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Cell & Gene Therapy Insights

Cell & Gene Therapy Insights

Spotlights 2020

2020 Vision




With 2019 having broken yet more new ground in terms of commercial advances, translational and clinical R&D progress and platform technology innovation, the Twenty-Tens will surely go down as the most significant decade to date for the cell & gene therapy field. This month, CGTI reviews and celebrates remarkable recent progress, and previews a new year and decade through analysis of 2019’s major trends and talking points – what will the Twenties hold in store for cell & gene therapy?
    • KOL insights, reflections and horizon-gazing – just how far have we come and where do we go next?
    • What were the most significant advances and issues of 2019 in terms of providing pointers to the future?
    • Identifying both the key short-, mid- and longer-term challenges facing the global cell & gene therapy sector and next steps to their resolution.
    • Geographical analysis – how is the world of cell & gene therapy likely to evolve? (Commercialisation, finance, manufacturing, R&D, regulation)
    This special edition will also focus on enabling tools and technologies at the cutting edge with the potential to boost both safety and efficacy:
    • Synthetic biology
    • Gene editing
    • Microfluidics
    • On/off switches to control T cell activation

Preclinical and translational R&D insights

GUEST EDITOR: Karen Kozarsky, PhD, Founder & CSO, SwanBio Therapeutics
  • 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?

Raw and starting materials: troubleshooting supply, management and optimization issues

GUEST EDITOR: Dr Steven Goodman, Senior Director, Drug Product Manufacturing, bluebird bio
  • 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

Viral vector bioprocessing & analytics: today’s key tools and innovation requirements to meet future demand

GUEST EDITOR: Nolan Sutherland, Senior Associate Scientist, Vector/Cellular Process Development, bluebird bio
  • 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?

Trends and advances in gene therapy delivery and gene editing

  • 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?

Clinical development strategy, tools and trial designs

    • 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 pathways

Immuno-oncology: manufacturing and commercial business models for the new decade

GUEST EDITOR: Dr Usman Azam, President & CEO, Tmunity Therapeutics Inc
  • 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

Market access: evolving commercialisation trends and strategies

GUEST EDITOR: W. Neil Palmer, President and Principal Consultant, PDCI Market Access
  • 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?

Scaling up/out: cost-effective and robust transitioning through the clinic to commercial manufacture

GUEST EDITOR: Jan Thirkettle, Chief Development Officer, Freeline Therapeutics
  • 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 cellular immunotherapy: next-gen platforms and modalities

Guest Editor: Dr David Morrow, ATMP & Vaccine Scientific Programme Manager, Translational Medicine & Drug Development, EATRIS
  • 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.

Cell therapy bioprocessing and analytics: today’s key tools and innovation requirements to meet future demand

GUEST EDITOR: John Tomtishen, Director of Manufacturing, CMC Technical Operations, Legend Biotech USA Inc.
  • 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?

Tools of Tomorrow

Cell & Gene Therapy Insights’ annual exploration of cutting edge enabling and platform technologies. Find out about the innovation arriving over the horizon in 2021 to help drive address lingering R&D and manufacturing challenges, and drive cell and gene therapy products into and through the clinic towards commercialization.

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