DAY 1 | Monday, January 24th 2011

Morning plenary session
So you’ve got a C&GT product that works – now how are you going to make money from it?

• How are commercial cell/gene therapies and tissue-engineered products performing in the global marketplace? Delivering the facts and figures
• Public/private sector payers' perspectives: How have Provenge and other commercial cell/gene therapy and tissue-engineered products been assessed for reimbursement?
  • What lessons are there for developers of future cell & gene therapy products?
  • What degree of benefit do payers need to see from novel modalities such as cell & gene therapy? What benefit do they need to see from cell or gene therapies in order to give them the green light to be added to, or to replace, the Standard of Care?
• Industry/payer roundtable discussion
  • Analyzing the pricing, reimbursement and marketing strategies behind commercial products
    • How to argue your price point - what data do you need?
    • How does one tie one's R&D strategy and business model to a feasible future price point given current uncertainty in the healthcare marketplace?
      • How to model and integrate commercial economic considerations during the early clinical phases?
    • Examining future plans: How will we realize the full commercial potential of our products moving forward?
      • Ongoing roll-out and market development on a global basis
      • Label expansion
      • Combination with adjuvants/other therapeutic modalities
      • What are our expectations/estimations in terms of off-label or unreimbursed use?
      • What steps are we taking to defend our patents?
  • How to realize the commercial potential of niche indications and orphan diseases in the context of the evolving pricing and reimbursement environment?
    

FOLLOWED BY YOUR CHOICE OF 3 PARALLEL BREAKOUT SESSIONS:

Cell therapy road to commercialization stream
Focus session 1
(Shared with participants in the Washington Vaccine Forum 2010)
Addressing the specific manufacturing, regulatory and logistical challenges facing autologous cell therapy products at commercial scale

• Learning lessons from the development history of autologous cell therapies: A series of presentations, each followed by an extended period for Q&A/discussion
  • What CMC/QA challenges did they face over the course of their full development history, and how were they addressed?
  • Ramping up to commercial capacity: What are the key strategic differences between operating at clinical and commercial scales?
• Examining the full process at commercial scale: From extraction of a patient's cells to reinsertion - defining the key steps and issues at each point
  • When and where do the key issues/challenges occur and how are they being addressed?
    • Transportation/storage/security - what methods are we using to track individual patients' cells? How are shelf life limitations impacting the business model? How would we address this aspect differently for future therapies?
  • Management of the clinical site - how to ensure that multiple sites comply to GMP requirements without the element of control provided by the clinical trial environment?
    • How are we educating physicians and patients alike?
  • How is product testing conducted at each step of the process, from pre-collection through final release, to post-therapy follow up?
• Panel discussion: What are the key lessons for the industry to avoid other players having to 'reinvent the wheel'?
  • How have enabling technologies developed since Provenge entered Phase III, particularly with regard to boosting efficacy/potency, and how can the industry harness these advances to accelerate the pivotal trial process?
  • In oncology, is Progression-Free Survival or Overall Survival the optimal pivotal trial endpoint?
    

OR | Focus session 2
eSC's and iPS cells: Separating the hype from the real therapeutic potential

• FDA perspective: How do we view eSC's and iPS cells, particularly in the therapeutic setting moving forward, and with regard to safety and ethical issues?
• Embryonic stem cell therapeutics
  • Updates on product candidates entering the clinic: What is the latest data, and how are regulatory issues (eg. regarding safety, use of devices, use of genetically-engineered cells) being addressed given the lack of specific guidelines?
  • Questions to the speakers & discussion: What are the highest hurdles to the further development of eSC therapies in our view? What specific technical aspects are the most challenging? What technological approaches are we thinking about and/or developing in terms of cell tracking in vivo?
• Induced pluripotent stem cells: Academic and industry perspectives
  • Where does the iPS cell field stand today?
  • What are our expectations for future application areas?
  • What future role (if any) will they play as a therapeutic modality?
  • What is the latest progress with genetic manipulation of iPS cells?
  • Questions from the audience & panel discussion: What is big pharma's view on the current and future utility of iPS cells? Where are they with their evaluation and/or implementation of this technology area?

