Phacilitate Cell & Gene Therapy Forum 2007
Presentations
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Monday, January 22nd, 2007
Day 1
Morning plenary session
Preparing cell and gene therapy products for the marketplace
• Informing your product development strategy - how will the first licensed products be commercialized?
Chair’s introduction
Where are we currently in terms of the development of cell and gene therapies at large?
Dr Scott R. Burger, Advanced Cell & Gene Therapy
Keynote US healthcare provider perspective
How will cell and gene therapy products be evaluated for reimbursement?
Dr Naomi Aronson, BlueCross & BlueShield Association
Keynote Address
How should cell and gene therapy products be marketed once licensure is achieved?
Michael S. Fox, Johnson & Johnson Pharmaceutical Services, LLC
Cell therapy case study
Heading to market: from product development to preparing for commercialization of potentially the first active cellular immunotherapy
Dr Mitchell H. Gold, Dendreon Corporation
Gene therapy case study
Preparing for launch - is it any different for gene therapy?
Paul Higham, Ark Therapeutics Group plc
Breakout session 1
Business strategy session: Transitioning cell and gene therapy business models to secure the long-term future of your organization
Moderator’s introduction
Financial analyst’s perspective
What are the keys to successfully diversifying a cell or gene therapy business model?
Stephen G. Brozak, WBB Securities, LLC
Leveraging vectors for applications in biotechnology and medicine to support internal R&D
programs
Greg Feulner, PhD, Lentigen Corporation
Capitalizing on the stem cell supply opportunity to support in-house cell therapy development
Dr Jane S. Lebkowski, Geron Corporation – NOT AVAILABLE
Panel discussion
Debating the business and research landscape of stem cell therapies
Panellist:
Dr Andrew Balber, Aldagen – NO SLIDES PRESENTED
Stephen G. Brozak, WBB Securities, LLC
Breakout session 2
Regulatory workshop: How will you need to prepare to take the final steps towards licensure?
• Successfully filing a BLA and navigating pre-approval inspection requirements
Moderator’s introduction
Dr Robert E. Sobol, Introgen Therapeutics Incorporated
US FDA perspective
Clarifying the BLA filling process
Daniel Takefman, PhD, US Food & Drug Administration
Cell therapy product candidate
Late stage development requirements and strategies for GVAX immunotherapy for prostate cancer
Shirley Clift, Cell Genesys
Cell therapy product candidate
Navigating a critical path for product registration: US and EU frameworks for sponsor-agency cooperation
Yaron Daniely, PhD, MBA, Gamida Cell Ltd
Gene therapy product candidate
Critical Path and oncology biomarker qualification initiatives for the late-phase development of Advexin p53 tumor suppressor therapy
Dr Robert E. Sobol, Introgen Therapeutics Incorporated - NOT AVAILABLE
A Canadian perspective on pre-approval inspections
Dr Anthony Ridgway, Health Canada
Panel discussion
Addressing the challenges in taking the final steps toward licensure
NO SLIDES PRESENTED
Breakout session 3
Gene therapy clinical development update: In which indications is the greatest progress being made?
Moderator’s introduction
Global overview of gene therapies in the clinic – what are the therapeutic and technology areas of greatest promise currently?
Dr Karen Kozarsky, GlaxoSmithKline
Cardiovascular
Therapeutic arteriogenesis with a constitutively active HIF-1alpha transgene: Scientific rationale and human experience
Dr Ralph A. Kelly, Genzyme Corporation
CNS
CERE-120 (AAV-neurturin) for Parkinson’s disease: From concept to clinical testing and beyond
Raymond T. Bartus, PhD, Ceregene, Inc
HIV
Anti-HIV gene therapy: A step closer to market
Dr Laurent Humeau, VIRxSYS Corporation – NOT AVAILABLE
Ophthalmology
Gene therapy for congenital blindness: From concept to clinical testing
Dr Jean Bennett | Part 1 | Part 2 | Part 3 |, University of Pennsylvania
Afternoon plenary session
Exploring the commercial potential of cell and gene therapies in combination with medical devices
Chair’s introduction
What are the major strategic considerations in undertaking the co-development of a cell or gene therapy product with a device?
