Phacilitate Cell & Gene Therapy Forum 2005
Presentations
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Pre-conference workshop | Monday, January 24th 2005
Overcoming major challenges in the development and regulation of cell and gene-based therapies
Morning Plenary
Clarifying and comparing the regulatory environment for cell & gene-based therapies in North America, Europe and Asia
Chair’s introduction
Dr Lincoln Tsang
What work is in progress at CBER/FDA to help the industry reach and progress through clinical development faster?
Dr Cynthia A. Rask
Panel discussion
How do national and local review bodies and ethics committees impact the regulatory process?
Dr Kathryn Harris
Dr Monika Preuss
Professor Walter Günzberg
Doug Jolly, PhD
NO SLIDES PROVIDED
Clinical trials exemption – what degree of protection from IP constraints does it now provide for preclinical and clinical research?
Rochelle K. Seide, PhD
Workshop 1
Cell therapy and tissue engineering regulation in North America and Europe
Moderator’s introduction
Dr Chris Holloway
NO SLIDES PROVIDED
Historical overview of cell therapy regulation in the European Union
Dr Chris Holloway
Latest developments in cell therapy regulation in the European Union
Shayesteh Fuerst-Ladani
Cell therapy regulation in the US
John W. Cross
Case study
Applying EMEA regulatory requirements to an encapsulated cell therapy orphan medicinal product for pancreatic cancer
Brian Salmons, PhD
Case study
Dr Chris Holloway
Workshop 2
Product development and regulation of gene therapies and vectors
Chair’s opening address
Planning ahead for the impact of stacked royalties and building in mechanisms to control their impact on gene therapy product development
Doug Jolly, PhD
Case study
Implementing an incremental GMP strategy – what is required at each stage of development?
Dr Samuel C. Wadsworth, PhD
Case study
Addressing the challenges and complexities of interacting with regulatory authorities in 16 countries to support Phase II/III studies in viral gene therapy
Francisco J. Castillo
Gene delivery vectors for vaccine development
Dr John Shiver
Case study
What are the practical considerations when developing lentiviral vectors for human clinical trials?
Dr Xiaobin Lu
Case study
Plasmid-based gene therapies: Reviewing performance, safety and efficacy of current technologies and undertaking a gap analysis
Alain Rolland, Pharm D, PhD
Panel discussion
What are the planning lessons in terms of budgeting and scheduling for all elements of gene therapy product development?
Panellist:
Rochelle K. Seide, PhD
NO SLIDES PROVIDED
Afternoon plenary session
Panel discussion
Sourcing and managing materials for cell and gene therapy
Moderator:
Dr Shelly Heimfeld
Panellists:
Dr Glyn Stacey
Dr Samuel C. Wadsworth
NO SLIDES PROVIDED
Main Conference Day 1 | Tuesday, January 25th 2005
Morning Plenary
Which disease areas are cell and gene therapies being most successfully applied to, and how are they being evaluated by big pharma and big biotech?
Chair’s introduction
How and when will cell and gene therapies reach the market and what are the major challenges still to be overcome?
Dr Scott R. Burger
KEYNOTE ADDRESS
Big pharma perspective on cell and gene therapy: Impacting human health
Patrice M. Milos, PhD
Focusing on R&D activity in major therapeutic areas – what role might cell and gene therapy play and what are the successes so far?
ONCOLOGY
KEYNOTE ADDRESS
Building the Novartis Oncology pipeline via business development activities: Is there a place for cell & gene therapies?
George Golumbeski, PhD
Questions & discussion
Panellists:
M. Laurie Phillips, PhD
David L. Urdal, PhD
CARDIOVASCULAR
Cardiovascular applications of cell and gene therapy at Eli Lilly: Development of arteriogenic agents for the treatment of coronary and peripheral ischemic vascular disease
Dr Jaipal Singh
Key considerations for device-based cardiovascular gene and cell therapy
Jim Barry, PhD
Panel discussion
Capitalizing on the strengths of cell and gene therapy in terms of their application to specific disease areas
Moderator:
Dr Alan Boyd
Panellists:
Dr Katherine High
George Golumbeski, PhD
NO SLIDES PROVIDED
Dr John Shiver
NO SLIDES PROVIDED
Dr Lothar Krinke
NO SLIDES PROVIDED
Dr Reiner Spaethe
NO SLIDES PROVIDED
David L. Urdal PhD
NO SLIDES PROVIDED
Main afternoon session
Driving gene therapies through preclinical and phase I clinical development
Chair’s introduction
Dr John Shiver
NO SLIDES PROVIDED
Knowing when your product and your business plans are robust enough to take the
risk of progressing into the clinic
Dr Jeffrey M. Ostrove
Proving preclinical safety including near-final vector processing
Dr Kenneth Cornetta,
Cell and gene therapy product development using adoptive transfer of autologous
human T cells
Dale Ando, MD
Case study
Optimizing clinical trial design: Contrasting risks and benefits in CNS and hematology clinical targets
Dr Glenn Pierce
Case study
Optimizing clinical trial design: Oncology
Dr Robert E. Sobol
Panel discussion
What are the key elements of a rigorous approach to early-stage clinical development?
