Novartis Purchases Cystinosis Gene Therapy from AVROBIO

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The $87.5 million purchase grants Novartis rights to an investigational gene therapy program intended for treatment of cystinosis.

Novartis announced the purchase of an investigational hematopoietic stem cell gene therapy program from AVROBIO, a pharmaceutical company specializing in clinical-stage gene therapy treatments, on May 22, 2023. Novartis will pay $87.5 million for the asset, which is intended for treatment of cystinosis, a rare genetic disease. According to a company press release, the move is designed 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, interim CEO and current chief finance officer, AVROBIO, in the release.

“Novartis acquiring the cystinosis program is a wonderful validation for the whole hematopoietic gene therapy field and great news for cystinosis patients,” said Geoff MacKay, former CEO of AVROBIO, in a statement on LinkedIn (1). MacKay stepped down from his long-time position as CEO earlier this month to join the leadership team at an early-stage company (2).

“Novartis is acquiring AVROBIO’s investigational hematopoietic stem cell gene therapy for the treatment of cystinosis, a rare, genetic, metabolic, lysosomal storage disease that causes cystine accumulation which can lead to widespread tissue and organ damage, including end stage renal disease,” said Novartis in a statement sent via email. “The acquisition of a potentially transformative and first-in-class therapy for a severe, rare condition with no disease-modifying options aligns with Novartis’ platform strategy in Gene & Cell Therapy. Ongoing development of the therapy, AVR-RD-04, will be supported by Novartis’ experience and capabilities in the development and delivery of gene and cell therapies for rare diseases.”




1. MacKay, G. Personal Statement. LinkedIn, May 23, 2023, (accessed May 30, 2023)

2. AVROBIO. AVROBIO Announces Leadership Transition. Press Release, May 1, 2023.