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.

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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

GENE THERAPY MANUFACTURING WORKSHOP

7:15am – 8:45am | Learning Theater

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?

Speakers:
James Brown, Ph.D., VP, Corporate Development, Aldevron
Michael Roberts, Ph.D., CSO, Synpromics

9:00am | GENERAL SESSION
9:00am – 9:15am WELCOME REMARKS
Speaker:
Robert Preti, Ph.D., Chairman, Alliance for Regenerative Medicine; President, PCT, A Caladrius Company; SVP, Manufacturing & Technical Operations, Caladrius Biosciences
9:15am – 10:15am PLENARY SESSION: CLINICAL PROGRESS IN IMMUNO-ONCOLOGY
Chair:
Amy Butler, Ph.D., VP & General Manager, Cell Biology, Thermo Fisher Scientific
Speaker:
Thomas Farrell., President & CEO, Bellicum Pharmaceuticals
10:15am | PARTNERING OPENS
10:15am – 10:45am | MORNING BREAK
10:45am – 12:00pm | CONCURRENT TRACKS
COMPANY PRESENTATIONS {Ballroom 1}
10:45am – 11:00am Pluristem Therapeutics
11:00am – 11:15am Caladrius Biosciences
11:15am – 11:30am Celyad
11:30am – 11:45am bluebird bio
11:45am – 12:00pm Capricor Therapeutics
COMPANY PRESENTATIONS {Ballroom 2}
10:45am – 11:00am Editas Medicine
11:00am – 11:15am InVivo Therapeutics
11:15am – 11:30am Asterias Biotherapeutics
11:30am – 11:45am StemCells, Inc.
11:45am – 12:00pm ViaCyte
12:00pm – 1:15pm | LUNCH
1:15pm – 3:30pm | CONCURRENT TRACKS
PANEL: PAYER PERSPECTIVES {Ballroom 1}
Sponsored by Quintiles
1:15pm – 2:15pm
Chair:
John Doyle, Dr.P.H., SVP, Advisory Services, Quintiles
Speaker:
Nick Crabb, Ph.D., Programme Director – Scientific Affairs, Centre for Health Technology Evaluation, National Institute for Health and Care Excellence (NICE)
COMPANY PRESENTATIONS {Ballroom 2}
1:15pm – 1:30pm Accurexa
1:30pm – 1:45pm BioCardia
1:45pm – 2:00pm StemBioSys
2:00pm – 2:15pm Break – No Presentation
COMPANY PRESENTATIONS {Ballroom 1}
2:15pm – 2:30pm MiMedx
2:30pm – 2:45pm Sangamo BioSciences
2:45pm – 3:00pm AGTC
3:00pm – 3:15pm Oxford BioMedica
3:15pm – 3:30pm ReNeuron
COMPANY PRESENTATIONS {Ballroom 2}
2:15pm – 2:30pm Juventas Therapeutics
2:30pm – 2:45pm Hemostemix
2:45pm – 3:00pm Cell Therapy Ltd.
3:00pm – 3:15pm Commence Bio
3:15pm – 3:30pm Kiadis Pharma
3:30pm – 4:00pm | AFTERNOON BREAK
4:00pm – 6:00pm | CONCURRENT TRACKS
PANEL: FINANCING CELL & GENE THERAPIES {Ballroom 1}
COMPANY PRESENTATIONS {Ballroom 2}
4:00pm – 4:15pm BioLife Solutions
4:15pm – 4:30pm Terumo BCT
4:30pm – 4:45pm MaxCyte
4:45pm – 5:00pm TaiwanBio
COMPANY PRESENTATIONS {Ballroom 1}
5:00pm – 5:15pm End of Payer Panel (see above)
5:15pm – 5:30pm Athersys
5:30pm – 5:45pm Healios
5:45pm – 6:00pm Vericel
COMPANY PRESENTATIONS {Ballroom 2}
5:00pm – 5:15pm BrainStorm Cell Therapeutics
5:15pm – 5:30pm GenVec
5:30pm – 5:45pm REGENXBIO
5:45pm – 6:00pm Avita Medical
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
7:30am – 9:00am | CONCURRENT WORKSHOPS

REIMBURSEMENT WORKSHOP

7:15am – 8:45am | Magnolia Room

REIMBURSEMENT WORKSHOP

Speaker:
Erik Faulkner, VP, Precision and Transformative Technology Solutions, Evidera

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.).