Dr Maria Palasis, Boston Scientific Corporation
Panel discussion
Panellists:
Dr Robert F. Willenbucher, Johnson & Johnson Development Corporation – NO SLIDES PRESENTED
Beverly H. Lorell, MD, FACC, King & Spalding, LLP
Dr Joyce Frey-Vasconcells, PharmaNet Inc
Tuesday, January 23rd, 2007
Day 2
Morning plenary session
Delivering a strategy for success in meeting the demands of the global regulatory environment
Chair’s introduction
Dr Ralph A. Kelly, Genzyme Corporation – NO SLIDES PRESENTED
US FDA Keynote address
Key areas of focus for the US FDA - combination products and product potency
Stephanie L. Simek, PhD, US Food & Drug Administration
Opportunities within the new European regulatory framework for Advanced Therapy Products
Dr Chris Holloway, ERA Consulting Group
Private and public sector stakeholders’ panel discussion
Panellists:
Dr Sol Ruiz, Spanish Medicines Agency – NO SLIDES PRESENTED
Dr Shelly Heimfeld, Fred Hutchinson Cancer Research Center – NO SLIDES PRESENTED
Duncan MacKay, Genzyme Corporation – NO SLIDES PRESENTED
Breakout session 1
Business strategy session: Overcoming comparability and scale-up challenges for cell therapy and tissue engineered product candidates
Moderator’s introduction
Dr Scott R. Burger, Advanced Cell & Gene Therapy
Engineered cellular therapeutics: Optimizing product potency utilizing scalable manufacturing
processes
Dr Madhusudan V. Peshwa, Maxcyte, Inc – NOT AVAILABLE
Building a scaleable stem cell processing platform
Dr Ram Mandalam, Cellerant Therapeutics, Inc
Tissue engineered products vs cell therapy products: Is there a regulatory difference?
Dr Gary C. du Moulin, Genzyme BioSurgery
Case study
What are the issues in setting up and maintaining a GMP cell processing facility?
Dr John D. McMannis, MD Anderson Cancer Center
How is the commercial relationship between academic controls and the industry set to develop?
Dr Theresa Whiteside, University of Pittsburgh Medical Center
Case study
A successful business model for the migration of an academic centre to CMO
Raymond Wood, Cell Therapies Pty Ltd
Breakout session 2
Regulatory workshop: Navigating the European regulatory mechanisms for Advanced Therapy Products
• A timely, in-depth exploration of the nuts and bolts of the new European regulatory framework for cell and gene therapies and tissue engineered products
Chair’s introduction
Dr Chris Holloway, ERA Consulting Group
The new EU regulation for 'Advanced Therapy Medicinal Products'
Dr Lincoln Tsang, Arnold & Porter LLP, London
Discussion of the key guidelines and specific regulatory issues
Dr Chris Holloway, ERA Consulting Group
Regulatory strategies to support the development of 'Advanced Therapy Medicinal Products' in
the EU
Dr Richard Turner, ERA Consulting Group
Panel discussion
Moderator:
Dr Chris Holloway, ERA Consulting Group – NO SLIDES PRESENTED
Panellists:
Dr Sol Ruiz, Spanish Medicines Agency – NO SLIDES PRESENTED
Dr Lincoln Tsang, Arnold & Porter LLP, London – NO SLIDES PRESENTED
Christopher A. Bravery, UK Medicines & Healthcare Products Regulatory Agency (MHRA) – NO SLIDES PRESENTED
Dr Richard Turner, ERA Consulting Group – NO SLIDES PRESENTED
Breakout session 3
Designing and delivering optimal preclinical data packages for cell and gene therapies
• Which animal models should you choose?
Moderator’s introduction
Raymond T. Bartus, PhD, Ceregene, Inc – NO SLIDES PRESENTED
US FDA perspective
Preclinical perspective on cell and gene therapy products
Dr Theresa Chen, US Food & Drug Administration
Case study: Cell therapy
GVAX® immunotherapy for prostate cancer
Dr Karin Jooss, Cell Genesys – NOT AVAILABLE
Case study: Cell therapy
Matching clinical disease targets with predictive animal models for treating CNS disorders with human neural stem cells
Ann Tsukamoto, PhD, StemCells, Inc
Case study: Gene therapy
DNA targeted therapeutics: Species specificity issues in efficacy and safety studies
Dr Dale Ando | Part 1 | Part 2 |, Sangamo Biosciences, Inc (IN TWO PARTS)
Case study: Gene therapy
Translational development - from bench to clinic: rAd-IFN pathway to phase I
Beth Hutchins, PhD, Schering Plough Biopharma
Afternoon Plenary session
VC panel discussion
Short presentations and panel discussion
Moderator:
Dr Akihiro Shimosaka, EPS Co. Ltd
Panellists:
Dr Dale R. Peterson, The Vertical Group
Manish Singh, PhD, California Technology Ventures
Kathryne F. Carr, Tall Oaks Capital Partners, LLC
Douglas D. Lind, MD, Greenwich Biotech Partners
Linda Powers, Toucan Capital Fund II & Maryland Stem Cell Commission (awaiting from Ms Powers)
Wednesday, January 24th, 2007
Day 3
Morning plenary session
Accessing key potential sources of funding for cell and gene therapy R&D
Chair’s introduction
Big pharma perspective
What is their current assessment of the cell and gene therapy area and ongoing strategy for involvement in the field?