Panellists:
Dr Stephen Grant
Allan Shipp
Afternoon Breakout
Cell and tissue therapies product development and manufacture
Moderator’s introduction
What commercialization models are emerging to cope with the highly complex
manufacture required by cell therapies?
Dr Chris Holloway
Case study
Autologous and allogeneic cancer vaccine product development
Flavia Borellini, PhD
Case study
Cell-based tissue engineering product using fibrin technology
Dr Reiner Spaethe
Processing autologous cell products – discussing the regulatory and commercial
impact of patient-specific therapies
Dr Andra Miller
Panel discussion
Optimizing product development, manufacture and commercialization of autologous
and allogeneic cell therapies
Afternoon plenary session
Panel discussion
Overcoming the issues involved in using iterative development models
Moderator:
Professor Adrian P. Gee
Panellists:
Karen Kozarsky, PhD
NO SLIDES PROVIDED
M. Laurie Phillips, PhD
NO SLIDES PROVIDED
Main Conference Day 2 | Thursday, November 4, 2004
Morning Plenary
How can cell and gene therapy companies add value and leverage their unique selling points with the investor community and potential licensing partners?
Chair’s introduction
Big pharma perspective - what will it take to secure their investment?
Robert Ward
KEYNOTE ADDRESS
Learning from the successes and failures in the field: What business models are the
financial community looking for now?
G. Steven Burrill
Cell therapy product development case study
Autologous chondrocyte transplantation: lessons learned and future challenges for cell therapy product development
John M. McPherson, PhD
RNAi case study
Attracting funding and partnerships for discovery research using siRNA in animal disease models and for siRNA therapeutics
Dr Martin Woodle – NOT AVAILABLE
Panel debate between bull and bear investors in cell and gene therapy
Moderator:
Dr Jeffrey M. Ostrove
NO SLIDES PROVIDED
Panellists:
G. Steven Burrill
NO SLIDES PROVIDED
David H. Mack, PhD
NO SLIDES PROVIDED
Matt Kaplan
R. Nelson Campbell
NO SLIDES PROVIDED
Dr Chris Kroeger
NO SLIDES PROVIDED
Dr Krisztina M. Zsebo
NO SLIDES PROVIDED
Main afternoon session
Late phase clinical development of cell and gene therapies
Chair’s introduction
Karen Kozarsky, PhD
CELL THERAPY CASE STUDIES
The MAGIC trial: Pioneering cardiac cell therapy
Bruce M. Wentworth, PhD
Clinical development of the allogeneic GVAX® prostate cancer vaccine
Dr Natalie Sacks
NO SLIDES PROVIDED
GENE THERAPY CASE STUDIES
Developing AAV vector gene therapy for Cystic Fibrosis lung disease
Dr Barrie J. Carter
Phase II clinical development of Trovax®, a gene-based immunotherapy for cancer
Professor Alan Kingsman
The GENASIS trial: clinical development of Vascular Endothelial Growth Factor-2 (VEGF-2) for the treatment of refractory myocardial ischemia
Richard E. Otto
Afternoon breakout
Business models, partnering and licensing strategies for cell and gene therapies
Moderator’s introduction
Comparing and contrasting the business models that are appropriate for cell therapy and for gene therapy
R. Nelson Campbell
NO SLIDES PROVIDED
GENE THERAPY BUSINESS MODELS IN ACTION
What have been the differences in approach to partnering and licensing strategies for
cell and gene therapy at GenVec?
E. Michael Egan
Case study
Cell and gene therapy business models for a small molecule world
Eric Rhodes
CELL THERAPY BUSINESS MODELS IN ACTION
Business models for partnering at the preclinical and clinical stage: Stem cell products case study
Ehud Marom – NOT AVAILABLE
Practical considerations for commercialization
James Cour
Panel discussion
How can the dislocation between cell and gene therapy business models and those favoured by big pharma and big biotech be reduced?
Robert Ward
NO SLIDES PROVIDED
Afternoon Plenary Session
Business models, partnering and licensing strategies for cell and gene therapies
Panel discussion
Commercializing cell and gene therapies – bringing profitable cell and gene therapies to the market faster
Panellists:
M. Laurie Phillips, PhD, Dr Barrie J. Carter, Dr Glyn Stacey
NO SLIDES PROVIDED
