITY ACROSS THE LIFECYCLE {Ballroom 1}
1:15pm – 2:15pm
Regenerative and advanced therapies are now entering a changing and increasingly restrictive global environment. There is movement towards value assessment across the lifecycle by both regulators, HTA agencies and payers in the U.S. and EU. There is also growing focus on the affordability of costly therapies. Success in the emerging environment requires focus on value demonstration across the entire lifecycle from the earliest stages of development thru post-market differentiation as new therapies become available. This session will bring together regulators, manufacturers and payers to discuss what “good” looks like in the new global healthcare environment.

Chair:
Eric Faulkner, VP, Precision and Transformative Technology Solutions, Value Demonstration, Access and Commercial, Evidera
Speakers:
Pamela Keith, Director, Oncology Reimbursement, Access, and Value Marketing, Juno Therapeutics, a Celgene company
Ron Philip, SVP, Head of Global Commercial, Spark Therapeutics
Pilar Pinilla-Dominguez, Senior Scientific Adviser, National Institute for Health and Care Excellence (NICE)
Richard Powell, M.D., Chief Medical Officer, MedPOINT Management

COMPANY PRESENTATIONS {Ballroom 2}

1:15pm – 2:15pm

COMPANY PRESENTATIONS {Ballroom 1}

2:15pm – 3:45pm

COMPANY PRESENTATIONS {Ballroom 2}

2:15pm – 3:45pm

3:45pm – 4:00pm | AFTERNOON BREAK
Sponsored by AD Biosciences, PeproTech and WiCell

4:00pm – 6:00pm | CONCURRENT TRACKS

PANEL: OPPORTUNITIES AND CHALLENGES IN RARE DISEASE {Ballroom 1}
4:00pm – 5:00pm
Cell and gene therapies have moved from promise to reality in 2018, with rare diseases often in the vanguard of these remarkable new living medicines. Many firms have built substantial franchises in rare diseases, but there is still great unmet need and the potential to displace and disrupt existing therapies. This panel will explore the opportunities and challenges in rare diseases across cell and gene therapy modalities as these therapies come to market; exploring technical challenges, licensing, adoption and reimbursement in healthcare systems and public or patient attitudes to this medical revolution.

Chair:
Keith Thompson, CEO, Cell and Gene Therapy Catapult
Speakers:
Geoff MacKay, President and CEO, AVROBIO
Mark Rothera, President and CEO, Orchard Therapeutics
Alvin Shih, CEO, Enzyvant

COMPANY PRESENTATIONS {Ballroom 2}

4:00pm – 5:00pm

COMPANY PRESENTATIONS {Ballroom 1}

5:00pm – 6:00pm

COMPANY PRESENTATIONS {Ballroom 2}

5:00pm – 6:00pm

6:00pm | PARTNERING CLOSES

6:30pm – 9:30pm | GALA RECEPTION
Sponsored by BlueRock Therapeutics and CCRM

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

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

PATIENT AND PUBLIC ATTITUDES TOWARD GENE THERAPY WORKSHOP

Magnolia Room | 7:15am – 8:45am

PATIENT AND PUBLIC ATTITUDES TOWARD GENE THERAPY WORKSHOP

7:15am – 7:20am | Welcome Remarks
Speaker:
Dena Ladd, Executive Director, Missouri Cures

7:20am – 7:40am | Perceptions and Misconceptions: What is the current landscape?
This discussion will address existing patient and public attitudes toward gene therapy, and examine gaps in awareness and understanding as well as ideas on tools to fill them going forward.
Speakers:
Michelle Berg, VP, Patient Affairs and Community Engagement, Abeona Therapeutics
Susan Sikora, Program Director, ARM Foundation for Cell & Gene Medicine

7:40am – 8:20am | Successful Partnerships Between Industry and Patient Organizations
This presentation will explore case studies on how industry can successfully partner with the patient communities they serve, and how the patient perspectives can be integrated into clinical research and educational programs.
Speakers:
Amy Fisher, Patient Advocacy Lead, Spark Therapeutics
Kristin Smedley, President and Co-Founder, Curing Retinal Blindness Foundation
Kimberly Trant, Director, Head of Patient Advocacy and Engagement, Audentes Therapeutics

