AVROBIO Sells Cystinosis Gene Therapy Program to Novartis

Anna Osborne
24 May 2023
Clinical Trials
Collaborations, Mergers & Acquisitions
Finance & Investment
Gene Therapy
Viral/Non-Viral Vectors
Leading gene therapy company AVROBIO has announced an agreement to sell its investigational hematopoietic stem cell (HSC) gene therapy program for cystinosis treatment to Novartis for $87.5 million.

According to the asset purchase agreement, Novartis will acquire certain assets related to the cystinosis program, and AVROBIO will exclusively license other assets and intellectual property to Novartis for use in cystinosis treatment. Financial advisory services for AVROBIO are being provided by TD Cowen and Wells Fargo Securities, LLC.

Under a separate agreement, AVROBIO will also provide transition and knowledge transfer services to support Novartis.

This strategic move allows AVROBIO to focus on its portfolio of first-in-class HSC gene therapies for other genetic diseases; Gaucher disease type 1 and 3, Hunter syndrome and Pompe disease.

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The proceeds from the $87.5 million cash transaction will 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.

The investigational gene therapy for cystinosis, a rare multisystem genetic disorder resulting in the accumulation of cystine in the patient’s tissues and organs, is under an ongoing Phase I/II clinical evaluation with AVROBIO and the University of California.

The study was funded in part by California Institute for Regenerative Medicine (CIRM), Cystinosis Research Foundation (CRF), and the National Institutes of Health (NIH).

The gene therapy targeting Cystinosis introduces a functional copy of the affected gene into a patient’s own hematopoietic stem cells, aiming to achieve durable expression of the therapeutic protein throughout the body.

Results from the clinical trial are to be expected in the first quarter of 2025.

Source: AVROBIO Press Release


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