Emma Ungstrup’s Post

Was a fascinating Ad Com yesterday @FDA for the first-ever therapy to use CRISPR/Cas9 gene editing (Vertex's very impressive exa-cel gene therapy for sickle cell disease)- "Off target" gene editing, genomic heterogeneity, genome sequencing of edits, and the long term biological consequences of undesired edits was up for discussion. Take homes are its really important to analyse the genetic changes after editing and to hold samples back for long-term follow up (maybe a new guidance coming soon). The potential approval of this product next month is an important step for the CRISPR/Cas9 gene editing field of which there are dozens of other therapies in development.