8:20am – 8:40am | Externally-led Patient-Focused Drug Development Meetings: What are they and what are the benefits?
This portion of the workshop will discuss how these meetings are organized and the systematic approach they provide to ensure that patient’s experiences, perspectives, needs and priorities are meaningfully incorporated into drug development and evaluation.
Speaker:
Jen Farmer, Executive Director, Friedreich’s Ataxia Research Alliance

8:40am – 8:45am | Closing Remarks
Speaker:
Dena Ladd, Executive Director, Missouri Cures

READINESS STRATEGIES FOR CELL THERAPY COMMERCIAL MANUFACTURING

Learning Theater | 7:15am – 8:45am
Sponsored by: Hitachi Chemical Advanced Therapeutics Solutions

READINESS STRATEGIES FOR CELL THERAPY COMMERCIAL MANUFACTURING

After several decades of steady progress in cancer research, the cell-based immunotherapy approach has shown significant potential with two-major milestone CAR-T cell therapies recently being approved. While multiple clinical trials are underway and being planned, the industry continues to be faced with challenges on how to commercialize, and to properly prepare for sustainable commercialization of these promising therapies. Participants in this moderated workshop will discuss considerations for readiness strategies to help prepare and innovate for global, scalable, cost efficient cell therapy commercial manufacturing and manufacturing platforms.

7:15am – 7:20am | Welcome Remarks
Chair:
Robert Preti, Ph.D., President and CEO, Hitachi Chemical Advanced Therapeutics Solutions

7:20am – 7:45am | Section 1: Commercialization
Chair:
Sanjin Zvonić, Ph.D., Senior Director, Business Leader, Clinical and Commercial Manufacturing, Hitachi Chemical Advanced Therapeutics Solutions

7:45am – 8:10am | Section 2: Global Footprint
Chair:
Kazuchika Furuishi, Ph.D., Deputy General Manager, Hitachi Chemical Co.

8:10am – 8:35am | Section 3: Global Footprint
Chair:
David Kneen, VP, Cell Therapy, Invetech

8:35am – 8:45am | Closing Remarks
Chair:
Robert Preti, Ph.D., President and CEO, Hitachi Chemical Advanced Therapeutics Solutions

EVOLVING THE SUPPLY CHAIN FOR ADVANCED THERAPIES WORKSHOP

Ballroom 2 | 7:15am – 8:45am
Sponsored by World Courier

EVOLVING THE SUPPLY CHAIN FOR ADVANCED THERAPIES WORKSHOP
This session will use brief presentations to highlight the lessons learned within the supply chain, viewed through an industrial, academic, CMO and orchestration lens. These perspectives will then be used to discuss opportunities for the advanced therapy industry to learn from the past and create commercially viable logistics platforms.

This workshop will highlight topics such as how to manage:
• Cost by reducing complexity
• Vein to vein journey as a single inter-related system
• Scale up/out by utilizing a logistics platform early in the development pathway

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

7:25am – 7:35am | Presentation 1
Speaker:
Sven Kili, M.D., Principle, Sven Kili Consulting

7:35am – 7:45am | Presentation 2
Speaker:
Bruce Thompson, Ph.D., Senior Scientific Director, Therapeutic Products Program, Fred Hutchinson Cancer Center

7:45am – 7:45am | Presentation 3
Speaker:
Thomas Fellner, Ph.D., Head of Cell and Gene Therapy, Lonza

7:55am – 8:05am | Presentation 4
Speaker:
Amy DuRoss, CEO, Vineti

8:05am – 8:45am | Moderated Discussion w/ All Speakers

9:00am | GENERAL SESSION AND PARTNERING OPENS

9:00am – 9:15am

OVERVIEW OF THE ALLIANCE FOR REGENERATIVE MEDICINE’S INITIATIVES {Ballroom 1}

Speaker:

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

PANEL: LARGE PHARMA/BIOTECH’S LEADERSHIP ROLE {Ballroom 1}
9:15am – 10:15am

Speakers:
Brian Bronk, Ph.D., Head of External Innovation, Rare Diseases, Sanofi
Martin Golden, SVP, Head of Global Marketing Strategy, Astellas Pharma
Gabriele Proetzel, Ph.D., Director, Regenerative Medicine, Takeda Pharmaceuticals
Bob Smith, SVP, Global Gene Therapy Business, Pfizer

COMPANY PRESENTATIONS {Ballroom 2}

9:15am – 10:15am

10:15am – 10:45am | MORNING BREAK
Sponsored by AD Biosciences, PeproTech and WiCell

