Assessing emerging technological innovation and collaborative strategies to drive time/cost efficiencies and valuable translational insights in non-clinical development

• As the cell and gene therapy field expands into new, more complex diseases, pathways and targets, what are the key methods and tools, and where are most pressing innovation requirements, for preclinical/ translational R&D?
• Where is tangible progress being made in developing animal models that can deliver clinically translatable insights?
• How are novel/alternative non-clinical models and methods shaping up in terms of enhancing translatability to human diseases?
• Preclinical assay 101: defining the key considerations/requirements and clarifying timeframes relating to successful preclinical assay development
• Integrating early process development with preclinical/translational development

Examining risk mitigation best practices and exploring emerging challenges/opportunities for an expanding cell and gene therapy sector

• What lessons has the cell and gene therapy field learned about the raw and starting materials supply chain from the COVID-19 pandemic?
• What is the latest progress in ensuring a sufficient supply of starting materials for specific, rapidly expanding cell and gene therapy technology areas?
  • Eg. how are donor management strategies and cell conversion methods evolving to enable the allogeneic cell therapy field?
• Driving improvements in the consistency, reproducibility and traceability of reagents and other critical raw/ancillary materials for cell and gene therapy manufacture
• How to predict, measure and manage the impact downstream of variability in cell therapy starting materials?
• Key considerations and preparations for effectively managing materials supply risk and meeting regulatory requirements in the post-market approval setting.

Two decades on from the advent of commercial-scale protein therapeutics manufacture, is the viral vector space ready to make its own rapid advance towards industrialization?

• Are we ready to deliver improvements required in upstream yield and downstream throughput to finally alleviate the current bioprocessing bottlenecks affecting both AAV and lentiviral/retroviral vector production?
• Customized versus repurposed bioprocessing tools – what has been the impact on key steps in gene therapy processing from the emergence of more fit-for-purpose enabling technologies (eg. in the separation area)?
• What is the latest progress in terms of cost and time savings in viral vector bioprocessing, and what elements should we target for further gains moving forward?
• Key considerations for both in-house and outsourced viral vector manufacture in the current environment

Learning lessons from recent setbacks, utilizing novel technological innovation, and defining key steps from early development towards delivering robust data packages

• How will evolving regulatory guidance and manufacturing strategy trends impact approaches to demonstrating comparability and potency of cell therapy products?
• Examining challenges in, and benefits from, implementing Quality by Design and Design of Experiments methodologies for cell therapy manufacture
• Technology evolution: what efficiencies and consistency/qualityrelated benefits are emerging process analytical tools delivering in practice to cell therapy developers and manufacturers?
• Automation and associated analytics: what is the current state-of-theart in cell therapy?
• How to effectively harness and extract value from large analytical data sets to the benefit of cell therapy manufacturers?
• What progress in accelerating release testing to help further reduce manufacturing timeframes?

How sustainable are the manufacturing and commercialization models of leaders in the cellular immunotherapy field?

• What can we glean from macro investment, biophama innovation, and cancer healthcare trends to inform future cell and gene therapy clinical development, manufacturing and commercialization strategies?
• How are leading developers of both autologous and allogeneic cellular immunotherapies and supporting tool and service providers alike addressing challenges relating to:
  • Cost of goods control?
  • Minimizing manufacturing and supply chain timeframes?
  • Optimizing bioprocess and supply chain quality/robustness?
• What has the cellular immunotherapy sector learned from the COVID-19 pandemic?

How are cutting-edge R&D/technological innovation and a growing understanding of human and disease biology driving the expansion of gene therapy and gene editing into new, larger markets and indications?

• Predicting next steps for gene therapy R&D in the traditional stronghold of rare monogenic diseases
• Continuing the drive of gene editing into clinical application:
  • How are genome editing platforms evolving in the context of their applications in the burgeoning allogeneic cell therapy field?
  • Reviewing emerging gene editing platforms and their relative pros and cons versus established tools
• How successfully are current R&D approaches, such as next-gen vector engineering, at tackling key obstacles to the widespread and long-term success of in vivo gene therapy?
• Brave new world: assessing evidence of early progress in terms of gene therapies delivering both clinical efficacy and safer delivery strategies in larger indications

