Program

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.

• Workshop: Safety

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.

• Workshop: Clinical

Chairs: Alessia Cavazza (University College London) and Waseem Qasim (UCL Great Ormond Street Institute of Child Health)

• Track 5: Pre-clinical
• Track 6: Clinical Trials

Chairs: Marcello Maresca (AstraZeneca) and Anna Cereseto (University of Trento, Italy)

• Track 1: Tools
• Track 2: Delivery

Chairs: Alessia Cavazza (University College London) and Waseem Qasim (UCL Great Ormond Street Institute of Child Health)

• Track 5: Pre-clinical
• Track 6: Clinical Trials

Chairs: Marcello Maresca (AstraZeneca) and Anna Cereseto (University of Trento, Italy)

• Track 1: Tools
• Track 2: Delivery

A 1-hour guided boat trip in the Copenhagen canals is the perfect way to cap off your stay at the CRISPR Medicine Conference. Participants will walk from the venue site to the boat (25-min walk) after the conference programme formally ends at 15:00 PM. Registration is necessary and can be done here. Price: 10 EUR.