Pre-Conference Workshop Day

Monday 30 May

09:00 - 11:00 | Workshop A: Ex Vivo Gene Therapy Analytics Focus Session

With a lack of optimal analytical tools to support with the characterisation of transduced cells, this workshop aims to address the approaches being leveraged to support with the analytics for the unique cell therapy component of ex vivo therapies. The workshop will dive into:

  • High resolution single cell profiling of human hematopoietic stem cell drug products
  • Limitations of current methods for CD34+ drug product characterization and why we need a step change
  • How sc-RNAseq allows high resolution capture of Hematopoietic Stem/Progenitor Cells (HSPC) heterogeneity at steady state from multiple sources and upon exvivo gene transfer
  • How the information generated by single cell mapping of human HSPCs can be leveraged to design new potency assays for routine use in gene therapy and transplantation

Workshop Speaker:

Luca AvroBio

Luca Biasco
Director of Translational research
AvroBio

11:30 - 1:30 | Workshop B: Phase-Appropriate Development of Potency Assays for Starting Materials and UCART Cell-Based Gene Therapy

This workshop offers an in depth review of the critical components required to effectively demonstrate potency of cell based gene therapies. The presentation will provide insights into how to optimise the strategy for potency assays for starting materials and navigate the inherent complexities. We will address:

  • Potency assay development for UCART drug products is complex and often multimodal
  • Designing phase appropriate potency assays to support product characterization and development
  • Developing potency assays for starting material during early phase

Workshop Speaker:

Guillaume-Cornelis

Guillaume Cornelis
Senior Scientist, Team Leader
Cellectis

2:00 - 4:00 | Workshop C: Expression Based Assays and Functional Potency Assays: Determining the How and When

  • Among critical quality attributes which need to be monitored during drug development, potency is considered essential for its direct link to product efficacy. Developing the relevant set of potency assays for cell and gene medicinal products comes with numerous challenges due to the complex nature of the products and absence of standard approach.
  • This workshop will highlight several pragmatic approaches to consider when
    designing the potency assay strategy in the framework of current EU and USA
    regulatory expectations.

Workshop Speaker: 

François_Gianelli_VCLS

François Gianelli
Senior Director, CMC
Voisin Consulting Life Sciences

Lavi (2)

Lavi Thanabalasundaram
Regulatory Scientist
Voisin Consulting Life Sciences