Workshop Chair:
Robert Preti, Ph.D., Chairman, Alliance for Regenerative Medicine; President, PCT, A Caladrius Company; SVP, Manufacturing and Technical Operations, Caladrius Biosciences

Session Organizers:
Jiwen Zhang, Ph.D., Senior Director, Regulatory Affairs, Cell Therapy and Regenerative Medicine, GE Healthcare
Michael Mendicino, Ph.D., Director of Regulatory Affairs, Mesoblast

Expert Panelists:
Nicolas Ferry, M.D., Ph.D., Member, Committee for Advanced Therapies (CAT), European Medicines Agency (EMA)
Steven 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)

Industry Speakers:
Stewart Abbot, Ph.D., Chief Development Officer, Fate Therapeutics
Vijay Chiruvolu, Ph.D., Senior Director, Product Sciences, Kite Pharma
John Harrington, Ph.D., EVP & 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 – 9:10am WELCOME REMARKS
Speakers:
Morrie Ruffin, Managing Director, Alliance for Regenerative Medicine (ARM)
Michael Werner, Executive Director, Alliance for Regenerative Medicine (ARM)
9:10am – 9:40am FEATURED PRESENTATION
Featured 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
9:40am – 10:15am FEATURED PRESENTATION
Speaker: TBD
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
The objective of this international regulatory session is to explore opportunities in various regions to accelerate development and approval for regenerative medicines. The panel will focus on challenges to developers wishing to license their product using accelerated pathways and will focus specifically on the similarities and differences of the schemes in various regions.
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)
Yoshiaki Maruyama, Ph.D., Review Director, Office of Cellular and Tissue-based Products (OCTP), Pharmaceuticals and Medical Devices Agency (PMDA)
COMPANY PRESENTATIONS {Ballroom 2}
10:45am – 11:00am Videregen
11:00am – 11:15am SCM Lifescience
11:15am – 11:30am jCyte
11:30am – 11:45am Cellular Dynamics International
11:45am – 1:00pm | LUNCH
1:00pm – 2:45pm | CONCURRENT TRACKS
COMPANY PRESENTATIONS {Ballroom 1}
1:00pm – 1:15pm Organovo
1:15pm – 1:30pm Fate Therapeutics
1:30pm – 1:45pm Intellia Therapeutics
1:45pm – 2:00pm Cellectis
2:00pm – 2:15pm uniQure
2:15pm – 2:30pm Inovio Pharmaceuticals
2:30pm – 2:45pm Osiris Therapeutics
COMPANY PRESENTATIONS {Ballroom 2}
1:00pm – 1:15pm MEDIPOST America
1:15pm – 1:30pm TiGenix
1:30pm – 1:45pm Histogenics
1:45pm – 2:00pm TissueGene
2:00pm – 2:15pm OrthoCell
2:15pm – 2:30pm Cesca Therapeutics
2:30pm – 2:45pm RepliCel
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
Twenty years ago it was impossible to get any investor, venture or otherwise, to take a serious look at a gene therapy company. What is the difference between today’s environment and where we were in the mid-1990s? What are the key technology breakthroughs and clinical insights that have led to this resurgence in interest? No doubt there are challenges to successful commercialization, but why do investors believe these hurdles can be overcome and that valuations will remain strong? This panel of venture and institutional investors will discuss the current financing environment for gene therapy companies and how access to capital can be maintained over the long term.
Chair:
Joseph Pantginis, Ph.D., Head of Biotechnology Research & Senior Research Analyst, ROTH Capital Partners
Speakers:
Adam Bristol, Ph.D., Analyst & Director of Research, Aquilo Capital Management
Fariba Ghodsian, Ph.D., Chief Investment Officer, DAFNA Capital Management
Daniel Krizek, Director, Brookside Capital
Kush Parmar, M.D., Ph.D., Partner, 5AM Ventures
COMPANY PRESENTATIONS {Ballroom 2}
3:15pm – 3:30pm Daedalus Bio
3:30pm – 3:45pm Stanford University
3:45pm – 4:00pm TC BioPharm
4:00pm – 4:15pm Regenerative Patch
COMPANY PRESENTATIONS {Ballroom 1}
4:15pm – 4:30pm MolMed
4:30pm – 4:45pm Argos Therapeutics
4:45pm – 5:00pm Fibrocell Science
COMPANY PRESENTATIONS {Ballroom 2}
4:15pm – 4:30pm Abeona Therapeutics
4:30pm – 4:45pm Cyfuse Biomedical
4:45pm – 5:00pm Cynata Therapeutics
5:00pm | PARTNERING CLOSES

