Agenda

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 poster abstracts by 40 leading scientists from around the globe.

This agenda is preliminary. New speakers will be added frequently so check back soon for updates!

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7:00am – 9:30am | REGISTRATION & BREAKFAST
7:15am – 8:45am | CONCURRENT WORKSHOPS

DOING BUSINESS IN JAPAN WORKSHOP

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

DOING BUSINESS IN JAPAN WORKSHOP
Implications of the New Regulatory Framework

GENE THERAPY MANUFACTURING WORKSHOP

7:15am – 8:45am | Learning Theater
Sponsored by Brammer Bio & MaxCyte

GENE THERAPY MANUFACTURING WORKSHOP
Strategies for BioManufacturing Viral-based Gene Therapy Vectors – Early Stage Development Through Clinical Production

Workshop Topics to Include:

  • Manufacturing in early development – construct optimization and comparative analysis, assay development, validation
  • AAV and lentivirus process development – scale-up, quality, and the need for standards
  • Establishing novel stable producer cell lines -strategies and current state of the science
  • In house versus out-sourced bioproduction – how and when to transfer the technology, best practices for coordination and demonstration of comparability, advantages and disadvantages of outsource v in-house manufacturing, current problems with the supply chain, current efforts of / by therapeutic companies to secure short, mid and long term supply
  • Toward large scale clinical and commercial production – scale up, vector analytics, COGS, regulatory requirements; compare and contrast requirements for ‘direct use’ v ‘use as reagent in cell-based gene therapy’
  • How (if at all) are gene editing technologies disruptive to the use of viral-based gene vectors?  What should therapeutics companies be thinking about?  What should vector manufacturing firms be preparing for/thinking about?

Chair:
Doug Doerfler, President and CEO, MaxCyte

Speakers:
James Brown, Ph.D., VP, Corporate Development, Aldevron
Matthew Caple, Ph.D., VP, Site Operations, Brammer Bio
Mike Kelly, Ph.D., Director, Asset Leader, Gene Therapy, Biogen
Michael Roberts, Ph.D., Chief Scientific Officer, Synpromics

