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Cell & Gene Therapy Insights

Cell & Gene Therapy Insights

Spotlights 2020

Preclinical and translational R&D insights

GUEST EDITOR: TBC
  • 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?
COMING IN FEBRUARY 2020

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

GUEST EDITOR: TBC
  • 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
COMING IN MARCH 2020

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

GUEST EDITOR: TBC
  • 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?
COMING IN APRIL 2020

Trends and advances in gene therapy delivery and gene editing

GUEST EDITOR: TBC
  • 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?
COMING IN MAY 2020

Clinical development strategy, tools and trial designs

GUEST EDITOR: TBC
  • 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
  • COMING IN MAY 2020

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

GUEST EDITOR: TBC
  • 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
COMING IN JUNE 2020

Market access: evolving commercialisation trends and strategies

GUEST EDITOR: TBC
  • 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?
COMING IN AUGUST 2020

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

GUEST EDITOR: TBC
  • 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?
  • COMING IN SEPTEMBER 2020

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.
COMING IN OCTOBER 2020

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

GUEST EDITOR: TBC
  • 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?
COMING IN NOVEMBER 2020

Round-up of the year and expert predictions for 2021

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.

COMING IN DECEMBER 2020
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