10:45am – 12:00pm | CONCURRENT TRACKS

FEATURED TALK {Ballroom 1}
10:45am – 11:00am

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

COMPANY PRESENTATIONS {Ballroom 2}

10:45am – 12:00pm

PANEL: RMAT REGULATORY CONVERGENCE: STARTING THE CONVERSATION {Ballroom 1}
11:00am – 12:00pm
Sponsored by Cell and Gene Therapy Catapult and Janssen R&D

Chair:
Melody Eble, Pharm.D., Director, Global Regulatory Affairs, Scientific Innovation Projects – Digital Technology and Regenerative Medicine, Janssen R&D
Speaker:
Peter Marks, M.D., Ph.D., Director, Center for Biologics Evaluation and Research, U.S. Food and Drug Administration (FDA)

12:00pm – 1:15pm | LUNCH

1:15pm – 2:45pm | CONCURRENT TRACKS

SPOTLIGHT SESSION: TISSUE ENGINEERING AND ORGAN TRANSPLANTATION {Ballroom 1}
1:15pm – 1:45pm
Tissue engineering holds the promise to eliminating the organ transplant waiting list, where over 120,000 patients currently wait, but how close are real solutions? Commercially, tissue engineering products have been limited to acellular or relatively thin constructs that lack vasculature and the complexity of functional tissue. This panel will discuss how recent advancements in decellularization technology and biologic scaffolds are addressing the vascular challenge. The speakers will describe the current pipeline of functional tissue engineered products including whole organs, how advancements in cell therapy have accelerated the path to market, the manufacturing, cell sourcing and distribution considerations when commercializing tissue engineering products and how recent regulatory programs – including RMAT – have fast-tracked the process.

Chair:
Jeff Ross, CEO, Miromatrix
Speakers:
Jim McGorry, CEO, Biostage
Jason Wertheim, M.D., Ph.D., Associate Professor of Surgery – Organ Transplantation, Northwestern University

COMPANY PRESENTATIONS {Ballroom 2}

1:15pm – 1:45pm

COMPANY PRESENTATIONS {Ballroom 1}

1:45pm – 2:45pm

COMPANY PRESENTATIONS {Ballroom 2}

1:45pm – 2:45pm

2:45pm – 3:15pm | AFTERNOON BREAK
Sponsored by AD Biosciences, PeproTech and WiCell

3:15pm – 5:30pm | CONCURRENT TRACKS

PANEL: NEXT GENERATION CELL AND GENE THERAPY IN ONCOLOGY {Ballroom 1}
3:15pm – 4:15pm

Speakers:
Stewart Abbot, Ph.D., Chief Scientific Officer, Adicet Bio
Maria Fardis, Ph.D., President and CEO, Iovance Biotherapeutics
Sanjaya Singh, Ph.D., VP and Global Head, Janssen BioTherapeutics, Janssen R&D, Janssen Pharmaceutical Companies of Johnson & Johnson
Jeffrey Walsh, Chief Financial and Strategy Officer, bluebird bio

COMPANY PRESENTATIONS {Ballroom 2}

3:15pm – 4:15pm

COMPANY PRESENTATIONS {Ballroom 1}

4:15pm – 5:30pm

COMPANY PRESENTATIONS {Ballroom 2}

4:15pm – 5:30pm

5:30pm | PARTNERING CLOSES

5:45pm | PUBLIC FORUM OPENS

6:00pm – 6:45pm

FEATURED PRESENTATION

Speaker:

Helen Blau, Ph.D., Donald E. and Delia B. Baxter Foundation Professor; Director, Baxter Laboratory for Stem Cell Biology, Stanford University

6:45pm – 7:30pm | RECEPTION AND NETWORKING
Sponsored by Mesoblast

7:30pm | PUBLIC FORUM CLOSES

7:00am – 8:00am | REGISTRATION AND BREAKFAST
Sponsored by Polyplus-transfection

8:00am | GENERAL SESSION

8:00am – 8:15am

WELCOME REMARKS

Speaker:

Alysson Muotri, Ph.D., Chair, Scientific Symposium Steering Committee; Director, Stem Cell Program; Professor, Department of Pediatrics and Cellular Molecular Medicine, UC San Diego

8:15am – 8:55am

MORNING KEYNOTE PRESENTATION
Sponsored by Homology Medicines

Keynote Speaker:

