Avrobio sells gene therapy program to Novartis

May 23, 2023

Massachusetts-based Avrobio announced this week that it will sell its investigational hematopoietic stem cell (HSC) gene therapy program for the treatment of cystinosis to Novartis in an all-cash transaction valued at $87.5 million.

Under the terms of the agreement, Novartis will pay Avrobio for the sale and transfer of the assets associated with the cystinosis program. Cystinosis is a genetic disorder characterized by an accumulation of cystine in tissue and organs.

Avrobio will retain the rest of its portfolio of HSC gene therapies for Gaucher disease type 1 and type 3, Hunter syndrome, and Pompe disease. With the transaction, the biotech says it will have enough cash runway through the fourth quarter of next year.

Cystinosis is classified as a lysosomal storage disorder, as its onset is caused by a lack of a specific amino acid transporter that causes cystine to accumulate in lysosomes. Early symptoms in infancy include poor feeding, vomiting, and dehydration.  Although standard care treatment has improved and the condition can improve after a kidney transplant, cystinosis still causes debilitating symptoms that can significantly shorten patients’ life expectancy. 

Following the announcement of the transaction, Avrobio’s shares went up 69%.