OR | Gene therapy R&D strategies stream
Workshop
(Highly interactive session for a maximum of 25 participants)
Delivering step-by-step strategic insights into the efficient, successful development of gene therapies from preclinical through to pivotal trials

• How can we deliver the required toxicology studies given the relatively small range of animal models that are readily acceptable to do the regulators? When to engage the regulators?
• Clinical development through the phases: Case studies examining product candidates at each stage
  • Weighing up the various options for clinical development/patient recruitment on a global basis: What are the regulatory considerations?
  • Integrating product development and process scale-up requirements with clinical strategy/trial design
  • Endpoint selection and the role of biomarkers
• Questions from the audience & panel discussion
  • Endpoint selection at the pivotal trial stage: Sharing regulator insights and lessons learned from past experiences
  • How to get regulatory acceptance from multinational authorities prior to starting your phase III trial?

THEN | Afternoon plenary session
Global regulatory update: Who's driving the sector forward and what are the corresponding R&D/commercial opportunities for the C&GT industry?

• Regulator updates - short presentations: How are regulatory pathways developing in key Asian nations including Japan, South Korea, China, Taiwan and India?
  • What are the top 5 recent regulatory issues/developments/experiences relating to cell & gene therapies in each region, and how are these shaping future guidelines and legislation?
• Regulator/industry roundtable discussion
  • What are cell & gene therapy companies' experiences to date in navigating the regulatory pathways previously described?
  • What are the similarities and differences between Asian regulatory environments and those in the US and Europe, and what are the repercussions (and potential clinical development/commercial opportunities) for industry?
    • What are the US and European regulators' views and experiences regarding clinical data quality, variations in standards of practice, and similarity of study populations based on their evaluation of multinational clinical trials?
      

Close of day 1, followed by a cocktail reception in the exhibition area

DAY 2 | Tuesday January 25th 2011

Morning plenary session
Devising cell & gene therapy financing/business models for success in the 'post-VC world'
• Are big pharma/big biotech fully engaged as yet? If not, why not?
• Are the public markets still a viable option?
• What alternative public/private sector funding options are available now?

• Is the VC model really broken in the cell & gene therapy space?
  • What financing trends can we expect to see developing over the short- to mid-term?
• Big pharma/big biotech perspectives
  • Short presentations - exploring the specific activities of established and new big pharma/big biotech players in the cell & gene therapy area
    • How are we developing/readjusting in-house to better understand cell & gene therapy approaches, and to be able to potentially accommodate them?
    • Are we looking to partner and, if so, what do we need to see from potential collaborators (eg. in terms of product characterization/Mechanism of Action data)?
    • What is the focus of our own internal R&D efforts? How are we seeking to combine cell/gene therapy with other therapeutic modalities to differentiate our technologies/product candidates?
    • How will the trend for increasing big pharma/big biotech interest/involvement in orphan indications/niche markets develop? What role will cell & gene therapy play in the further pursuit of these markets, and what is the right timing for deal-making in big pharma/big biotech's eyes?
  • Case studies: Analysis of closed deals between big pharma/big biotech and cell & gene therapy companies
    • Examining the strategic drivers behind these collaborations: What data/business model aspects convinced the big pharma/big biotech company to get involved in each example?
  • Roundtable discussion
    • How do big pharma/big biotech companies view and define 'Proof of Concept in man' with regard to their own internal R&D projects? What specifically do they look for and what clinical trial endpoints do they select as a consequence? What are the lessons for cell & gene therapy companies seeking a big pharma/big biotech partner?
    • What is the opportunity for deals to be done earlier in the R&D process (pre-phase II) to mitigate risk and accelerate development? (eg. Option agreements). What are the keys to getting such deals done?
    • How to bridge the disconnect between big pharma and the cell & gene therapy field when it comes to the autologous therapy commercial model?
• Wall Street analyst's perspective
  • What is our current assessment of the cell & gene therapy space as whole?
  • What do we look for in a public company in terms of choosing whether or not to invest?
• Multiple stakeholders' roundtable discussion: How are alternative public (eg. State/Government funding for stem cell R&D, NIH grants, Tax Credits) and private (eg. Foundation funding) financing opportunities and initiatives evolving to meet the challenge of delivering approved cell & gene therapy products to patients?
  • What support is out there for clinical-stage companies? Does each stakeholder have a different/specific role to play when it comes to early- to mid-stage financing of the sector? What are the opportunities for companies without a US-based affiliate/subsidiary?
    