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5:30pm | PUBLIC FORUM OPENS
5:45pm – 6:45pm FEATURED PRESENTATION: THE PROMISE OF REGENERATIVE MEDICINE: ARE WE THERE YET?
Introduction By:
Donna Skerrett, M.D., Chief Medical Officer, Mesoblast
Featured Speaker:
Jennifer Elisseeff, Ph.D., Professor, Jules Stein Chair in Ophthalmology, Wilmer Eye Institute; Director, Translational Tissue Engineering Center, Johns Hopkins University
6:45pm – 7:30pm | RECEPTION & NETWORKING
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:
Roger Bingham, Co-Founder & Director, The Science Network; Member, Computational Neurobiology Laboratory, Salk Institute for Biological Studies
Speaker:
C. Randal Mills, Ph.D., President & CEO, California Institute for Regenerative Medicine (CIRM)
8:15am – 8:55am STEM CELLS IN SILENCE, ACTION & CANCER
Keynote Speaker:
Elaine Fuchs, Ph.D., Investigator, Howard Hughes Medical Institute; Rebecca C. Lancefield Professor, Robin Chemers Neustein Laboratory of Mammalian Cell Biology & Development, Rockefeller University
8:55am – 10:15am PANEL I: TRIALS AT THE SANFORD STEM CELL CLINICAL CENTER
This panel will describe new data and design for upcoming and ongoing cell-therapy clinical trials of the Sanford Stem Cell Clinical Center. The Sanford Stem Cell Clinical Center supports ethically conducted stem cell-based and stem-cell targeted clinical trials that are strongly based on evidence and have rigorous trial design. These two elements are essential for the conduct of Phase I stem cell trials. The third essential element is strong collaboration among many scientific and medical partners. The CIRM Alpha Stem Cell Clinic at UC San Diego provides infrastructural strength to enable the complex interaction required for success.
Chair / Introduction By:
Lawrence Goldstein, Ph.D., Distinguished Professor, Departments of Cellular and Molecular Medicine and Neurosciences; Director, Sanford Stem Cell Clinical Center; Director, UC San Diego Stem Cell Program; Scientific Director, Sanford Consortium for Regenerative Medicine
First-in-Human Phase I Single Site Safety Study of Human Spinal Cord-derived Neural Stem Cell Transplantation for the Treatment of Chronic SCI
Joseph Ciacci, M.D., Clinical Professor of Surgery, UC San Diego; Chief of Neurosurgery, VA San Diego Healthcare System
Visualizing the Behavior of Cytotherapy in Clinical Trials Using MRI
Eric Ahrens, Ph.D., Professor of Radiology & Director, Stem Cell Molecular Imaging, UC San Diego
Towards the Clinical Application of Lentiviral Hematopoietic Stem Cell Gene Therapy for Cystinosis
Stephanie Cherqui, Ph.D., Assistant Professor in Residence, Department of Pediatrics, UC San Diego
10:15am – 10:40am | MORNING BREAK / POSTER VIEWING – EVEN NUMBER ABSTRACTS
10:40am – 12:00pm PANEL II: NEURONAL DIVERSIFICATION USING SPINAL NEURONS AS A MODEL
There is enormous cellular diversity in the central nervous system which underlies the specific deficits linked to different neurological diseases. In this session the speakers will use the spinal cord as a model system to highlight discoveries that have enlightened our understanding of neuronal diversification using stem cells. The talks will cover cell signaling, transcriptional mechanisms, chromatin mechanisms and microRNA functions. These findings are leading to a better understanding of the molecular pathways and targets that are relevant to neurodevelopment and birth defects, and helping to provide a foundation for regenerative medicine involving the replacement of specific cell types.
Chair / Introduction By:
Samuel Pfaff, Ph.D., Benjamin H. Lewis Chair, Professor, Gene Expression Laboratory, Salk Institute for Biological Studies
Editing Hedgehog Response Genes in Embryonic Stem Cells Reveals New Mechanisms of Smoothened Regulation by Ptch1/2
