First patients treated with CRISPR

crispr

First patients treated with CRISPR

The University of Pennsylvania in Philadelphia, USA, reported that the first two patients in a cancer immunotherapy trial have received CRISPR-edited medicinal product.

In this protocol, CRISPR is not applied in vivo, but ex vivo to eliminate three endogenous genes, the α and β chains of the endogenous T cell receptor (TCR) and Programmed Cell Death Protein-1 (PD-1), whereas a lentivirus is used to introduce into autologous T Cells a T cell receptor targeting the cancer antigen NY-ESO-1. The rationale for using CRISPR to supplement the chimeric T cell receptor approach is to reduce the risk of autoimmunity, reduce TCR mispairing and promote the expression of exogenous NY-ESO-1 TCR.

This data will represent the first safety data for CRISPR in properly controlled and regulated human studies.

Asphalion is companionship
Free Webinar to guide you through the process of the Centralised Procedure