9:00am | GENERAL SESSION & PARTNERING OPENS
9:00am – 9:15am WELCOME REMARKS
Speaker:
Robert Preti, Ph.D., Chairman, Alliance for Regenerative Medicine; President, PCT, A Caladrius Company; SVP, Manufacturing and Technical Operations, Caladrius Biosciences
9:15am – 10:15am PLENARY SESSION: CLINICAL PROGRESS IN IMMUNO-ONCOLOGY
Chair:
Amy Butler, Ph.D., VP and General Manager, Cell Biology, Thermo Fisher Scientific
Speakers:
David Chang, M.D., Ph.D., Chief Medical Officer and EVP, Research and Development, Kite Pharma
Thomas Farrell, President and CEO, Bellicum Pharmaceuticals
Christian Homsy, M.D., CEO, Celyad
10:15am – 10:45am | MORNING BREAK
10:45am – 12:00pm | CONCURRENT TRACKS
GENE EDITING INTERVIEW {Ballroom 1}
10:45am – 11:15am
Chair:
Blythe Thomson, M.D., Senior Director, Medical Affairs, Hematology and Oncology, Medpace
Speaker:
Alexandra Glucksmann, Ph.D., Chief Operating Officer, Editas Medicine
COMPANY PRESENTATIONS
11:15am – 11:30am Celyad
11:30am – 11:45am Mesoblast
11:45am – 12:00pm Capricor Therapeutics
COMPANY PRESENTATIONS {Ballroom 2}
10:45am – 11:00am Nohla Therapeutics
11:00am – 11:15am GenVec
11:15am – 11:30am Abeona Therapeutics
11:30am – 11:45am NovaHep
11:45am – 12:00pm Juventas Therapeutics
12:00pm – 1:15pm | LUNCH
1:15pm – 3:30pm | CONCURRENT TRACKS
PANEL: THE IMPACT OF A NEW HEALTHCARE MODEL: WHAT DO PAYERS THINK OF GENE THERAPIES? {Ballroom 1}
1:15pm – 2:15pm
Gene therapies offer potentially curative treatments to alleviate unmet need and improve patient outcomes. Cost and uncertain financing, however, present challenges to patient access. This panel will discuss the unique challenges presented by gene therapies with the current financing and reimbursement mechanisms and processes. As well, the panel will explore the impact these potentially curative therapies have on the current healthcare model designed to manage chronic diseases. This session will elevate the discussion from theoretical to the more practical considerations within existing or proposed financing models.
Sponsored by Quintiles
Chair:
John Doyle, Dr.P.H., SVP, Advisory Services, Quintiles
Speakers:
Nick Crabb, Ph.D., Programme Director – Scientific Affairs, Centre for Health Technology Evaluation, National Institute for Health and Care Excellence (NICE)
Michael Fine, M.D., Medical Director, Health Net of California
Mark Trusheim, Founder and President, Co-Bio Consulting; Visiting Scientist, MIT Sloan School of Management
COMPANY PRESENTATIONS {Ballroom 2}
1:15pm – 1:30pm MaxCyte
1:30pm – 1:45pm 4D Molecular Therapeutics
1:45pm – 2:00pm Synpromics
2:00pm – 2:15pm ToolGen
COMPANY PRESENTATIONS {Ballroom 1}
2:15pm – 2:30pm ReNeuron
2:30pm – 2:45pm Oxford BioMedica
2:45pm – 3:00pm AGTC
3:00pm – 3:15pm GenSight Biologics
3:15pm – 3:30pm Audentes Therapeutics
COMPANY PRESENTATIONS {Ballroom 2}
2:15pm – 2:30pm Precision Biosciences
2:30pm – 2:45pm Universal Cells
2:45pm – 3:00pm Commence Bio
3:00pm – 3:15pm TxCell
3:15pm – 3:30pm Terumo BCT
3:30pm – 4:00pm | AFTERNOON BREAK
4:00pm – 6:00pm | CONCURRENT TRACKS
PANEL: FINANCING GENE & CELL THERAPIES {Ballroom 1}
4:00pm – 5:00pm
COMPANY PRESENTATIONS {Ballroom 2}
4:00pm – 4:15pm Irvine Scientific
4:15pm – 4:30pm StemBioSys
4:30pm – 4:45pm CTI Clinical Trial & Consulting Services
4:45pm – 5:00pm Vitruvian Networks
COMPANY PRESENTATIONS {Ballroom 1}
5:00pm – 5:15pm Organovo
5:15pm – 5:30pm Histogenics
5:30pm – 5:45pm Osiris Therapeutics
5:45pm – 6:00pm Vericel Corporation
COMPANY PRESENTATIONS {Ballroom 2}
5:00pm – 5:15pm Cryoport Systems
5:15pm – 5:30pm Regencor
5:30pm – 5:45pm Cells for Cells
5:45pm – 6:00pm Stem Cell & Device Laboratory
6:00pm | PARTNERING CLOSES
6:30pm – 9:30pm | GALA RECEPTION
Sponsored by CCRM & Dohmen Life Science Services

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7:00am – 9:30am | REGISTRATION & BREAKFAST
7:15am – 8:45am | CONCURRENT WORKSHOPS

ADDRESSING UNCERTAINTY IN REGENERATIVE MEDICINE VALUE DEMONSTRATION

7:15am – 8:45am | Magnolia Room

ADDRESSING UNCERTAINTY IN REGENERATIVE MEDICINE VALUE DEMONSTRATION
What is Mission Critical vs. Mission Impossible?

Early payer evaluations of regenerative therapies have identified uncertainty in value demonstration as the most important global acceptance barrier. What are the core areas of payer uncertainty critical for regenerative medicine developers to address? Where have past technologies fallen short? What examples from the marketplace highlight successes and point to solutions to bridge the uncertainty chasm? How should approaches differ between rare versus common diseases? What solutions may work across highly variable global reimbursement systems? This panel will debate the implications of regenerative and potentially curative therapies and consider how regenerative medicine developers can overcome the uncertainty gaps associated with these technologies.

Chair:
Eric Faulkner, VP, Precision and Transformative Technology Solutions, Evidera

Speakers:
Dawn Driscoll, Principal, DCi Biotech
Donald Han, Payer Insights and Access Cluster Lead, Pfizer
Panos Kefalas, Ph.D., Head of Health Economics and Market Access, Cell and Gene Therapy Catapult

PROCESS IMPROVEMENT & ACCELERATED CMC DEVELOPMENT

7:15am – 8:45am | Learning Theater
Sponsored by PCT, a Caladrius Company

PROCESS IMPROVEMENT & ACCELERATED CMC DEVELOPMENT

The objective of this workshop is to discuss the needs of continuous process improvement for cell therapy manufacturing compared to major process changes for traditional drug/biologic manufacturing, the thinking or rethinking on comparability assessment and the need for potential regulatory mechanisms to help with accelerated CMC development to match accelerated clinical development under various existing regulatory pathways (conditional approval etc.).