Henk Roelink, Ph.D., Associate Professor of Genetics, Genomics & Development, UC Berkeley
Unraveling Transcriptional Circuits in the Developing Spinal Cord
Todd Macfarlan, Ph.D., Earl Stadtman Investigator, The Eunice Kennedy Shriver National Institute of Child Health & Human Development, National Institutes of Health
Programming the Functional Diversity of Stem Cell-derived Motor Neurons
Bennett Novitch, Ph.D., Associate Professor, Department of Neurobiology, Eli & Edythe Broad Center for Regenerative Medicine & Stem Cell Research, UCLA / David Geffen School of Medicine
Post Transcriptional Mechanisms Regulating Neural Development & Survival
Samuel Pfaff, Ph.D., Benjamin H. Lewis Chair, Professor, Gene Expression Laboratory, Salk Institute for Biological Studies
12:00pm – 12:10pm HERTZBERG-SCHECHTER PRIZE FOR STEM CELL RESEARCH
Presented By:
Alysson Muotri, Ph.D., Associate Director, Stem Cell Program; Associate Professor, Department of Pediatrics & Cellular Molecular Medicine, UC San Diego
12:10pm – 1:15pm | LUNCH / POSTER VIEWING – ODD NUMBER ABSTRACTS
1:15pm – 2:35pm PANEL III: ENGINEERING BLOOD CELLS IN THE FIGHT AGAINST CANCER & OTHER DISEASES
The talks in this session will describe research on improved methods for using hematopoietic stem cells for gene therapy for immune deficiencies and for generating effector cells to fight cancer and leukemia. It will also describe new methods for generating immune T lymphocytes that are more effective at targeting cancer cells.
Chair / Introduction By:
Mitchell Kronenberg, Ph.D., President & Chief Scientific Officer, La Jolla Institute for Allergy & Immunology
Expanding Antigen Specific Adoptive T Cell Immunotherapy for Treatment of Cancer
Stephen Forman, M.D., Francis & Kathleen McNamara Distinguished Chair in Hematology & Hematopoietic Cell Transplantation, City of Hope
Natural Killer T Cells: A Versatile Platform for Cell Mediated Immune Therapies
Mitchell Kronenberg, Ph.D., President & Chief Scientific Officer, La Jolla Institute for Allergy & Immunology
Development of a Preclinical Patient-derived Xenograft Model of Personalized Cancer Immunotherapy
Stephen Schoenberger, Ph.D., Professor, Laboratory of Cellular Immunology, La Jolla Institute for Allergy & Immunology
2:35pm – 3:00pm | AFTERNOON BREAK / POSTER VIEWING – EVEN NUMBER ABSTRACTS
3:00pm – 4:20pm PANEL IV: TISSUE ENGINEERING
This panel will present cutting-edge research in advanced biomaterials and tissue regeneration. Specific topics will include an epicardial biomimetic that regenerates the adult mammalian heart, activating host cells using engineered extracellular matrices for tissue repair, and using a synthetic molecular band-aid to treat Duchenne muscular dystrophy.
Co-Chairs / Introduction By:
Shaochen Chen, Ph.D., Founding Director, Biomaterials & Tissue Engineering Center, UC San Diego
Mark Mercola, Ph.D., Professor, Department of Bioengineering, Jacobs School of Engineering, UC San Diego; Professor & Director, Muscle Development & Regeneration Program, Sanford Burnham Prebys Medical Discovery Institute
Activating Host Cells Using Engineered Extracellular Matrices for Tissue Repair
Shyni Varghese, Ph.D., Director, Bio-Inspired Materials & Stem Cell Engineering Laboratory, UC San Diego
Engineered Epicardium & Cardiac Regeneration
Pilar Ruiz-Lozano, Ph.D., Associate Professor, Pediatrics – Cardiology, Stanford University
Synthetic Molecular Band-aid for Duchenne Muscular Dystrophy
Joseph Metzger, Ph.D., Professor & Chair, Department of Integrative Biology & Physiology, University of Minnesota
4:20pm – 5:00pm HEMATOPOIETIC STEM CELL GENE THERAPY
Keynote Speaker:
Donald Kohn, M.D., Professor, Department of Microbiology, Immunology & Molecular Genetics (MIMG) & Department of Pediatrics, UCLA
5:00pm – 6:30pm | NETWORKING RECEPTION / POSTER VIEWING
6:30pm | SCIENTIFIC SYMPOSIUM CLOSES