Ali Brivanlou, Ph.D., Robert and Harriet Heilbrunn Professor, Head of Laboratory of Stem Cell Biology and Molecular Embryology, The Rockefeller University

8:55am – 10:15am

PANEL: STEM CELL GENE THERAPY

Chair / Introduction By:

Stephanie Cherqui, Ph.D., Associate Professor, Department of Pediatrics, Division of Genetics, UC San Diego

Speakers:

Florian Eichler, M.D., Associate Professor of Neurology, Harvard Medical School; Assistant in Neurology, Massachusetts General Hospital

Matthew Porteus, M.D., Ph.D., Associate Professor of Pediatrics – Stem Cell Transplantation, Stanford University

Stephanie Cherqui, Ph.D., Associate Professor, Department of Pediatrics, Division of Genetics, UC San Diego

10:15am – 10:40am | MORNING BREAK
Sponsored by AD Biosciences, PeproTech and WiCell

10:15am – 10:40am | POSTER VIEWING
Sponsored by: Brammer Bio

10:40am – 12:00pm

PANEL: ADVANCED THERAPIES FOR SKELETAL MUSCLES

Chair / Introduction By:

Alessandra Sacco, Ph.D., Associate Professor, Development, Aging and Regeneration Program; Associate Dean of Curriculum, Graduate School of Biomedical Sciences, Sanford Burnham Prebys Medical Discovery Institute

Speakers:

Andrew Brack, Ph.D., Associate Professor, Orthopaedic Surgery, UC San Francisco

Rita Perlingeiro, Ph.D., Lillehei Professor in Stem Cell and Regenerative Cardiovascular Medicine, Lillehei Heart Institute; Professor of Medicine, Cardiovascular Division, University of Minnesota

Pier Lorenzo Puri, M.D., Ph.D., Professor, Development, Aging and Regeneration Program, Sanford Burnham Prebys Medical Discovery Institute

12:00pm – 1:15pm | LUNCH

12:00pm – 1:15pm | POSTER VIEWING
Sponsored by: Brammer Bio

1:15pm – 2:35pm

PANEL: GENE THERAPY FOR NEURODEGENERATIVE DISEASES

Chair / Introduction By:

Mark Tuszynski, M.D., Ph.D., Director, Center for Neural Repair; Professor, Department of Neurosciences, UC San Diego

Speakers:

Brian Kaspar, Ph.D., Principal Investigator, Center for Gene Therapy; Associate Professor, Department of Pediatrics and Department of Neuroscience, The Ohio State University; Chief Scientific Officer, AveXis

John Ravits, Ph.D., Professor of Clinical Neuroscience, UC San Diego

Mark Tuszynski, M.D., Ph.D., Director, Center for Neural Repair; Professor, Department of Neurosciences, UC San Diego

2:35pm – 3:00pm | AFTERNOON BREAK
Sponsored by AD Biosciences, PeproTech and WiCell

2:35pm – 3:00pm | POSTER VIEWING
Sponsored by: Brammer Bio

3:00pm – 4:20pm

PANEL: USING STEM CELLS TO STUDY NEUROPSYCHIATRIC DISORDERS

Chair / Introduction By:

Evan Snyder, M.D., Ph.D., Director, Center for Stem Cells and Regenerative Medicine; Professor, Human Genetics Program, Sanford Burnham Prebys Medical Discovery Institute

Speakers:

Guo-Li Ming, M.D., Ph.D., Professor of Neuroscience, Perelman School of Medicine, University of Pennsylvania

Kristen Brennand, Ph.D., New York Stem Cell Foundation – Robertson Investigator; Associate Professor, Departments of Genetics and Genomics, Neuroscience and Psychiatry, Icahn School of Medicine, Mount Sinai

Carol Marchetto, Ph.D., American Cancer Society Professor, Senior Staff Scientist, Laboratory of Genetics, Salk Institute for Biological Studies

4:20pm – 5:00pm

AFTERNOON KEYNOTE PRESENTATION

Keynote Speaker:

Kathy Niakan, Ph.D., Group Leader, The Francis Crick Institute

5:00pm – 6:30pm | NETWORKING RECEPTION

5:00pm – 6:30pm | POSTER VIEWING
Sponsored by: Brammer Bio

6:30pm | SCIENTIFIC SYMPOSIUM CLOSES