7:15am – 7:25am | Introduction & Opening Remarks
Speaker:
Robert Preti, Ph.D., Chairman, Alliance for Regenerative Medicine; President, PCT, A Caladrius Company; SVP, Manufacturing and Technical Operations, Caladrius Biosciences

7:25am – 8:00am | Panel Discussion: Process Improvement & Comparability Assessment
Chair:
Robert Preti, Ph.D., Chairman, Alliance for Regenerative Medicine; President, PCT, A Caladrius Company; SVP, Manufacturing and Technical Operations, Caladrius Biosciences

8:00am – 8:35am | Panel Discussion: Accelerated CMC Development
Chair:
Jiwen Zhang, Ph.D., Senior Director, Regulatory Affairs, Cell Therapy and Regenerative Medicine, GE Healthcare

8:35am – 8:45am | Closing Remarks & Takeaways
Michael Mendicino, Ph.D., CEO, Hybrid Concepts International

Expert Panelists from Regulatory Agencies:
Nicolas Ferry, M.D., Ph.D., Former Member, Committee for Advanced Therapies (CAT), European Medicines Agency (EMA)
Steven S. Oh, Ph.D., Chief, Cell Therapies Branch, Office of Cellular, Tissue and Gene Therapies, Center for Biologics Evaluation and Research (CBER), U.S. Food and Drug Administration (FDA)
Yoshiaki Maruyama, Ph.D., Review Director, Office of Cellular and Tissue-based Products (OCTP), Pharmaceuticals and Medical Devices Agency (PMDA)

Expert Panelists from Industry:
Stewart Abbot, Ph.D., Chief Development Officer, Fate Therapeutics
Vijay Chiruvolu, Ph.D., Senior Director, Product Sciences, Kite Pharma
John Harrington, Ph.D., EVP and Chief Scientific Officer, Athersys
Francis Meacle, Ph.D., Director, Cell Therapy Manufacturing, Janssen R&D
Keith Wonnacoutt, Ph.D., Director, Regulatory Affairs, Novartis

 