Followed by your choice of 3 highly interactive parallel sessions:

Cell therapy road to commercialization stream
Focus session 1
Tackling the key logistical challenges in the commercial delivery of autologous and allogeneic cell therapies

• A view from the trenches: A clinician's perspective on what works - and what doesn't - incell therapy processing and delivery to the patient
  • What should be the key areas of focus for the industry in terms of educating patients and physicians alike as both autologous and allogeneic cell products scale up to commercial levels?
• Case studies followed by audience questions/panel discussion: Comparing and contrasting the post-manufacturing, pre-treatment pathways for autologous and allogeneic cell therapies
  • What are the key steps/technical options for distribution, transportation and storage?
  • How do you coordinate product release for autologous cell therapy products with the clinical site?
  • What are the optimal mechanisms for tracking patient-specific products?
  • Storage/handling of cell therapies at the clinical site: How to regulate this and what are liability issues? How to alleviate them? At what point/under what circumstances does liability shift from company to physician? Which emerging processing technologies can address potential liability issues?
• Questions from the audience & panel discussion: Debating the pros and cons of clinical site vs. factory-based manufacturing options in terms of logistical issues and scaling-up challenges
  

OR | Gene therapy R&D strategies stream
Focus session 2
Cell therapy/gene therapy crossover products: How are leading candidates performing in the clinic and how are safety issues being addressed?

• What preclinical toxicology work do you need to do (in animal models and single cells alike) and what clinical monitoring is required to guard against potential safety issues moving forward?
  • Addressing the risks associated with vector integration: How are the regulators viewing this aspect?
• Case studies: Clinical development updates from a range of novel ex vivo cell products: Reviewing the latest safety/efficacy data and clinical trial designs
  • What are the prospects for the various approaches: How will they realize their therapeutic potential?
  • What are the differentiators between the companies involved in terms of business model? How do they relate to each specific technical/scientific approach?
• Questions from the audience and panel discussion: How commercially viable are these products? What might the commercialization model look like?
  • What is the product - the virus or the cell?
  • Who should release the transplant and who should transfect the cells? What are the pros and cons of centralized versus onsite processing solutions in the context of GMP compliance?

OR | Workshop
(Highly interactive session for a maximum of 25 participants)
Delivering a new business paradigm for cell & gene therapy companies: Weighing up financing models and collaborative strategies for efficient R&D progress

• Case studies from cell and gene therapy companies that have recently secured significant financing or closed partnering deals: What were the secrets of their success?
  • How to secure VC backing in the current environment?
  • Partnering deal
• Short presentation - case study: Capitalizing on opportunities to partner with non-pharma industry companies
• ...followed by panel discussion/audience Q&A
  • What other opportunities are there for cell & gene therapy companies to partner with non-pharma companies (eg. those from devices, imaging, biomaterials sectors)?
    • What are the challenges specific to each in terms of aligning goals and business models and how are they being addressed?
    • How will the general life science/healthcare trend of focusing on biology at the cellular/molecular level drive further collaboration between cell & gene therapy companies and non-traditional partners? How can the cell & gene therapy industry engage these sectors more aggressively?
  • How can cell & gene therapy companies harness alternative revenue streams to support their R&D?
• Short presentation - case study: Making the case for the 'virtual biotech' - is this model the answer for the quick, cost-effective progression of cell & gene therapies to Phase II and beyond?
• ...followed by panel discussion/audience Q&A
  • Examining the arguments of companies that have maintained core competencies in R&D and manufacturing versus those that have pursued an outsourcing-based model: Understanding the key drivers behind the decision in each case
  • Assessing the pros and cons of outsourcing with CROs and CMOs over the short- and long-terms as the services sector surrounding the cell & gene therapy industry continues to mature
    • What will be the repercussions for Western cell & gene therapy companies if the current trend for CRO migration to Asia continues?

Then | Afternoon plenary session
Product characterization and Mechanism of Action of cell & gene therapies: What data do the regulators require in Phase III?

