Novartis buys the AVROBIO cystinosis gene therapy program

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

AVROBIO owns the full rights to the class one HSC gene therapy portfolio for Type 1 and Type 3 Gaucher disease, Hunter syndrome and Pompe disease. The proceeds from this transaction are expected to extend the company’s cash foundation into the fourth quarter of 2024.

“This transaction strengthens AVROBIO’s balance sheet, focuses our channel strategy and is a strong endorsement of both our HSC gene therapy approach and our plateau 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 of the sale and transfer of certain assets related to the cystinosis program. In addition, AVROBIO exclusively licenses certain Novartis assets, know-how and other intellectual property associated with the AVROBIO gene therapy platform for use in cystinosis.

To support program transitions, AVROBIO has also 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 accumulation of the amino acid cystine dimer in various tissues and organs of the body, with the kidneys and eyes especially susceptible, although it can include the muscles, liver, thyroid, pancreas and brain.


Massachusetts-based AVROBIO targets genetic disease-causing agents by inserting functional copies of affected genes into patients’ own hematopoietic stem cells (HSCs), with the goal of long-lasting expressing therapeutic proteins throughout the body, including the central nervous system.

The company’s class one pipeline includes clinical programs for Gaucher disease and Hunter syndrome, as well as preclinical programs for Pompe disease.

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

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