9:00am | GENERAL SESSION & PARTNERING OPENS
9:00am – 9:15am WELCOME REMARKS
Speakers:
Morrie Ruffin, Managing Director, Alliance for Regenerative Medicine (ARM)
Michael Werner, Executive Director, Alliance for Regenerative Medicine (ARM)
9:15am – 9:45am FEATURED PRESENTATION {Ballroom 1}
Speaker:
Yuzo Toda, Director EVP, FUJIFILM Corporation; Chairman, Forum for Innovative Regenerative Medicine (FIRM)
Moderated Discussion By:
Robert Preti, Ph.D., Chairman, Alliance for Regenerative Medicine; President, PCT, A Caladrius Company; SVP, Manufacturing and Technical Operations, Caladrius Biosciences
COMPANY PRESENTATIONS {Ballroom 2}
9:15am – 9:30am Fibrocell
9:30am – 9:45am Cellular Dynamics International
9:45am – 10:00am TrakCel
10:00am – 10:15am Thermo Fisher Scientific
9:45am – 10:15am FEATURED PRESENTATION: AN UPDATE ON THE OFFICE OF CELLULAR, TISSUE AND GENE THERAPIES {Ballroom 1}
Speaker:
Celia Witten, M.D., Ph.D., Deputy Director, Center for Biologics Evaluation and Research (CBER), U.S. Food and Drug Administration (FDA)
10:15am – 10:45am | MORNING BREAK
10:45am – 11:45am | CONCURRENT TRACKS
PANEL: EVOLVING REGULATORY SCIENCE TO SUPPORT INNOVATION {Ballroom 1}
Improvement in Current Regulatory Processes for Cell and Gene Therapy Products
10:45am – 11:45am
This panel will focus on key lessons learned from recent initiatives that have been put in place by regulators in different regions to accelerate the development of medicinal products, including cell and gene therapy products, as well as how existing regulatory processes could be improved further to support innovation in this field. The session will also include how international synergies could further support these types of innovations.
Sponsored by Cell & Gene Therapy Catapult & Janssen R&D
Chair:
Anne-Virginie Eggimann, VP, Regulatory Science, bluebird bio
Speakers:
Nicolas Ferry, M.D., Ph.D., Former Member, Committee for Advanced Therapies (CAT), European Medicines Agency (EMA)
Soichiro Isobe, Ph.D., Director, Medical Device Evaluation Division, Pharmaceutical Safety and Environmental Health Bureau, Ministry of Health, Labor and Welfare, Government of Japan
Christiane Niederlaender, Ph.D., Senior Quality Assessor and Deputy Unit Manager, Biologicals Unit, Licensing Division, Medicines and Healthcare Products Regulatory Agency (MHRA)
Celia Witten, M.D., Ph.D., Deputy Director, Center for Biologics Evaluation and Research (CBER), U.S. Food and Drug Administration (FDA)
COMPANY PRESENTATIONS {Ballroom 2}
10:45am – 11:00am Cynata Therapeutics
11:00am – 11:15am TiGenix
11:15am – 11:30am Pluristem Therapeutics
11:30am – 11:45am Caladrius Biosciences
11:45am – 1:00pm | LUNCH
1:00pm – 2:45pm | CONCURRENT TRACKS
COMPANY PRESENTATIONS {Ballroom 1}
1:00pm – 1:15pm Sangamo BioSciences
1:15pm – 1:30pm bluebird bio
1:30pm – 1:45pm Editas Medicine
1:45pm – 2:00pm Intellia Therapeutics
2:00pm – 2:15pm Dimension Therapeutics
2:15pm – 2:30pm Voyager Therapeutics
2:30pm – 2:45pm Adverum Biotechnologies
COMPANY PRESENTATIONS {Ballroom 2}
1:00pm – 1:15pm Orchard Therapeutics
1:15pm – 1:30pm ViaCyte
1:30pm – 1:45pm Regeneus
1:45pm – 2:00pm MEDIPOST America
2:00pm – 2:15pm ORIG3N
2:15pm – 2:30pm Calimmune
2:30pm – 2:45pm Immusoft
2:45pm – 3:15pm | AFTERNOON BREAK
3:15pm – 5:00pm | CONCURRENT TRACKS
PANEL: GENE THERAPY: COMMERCIALIZING A THERAPEUTIC PRODUCT {Ballroom 1}
3:15pm – 4:15pm
This panel will discuss aspects of the commercialization process that are unique to the gene therapy sector. Discussion will include market access issues, how companies are assembling commercially and what types of partnerships are available for these organizations.
Speakers:
Andrea Hunt, VP New Product Therapeutic Area Lead Gene Therapy, Neuroscience and Ophthalmology, Shire
Sven Kili, M.D., VP & Head of Gene Therapy Development, GlaxoSmithKline
Matt Patterson, President and CEO, Audentes Therapeutics
Sue Washer, President and CEO, AGTC
Elizabeth White, Ph.D., Assistant VP, Early Commercial Planning, Rare Disease and Gene Therapy, Pfizer Innovative Health
COMPANY PRESENTATIONS {Ballroom 2}
3:15pm – 3:30pm Biostage
3:30pm – 3:45pm Avita Medical
3:45pm – 4:00pm DiscGenics
4:00pm – 4:15pm InVivo Therapeutics
COMPANY PRESENTATIONS {Ballroom 1}
4:15pm – 4:30pm MolMed
4:30pm – 4:45pm Argos Therapeutics
4:45pm – 5:00pm Kiadis Pharma
COMPANY PRESENTATIONS {Ballroom 2}
4:15pm – 4:30pm Scottish National Blood Transfusion Service
4:30pm – 4:45pm Promethera Biosciences
4:45pm – 5:00pm SCM Lifescience
5:00pm | PARTNERING CLOSES

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5:30pm | PUBLIC FORUM OPENS
5:45pm – 6:45pm FEATURED PRESENTATION
Introduction By:
Donna Skerrett, M.D., Chief Medical Officer, Mesoblast
Featured Speaker:
Elena Cattaneo, Ph.D., Full Professor, Laboratory of Stem Cell Biology and Pharmacology of Neurodegenerative Diseases, Department of Biosciences, University of Milan
6:45pm – 7:30pm | RECEPTION & NETWORKING
Sponsored by Mesoblast