• Short presentation: FDA update on regulatory requirements for the final drug product
  • Use of products of animal origin, shelf life, potency assay
    • What do you do/how do you approach the regulator where there is no tried and tested potency assay for your application?
• ...followed by audience Q&A/panel discussion: How to begin addressing these challenges in earlier phases?
• Short presentation: How are cell/vector tracking technologies improving to help meet MoA data requirements?
• ...followed by audience Q&A/panel discussion
  • What are the pros and cons of the various emerging technologies for biodistribution tracking?
    • How are issues such as the toxicity and potentially short lifespan of 'labels' being addressed by innovative approaches?
  • How can we be more proactive at sharing cell/vector distribution data to save each individual company/organization having to do the same work?

Close of day 2, followed by a cocktail reception in the exhibition area

DAY 3 | Wednesday, January 26th 2011

Morning plenary session
Who's next to market? Evaluating the prospects of cell & gene therapies in pivotal trials
• How are both lessons from the past, and cutting edge technologies, informing clinical development and regulatory strategies?

• Case studies: Clinical development updates on leading gene therapy, stem cell therapy and tissue engineered product candidates in pivotal trials
  • What is the latest clinical data?
  • What specific trial designs are being employed and why? What is the regulatory rationale? How have we engaged with the regulators?
  • How are we tackling the increasingly acute challenge of patient recruitment? How to structure your clinical development program given the comparative lack of patients for orphan disease indications?
  • How does our clinical development strategy integrate with our overall business/commercialization model?
  • In the case of the stem cell therapies, specifically: How are the companies involved differentiating their cell platforms through manipulation/enhancement of the cells? What are the regulators asking for in terms of tracking data?
• Questions for the speakers, followed by industry/regulator/investor roundtable discussion
  • What insights regarding the selection of pivotal trial endpoints can be drawn from past trials in both cell and gene therapy?
    • What work can be done pre-phase III to inform the endpoint selection process?
  • What are the keys to successfully undertaking a multinational clinical trial programme?
    • What are the mechanics, step-by-step?
    • What are the major regulatory/strategic considerations in running trials simultaneously in the US, Europe and other regions of the world?

FOLLOWED BY YOUR CHOICE OF 3 PARALLEL BREAKOUT SESSIONS:

Cell therapy road to commercialization stream
Focus session 1
Piecing together the technology jigsaw to enable the economically-feasible commercial scale-up of cell therapies

• When should you delay clinical advancement in favour of further process/product development to improve the cost/performance aspect?
  • How do you assess what your product price point might be, and then accurately estimate the improvements that might be made on the unit cost and product performance?
  • What impact might the continued development have both on costs and on potential price?
• Cell therapy company case study: Assembling technology components into a platform - what were the decision-making processes and rationale behind our choices?
  • Which specific components are required to balance the cost of goods/manufacturing vs. product performance equation, and how are we strategizing to optimally access and integrate them?
    • How much to invest in optimizing the process with consideration to CoG?
  • How to optimize strategic decision-making in terms of biomaterials sourcing?
• When/where/how should automation technology be applied to minimize costs and improve quality/regulatory compliance?
• Short presentation(s) followed by panel discussion: Addressing scale-up issues with tissue/cell therapy-device combination products
  • When to engage a device specialist and when to develop your own? Examining the pros and cons of each approach, and the decision-making processes for companies that have chosen either pathway

OR | Gene therapy R&D strategies stream
Focus session 2
Focus on the eye: Comparing and contrasting the scientific approaches and clinical data for innovative gene and RNAi therapies in the ophthalmology area

• Case studies: Clinical development updates in the age related macular degeneration (AMD) area with varying gene and RNAi therapy technologies/methods of delivery
  • Overcoming targeted delivery issues: What specific approach is each company employing and why?
  • How do the clinical development strategies tie in with the company's overall business models?
• Questions for the speakers, followed by panel discussion
  • Comparing and contrasting viral and non-viral delivery platforms for ophthalmology therapeutics - which are achieving the desired safety and efficacy profiles? Where do challenges remain?
  • Examining clinical trial endpoints for gene and RNAi therapies in ophthalmology indications - are they good enough? What do these candidates really need to show in trials to ensure there is going to be a viable product at the end?
  • What is the ideal combination of delivery device and vector for ophthalmology indications?

OR | Workshop
(Highly interactive session for a maximum of 25 participants)
In-depth regulatory workshop

• Addressing specific issues arising from disharmonization between US and European regulations for cell & gene therapies

Close of the Phacilitate Cell & Gene Therapy Forum 2011 followed by buffet lunch in the exhibition area