Avrobio Sells Cystinosis Gene Therapy Program to Novartis for $87.5 Million

Rare Daily Staff

Gene therapy developer Avrobio said it has entered into an agreement to sell its investigational hematopoietic stem cell 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 chief financial officer.

Under the terms of the agreement, 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 Abrobio’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.

Erik Ostrowski, Avrobio’s interim CEO and current chief financial officer