Ensuring strategic and operational excellence in the increasingly competitive world of cell and gene therapy clinical development

• Assessing the longer-term repercussions of the COVID-19 pandemic for cell and gene therapy clinical development
• How is competition for patients in key indications driving innovation in patient recruitment within the cell and gene therapy space?
• Weighing up the pros and cons of recent innovation in both clinical development tools and trial designs
• How should the cell and gene therapy field capitalize on the potential of Patient Reported Outcomes to bring both efficiencies and increased robustness to clinical development?
• Adaptive trial designs
• Biomarkers and surrogate markers linked to evidence of clinical effectiveness and response to treatment in cell and gene therapy: Recent progress and regulatory implications.
• Capitalizing on increasing interaction and convergence between the cell and gene therapy, in vitro diagnostics, and medical device fields

Identifying and addressing critical scalability bottlenecks in cell therapy and viral vector bioprocessing and supply chain to enable large-scale manufacture of advanced therapies

• Considerations for effective viral vector scale-up and scale-out with both insect and mammalian cell culture systems
• Improving the scalability of allogeneic cell therapy platforms (eg. iPSCs, extracellular vesicles)
• What should the future of scaled-out commercial autologous cell therapy manufacture actually look like?
• Assessing the advantages and limitations of current tools and processes through this lens
• How to ensure that PAT-derived data management and integration doesn’t apply the handbrake to rapid, robust scale-up/-out?
• What progress in delivering turnkey manufacturing platforms and solutions for the cell and gene therapy space to enable a stronger early-stage focus on scale-up?
• Key do’s and don’ts for an effective integrated approach to materials supply chain and bioprocess scale-up

Is recent innovation in analytical tools delivering the required improvements in cost, speed and accuracy for vector manufacturing?

• How and where are process analytical tools being successfully incorporated/integrated into viral vector bioprocess tools/steps, and where is further innovation required in this regard?
• What is the state-of-the-art in accelerated release testing for gene therapy products?
• Manufacturer and regulator perspectives on viral vector purity testing requirements and the utility of current tools
• How will evolving regulatory guidance and manufacturing strategy trends impact approaches to demonstrating comparability and potency of gene therapy products?
• Examining challenges in, and benefits from, implementing Quality by Design and Design of Experiments methodologies for gene therapy manufacture
• Is it time to prepare for continuous manufacture of gene therapy products? If so, what are the initial steps?

An exploration of the cutting-edge in next-gen cell therapy technology platforms and applications spanning the cellular immunotherapy and stem cell therapy realms

• Update on preclinical and early clinical-stage cell therapy platforms, with analysis of data generated to date, and discussion of how specific challenges and considerations relating to their ongoing development will be addressed – to include:
• Cellular Immunotherapies (eg. novel CAR T, TCR and NK cell therapy approaches)
  • Progress on the migration into solid tumor indications – how are obstacles presented by the tumor microenvironment being approached? What is the latest thinking on the potential role/utility of cellular immunotherapy in the combination therapy setting?
  • Profiling the continuing expansion of engineered cells (eg. T cells, NK cells, macrophages) into non-cancer therapeutic areas and indications
• Are MSCs back for the long-term?
• Exosomes
  

How recent technological advances and economic/healthcare/regulatory evolution have helped ignite the centralized versus distributed manufacturing debate

• How far have we come in automating cell therapy manufacture, and how far have we got to go? Assessing successes and setbacks to date, as well as key areas for future focus
  • How has the ‘unit operations vs. GMP-in-a-box’ debate moved on over recent times? And where next for either approach?
  • Automation of analytics – reviewing recent advances in enabling fully closed system manufacture, and creating the feedback loops necessary for true manufacturing automation
• What do the latest cost analyses for centralized and various distributed cell therapy manufacturing models tell us about their current relative efficiency? How is this picture evolving?
• Just how far away – and how desirable - is point-of-care manufacturing? Healthcare sector, industry, academic and regulatory perspectives
• Exploring the interface between tool development and trends in cell therapy manufacturing facility design – what do they mean for the cell factory of the future?

Cell & Gene Therapy Insights’ annual review of the year just gone, with trends analysis providing pointers to the likely big stories and developments in the 12 months ahead.

Combined with an exploration of enabling tools and therapeutic technology platforms likely to make a splash in 2022.