7:30pm | PUBLIC FORUM CLOSES

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7:00am – 8:00am | REGISTRATION & BREAKFAST
8:00am | GENERAL SESSION
8:00am – 8:15am WELCOME REMARKS
Introduction:
Alysson Muotri, Ph.D., Chair, Scientific Symposium Steering Committee; Associate Director, Stem Cell Program; Associate Professor, Department of Pediatrics and Cellular Molecular Medicine, UC San Diego
8:15am – 8:55am MORNING KEYNOTE SPEAKER
Keynote Speaker:
Jun Takahashi, M.D., Ph.D., Professor, Center for iPS Cell Research & Application (CiRA), Kyoto University
8:55am – 10:15am PANEL I: ORGANOIDS IN A DISH
Human pluripotent stem cells can be used to generate different types of cells in a dish. Recently, this technology has evolved to produce organoids, tridimensional self-assemble structures that possess certain features of human tissues, including the spatial organization of multi-cellular systems. Organoids could serve as unprecedentedly accurate models of study for a wide range of human diseases. This session will highlight recent advances on the use of this technology to gain novel insights into human development.
Chair / Introduction By:
Alysson Muotri, Ph.D., Associate Director, Stem Cell Program; Associate Professor, Department of Pediatrics and Cellular Molecular Medicine, UC San Diego
Speakers:
Alysson Muotri, Ph.D., Associate Director, Stem Cell Program; Associate Professor, Department of Pediatrics and Cellular Molecular Medicine, UC San Diego
Trinh (Tracy) Tran, Ph.D. Student, The McMahon Laboratory, Keck School of Medicine, University of Southern California (USC)
Juan Carlos Izpisua Belmonte, Ph.D., Professor, Gene Expression Laboratory; Roger Guillemin Chair, Salk Institute for Biological Studies
10:15am – 10:40am | MORNING BREAK / POSTER VIEWING – EVEN NUMBER ABSTRACTS
10:40am – 12:00pm PANEL II: CELL THERAPY FOR HEMATOPOIETIC DISORDERS AND BEYOND
Chair / Introduction By:
Dan Kaufman, M.D., Ph.D., Director, Cell Therapy Program; Professor, Department of Medicine, UC San Diego
Speakers:
Claudio Brunstein, M.D., Ph.D., Associate Professor of Medicine, Division of Hematology, Oncology and Transplantation, University of Minnesota
Dan Kaufman, M.D., Ph.D., Director, Cell Therapy Program; Professor, Department of Medicine, UC San Diego
Sandip Patel, M.D., Assistant Professor, Medical Oncology/Hematology, UC San Diego Moores Cancer Center
12:00pm – 1:15pm | LUNCH / POSTER VIEWING – ODD NUMBER ABSTRACTS
1:15pm – 2:35pm PANEL III: GENOMICS & GENETICS
Chair / Introduction By:
Stefan Aigner, Ph.D., Project Scientist, Department of Cellular & Molecular Medicine, UC San Diego
Speakers:
Clodagh O’Shea, Ph.D., Associate Professor, Molecular and Cell Biology Laboratory; William Scandling Developmental Chair, Salk Institute for Biological Studies
Anjana Rao, Ph.D., Professor, Division of Signaling and Gene Expression, La Jolla Institute for Allergy and Immunology
2:35pm – 3:00pm | AFTERNOON BREAK / POSTER VIEWING – EVEN NUMBER ABSTRACTS
3:00pm – 4:20pm PANEL IV: UNBIASED DISCOVERY METHODS IN THE DEVELOPMENT OF CELL-BASED THERAPIES
Co-Chairs / Introduction By:
Anne Bang, Ph.D., Director, Cell Biology, Sanford Burnham Prebys Medical Discovery Institute
Luke Lairson, Ph.D., Assistant Professor, Department of Chemistry, The Scripps Research Institute; Principal Investigator, California Institute for Biomedical Research
Speakers:
Xiang-Dong Fu, Ph.D., Professor, Cellular and Molecular Medicine, UC San Diego
Prashant Mali, Ph.D., Assistant Professor of Bioengineering, UC San Diego
Lee Rubin, Ph.D., Professor, Department of Stem Cell and Regenerative Biology, Harvard Stem Cell Institute
4:20pm – 5:00pm AFTERNOON KEYNOTE SPEAKER
Keynote Speaker:
Lorenz Studer, M.D., Director, Center for Stem Cell Biology, Memorial Sloan Kettering Cancer Center
5:00pm – 6:30pm | NETWORKING RECEPTION / POSTER VIEWING
6:30pm | SCIENTIFIC SYMPOSIUM CLOSES