All-cash transaction, with full $87.5 million to be paid at closing

Proceeds expected to extend cash runway into Q4 2024

CAMBRIDGE, MA, USA I May 22, 2023 IAVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company working to free people from a lifetime of genetic disease, today announced an agreement 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
Pursuant to the terms of an asset purchase 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 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. TD Cowen and Wells Fargo Securities, LLC are acting as financial advisors to AVROBIO in the transaction.

Our vision is to bring personalized gene therapy to the world. We target the root 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. Our first-in-class pipeline includes clinical programs for Gaucher disease and Hunter syndrome, as well as a preclinical program for Pompe disease. Our proprietary plato® gene therapy platform is scalable for planned global commercialization. We are headquartered in Cambridge, Mass. For additional information, visit, and follow us on Twitter and LinkedIn.

SOURCE: Avrobio