No longer Conspiracy Theory: a pill (or injection) that modifies genes…

07-01-22

The first CRISPR gene-editing drug is coming—possibly as soon as next year

The first CRISPR gene-editing drug, designed to treat blood disorders, could be on the market by 2023. Here’s what it means for the future of drug development.

In a $900 million collaboration, rare disease specialist Vertex and CRISPR Therapeutics developed the therapy, dubbed exa-cel (short for exagamglogene autotemcel). It has already amassed promising evidence that it can help patients with beta thalassemia and sickle cell disease (SCD), both of which are genetic blood diseases that are relatively rare in the U.S. but somewhat more common inherited conditions globally.