• In vivo and in vitro tools in application – which ones are providing the greatest depth of insight in terms of predicting clinical safety and efficacy?
• How to address the shortfalls in current hPSC-based preclinical models?
• How to optimise integration of bioprocess development with preclinical R&D?
• Regulatory and operational best practice for preclinical-clinical translation of cell & gene therapies
• How are regulators’ expectations and requirements changing as knowledge and experience continues to build in cell & gene therapy?
• What to outsource and what to keep in-house

• Starting material variability and its impact on reproducibility – managing regulatory and commercial repercussions
• Weighing up emerging allogeneic cell sources: pros and cons in practice
  • iPSCs
  • Cord blood and tissue
• Delivering apheresis/leukapheresis best practices
• Securing supply of critical raw materials through scale-up
• Ensuring maximum quality at minimal cost

• How to boost yield and titer throughout upstream and downstream bioprocessing?
• Cutting edge closed, automated systems – can we quantify the impact on cost, quality and productivity?
• Viral vector process controls and analytics – how close are we to an era of precision manufacturing in gene therapy?
• Assessing novel tools in practical application – how are they impacting cost, speed and quality?
• Where are the critical remaining gaps in the toolbox

• Innovations in viral and non-viral vector engineering and bioprocessing
• Next-generation gene editing tools – assessing the relative pros and cons of novel platforms
• Pathway to the future application of gene editing platforms in clinical application
• How to tackle the issue of immunogenicity for both viral vectors and gene editing in in vivo applications

• How are clinical trial designs and overall strategy evolving in the rare disease arena?
• Combination therapy development in immuno-oncology – do’s and don’ts in trial design.
• Clinical operations – what are the specific considerations with cell & gene therapy products, particularly for multinational trials
• Critical considerations for cell & gene therapy development in paediatric patient populations?
• Biomarkers and surrogate endpoints linked to evidence of clinical effectiveness and response to treatment: case examples in cell & gene therapy and regulatory implications
• Harnessing clinical patient outcomes data for biomarker development
• What is the latest progress in identifying responders and non-responders in the immuno-oncology sphere?
• The growing influence of adaptive trial designs in cell & gene therapy
• Key lessons learned from expedited regulatory pathway

• Analysing early commercial experiences with cellular immunotherapies – what lessons for the next generation of product candidates making the transition from clinical to commercial? (Eg. in terms of cost control? Market and patient access strategies?)
• Enabling allogeneic approaches
• Are iPSCs ready to step forward? What are developers’, manufacturers’ and regulators’ key concerns?
• Cell transduction/engineering tools and techniques – what does the future hold for viral and non-viral vectors and gene editing?
• How will decentralised manufacturing models continue to evolve and emerge?
• Global supply chain optimisation

• Global analysis of the ongoing evolution of valuation, pricing and reimbursement models
• How will cell & gene therapies compete with each other on the market, and with what impact on pricing and reimbursement?
• What are the key differentiators and sources of competitive advantage for cell & gene therapy products in key indications and therapeutic areas such as hematological malignancies and monogenic disorders?
• How will the First-to-Market vs. Best-in-Class question play out in cell & gene therapy?
• How are emerging markets for cell & gene therapy products (eg. China) developing, and what are the keys to accessing them?

• Examining current trends in in-house and outsourced manufacture – where is the cell & gene therapy space heading, and why?
• Where are the key opportunities to target cost of goods reductions in both cell therapy and gene therapy today? How to capitalise upon them?
• In process and release testing – how are next-generation analytics driving improvements in product quality and accelerating manufacturing timelines?
• How to demonstrate comparability with both cell therapy and gene therapy products through the transitions between early clinical, pivotal trial and commercial phases?
• Continuous manufacture: is it likely to impact the cell & gene therapy space? If so, how and where?
• Standardisation: what are the critical next steps to further enable cell & gene therapy manufacturing?

• New horizons in immuno-oncology:
  • How are emerging autologous and allogeneic approaches and immune cell types performing in preclinical and early clinical studies?
    • What is the evidence to date that they can improve rates and durability of response and address lingering safety concerns?
    • Next steps in targeting and tackling solid tumours: what have we learned from earlier approaches?
  • Preclinical to clinical translatability: overcoming in vivo hurdles for immuno-oncology therapies
  • Window on future enabling technologies: what impact will tools such as genome and epigenome editing have on the immunooncology space moving forward?
  • How are novel therapeutics combinations performing in clinical applications?
• The dawn of cellular immunotherapy in non-cancer: evaluating the promise of novel approaches in tolerisation, autoimmune diseases and diabetes.

• Step-by-step assessment of cell therapy bioprocessing tools – showcasing the state-of-the-art (including closed, automated systems and single-use technologies) for:
  • Isolation
  • Transduction
  • Expansion
  • Harvest, concentration and washing
  • Formulation and fill-finish
  • Packaging and cryopreservation
• GMP in a box: what will the next wave of ‘all-in-one’ solutions look like?
• Cell therapy process controls and analytics – where is progress being made in improving robustness and accelerating timelines in cell therapy manufacture?
  • How is the latest innovation in QC analytics helping reduce release testing waiting times?

A wrap-up of the year focused variously on different geographical regions, cell & gene therapy technology areas, indications and functions. Thought leaders from across the cell & gene therapy community will reflect on the significant events and talking points of 2019 and share their expectations for 2020 and beyond.