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08:30 - 09:00 30 mins
Main agenda
Registration and Morning Coffee
09:00 - 16:00 420 mins
Main agenda
Applying new technologies to ATMPs: Gene Editing and CRISPR
  • James Smith - Principal, Biolacuna and Research Associate, CASMI, UK
  • Sarah Robinson - Associate, Biolacuna, UK

As the ATMP industry matures and therapeutics see more and more regulatory success, the gene editing and particularly CRISPR development market has been growing in the background. The first CRISPR therapeutics are entering the clinic, and the recent boom in activity in expected to continue.

In this workshop, designed as an introductory course for industry professionals less familiar with CRISPR, we will cover

  • A brief introduction and assessment of CRISPR technology and other gene editing approaches: pros and cons
  • Case studies of major players in the CRISPR space
  • The CRISPR market and how it is expected to grow and change in the near future based on analysis of pre-clinical research
  • Update on the IP landscape for CRISPR
  • Analysis of the research literature to determine long term expected change
  • The application of CRISPR to cell therapies, e.g. CAR-T
  • Early regulatory considerations for CRISPR – analysis of ATMPs and extrapolation to CRISPR

  • Workshops will finish no later than 16:00
  • Refreshments, documents and a lunch will be provided
09:00 - 16:00 420 mins
Main agenda
Comparability for ATMPs
  • Christopher Bravery - Director, Consulting on Advanced Biologicals Ltd., UK
  • Karin Hoogendoorn - Scientist ATMP Product Development, Leiden University Medical Centre (LUMC), The Netherlands

Change is inevitable and necessary both in development and over the post-approval product lifecycle. Whenever changes are made it is necessary to confirm they do not adversely impact the quality and therefore safety and efficacy of the product; this requires data beyond meeting current specifications. With any biological product this is challenging, for cell, gene and tissue products that cannot be fully characterised the challenges are greater still. Concerns about comparability undertaken during development are common issues raised during review and often delay market approval or contribute to failure. This course explains what comparability is and how to develop a successful comparability protocol.

Workshop Learning Overview

• What is comparability?

• Why is meeting existing specifications not comparability?

• How do I apply the principles of comparability to highly variable products?

• Case studies: Common mistakes with comparability and their consequences.

• Interactive exercise: Spot the weaknesses and propose improvements to a worked comparability study.

12:00 - 16:00 240 mins
Main agenda
Viral Safety for ATMPs
  • Alison Wilson - Principal Consultant, CellData Services
  • Thomas R. Kreil - Senior Director, Global Pathogen Safety, Shire, Austria
  • Albert Stühler - Deputy Head, Virus Safety, Paul-Ehrlich-Institut

Topics to be discussed include:

  • Regulatory Overview
  • Human-derived Raw Materials
  • Animal-derived & Recombinant Raw Materials
  • Viral Safety Concepts for ATMPs