Novartis has made a deal to buy Avrobio’s investigational hematopoietic stem cell (HSC) gene therapy program focusing on cystinosis treatment of cystinosis, a rare genetic disease. This was a $87.5 million cash deal expected to extend Avrobio’s cash runway into the fourth quarter of 2024.
 
Avrobio is a leading company in clinical-stage gene therapy and will continue to have full rights to its HSC gene therapy portfolio. This first-in-class HSC gene therapy focuses on lifetime generic diseases such as Hunter syndrome, Gaucher disease type 1 and type 3, and Pompe disease.