AVROBIO has announced a deal to sell its investigational haematopoietic stem cell (HSC) gene therapy programme, designed to treat cystinosis, to Novartis in an all-cash deal valued at $87.5m.

Novartis will receive an exclusive licence for intellectual property related to the gene therapy platform of Avrobio for use in cystinosis and for other assets.

Avrobio will retain complete rights to its HSC gene therapies portfolio developed to treat Pompe disease, Gaucher disease type 1 and type 3, and Hunter syndrome.

The company expects that the proceeds obtained from the deal will extend its cash runway into the fourth quarter of 2024.

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

The company will also provide knowledge transfer and other related services to support the programme’s transition.

Wells Fargo Securities and TD Cowen are serving as financial advisors to Avrobio.

In 2022, Avrobio’s investigational gene therapy, AVR-RD-04, received a rare paediatric disease designation from the US Food and Drug Administration.

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