Novartis buys AVROBIO cystinosis gene therapy program for $87.5M


AVROBIO, Inc., a clinical-stage gene therapy company working to free people from a lifetime of genetic disease, has agreed to sell its investigational hematopoietic stem cell (HSC) gene therapy program for the treatment of cystinosis to Novartis for $87.5 million in cash. 

AVROBIO retains full rights to its portfolio of first-in-class HSC gene therapies for Gaucher disease type 1 and type 3, Hunter syndrome and Pompe disease. Proceeds from this transaction are expected to extend the company’s cash runway into the fourth quarter of 2024. 

“This transaction strengthens AVROBIO’s balance sheet, focuses our pipeline strategy and is a strong endorsement of our HSC gene therapy approach and plato gene therapy platform,” said Erik Ostrowski, AVROBIO’s interim CEO and current CFO.

Transaction details 

Novartis will pay AVROBIO $87.5 million in cash at closing, in consideration for the sale and transfer of certain assets related to the cystinosis program. In addition, AVROBIO has exclusively licensed to Novartis certain other assets, know-how and other intellectual property related to AVROBIO’s gene therapy platform for use in cystinosis. 

To support the transition of the program, AVROBIO also has agreed to provide under a separate agreement certain transition, knowledge transfer and other related services. 

About cystinosis

Cystinosis is a rare genetic disorder characterized by the accumulation of the amino acid dimer cystine in different tissues and organs of the body, with the kidneys and eyes particularly susceptible, although it can include muscles, the liver, thyroid, pancreas and brain. 


Massachusetts-based AVROBIO targets the cause of genetic disease by introducing a functional copy of the affected gene into patients’ own hematopoietic stem cells (HSCs), with the goal of durably expressing the therapeutic protein throughout the body, including the central nervous system. 

The company’s first-in-class pipeline includes clinical programs for Gaucher disease and Hunter syndrome, as well as a preclinical program for Pompe disease. 

Its plato gene therapy platform is scalable for planned global commercialization. 

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