Facilitator: Antonio Casini PhD (Alia Therapeutics)

This workshop will focus on discussing the advantages and disadvantages of different gene-editing technologies and how to select the right one for your approach. We will discuss the following topics:

- Comparing CRISPR-mediated knockout and knock-in strategies such as homologous recombination, HITI and MMEJ with novel, DSB-free technologies such as base- and prime editing, focusing on the ideal uses of each technology and their advantages and disadvantages.

- How to decide between different Cas9 and base-editor variants depending on the context of your experiments.

- Use of protein, RNA or plasmid formulations for Cas9 transfection into cells.

Facilitator: Alessandro Umbach PhD (Laboratory of Molecular Virology, University of Trento, Italy)

This workshop will focus on discussing the difficulties that arise when trying to deliver Cas9 into cells or specific tissues, and the different delivery technologies that currently exist. We will discuss the following topics:

- In vitro CRISPR delivery systems for efficient transfection of CRISPR reagents in different cell types.

- Viral delivery systems and their applications and limitations for in vivo CRISPR delivery.

- Non-viral delivery systems for transient activity of CRISPR in target tissues.

- Hurdles, bottlenecks and future perspectives for CRISPR delivery and its clinical relevance.

Facilitators: Carla Fuster García PhD, Julia Klermund PhD and Manuel Rhiel PhD (The University Medical Center Freiburg)

This workshop will focus on the pre-clinical analyses that are essential to guarantee the safety of a gene-editing approach before it can be brought to the clinic. We will discuss the kind of safety data needed in order to obtain approval for a clinical trial, and the best methodologies for obtaining it. The main topics will be:

- Regulatory guidelines for gene-editing safety.

- Types of pre-clinical safety data necessary for approval.

- Producing quality pre-clinical safety data to ensure approval.

- The future of gene-editing regulations and how they will affect pre-clinical studies.

This workshop will focus on the often strenuous process of obtaining approval for a gene-editing clinical trial, aiming to give you the best tools to produce and present all the necessary data and documentation so that your approval process is as smooth as possible. We will discuss:

- Pre-clinical data necessary for approval, including potency, dose-response and safety studies.

-GLP and GMP guidelines relevant for gene editing.

-Necessary documentation for approval.