Dr Robert F. Willenbucher, Johnson & Johnson Development Corporation
Analyst’s perspective
Defining short-, mid- and long-term expectations for the cell and gene therapy sector
Matt Kaplan, Punk, Ziegel & Co
NIH perspective
Improving the translation of products from academic to industrial settings
Dr Anthony Hayward, NIH
Show me the money: Getting state funding for cell therapy
Michael J. Werner, The Werner Group
Case study
Maryland’s stem cell funding program
Linda Powers, Toucan Capital Fund II &Maryland Stem Cell Commission
Breakout session 1
Cell therapy clinical development update: Adult stem cell therapies for cardiac indications
• What is the most promising stem cell source and delivery method for cell therapy moving forwards?
Moderator’s introduction
Pharmacological mobilization of progenitor cells as a future therapeutic strategy
Dr Jaipal Singh, Eli Lilly and Company
Case study
Adult, autologous CD34+ stem cells: A near term solution
Andrea Hunt, Baxter Healthcare Corporation – NOT AVAILABLE
Case study
Megadoses of stem and progenitor cells for the regeneration of failing hearts: A new application for tissue repair cells
Elmar Reinhold Burchardt, MD, PhD, Aastrom Biosciences, Inc
Case study
ALDH-bright cells as therapeutic agents for cardiovascular diseases
Dr Andrew Balber, Aldagen
Case study
Adipose tissue: a unique source for real-time, autologous cell therapy
Dr Kai Pinkernell, Cytori Therapeutics, Inc – NOT FOR POST EVENT ZONE
Short presentation & panel discussion
Addressing potential IP-related obstacles as adult stem cell therapies progress towards the market
Moderator:
Dr Nanda P. B. A. Kumar, Reed Smith LLP
Panellist:
Yaron Daniely, PhD, MBA, Gamida Cell Ltd – NO SLIDES PRESENTED
Breakout session 2
How do you develop a potency assay that will meet the regulator’s requirements?
• Defining and integrating product characterization requirements at each step of the R&D process
Moderator’s introduction
Dr Joyce Frey-Vasconcells, PharmaNet Inc
Lessons from another biologicals sector – how the vaccine industry has addressed the challenge
of successfully developing potency assays to meet the regulator’s requirements
John P. Hennessey, Jr, PhD, Merck Research Laboratories
Case study: Gene therapy
Transitioning plasmid-based products from Phase II to Phase III
Alain Rolland, Pharm D, PhD, Vical
Case study: Gene therapy
European perspective: Development of CereproTM
Dr Alan Boyd, alanboyd consultants ltd (Consultant, Ark Therapeutics Ltd)
Case study
Provenge (sipuleucel-T) product characterization and potency assay development
Dr David Urdal, Dendreon Corporation
Developing a cell potency approach depends on autologous vs allogeneic source of cells
David L. Amrani, PhD, FAHA, Baxter Healthcare Corporation – NOT AVAILABLE
Breakout session 3
Finance workshop: What fund-raising options are open to your organization at each stage of R&D?
Moderator’s introduction
Funding opportunities for cell and gene therapy companies
Dr Doug Jolly, Advantagene
Case study
Reviewing an IPO – what were the keys for success and what comes next?
Angelene Simonello, ViaCell, Inc - CANCELLED
Case study
How to secure funding for orphan disease indications
Sheila A. Mikhail, Asklêpios BioPharmaceutical, Inc
Case study
Capitalizing on public and private sector funding opportunities in the Far East
Dr Kwan Hee Lee, TissueGene, Inc
Case study: Partnering with a NGO
Ralph Paul, Targeted Genetics Corporation – NOT AVAILABLE
Afternoon plenary session
Closing panel discussion
What have been the key take-home messages from the event?
Moderator:
Dr Alan Boyd, alanboyd consultants ltd – NO SLIDES PRESENTED
